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236 Trials

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Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Rare Diseases
Alta, NCT03061201: A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A

Active, not recruiting
2
11
US
SB-525 (PF-07055480)
Pfizer
Hemophilia A
06/24
06/24
NCT05092815: The Efficacy and Safety of HLX208 in Adult Langerhans Cell Histiocytosis (LCH) and Erdheim-Chester Disease (ECD) With BRAF V600E Mutation

Active, not recruiting
2
25
RoW
HLX208, BRAF V600E inhibitor
Shanghai Henlius Biotech
Langerhans Cell Histiocytosis, Erdheim-Chester Disease, LCH, ECD
06/24
10/24
AHAEmi, NCT05345197: Emicizumab in Patients With Acquired Hemophilia A

Recruiting
2
51
US
emicizumab, Hemlibra (R)
University of Washington, Genentech, Inc.
Acquired Hemophilia A
07/24
01/25
NCT06386744: Dusquetide for the Treatment of Behcet's Disease

Not yet recruiting
2
25
RoW
Dusquetide, SGX945, SGX942
Soligenix
Behçet Disease
02/25
03/25
PAGETEX, NCT03713203: ® Photodynamic Therapy Device for the Treatment of Extra Mammary Paget's Disease of the Vulva (EMPV).

Recruiting
2
24
Europe
pagetex PDT
University Hospital, Lille, Galderma R&D, Institut National de la Santé Et de la Recherche Médicale, France
Paget Disease of the Vulva, Paget Disease, Extramammary
08/24
08/24
NCT04585893: Safety and Efficacy of Rituximab for Treatment of Multicentric Castleman Disease in Malawi

Recruiting
2
27
RoW
Rituximab, Rituxan, Etoposide, Toposar, VP-16
UNC Lineberger Comprehensive Cancer Center, Fogarty International Center of the National Institute of Health
Multicentric Castleman Disease
08/24
08/26
REDEEM-1, NCT05116774 / 2020-004438-39: BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy

Terminated
2
12
Europe
BCX9930, Eculizumab, Soliris, Ravulizumab, Ultomiris, ALXN1210, ravulizumab-cwvz
BioCryst Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria (PNH)
09/23
09/23
NCT05650736: Janus Kinase Inhibition in Granuloma Annulare

Active, not recruiting
2
10
US
Abrocitinib 200 mg
William Damsky, Yale University, Pfizer
Granuloma Annulare
08/24
10/24
NCT04094675: Sirolimus for Cowden Syndrome With Colon Polyposis

Active, not recruiting
2
10
US
Sirolimus, Rapamycin, Rapamune
Ohio State University, PTEN Research, Pfizer
PTEN Gene Mutation, PTEN Hamartoma Tumor Syndrome, PTEN Hamartoma Syndrome, Cowden Syndrome, Bannayan Syndrome, Bannayan Zonana Syndrome, Polyposis
02/25
02/25
SYNAPSE-SMA, NCT05794139 / 2022-002301-24: Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

Recruiting
2
54
Europe, Canada, US
NMD670, Placebo
NMD Pharma A/S, NMD Pharma A/S
Spinal Muscular Atrophy
12/24
12/24
NCT06303570: A Study to Evaluate the Efficacy and Safety of CBL-514 Compared to Placebo in Participants With Dercum's Disease Lipomas

Not yet recruiting
2
30
NA
CBL-514 injection, 0.9% Sodium chloride
Caliway Biopharmaceuticals Co., Ltd.
Dercum's Disease
09/25
09/25
NCT04702568 / 2020-000501-93: A Long Term Safety Study of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Terminated
2
19
Europe, RoW
BCX9930
BioCryst Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria, PNH
10/23
10/23
BGB-3111-2005-IIT, NCT05356858: An Open Label Study of the Effects and Safety of Zanubrutinib in NMOSDs Adult Patients

Recruiting
2
20
RoW
zanubrutinib
Xuanwu Hospital, Beijing
Neuromyelitis Optica
10/24
10/24
NCT06051357: Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria

Active, not recruiting
2
26
RoW
HRS-5965 tablets
Chengdu Suncadia Medicine Co., Ltd.
Paroxysmal Nocturnal Hemoglobinuria
10/24
10/24
NCT05659368: Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1

Recruiting
2
10
Europe
Tirzepatide
Ospedale San Raffaele
Wolfram Syndrome
10/24
12/24
NCT05551598: Efficacy and Safety of Mitoxantrone Hydrochloride Liposome Injection in the Treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD)

Not yet recruiting
2
45
NA
Mitoxantrone Hydrochloride Liposome Injection
CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.
Neuromyelitis Optica Spectrum Disorder
10/24
04/25
SWOG S1609, NCT02834013: Nivolumab and Ipilimumab in Treating Patients With Rare Tumors

