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 42 Trials 
61 Trials

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Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Viskochil, David
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
NCT05005845: NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)

Completed
2
199
US
NFX-179 gel, NFX-179 topical gel, Vehicle gel, NFX-179 vehicle gel
NFlection Therapeutics, Inc.
Cutaneous Neurofibroma, Neurofibromatosis 1
10/23
10/23
NCT05238324: Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

Withdrawn
1
9
Canada, US
Genetic HMI-203
Homology Medicines, Inc
Mucopolysaccharidosis II
10/24
01/29
NCT05795361: Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Available
N/A
Europe, US, RoW
Idursulfase-IT, HGT-2310, TAK-609
Takeda
Hunter Syndrome
 
 
Cottin, Vincent
NCT03626688 / 2018-001187-33: A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH Patients

Recruiting
3
1000
Europe, Canada, US, RoW
Ralinepag, APD811, Placebo
United Therapeutics
PAH, Pulmonary Hypertension, Pulmonary Arterial Hypertension, Hypertension, Connective Tissue Diseases, Familial Primary Pulmonary Hypertension, Vascular Diseases, Cardiovascular Diseases, Hypertension, Pulmonary, Lung Diseases, Respiratory Tract Disease
12/25
12/25
IMPALA-2, NCT04544293 / 2020-001263-85: Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

Active, not recruiting
3
160
Europe, Canada, Japan, US, RoW
Molgramostim, Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF), Placebo
Savara Inc., Savara ApS,
Autoimmune Pulmonary Alveolar Proteinosis
11/23
11/24
TETON-2, NCT05255991 / 2021-005881-17: Multinational Study of Efficacy and Safety of Inhaled Treprostinil in Subjects With Idiopathic Pulmonary Fibrosis

Active, not recruiting
3
597
Europe, RoW
Placebo, Inhaled Treprostinil, Tyvaso, Treprostinil Ultrasonic Nebulizer
United Therapeutics
Idiopathic Pulmonary Fibrosis, Interstitial Lung Disease
07/25
07/25
IMPAHCT, NCT05036135 / 2021-001910-13: A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH)

Terminated
2/3
202
Europe, Canada, US, RoW
AV-101, Placebo
Aerovate Therapeutics, Aerovate Therapeutics, Inc.
Pulmonary Arterial Hypertension
08/24
08/24
ELEVATE 2, NCT04712669 / 2020-004971-42: A Study of Rodatristat Ethyl in Patients With Pulmonary Arterial Hypertension

Completed
2
108
Europe, Canada, US, RoW
rodatristat ethyl 300 mg tablet BID, rodatristat ethyl 600 mg BID, Placebo
Altavant Sciences GmbH
Pulmonary Arterial Hypertension
06/23
08/23
Tesar, Vladimir
FALCON, NCT03918447 / 2018-004651-20: A Trial of Bardoxolone Methyl in Patients With ADPKD -

Terminated
3
667
Europe, Japan, US, RoW
Bardoxolone methyl oral capsule, RTA 402, Placebo oral capsule
Reata, a wholly owned subsidiary of Biogen, Reata Pharmaceuticals, Inc., REATA PHARMACEUTICALS, INC
Autosomal Dominant Polycystic Kidney, ADPKD
08/23
08/23
ALIGN, NCT04573478: Atrasentan in Patients With IgA Nephropathy

Active, not recruiting
3
404
Europe, Canada, Japan, US, RoW
Atrasentan, CHK-01, Atrasentan Hydrochloride, ABT-627, Placebo
Chinook Therapeutics, Inc.
IgA Nephropathy, Immunoglobulin A Nephropathy
12/26
12/26
NCT05123586: A IMMA Master Protocol: A Study of LY3361237 in Participants With at Least Moderately Active Systemic Lupus Erythematosus

Terminated
2
85
Europe, US, RoW
LY3361237, Placebo
Eli Lilly and Company
Systemic Lupus Erythematosus
12/23
12/23
NCT06274489: A Study to Evaluate Setanaxib in Patients With Alport Syndrome