Checkmark From cohort 36 of DART trial in combination with Yervoy for metaplastic breast cancer
Jun 2020 - Jun 2020: From cohort 36 of DART trial in combination with Yervoy for metaplastic breast cancer
Checkmark From cohort 36 of DART trial in combination with Opdivo for metaplastic breast cancer
Jun 2020 - Jun 2020: From cohort 36 of DART trial in combination with Opdivo for metaplastic breast cancer
Checkmark In combination with yervoy in aggressive neuroendocrine tumors
More
Active, not recruiting
2
818
US, RoW
Biospecimen Collection, Biological Sample Collection, Biospecimen Collected, Specimen Collection, Computed Tomography, CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, tomography, Echocardiography, EC, Ipilimumab, Anti-Cytotoxic T-Lymphocyte-Associated Antigen-4 Monoclonal Antibody, BMS-734016, Ipilimumab Biosimilar CS1002, MDX-010, MDX-CTLA4, Yervoy, Magnetic Resonance Imaging, Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, MRIs, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging, sMRI, Structural MRI, Nivolumab, ABP 206, BCD-263, BMS-936558, CMAB819, MDX-1106, NIVO, Nivolumab Biosimilar ABP 206, Nivolumab Biosimilar BCD-263, Nivolumab Biosimilar CMAB819, ONO-4538, Opdivo
National Cancer Institute (NCI)
Acinar Cell Carcinoma, Adenoid Cystic Carcinoma, Adrenal Cortical Carcinoma, Adrenal Gland Pheochromocytoma, Anal Canal Neuroendocrine Carcinoma, Anal Canal Undifferentiated Carcinoma, Angiosarcoma, Apocrine Neoplasm, Appendix Mucinous Adenocarcinoma, Bartholin Gland Transitional Cell Carcinoma, Basal Cell Carcinoma, Bladder Adenocarcinoma, Breast Metaplastic Carcinoma, Cervical Adenocarcinoma, Cholangiocarcinoma, Chordoma, Colorectal Squamous Cell Carcinoma, Desmoid Fibromatosis, Endometrial Transitional Cell Carcinoma, Endometrioid Adenocarcinoma, Esophageal Neuroendocrine Carcinoma, Esophageal Undifferentiated Carcinoma, Extrahepatic Bile Duct Carcinoma, Extramammary Paget Disease, Fallopian Tube Adenocarcinoma, Fallopian Tube Transitional Cell Carcinoma, Fibromyxoid Tumor, Gallbladder Carcinoma, Gastric Neuroendocrine Carcinoma, Gastric Squamous Cell Carcinoma, Gastric Undifferentiated Carcinoma, Gastrointestinal Stromal Tumor, Gestational Trophoblastic Tumor, Giant Cell Carcinoma, Human Papillomavirus-Independent Cervical Adenocarcinoma, Clear Cell-Type, Intestinal Neuroendocrine Carcinoma, Intrahepatic Cholangiocarcinoma, Lung Neuroendocrine Tumor, Lung Sarcomatoid Carcinoma, Major Salivary Gland Carcinoma, Malignant Odontogenic Neoplasm, Malignant Peripheral Nerve Sheath Tumor, Malignant Solid Neoplasm, Malignant Testicular Sex Cord-Stromal Tumor, Metastatic Malignant Neoplasm of Unknown Primary, Metastatic Pituitary Neuroendocrine Tumor, Minimally Invasive Lung Adenocarcinoma, Mixed Mesodermal (Mullerian) Tumor, Mucinous Adenocarcinoma, Mucinous Cystadenocarcinoma, Nasal Cavity Adenocarcinoma, Nasal Cavity Carcinoma, Nasopharyngeal Carcinoma, Nasopharyngeal Low Grade Papillary Adenocarcinoma, Nasopharyngeal Undifferentiated Carcinoma, Oral Cavity Carcinoma, Oropharyngeal Undifferentiated Carcinoma, Ovarian Adenocarcinoma, Ovarian Germ Cell Tumor, Ovarian Mucinous Adenocarcinoma, Ovarian Squamous Cell Carcinoma, Ovarian Transitional Cell Carcinoma, Pancreatic Acinar Cell Carcinoma, Pancreatic Neuroendocrine Carcinoma, Paraganglioma, Paranasal Sinus Adenocarcinoma, Paranasal Sinus Carcinoma, Parathyroid Gland Carcinoma, PEComa, Penile Squamous Cell Carcinoma, Peritoneal Mesothelial Neoplasm, Placental Choriocarcinoma, Primary Peritoneal High Grade Serous Adenocarcinoma, Pseudomyxoma Peritonei, Rare Disorder, Scrotal Squamous Cell Carcinoma, Seminal Vesicle Adenocarcinoma, Seminoma, Serous Cystadenocarcinoma, Small Intestinal Adenocarcinoma, Small Intestinal Squamous Cell Carcinoma, Spindle Cell Neoplasm, Teratoma With Somatic-Type Malignancy, Testicular Non-Seminomatous Germ Cell Tumor, Thyroid Gland Carcinoma, Tracheal Carcinoma, Transitional Cell Carcinoma, Ureter Adenocarcinoma, Ureter Squamous Cell Carcinoma, Urethral Adenocarcinoma, Urethral Squamous Cell Carcinoma, Vaginal Adenocarcinoma, Vaginal Squamous Cell Carcinoma, Not Otherwise Specified, Vulvar Carcinoma
05/26
05/26
NCT05476887: To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104

Recruiting
2
35
RoW
KP104
Kira Pharmacenticals (US), LLC.
Paroxysmal Nocturnal Hemoglobinuria
11/24
02/25
NCT04186559: Topical Pentoxifylline Gel on Behcet's Disease Genital Ulcers

Not yet recruiting
2
60
US
Topical Pentoxifylline Gel (Vehicle +PTX), Trental, Topical Placebo Gel (Vehicle)
Silk Road Therapies, Inc.
Behcet Syndrome, Behcet Disease
11/24
12/24
NCT04396496: Treatment of POEMS Syndrome With Daratumumab

Completed
2
2
US
Daratumumab Injection, Darzalex
University of Arkansas, Janssen Scientific Affairs, LLC
POEMS Syndrome
08/23
10/23
NACHO COBI, NCT04079179: Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders

Recruiting
2
90
US
Cobimetinib, COTELLIC, RO5514041
Carl Allen, Baylor College of Medicine, North American Consortium for Histiocytosis, Genentech, Inc.
Langerhan's Cell Histiocytosis, Juvenile Xanthogranuloma, Erdheim-Chester Disease, Rosai Dorfman Disease, Neuro-Degenerative Disease, Histiocytic Sarcoma, Histiocytic Disorders, Malignant
12/24
12/29
NCT06134414: Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Not yet recruiting
2
40
NA
MY008211A tablets, MY008211A
Wuhan Createrna Science and Technology Co., Ltd
Paroxysmal Nocturnal Hemoglobinuria (PNH)
07/25
12/25
Jewelfish, NCT03032172 / 2016-004184-39: A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

Active, not recruiting
2
174
Europe, US
Risdiplam, RO7034067
Hoffmann-La Roche
Spinal Muscular Atrophy
12/24
12/24
iMCD, NCT04743687: Zanuburutinib in Relapsed and Refractory a Prospective, Single-center, Single-arm Trial

Recruiting
2
30
RoW
Zanubrutinib, brukinsa
Peking Union Medical College Hospital
Idiopathic Multicentric Castleman's Disease
01/25
01/25
NCT05376319: PR3-AAV Resilient Remission or PRRR

Terminated
2
6
US
Obinutuzumab, Rituximab
Mayo Clinic, Genentech, Inc.
Granulomatosis With Polyangiitis, Microscopic Polyangiitis, ANCA Associated Vasculitis
05/24
05/24
NURTURE, NCT02386553 / 2014-002098-12: A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Active, not recruiting
2
25
Europe, Canada, US, RoW
Nusinersen, ISIS 396443, BIIB058, ISIS SMNRx, Spinraza
Biogen
Spinal Muscular Atrophy
12/24
12/24
CBD_RE, NCT05803434: Cannabidiol in Children and Young Adults With Rare Disease-associated Severe Epilepsy

Not yet recruiting
2
30
NA
Cannabidiol oral solution
Meyer Children's Hospital IRCCS
Epilepsy, Rare Diseases
03/25
03/25
NCT06046222: Trial of Efficacy and Safety of NS-229 Versus Placebo in Patients With Eosinophilic Granulomatosis With Polyangiitis

Recruiting
2
45
Japan, US
NS-229, Placebo
NS Pharma, Inc., Nippon Shinyaku Co., Ltd.
Eosinophilic Granulomatosis With Polyangiitis, Churg-Strauss Syndrome
04/25
05/25
NCT06044350: A Clinical Trial of BAT4406F Injection in Patients With Neuromyelitis Optica Spectrum Disorders

Recruiting
2
162
RoW
BAT4406F Injection, BAT4406F Placebos
Bio-Thera Solutions
Optic Neuromyelitis Spectrum Disease
05/25
05/26
NCT03520647: Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

Recruiting
2
56
US
Cyclophosphamide, Peripheral Blood Stem Cells
National Heart, Lung, and Blood Institute (NHLBI)
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS), Paroxysmal Nocturnal Hemoglobinuria (PNH)
06/25
06/28
NCT02938793: Durvalumab in Combination With Tremelimumab in Subjects With Advanced Rare Solid Tumors

Recruiting
2
150
US
Durvalumab, MEDI4736, Tremelimumab, CP-675,206
Prisma Health-Upstate, AstraZeneca, MedImmune LLC
Cancer, Rare Disease
02/26
06/27
NCT05646524: Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

Not yet recruiting
2
12
NA
NM8074
NovelMed Therapeutics
Paroxysmal Nocturnal Hemoglobinuria
04/26
09/26
NCT05646563: Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris

Not yet recruiting
2
12
NA
NM8074, Soliris, Eculizumab
NovelMed Therapeutics
Paroxysmal Nocturnal Hemoglobinuria
08/26
01/27
Pupfish, NCT05808764: A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Recruiting
2
10
Europe, US
Risdiplam, Evrysdi
Hoffmann-La Roche
Muscular Atrophy, Spinal
08/25
08/25
PRESent-3, NCT05789537: A Study of SerpinPC in Participants With Hemophilia B (HemB) With Inhibitors

Recruiting
2
12
Europe, US, RoW
SerpinPC, Activated Protein C (APC) inhibitor
ApcinteX Ltd, Centessa Pharmaceuticals plc
Hemophilia B With Inhibitor, Hemophilia B
09/25
10/25
NCT00092222: Virotherapy and Natural History Study of KHSV-Associated Multricentric Castleman s Disease With Correlates of Disease Activity

Active, not recruiting
2
75
US
Etoposide, Interferon-alpha, Rituximab, Zidovudine, Liposomal Doxorubicin, Bortezomib, Valganciclovir, Doxorubicin, Vincristine, Cyclophosphamide, Filgrastim (G-CSF), Prednisone, Sirolimus, Observation Only
National Cancer Institute (NCI)
Lymphoproliferative Disorder, HHV-8, Malignancy, HIV
10/25
10/25
CALCIFADE, NCT05662111: Treatment of Ectopic Calcification in Fahr's Disease or Syndrome

Recruiting
2
98
Europe
Etidronate, Etidronate disodium, Placebo
UMC Utrecht, Netherlands Brain Foundation
Fahr Disease, Fahr Syndrome, Primary Familial Brain Calcification
03/26
03/26
R-HLH, NCT05762640: Ruxolitinib as First Line Treatment in Primary Haemophagocytic Lymphohistiocytosis

Not yet recruiting
2
20
NA
Ruxolitinib
Assistance Publique - Hôpitaux de Paris
Haemophagocytic Lymphohistiocytosis
11/26
11/27
NCT04965597: Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

Recruiting
2
40
US
Treosulfan, Dihydroxybusulfan, Ovastat, Treosulphan, Tresulfon, Trecondi, Fludarabine Phosphate, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, 9H-purin-6-amine, 2-fluoro-9-(5-O-phosphono-beta-D-arabinofuranosyl), Beneflur, Fludara, Fludarabine-5'-Monophosphate, SH T 586, Tacrolimus, FK 506, Fujimycin, Hecoria, Prograf, Protopic, Methotrexate, Abitrexate, Alpha-Methopterin, Amethopterin, Brimexate, Emthexat, Emtexate, Farmitrexat, Methotrexate LPF, Methylaminopterin, Methotrexatum, Metotrexato, Novatrex, Rheumatrex, Texate, Tremetex, Trexeron, Trixilem, WR-19039, Lapine T-Lymphocyte Immune Globulin, Anti-Thymocyte Globulin Rabbit, Grafalon, Rabbit Anti-Human Thymocyte Globulin (RATG), Rabbit Anti-Thymocyte Globulin, Rabbit Antithymocyte Globulin, Rabbit ATG, rATG, Thymoglobulin, Antithymocyte globulin rabbit ATG, Imtix, Peripheral Blood Stem Cell Transplantation, PBPC transplantation, PBSCT, Peripheral Blood Progenitor Cell Transplantation, PERIPHERAL BLOOD STEM CELL TRANSPLANT, peripheral stem cell support, Peripheral Stem Cell Transplant, peripheral stem cell transplantation, Allogeneic Bone Marrow Transplantation, Allo BMT, Allogeneic Blood and Marrow Transplantation, Allogeneic BMT, Quality-of-Life Assessment, Quality of Life Assessment, Echocardiography, EC, Multigated Acquisition Scan, Blood Pool Scan, Equilibrium Radionuclide Angiography, Gated Blood Pool Imaging, Gated Heart Pool Scan, MUGA, Multi-Gated Acquisition Scan, MUGA Scan, Radionuclide Ventriculogram Scan, Radionuclide ventriculography, RNVG, SYMA Scanning, Synchronized Multigated Acquisition Scanning, Bone Marrow Biopsy, Bone Marrow Aspiration, Biospecimen Collection, Biological Sample Collection, X-Ray Imaging, Conventional X-Ray, Diagnostic Radiology, Computed Tomography, Computed Axial Tomography, CAT Scan, CT Scan
Fred Hutchinson Cancer Center, Blood and Marrow Transplant Clinical Trials Network, National Cancer Institute (NCI), National Marrow Donor Program, National Heart, Lung, and Blood Institute (NHLBI)
Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Shwachman-Diamond Syndrome, Hematologic Neoplasm With Germline GATA2 Mutation, Hematologic Neoplasm With Germline SAMD9 Mutation, Hematologic Neoplasm With Germline SAMD9L Mutation
12/25
12/26
NCT05741346 / 2021-006776-17: Long-term Safety of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria

Active, not recruiting
2
28
Europe, RoW
BCX9930
BioCryst Pharmaceuticals, BioCryst Pharmaceuticals Inc
Paroxysmal Nocturnal Hemoglobinuria
02/26
02/26
PRESent-2, NCT05789524: The Efficacy and Safety of SerpinPC in Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

Recruiting
2
120
Europe, Canada, US, RoW
SerpinPC, Activated Protein C (APC) inhibitor
ApcinteX Ltd, Centessa Pharmaceuticals plc
Hemophilia A, Hemophilia B
03/26
06/26
SENS-NMO, NCT05974293: A Phase IIb Study of Nabiximols for Spasticity Due to Neuromyelitis Optica Spectrum Disorders

Not yet recruiting
2
44
US
Nabiximols, Sativex, Placebo
Michael, Levy M.D.,Ph.D., Jazz Pharmaceuticals
NMO Spectrum Disorder, Spasticity, Muscle
04/26
09/26
NCT05845723: Tocilizumab and Tofacitinib in the Treatment of Vascular Behçet's Syndrome