Recruiting
1/2
18
Europe, RoW
Setanaxib, Placebo
Calliditas Therapeutics AB
Alport Syndrome
01/25
01/25
REYNAUD-GAUBERT, Martine
BOSTON-2, NCT03656926 / 2018-003205-25: Efficacy + Safety of Liposome Cyclosporine a to Treat Bronchiolitis Obliterans Post Single Lung Transplant

Completed
3
220
Europe, US, RoW
Liposomal Cyclosporine A, L-CsA, Standard of Care, SoC
Zambon SpA, BREATH Therapeutics Inc.
Bronchiolitis Obliterans, Chronic Rejection of Lung Transplant, Lung Transplant Rejection, Lung Transplant; Complications, Lung Transplant Failure and Rejection, Chronic Lung Allograft Dysfunction
03/24
03/24
TETON-2, NCT05255991 / 2021-005881-17: Multinational Study of Efficacy and Safety of Inhaled Treprostinil in Subjects With Idiopathic Pulmonary Fibrosis

Active, not recruiting
3
597
Europe, RoW
Placebo, Inhaled Treprostinil, Tyvaso, Treprostinil Ultrasonic Nebulizer
United Therapeutics
Idiopathic Pulmonary Fibrosis, Interstitial Lung Disease
07/25
07/25
IMPAHCT, NCT05036135 / 2021-001910-13: A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH)

Terminated
2/3
202
Europe, Canada, US, RoW
AV-101, Placebo
Aerovate Therapeutics, Aerovate Therapeutics, Inc.
Pulmonary Arterial Hypertension
08/24
08/24
NCT05032066: A Multicenter Trial to Evaluate the Efficacy, Safety and Tolerability of HZN-825 in Subjects With Idiopathic Pulmonary Fibrosis

Jan 2024 - Dec 2024: Data from trial for diffuse cutaneous systemic sclerosis
Terminated
2
153
Europe, Canada, Japan, US, RoW
HZN-825, Placebo
Amgen
Idiopathic Pulmonary Fibrosis
07/24
01/25
Cibula, David
innovaTV 301, NCT04697628 / 2019-001655-39: Tisotumab Vedotin vs Chemotherapy in Recurrent or Metastatic Cervical Cancer

Active, not recruiting
3
502
Europe, Canada, Japan, US, RoW
tisotumab vedotin, TIVDAK, topotecan, vinorelbine, gemcitabine, irinotecan, pemetrexed
Seagen Inc., Genmab
Cervical Cancer
07/23
08/25
ENGOT-EN20, NCT05611931 / 2022-002540-42: Selinexor in Maintenance Therapy After Systemic Therapy for Participants with P53 Wild-Type, Advanced or Recurrent Endometrial Carcinoma

Jul 2024 - Dec 2024: Top-line data from XPORT-EC trial in participants with TP53 wild type endometrial cancer
Recruiting
3
220
Europe, Canada, US, RoW
Selinexor, KPT-330, Matching Placebo for selinexor
Karyopharm Therapeutics Inc, GOG Foundation, European Network of Gynaecological Oncological Trial Groups (ENGOT), Belgium and Luxembourg Gynaecological Oncology Group, North Eastern German Society of Gynaecological Oncology, Multicentre Italian Trials in Ovarian Cancer and Gynecologic Malignancies, Spanish Research Group in Ovarian Cancer, The Central and Eastern European Gynecologic Oncology Group, Israeli Society of Gynecologic Oncology, Australia New Zealand Gynaecological Oncology Group
Endometrial Cancer
01/25
01/28
FLORA-4, NCT04498117 / 2020-002270-26: Oregovomab Plus Chemo in Newly Diagnosed Patients With Advanced Epithelial Ovarian Cancer Following Optimal Debulking Surgery