Not yet recruiting
2
81
RoW
tocilizumab, ACTEMRA®, tofacitinib, Xeljanz®, cyclophosphamide, Endoxana
Peking Union Medical College Hospital
Aneurysm, Behcet Syndrome, Vascular Type
06/26
06/27
NCT05731050: Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Not yet recruiting
2
6
NA
NM8074
NovelMed Therapeutics
PNH - Paroxysmal Nocturnal Hemoglobinuria
08/26
10/26
SATELITE, NCT04871191: Study of Salvage Therapy to Treat Patients With Granulomatosis With Polyangiitis

Not yet recruiting
2
42
Europe
Rituximab, Mabthera, Tocilizumab, RoActemra, Abatacept, Orencia
Assistance Publique - Hôpitaux de Paris, URC-CIC Paris Descartes Necker Cochin
Granulomatosis With Polyangiitis, Anti-neutrophil Cytoplasmic Antibody-associated Vasculitis
09/26
01/27
UVB, NCT05874505: "Comparison of the Efficacy and Safety of Adalimumab to That of Tocilizumab in Severe Uveitis of Behçet's Disease"

Not yet recruiting
2
60
NA
Adalimumab, Tocilizumab
Assistance Publique - Hôpitaux de Paris
Behcet's Uveitis
11/26
07/27
NCT01821781: Immune Disorder HSCT Protocol

Recruiting
2
20
US
Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
Washington University School of Medicine
Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Hyper-IgM, DiGeorge Syndrome, Chediak-Higashi Syndrome, Common Variable Immune Deficiency, Immune Dysregulatory Disorders, Hemophagocytic Lymphohistiocytosis, IPEX, Autoimmune Lymphoproliferative Syndrome, X-linked Lymphoproliferative Syndrome
03/27
03/27
NCT05549258 / 2021-003528-33: Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

Recruiting
2
15
Europe, US
Inebilizumab
Amgen, Horizon Therapeutics Ireland DAC
Neuromyelitis Optica Spectrum Disorder
04/27
04/27
ATA129-EBV-205, NCT04554914 / 2020-000177-25: A Study to Evaluate Tabelecleucel in Participants With Epstein-barr Virus-associated Diseases

Hourglass Jan 2023 - Dec 2023 : From P2 trial for Epstein-Barr virus-associated sarcoma
Recruiting
2
190
Europe, US
Tabelecleucel, tab-cel®, ATA129, EBV-CTLs
Atara Biotherapeutics
Epstein-Barr Virus (EBV)-Associated Diseases, EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (EBV+ PID LPD), EBV+ Lymphoproliferative Disease With Acquired (Non-congenital) Immunodeficiency (EBV+ AID LPD), EBV+ Posttransplant Lymphoproliferative Disease in Central Nervous System (EBV+ CNS PTLD), EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD), Solid Organ Transplant Complications, Lymphoproliferative Disorders, Allogeneic Hematopoietic Cell Transplant, Stem Cell Transplant Complications, EBV+ Sarcomas, Leiomyosarcoma
06/27
05/29
NCT00716066: Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases

Active, not recruiting
2
53
US
Anti-Thymocyte Globulin, Antithymocyte Globulin, Antithymocyte Serum, ATG, ATGAM, ATS, Thymoglobulin, Autologous Hematopoietic Stem Cell Transplantation, Autologous Stem Cell Transplantation, Carmustine, BCNU, Becenum, Becenun, BiCNU, Bis(chloroethyl) Nitrosourea, Bis-Chloronitrosourea, Carmubris, Carmustin, Carmustinum, FDA 0345, Gliadel, N,N'-Bis(2-chloroethyl)-N-nitrosourea, Nitrourean, Nitrumon, SK 27702, SRI 1720, WR-139021, 154-93-8, Cytarabine, .beta.-Cytosine arabinoside, 1-.beta.-D-Arabinofuranosyl-4-amino-2(1H)pyrimidinone, 1-.beta.-D-Arabinofuranosylcytosine, 1-Beta-D-arabinofuranosyl-4-amino-2(1H)pyrimidinone, 1-Beta-D-arabinofuranosylcytosine, 1.beta.-D-Arabinofuranosylcytosine, 2(1H)-Pyrimidinone, 4-Amino-1-beta-D-arabinofuranosyl-, 2(1H)-Pyrimidinone, 4-amino-1.beta.-D-arabinofuranosyl-, Alexan, Ara-C, ARA-cell, Arabine, Arabinofuranosylcytosine, Arabinosylcytosine, Aracytidine, Aracytin, Aracytine, Beta-Cytosine Arabinoside, CHX-3311, Cytarabinum, Cytarbel, Cytosar, Cytosar-U, Cytosine Arabinoside, Cytosine-.beta.-arabinoside, Cytosine-beta-arabinoside, Erpalfa, Starasid, Tarabine PFS, U 19920, U-19920, Udicil, WR-28453, 147-94-4, Etoposide, Demethyl Epipodophyllotoxin Ethylidine Glucoside, EPEG, Lastet, Toposar, Vepesid, VP 16-213, VP-16, VP-16-213, 33419-42-0, Laboratory Biomarker Analysis, Melphalan, Alanine Nitrogen Mustard, CB-3025, L-PAM, L-Phenylalanine Mustard, L-Sarcolysin, L-Sarcolysin Phenylalanine mustard, L-Sarcolysine, Melphalanum, Phenylalanine Mustard, Phenylalanine Nitrogen Mustard, Sarcoclorin, Sarkolysin, WR-19813, 148-82-3, Peripheral Blood Stem Cell Transplantation, PBPC transplantation, Peripheral Blood Progenitor Cell Transplantation, Peripheral Stem Cell Support, Peripheral Stem Cell Transplantation, Prednisone, .delta.1-Cortisone, 1, 2-Dehydrocortisone, Adasone, Cortancyl, Dacortin, DeCortin, Decortisyl, Decorton, Delta 1-Cortisone, Delta-Dome, Deltacortene, Deltacortisone, Deltadehydrocortisone, Deltasone, Deltison, Deltra, Econosone, Lisacort, Meprosona-F, Metacortandracin, Meticorten, Ofisolona, Orasone, Panafcort, Panasol-S, Paracort, PRED, Predicor, Predicorten, Prednicen-M, Prednicort, Prednidib, Prednilonga, Predniment, Prednisonum, Prednitone, Promifen, Servisone, SK-Prednisone, 53-03-2, Syngeneic Bone Marrow Transplantation
Fred Hutchinson Cancer Center
Autoimmune Disease, Neurologic Autoimmune Disease, Autologous Transplant Autoimmune, Multiple Sclerosis Transplant, MS Stem Cell Transplant, Multiple Sclerosis Stem Cell Transplant, Stiff Person Syndrome, HCT for Neurologic Autoimmune Disorders, CIDP Transplant, Myasthenia Gravis Transplant, Autoimmune Nervous System Disorder, Central Nervous System Vasculitis, Cerebellar Degeneration, Chronic Inflammatory Demyelinating Polyneuropathy, Lambert Eaton Myasthenic Syndrome, Myasthenia Gravis, Neuromyelitis Optica, Opsoclonus Myoclonus Syndrome, Rasmussen Subacute Encephalitis
01/25
01/30
NCT03307980: Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

Active, not recruiting
2
21
Canada, US, RoW
PF-06838435 (formerly SPK-9001)
Pfizer
Hemophilia B
05/29
05/29
NCT06052618: Phase II Study of Pacritinib in Kaposi Sarcoma Herpesvirus (KSHV)-Associated Multicentric Castleman Disease and KSHV-Associated Inflammatory Cytokine Syndrome (KICS)

Not yet recruiting
2
65
US
Pacritinib
National Cancer Institute (NCI)
KSHV Inflammatory Cytokine Syndrome (KICS), Kaposi Sarcoma Herpesvirus -Associated Multicentric Castleman Disease
01/33
01/34
NCT05907759: Daratumumab for Relapsed/Refractory Primary Effusion Lymphoma

Recruiting
2
28
US
Daratumumab SC
National Cancer Institute (NCI)
Lymphoma, Primary Effusion
08/34
12/35
Hemophilia-7: Phase I/II clinical trial with NecLip-FVIIa

Completed
1/2
0
RestOfWorld
NecLip-rFVIIa (LongSeven, recombinant liposomal factor VIIa)
Recoly
Hemophilia
 
 
Hemophilia-8: Phase I/II clinical trial to assess BAX 335 in hemophilia B

Ongoing
1/2
16
RoW
FIX (BAX335: Gene Therapy)
Baxter
Factor IX
 
 
Hemophilia-5: Combined Phase 1/2 clinical trial involving NAV rAAV8-mediated gene transfer in patients with hemophilia B

Ongoing
1/2
0
RoW
rAAV8 vector-based factor IX gene therapy
REGENX BioScience
Hemophilia
 
 
NCT01465100: Liver Cell Transplant for Phenylketonuria

Terminated
1/2
1
US
Preparative Radiation Therapy, Hepatocyte Transplant, Immunosuppression, Liver Evaluation, Neuro-psychological Assessment, Whole body Phe oxidation testing, Liver Biopsy
Ira Fox
Phenylketonuria
08/15
06/16
2009-013227-28: A Double-Blind, Randomized, Crossover Study of the Recovery of FACTANE 100 versus 200 IU/ml followed by an Open-Label Safety Study of FACTANE 200 IU/ml in Previously Treated Patients With Severe (FVIII:C<1%) Haemophilia A

Ongoing
1/2
12
Europe
Human coagulation factor VIII (FACTANE 200 IU/ml): powder and solvent for solution intravenous use, FACTANE 100 UI/ml, FACTANE 200 IU/ml, FACTANE 100 IU/ml, FACTANE 100 IU/ml
LFB BIOTECHNOLOGIES
Haemophilia A
 
 
2015-000359-26: First study in patients to assess safety, tolerability and inittial efficacy of the new gene therapy product to treat MPSIIIA. Primer estudio en pacientes para evaluar la seguridad, tolerabilidad y eficacia inicial del nuevo producto de terapia génica para tratar MPSIIIA.

Ongoing
1/2
6
Europe
Vector viral adenoasociado de serotipo 9 que contiene el gen de la sulfamidasa humana, AAV9-CAG-coh-SGSH, Suspension for injection
Laboratorios del Dr. Esteve, S.A., Laboratorios del Dr. Esteve,S.A.
Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) is an inherited lysosomal storage disease caused by a specific lysosomal enzyme deficiency that leads to intracellular accumulation of the GAG heparan sulphate (HS). It is caused by a deficiency of one of the four enzymes involved in the lysosomal degradation of HS. In the case of subtype A is the heparan N-sulfatase (SGSH). La Mucopolisacaridosis tipo IIIA (síndrome de Sanfilippo A) es una enfermedad hereditaria de almacenamiento lisosomal causada por una deficiencia de la enzima lisosomal específica que conduce a la acumulación intracelular del Heparán Sulfato GAG (HS). Es causada por una deficiencia de una de las cuatro enzimas implicadas en la degradación lisosomal de HS. En el caso del subtipo A es el heparán N-sulfatasa (SGSH)., Inherited disease caused by the deficiency of Sulfamidase, the enzime that degrades Heparan Sulfate, leading to its accumulation in the cells causing the severe signs and symptoms of MPSIIIA Enfermedad hereditaria causada por la deficiencia de Sulfamidasa, enzima que degrada el Heparan Sulfato,conduciendo a su acumulación en las células causando los graves signos y síntomas de la MPS IIIA, Diseases [C] - Nutritional and Metabolic Diseases [C18]
 
 
2016-003430-25: A clinical study to learn about the effects of a virus that transfers the gene for human coagulation factor IX in adults with moderate/severe to severe inherited coagulations defects in the long term

Not yet recruiting
1/2
6
Europe
DTX101, Concentrate for solution for infusion
Ultragenyx Pharmaceutical, Inc., Ultragenyx Pharmaceutical, Inc.
Moderate/severe to severe hemophilia B, Moderate/severe to severe inherited blood coagulation disorder, Diseases [C] - Blood and lymphatic diseases [C15]
 
 
2017-000662-29: A Phase 1/2 study to evaluate the safety and efficacy of BMN 270 gene transfer in patients with severe hemophilia A and pre-existing antibodies to AAV5

Not yet recruiting
1/2
10
Europe
AAV5-hFVIII-SQ, BMN 270, Solution for infusion
BioMarin Pharmaceutical Inc., BioMarin Pharmaceutical Inc.
Haemophilia A, Bleeding Disorder, Diseases [C] - Blood and lymphatic diseases [C15]
 
 
TIGET-MLD, NCT01560182 / 2009-017349-77: Gene Therapy for Metachromatic Leukodystrophy (MLD)

Active, not recruiting
1/2
20
Europe
OTL-200 Gene Therapy, Previously GSK2696274
Orchard Therapeutics, Ospedale San Raffaele
Lysosomal Storage Disease, Metachromatic Leukodystrophy
04/18
03/25
2018-000206-28: A phase 1/2 study to evaluate the safety and tolerability of SB-318, a rAAV2/6-based Gene Therapy, in patients with Mucopolysaccharidosis I (MPS I)

Not yet recruiting
1/2
27
Europe
rAAV2/6 Left ZFN Vector, rAAV2/6 Right ZFN Vector, SB-A6P-HRL Donor Vector, SB-47171, SB-47898, SB-IDUA, Solution for infusion
Sangamo Therapeutics, Inc., Sangamo Therapeutics, Inc.
Mucopolysaccharidosis type I (MPS I), Hurler/Hurler-Scheie/Scheie syndrome, a recessive lysosomal storage disorder caused by a deficiency in iduronidase (IDUA) enzyme., Diseases [C] - Nutritional and Metabolic Diseases [C18]
 
 
2017-000806-39: Study to test the safety and how well patients with severe hemophilia A respond to treatment with BAY 2599023 (DTX 201), a drug therapy that delivers a healthy version of the defective Factor VIII gene into the nucleus of liver cells using an altered, non-infectious virus (AAV) as a “shuttle”.