Active, not recruiting
3
615
Europe, Canada, US, RoW
Oregovomab, MAb-B43.13, Paclitaxel, Taxol, Carboplatin, Paraplatin, Placebo
CanariaBio Inc., Gynecologic Oncology Group, Iqvia Pty Ltd
Carcinoma, Ovarian Epithelial, Ovarian Neoplasms, Ovarian Cancer, Ovarian Serous Adenocarcinoma, Fallopian Tube Neoplasms, Fallopian Tube Adenocarcinoma, Fallopian Tube Serous Adenocarcinoma, Peritoneal Cancer, Peritoneal Carcinoma, Peritoneal Neoplasms
09/25
08/27
Rajan, Deepa Soundara
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
NCT04251026 / 2019-004909-27: A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Active, not recruiting
1/2
47
Europe, Canada, US
tividenofusp alfa
Denali Therapeutics Inc.
Mucopolysaccharidosis II
07/27
07/27
NCT04007536: A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Completed
N/A
18
Europe, US
No Intervention
Denali Therapeutics Inc.
Mucopolysaccharidosis II
03/24
03/24
NCT03047369: The Myelin Disorders Biorepository Project

Recruiting
N/A
12000
US
Children's Hospital of Philadelphia, National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (NINDS), National Center for Advancing Translational Sciences (NCATS), Biogen, Eli Lilly and Company, Myrtelle Inc., Orchard Therapeutics Ltd., Passage Bio, Inc., Synaptix Biotherapeutics Ltd., Takeda, Boehringer Ingelheim, Ionis Pharmaceuticals, Inc., Sanofi Winthrop Industrie, Sana Biotechnology, Yaya Foundation for 4H Leukodystrophy, University of Pennsylvania, United MSD Foundation, Foundation to Fight H-ABC, Calliope Joy Foundation, Don't Forget Me Foundation
Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
12/30
12/30
Bailey, Carrie
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
NCT05005845: NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)

Completed
2
199
US
NFX-179 gel, NFX-179 topical gel, Vehicle gel, NFX-179 vehicle gel
NFlection Therapeutics, Inc.
Cutaneous Neurofibroma, Neurofibromatosis 1
10/23
10/23
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
NCT05781399: First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria

Recruiting
1
112
US, RoW
JNT-517 Suspension, Placebo Suspension, JNT-517 Tablet, Placebo Tablet
Jnana Therapeutics
Phenylketonuria
12/24
03/25
ENERGY, NCT05734196: The Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency

Recruiting
1
16
Europe, US
INZ-701, (rhENPP1-Fc).
Inozyme Pharma
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum
03/26
04/26
Magner, Martin
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
Hillman, Paul
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC

Available
N/A
US
Arimoclomol
ZevraDenmark
Niemann-Pick Disease, Type C
 
 
Jansa, Pavel
TripleTRE, NCT06317805: Initial Triple Therapy Including Parenteral Treprostinil vs Initial Double Oral Therapy in PAH Group I Patients

Recruiting
4
110
Europe, RoW
Generic treprostinil sodium + Standard of Care (Double Oral), Trisuva, Tresuvi, Treposa, Treprostinil Orpha-Devel, Treprostinil Amomed, Treprostinil OrPha, Treposuvi, Standard of Care - Double Oral
AOP Orphan Pharmaceuticals AG, ANOVA CRO s.r.o., PharmaLex Belgium, Aixial s.r.o., GCP-Service International Ltd. & Co. KG
Pulmonary Arterial Hypertension
06/27
06/27
NCT03626688 / 2018-001187-33: A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH Patients

Recruiting
3
1000
Europe, Canada, US, RoW
Ralinepag, APD811, Placebo
United Therapeutics
PAH, Pulmonary Hypertension, Pulmonary Arterial Hypertension, Hypertension, Connective Tissue Diseases, Familial Primary Pulmonary Hypertension, Vascular Diseases, Cardiovascular Diseases, Hypertension, Pulmonary, Lung Diseases, Respiratory Tract Disease
12/25
12/25
IMPAHCT, NCT05036135 / 2021-001910-13: A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH)

Terminated
2/3
202
Europe, Canada, US, RoW
AV-101, Placebo
Aerovate Therapeutics, Aerovate Therapeutics, Inc.
Pulmonary Arterial Hypertension
08/24
08/24
ELEVATE 2, NCT04712669 / 2020-004971-42: A Study of Rodatristat Ethyl in Patients With Pulmonary Arterial Hypertension