Not yet recruiting
1/2
30
Europe, RoW
recombinant AAV vector with hu37 serotype capsid (AAVhu37) encoding human FVIII, BAY 2599023 (DTX201), Concentrate for solution for infusion
Bayer AG, Bayer AG
Hemophilia A, Hemophilia A is an X-linked congenital bleeding disorder causing frequent bleedings and recurrent spontaneous bleeds into the soft tissue and joints, leading to joint damage and severe disability., Diseases [C] - Blood and lymphatic diseases [C15]
 
 
2019-001878-28: A Phase 1/2 study to evaluate the safety and efficacy of BMN 307 gene therapy in patients with phenylketonuria

Not yet recruiting
1/2
100
Europe
BMN 307, Solution for infusion
BioMarin Pharmaceutical Inc., BioMarin Pharmaceutical Inc.
Phenylketonuria, metabolic disorder, Diseases [C] - Nutritional and Metabolic Diseases [C18]
 
 
2019-004210-33: Safety and Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A

Not yet recruiting
1/2
18
Europe
BDD-hFVIII Producing Spheres, SIG-001, Suspension for injection
Sigilon Therapeutics, Inc., Sigilon Therapeutics, Inc.
Haemophilia A, Haemophilia A is a genetic deficiency in clotting factor VIII, which causes increased bleeding and usually affects males., Diseases [C] - Blood and lymphatic diseases [C15]
 
 
2020-000067-23: First in human study to test the safety and preliminary efficacy of PPSGG, an antibody catcher in patients with anti-MAG neuropathy

Not yet recruiting
1/2
48
Europe
PPSGG solution for infusion, PPSGG, Solution for infusion
Polyneuron Pharmaceuticals AG, Polyneuron Pharmaceuticals AG
anti-MAG neuropathy, Anti-MAG peripheral neuropathy is a rare disease. It occurs when the body’s own immune system develops antibodies against myelin (protein of the nerves, myelin-associated glycoprotein, MAG)., Diseases [C] - Nervous System Diseases [C10]
 
 
2019-003213-34: A Phase 1/2 study to evaluate the safety and efficacy of BMN 270 gene transfer in patients with severe hemophilia A and active or prior inhibitors

Not yet recruiting
1/2
20
Europe
AAV5-hFVIII-SQ, BMN 270, Solution for infusion, ROCTAVIAN
BioMarin Pharmaceutical Inc., BIOMARIN PHARMACEUTICAL INC., BioMarin Pharmaceutical Inc.
Hemophilia A, Bleeding Disorder, Diseases [C] - Blood and lymphatic diseases [C15]
 
 
2020-000739-28: A Phase I/IIb extension study assessing the long-term safety and efficacy of a gene therapy (AAV5-hFIX) previously administered to adult patients with severe or moderately severe haemophilia B during the CT-AMT-060-01 Phase I/II study.

Ongoing
1/2
10
Europe
AAV5-hFIX, AMT-060, Solution for infusion
uniQure biopharma B.V., uniQure biopharma B.V.
Haemophilia B, Haemophilia B - Bleeding disorder, Diseases [C] - Blood and lymphatic diseases [C15]
 
 
2020-003371-18: Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Efficacy of Marzeptacog alfa (activated) in Treatment of Episodic Bleeding in Subjects with Factor VII deficiency, Glanzmann thrombasthenia, and Haemophilia A with inhibitors on prophylaxis Studio per valutare farmacocinetica, farmacodinamica, sicurezza ed efficacia di marzeptacog alfa (attivato) nel trattamento degli episodi emorragici in soggetti con il deficit di fattore VII, tromboastenia di Glanzmann o l’emofilia A con inibitori attualmente in trattamento preventivo con una dose di emicizumab.

Not yet recruiting
1/2
24
Europe
Marzeptacog alfa (activated), [MarzAA, CB813d, PF-05280602], Powder for solution for injection
Catalyst Biosciences, Inc., Catalyst Biosciences, Inc.
Factor VII deficiency, Glanzmann thrombasthenia (GT) and Hemophilia A with inhibitors on emicizumab prophylaxis (HAwI-E) deficit di fattore VII, tromboastenia di Glanzmann (GT) e l’emofilia A con inibitori attualmente in trattamento preventivo con una dose di emicizumab (HAwI-E)., Inherited rare bleeding disorders in which blood cannot clot normally difetti ereditari della coagulazioni in cui il sangue non è in grado di coagulare normalmente, Diseases [C] - Blood and lymphatic diseases [C15]
 
 
2018-004024-11: A Phase 1/2 study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects

Not yet recruiting
1/2
21
Europe
SHP648 (TAK748), Solution for injection
Baxalta Innovations GmbH, Baxalta Innovations GmbH
Hemophilia B is a X-linked recessive bleeding disorder caused by mutations in the gene encoding clotting factor IX (FIX) that result in disruption of the normal clotting pathway. Hemophilia B affects 1 in 25,000 male births. Disease severity correlates directly with the concentration of functional FIX protein in the plasma. Severe disease is characterized as having <1% of normal plasma levels of FIX (100% = 1 IU activity/mL or approximately 5000 ng protein/mL)., Hemophilia B is an inherited bleeding disorder caused by a lack of the blood clotting factor IX (9) in your blood. Without enough factor IX, the blood cannot clot properly to stop bleeding., Diseases [C] - Blood and lymphatic diseases [C15]
 
 
NCT01966367: CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

Active, not recruiting
1/2
37
US
CD34 Stem Cell Selection Therapy
Diane George
Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndromes, Acquired Immunodeficiency Syndromes, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemia
02/30
02/30
NCT03602079: Study of A166 in Patients With Relapsed/Refractory Cancers Expressing HER2 Antigen or Having Amplified HER2 Gene