Completed
2
108
Europe, Canada, US, RoW
rodatristat ethyl 300 mg tablet BID, rodatristat ethyl 600 mg BID, Placebo
Altavant Sciences GmbH
Pulmonary Arterial Hypertension
06/23
08/23
Spicka
MajesTEC-4, NCT05243797 / 2021-002531-27: Phase 3 Study of Teclistamab in Combination With Lenalidomide and Teclistamab Alone Versus Lenalidomide Alone in Participants With Newly Diagnosed Multiple Myeloma as Maintenance Therapy Following Autologous Stem Cell Transplantation

Recruiting
3
1594
Europe, Canada, US, RoW
Teclistamab, JNJ-64007957, Lenalidomide
Stichting European Myeloma Network, Janssen Pharmaceutica
Multiple Myeloma
04/28
04/32
Flekac, Milan
NCT06383390: The Effect of Retatrutide Once Weekly on Cardiovascular Outcomes and Kidney Outcomes in Adults Living With Obesity (TRIUMPH-Outcomes)

Recruiting
3
10000
Europe, Canada, US, RoW
Retatrutide, LY3437943, Placebo
Eli Lilly and Company
Atherosclerotic Cardiovascular Disease (ASCVD), Chronic Kidney Disease (CKD)
02/29
02/29
ACHIEVE-4, NCT05803421: A Study of Daily Oral Orforglipron (LY3502970) Compared With Insulin Glargine in Participants With Type 2 Diabetes and Obesity or Overweight at Increased Cardiovascular Risk

Active, not recruiting
3
2749
Europe, US, RoW
Orforglipron, LY3502970, Insulin Glargine
Eli Lilly and Company
Type 2 Diabetes, Obesity, Overweight or Obesity, Overweight, Cardiovascular Diseases, Chronic Kidney Disease
09/25
01/26
NCT06037252: A Study of Investigational Tirzepatide (LY3298176) Doses in Participants With Type 2 Diabetes and Obesity

Recruiting
2
350
US, RoW
Tirzepatide, LY3298176, Placebo
Eli Lilly and Company
Type 2 Diabetes, Obesity
01/26
10/26
NCT06074562: A Study of LY3556050 in Adult Participants With Diabetic Peripheral Neuropathic Pain

Recruiting
2
410
Europe, Japan, US, RoW
LY3556050, Placebo
Eli Lilly and Company
Diabetic Peripheral Neuropathy
07/25
07/25
Kolar, Dawn
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
NCT04251026 / 2019-004909-27: A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Active, not recruiting
1/2
47
Europe, Canada, US
tividenofusp alfa
Denali Therapeutics Inc.
Mucopolysaccharidosis II
07/27
07/27
PROVIDE, NCT04411654: Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease

Active, not recruiting
1/2
15
Europe, US
LY3884961, Methylprednisolone, Sirolimus, Prednisone
Prevail Therapeutics, Eli Lilly and Company
Gaucher Disease, Type 2
05/28
05/28
NCT04007536: A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Completed
N/A
18
Europe, US
No Intervention
Denali Therapeutics Inc.
Mucopolysaccharidosis II
03/24
03/24
Dolezalova
EMERALD, NCT05001737 / 2021-001577-24: Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE

Active, not recruiting
3
41
Europe, Canada, Japan, US, RoW
Emapalumab, NI-0501, emapalumab-lzsg, ATC code: L04AA39 (WHO)
Swedish Orphan Biovitrum
Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease, Systemic Lupus Erythematosus, SJIA, AOSD, MAS
06/24
09/25
Hachulla
EMERALD, NCT05001737 / 2021-001577-24: Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE

Active, not recruiting
3
41
Europe, Canada, Japan, US, RoW
Emapalumab, NI-0501, emapalumab-lzsg, ATC code: L04AA39 (WHO)
Swedish Orphan Biovitrum
Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease, Systemic Lupus Erythematosus, SJIA, AOSD, MAS
06/24
09/25
Linkova, Lenka
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
Costello
PREVAIL, NCT05202509 / 2021-005092-39: Cardiovascular Outcome Study to Evaluate the Effect of Obicetrapib in Patients With Cardiovascular Disease