Completed
1/2
49
US
A166
Klus Pharma Inc.
HER2-positive Breast Cancer, HER2 Gene Mutation, HER-2 Gene Amplification, HER2 Positive Gastric Cancer, Salivary Gland Cancer, Salivary Gland Tumor, Salivary Gland Carcinoma, Salivary Gland Neoplasms, Lung Cancer, Colo-rectal Cancer, Rare Diseases, Solid Tumor, Recurrent Gastric Cancer, Recurrent Colon Cancer, Recurrent Breast Cancer, Head and Neck Cancer, Head and Neck Carcinoma, Bladder Cancer, Cervical Cancer, Liver Cancer, Bile Duct Cancer, Urologic Cancer, Pancreatic Cancer, Prostate Cancer, Recurrent Prostate Cancer, Rectal Cancer, Recurrent Ovarian Carcinoma, Recurrent Renal Cell Cancer, Rectal Cancer Stage II, Rectal Cancer Stage I, Rectal Cancer Stage III, Skin Cancer, Mouth Cancer, Lip Cancer Stage I, Tongue Cancer, Breast Neoplasm Malignant Primary, Larynx Cancer, Tonsil Cancer, Palate Cancer, Mucoepidermoid Carcinoma, Primary Peritoneal Carcinoma, Mucinous Adenocarcinoma Gastric, Mucinous Breast Cancer Recurrent, Cholangiocarcinoma
01/22
01/22
2018-000504-42: Gene Therapy Clinical Trial for Mucopolysaccharidosis IIIA in patient with middle and advanced phases of the disease Ensayo Clínico de terapia génica para la Mucopolisacaridosis tipo IIIA en pacientes con enfermedad MPS IIIA media y avanzada

Not yet recruiting
1/2
12
Europe
scAAV9.U1A.SGSH, ABO-102, Suspension for injection
Abeona Therapeutics Inc, Abeona Therapeutics Inc
MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progressive, withdeterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatmentcurrently available for the disease. MPS IIIA es una enfermedad de depósito lisosomal, causada por un defecto genético de la enzima N-sulfoglucosamina sulfohidrolasa. Los niños parecen normales al nacer, pero la enfermedad es progresiva, conel deterioro de las habilidades sociales y de adaptación, la disminución neurocognitiva y la muerte prematura. La muerte se produce normalmente a finales de la segunda o principios de la tercera década. Es de destacar que no existe un tratamiento disponible actualmente para la enfermedad., Mucopolysaccharidosis type IIIA is a genetic disease in children, caused by the toxicity of an accumulation of substances in the body that generate a progressive deterioration. La mucopolisacaridosis tipo IIIA es una enfermedad genética en niños, causada por la toxicidad de un acúmulo de sustancias en el organismo que generan un deterioro progresivo., Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
2018-002776-40: Study to assess the safety and pharmacokinetic of subcutaneous injection of OCTA101 in previously treated adult patients suffering from severe hemophilia A

Not yet recruiting
1/2
40
Europe
OCTA101 (human-cl rhFVIII and recombinant human von Willebrand Factor fragment dimer), Nuwiq, Powder and solution for solution for injection, Nuwiq
Octapharma AG, Octapharma AG
Severe hemophilia A, Haemophilia A patients have insufficient levels of an important factor (factor VIII) in their blood. Factor VIII is important to stop bleeding., Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
2021-001004-15: Study of ACE-1334 to Evaluate the Safety, Pharmacokinetics, Pharmacodynamic Effects, and Efficacy in Participants with Systemic Sclerosis with and without Interstitial Lung Disease Studio di di ACE-1334 per valutare la sicurezza, la farmacocinetica, gli effetti farmacodinamici in partecipanti affetti da sclerosi sistemica con e senza malattia polmonare interstiziale

Not yet recruiting
1/2
210
Europe
TGF-beta recettore di tipo II extracellulare, collegata a un dominio Fc umano modificato dell’immuno, TGF-beta receptor type-2 extracellular domain fused to human IgG1 Fc domain; TGF-beta recettore di t, [ACE-1334], Lyophilisate for suspension for injection, Lyophilisate for solution for injection
ACCELERON PHARMA INC., Acceleron Pharma Inc.
Systemic sclerosis (SSc), a rare connective tissue disorder characterized by fibrosis, inflammation, and microvascular injury with heterogeneous presentations; Interstitial lung disease (ILD), a common manifestation of SSc that tends to occur early in the course of the disease. La sclerosi sistemica (SSc) è una malattia rara del tessuto connettivo, caratterizzata da fibrosi, infiammazione e lesione microvascolare con presentazioni eterogenee. La malattia polmonare interstiziale (Interstitial Lung Disease, [ILD]) è una manifestazione comune della SSc che tende a manifestarsi precocemente nel corso della malattia, SSc is a rare disorder characterized by fibrosis of the skin and/or internal organs. SSc-ILD is a rare, progressive autoimmune disease described by inflammation and fibrosis of the lungs/other organs. SSc è una malattia rara caratterizzata da fibrosi della pelle e/o di organi interni. SSc-ILD è una malattia progressiva autoimmune rara e descritta da infiammazione e fibrosi dei polmoni/altri organi, Diseases [C] - Skin and Connective Tissue Diseases [C17]
 
 
2021-000608-39: This is a first in human study, in patients with severe bleeding disorder (hemophilia A), who has developed inhibitory antibodies against coagulation factor VIII.The aim is to study the safety and efficacy of dendritic cells, originating from the patient itself. The dendritic cells has been loaded with coagulation factor VIII for an improved immunological tolerance.

Not yet recruiting
1/2
9
Europe
ItolDC-028, ItolDC-028, Suspension for injection
Idogen AB, Idogen AB
Treatment of patients with Hemophilia A who have developed inhibitory antibodies to clotting Factor VIII and have failed Immune Tolerance Induction, Patients with severe bleeding disorder (hemophilia A), who has developed inhibitory antibodies against coagulation factor VIII, Body processes [G] - Immune system processes [G12]
 
 
NCT02268552 / 2014-002053-19: An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)

Completed
1/2
40
Europe, RoW
branaplam
Novartis Pharmaceuticals
Spinal Muscular Atrophy
12/22
12/22
pheNIX, NCT03952156: Gene Therapy Clinical Study in Adult PKU

Terminated
1/2
10
US
HMI-102
Homology Medicines, Inc
Phenylketonurias, PAH Deficiency
01/23
08/23
ChiCTR2000031254: Safety and pharmacokinetics of ACT001 Capsules for the treatment of patients with recurrent neuromyelitis optica spectrum disorder-related optic neuritis (NMOSD-ON) : an open-label, dose-escalation, phase I/IIa clinical study