Active, not recruiting
3
9541
Europe, Canada, Japan, US, RoW
Obicetrapib, Placebo
NewAmsterdam Pharma, Monash University
Atherosclerotic Cardiovascular Disease
11/26
11/26
CARTITUDE-6, NCT05257083 / 2021-003284-10: A Study of Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) Followed by Ciltacabtagene Autoleucel Versus Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) Followed by Autologous Stem Cell Transplant (ASCT) in Participants With Newly Diagnosed Multiple Myeloma

Recruiting
3
750
Europe, Canada, Japan, US, RoW
Daratumumab, Bortezomib, Lenalidomide, Dexamethasone, Cilta-cel, JNJ-68284528, Cyclophosphamide, Fludarabine
Stichting European Myeloma Network, Janssen Research & Development, LLC
Multiple Myeloma
06/33
08/40
Ceska
PREVAIL, NCT05202509 / 2021-005092-39: Cardiovascular Outcome Study to Evaluate the Effect of Obicetrapib in Patients With Cardiovascular Disease

Active, not recruiting
3
9541
Europe, Canada, Japan, US, RoW
Obicetrapib, Placebo
NewAmsterdam Pharma, Monash University
Atherosclerotic Cardiovascular Disease
11/26
11/26
Rojas, Carolina
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
NCT05553392: StrataXRT Versus Aquaphor for the Prevention and Management of Radiation Dermatitis

Terminated
N/A
13
NA
StrataXRT, Aquaphor
Stratpharma AG
Radiation Dermatitis
05/24
05/24
Jungova
MajesTEC-4, NCT05243797 / 2021-002531-27: Phase 3 Study of Teclistamab in Combination With Lenalidomide and Teclistamab Alone Versus Lenalidomide Alone in Participants With Newly Diagnosed Multiple Myeloma as Maintenance Therapy Following Autologous Stem Cell Transplantation

Recruiting
3
1594
Europe, Canada, US, RoW
Teclistamab, JNJ-64007957, Lenalidomide
Stichting European Myeloma Network, Janssen Pharmaceutica
Multiple Myeloma
04/28
04/32
CARTITUDE-6, NCT05257083 / 2021-003284-10: A Study of Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) Followed by Ciltacabtagene Autoleucel Versus Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) Followed by Autologous Stem Cell Transplant (ASCT) in Participants With Newly Diagnosed Multiple Myeloma

Recruiting
3
750
Europe, Canada, Japan, US, RoW
Daratumumab, Bortezomib, Lenalidomide, Dexamethasone, Cilta-cel, JNJ-68284528, Cyclophosphamide, Fludarabine
Stichting European Myeloma Network, Janssen Research & Development, LLC
Multiple Myeloma
06/33
08/40
Garcia, Marilyn
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
HERO, NCT04732429 / 2023-000785-33: Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia

Terminated
2
26
US, RoW
HST5040, Placebo
HemoShear Therapeutics, HemoShear Therapeutics
Methylmalonic Acidemia, Propionic Acidemia
10/23
10/23
OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC

Available
N/A
US
Arimoclomol
ZevraDenmark
Niemann-Pick Disease, Type C
 
 
Fainboim, Alejandro
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
Q
EMERALD, NCT05001737 / 2021-001577-24: Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE

Active, not recruiting
3
41
Europe, Canada, Japan, US, RoW
Emapalumab, NI-0501, emapalumab-lzsg, ATC code: L04AA39 (WHO)
Swedish Orphan Biovitrum
Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease, Systemic Lupus Erythematosus, SJIA, AOSD, MAS
06/24
09/25
Pachmanova, Barbora
NCT06274489: A Study to Evaluate Setanaxib in Patients With Alport Syndrome

Recruiting
1/2
18
Europe, RoW
Setanaxib, Placebo
Calliditas Therapeutics AB
Alport Syndrome
01/25
01/25

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