Recruiting
1/2
12
 
Subjects will receive single dose 100mg of ACT001 in the morning at Day 1 of Cycle 1, followed by a 48-hour washout period, and then receive consecutive treatment with ACT001 BID (100mg x 2times)since Day 3 till withdrawal from the study (30-day treatment period for Cycle 1, 28-day treatment period ;Subjects will receive single dose 200mg of ACT001 in the morning at Day 1 of Cycle 1, followed by a 48-hour washout period, and then receive consecutive treatment with ACT001 BID (200mg x 2times)since Day 3 till withdrawal from the study (30-day treatment period for Cycle 1, 28-day treatment period ;Subjects will receive single dose 400mg of ACT001 in the morning at Day 1 of Cycle 1, followed by a 48-hour washout period, and then receive consecutive treatment with ACT001 BID (400mg x 2times)since Day 3 till withdrawal from the study (30-day treatment period for Cycle 1, 28-day treatment period ;Subjects will receive single dose 600mg of ACT001 in the morning at Day 1 of Cycle 1, followed by a 48-hour washout period, and then receive consecutive treatment with ACT001 BID (600mg x 2times)since Day 3 till withdrawal from the study (30-day treatment period for Cycle 1, 28-day treatment period
The First Medical Center of PLA General Hospital; Accendatech Luoyang Co., Ltd., Some are funded by the government and others are self-funded
Recurrent NMOSD-ON
 
 
NCT03734588: Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

Completed
1/2
4
US
SPK-8016
Spark Therapeutics
Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Factor VIII (FVIII), Factor VIII (FVIII) Deficiency, Factor VIII (FVIII) Gene, Factor VIII (FVIII) Protein, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Gene Therapy, Gene Transfer, Hematologic Diseases, Hemorrhagic Disorders, Recombinant, Vector, Inhibitors
10/20
01/23
NCT02554773 / 2015-001395-21: An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

Calendar Jan 2024 - Dec 2024: Data from trial for hemophilia A and B
Checkmark Additional data from OLE study at ISTH 2017
Jul 2017 - Jul 2017: Additional data from OLE study at ISTH 2017
Checkmark Presentation from open label extension trial for hemophilia A or B without inhibitors at ASH 2016
Dec 2016 - Dec 2016: Presentation from open label extension trial for hemophilia A or B without inhibitors at ASH 2016
Completed
1/2
34
Europe, US, RoW
Fitusiran (SAR439774)
Genzyme, a Sanofi Company
Hemophilia A, Hemophilia B
03/23
03/23
NCT04198818: A Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HH2710 in Patient With Advanced Tumors

Terminated
1/2
37
US, RoW
HH2710
Haihe Biopharma Co., Ltd.
Advanced Tumors, Melanoma, Non-Small-Cell Lung Cancer, Erdheim-Chester Disease, Other RAS/RAF/MEK/ERK Mutated Tumors
03/23
03/23
MARVEL1, NCT04040049 / 2018-002097-51: A Fabry Disease Gene Therapy Study

Terminated
1/2
3
Europe, Canada, US
FLT190
Freeline Therapeutics
Fabry Disease, Lysosomal Storage Diseases
05/23
05/23
NCT03641703: A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy

Terminated
1/2
10
Europe
FLT180a
Freeline Therapeutics
Hemophilia B
05/23
05/23
NCT05164471: Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B

Terminated
1/2
6
Europe, US
verbrinacogene setparvovec
Freeline Therapeutics
Hemophilia B
05/23
05/23
2021-001079-18: A clinical trial to confirm the dose and safety of FLT180a in adult subjects with hemophilia B. Eine klinische Studie zur Bestätigung der Dosis und Sicherheit von FLT180a bei erwachsenen Patienten mit Hämophilie B.

Not yet recruiting
1/2
9
Europe
FLT180a, FLT180a, Solution for infusion
Freeline Therapeutics Ltd, Freeline Therapeutics Ltd
Haemophilia B., Hereditary bleeding disorder caused by a lack of blood clotting factor., Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
IRB2021-YX-187-01, NCT05154734: Efficacy and Safety of Belimumab in Neuromyelitis Optica Spectrum Disorders

Completed
1/2
14
RoW
Belimumab
Tianjin Medical University General Hospital
NMO Spectrum Disorder
06/23
04/24
NCT05027230: A Safety and Efficacy Study of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor

Completed
1/2
77
RoW
STSP-0601 for Injection
Staidson (Beijing) Biopharmaceuticals Co., Ltd
Hemophilia
07/23
07/23
EDELWEISS, NCT06185335: A Trial of the Safety and Efficacy of Single-Dose Administration of ANB-010 in Subjects With Hemophilia A

Recruiting
1/2
50
RoW
ANB-010, dose 1, ANB-010, dose 2, ANB-010, dose 3
Biocad
Hemophilia A
12/25
06/33
NCT02034630: Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in GRD

Recruiting
1/2
5
RoW
Busulfan
Seoul National University Hospital
Genetic Rare Disease
12/23
12/23
NCT03003533: A Gene Transfer Study for Hemophilia A

Completed
1/2
25
Canada, US, RoW
SPK-8011
Spark Therapeutics
Hemophilia A
12/23
12/23
NCT04676048: ASC618 Gene Therapy in Hemophilia A Patients

Recruiting
1/2
12
US
ASC618
ASC Therapeutics
Hemophilia A
12/23
12/26
RESET-Myositis, NCT06154252: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy

Recruiting
1/2
18
US
CAB-201 following preconditioning with fludarabine and cyclophosphamide
Cabaletta Bio
Idiopathic Inflammatory Myopathy, Dermatomyositis, Anti-Synthetase Syndrome, Immune-Mediated Necrotizing Myopathy
07/28
07/28
ChiCTR2200056833: Single arm and single center clinical study on safety, tolerance and efficacy of GC101 injection in patients with spinal muscular atrophy type 1

Completed
1/2
3
 
Intrathecal injection of GC101
Seventh Medical Center of PLA General Hospital; Seventh Medical Center of PLA General Hospital, E-Town cooperation & Development Foundation
type 1 spinal muscular atrophy (SMA1)
 
 
NCT06010953: SS109 and NovoSeven ® PK / PD Profile, and Preliminary Efficacy and Safety of SS109 on Demand Treatment

Recruiting
1/2
24
RoW
SS109
Jiangsu Gensciences lnc.
Male, Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Hemophilia
07/24
07/24
NCT02576795 / 2014-003880-38: Gene Therapy Study in Severe Haemophilia A Patients (270-201)

Checkmark Hemophilia A
Dec 2017 - Dec 2017: Hemophilia A
Active, not recruiting
1/2
15
Europe
valoctocogene roxaparvovec, BMN 270
BioMarin Pharmaceutical
Severe Haemophilia A
03/24
03/24
NCT06288230: An Open Label Study of Gene Therapy Product in Spinal Muscular Atrophy Patients

Not yet recruiting
1/2
6
NA
vesemnogene lantuparvovec, AAV-hSMN1
Lantu Biopharma
Spinal Muscular Atrophy
03/27
03/27
NCT05792462: Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders

Recruiting
1/2
12
RoW
Baricitinib
Tianjin Medical University General Hospital
NMO Spectrum Disorder
12/25
12/25
NCT06147856: A Dose-finding Study to Evaluate mRNA-3210 in Participants With Phenylketonuria

Recruiting
1/2
54
US, RoW
mRNA-3210
ModernaTX, Inc.
Phenylketonuria
08/26
08/27
 

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