Paroxysmal Nocturnal Hemoglobinuria  >>  Phase 2
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Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Paroxysmal Nocturnal Hemoglobinuria
2016-002067-33: An Open Clincal Trial of Coversin in Patients with PNH

Ongoing
2
10
Europe
Coversin, rVA576, Powder for solution for injection
Akari Therapeutics Plc, Akari Therapeutics Plc
Paroxysmal nocturnal haemoglobinuria (PNH), Paroxysmal nocturnal haemoglobinuria (PNH), Diseases [C] - Immune System Diseases [C20]
 
 
2019-003830-17: Study of the Oral Factor D (fD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and with background use of an Approved C5 Inhibitor

Not yet recruiting
2
26
Europe
ALXN2050, ACH-0145228, Capsule
Achillion Pharmaceuticals, Inc., a wholly owned subsidiary of Alexion Pharmaceuticals Inc., Alexion Pharmaceuticals Inc., Achillion Pharmaceuticals, Inc. a wholly owned subsidiary of Alexion Pharmaceuticals Inc., Alexion Pharmaceuticals Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Nocturnal Hemoglobinuria (PNH), Diseases [C] - Immune System Diseases [C20]
 
 
2020-005005-17: Pozelimab and Cemdisiran Combination Treatment in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy

Not yet recruiting
2
24
Europe
Pozelimab, Cemdisiran, REGN3918, ALN-CC5, Solution for injection/infusion, Solution for injection
Regeneron Pharmaceuticals, Inc., Regeneron Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, Paroxysmal Nocturnal Hemoglobinuria (PNH), Diseases [C] - Blood and lymphatic diseases [C15]
 
 
ChiCTR2000039675: Phase II clinical trial of the superiority of all-trans retinoic acid in the treament of paroxysmal nocturnal hemoglobinuria

Recruiting
2
20
 
Tretinoin 0.33mg/kg.d; methylprednisolone 0.8mg/kg.d ;methylprednisolone 0.8mg/kg.d
Department of Hematology, Second Medical Center, Chinese PLA General Hospital; Chinese PLA General Hospital, Hospital research projects
paroxysmal nocturnal hemoglobinuria
 
 
NCT04888507: Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy

Completed
2
6
Europe
Pozelimab, REGN3918, Cemdisiran, ALN-CC5
Regeneron Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
05/22
05/23
NCT03333486: Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

Active, not recruiting
2
31
US
Cyclophosphamide, (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, Ciclofosfamida, Ciclofosfamide, Cicloxal, Clafen, Claphene, CP monohydrate, CTX, CYCLO-cell, Cycloblastin, Cycloblastine, Cyclophospham, Cyclophosphamid monohydrate, Cyclophosphamidum, Cyclophosphan, Cyclophosphane, Cyclophosphanum, Cyclostin, Cyclostine, Cytophosphan, Cytophosphane, Cytoxan, Fosfaseron, Genoxal, Genuxal, Ledoxina, Mitoxan, Neosar, Revimmune, Syklofosfamid, WR- 138719, Fludarabine Phosphate, 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, Fludara, SH T 586, Laboratory Biomarker Analysis, Peripheral Blood Stem Cell Transplantation, PBPC transplantation, PBSCT, Peripheral Blood Progenitor Cell Transplantation, peripheral stem cell support, Peripheral Stem Cell Transplant, peripheral stem cell transplantation, Total-Body Irradiation, TOTAL BODY IRRADIATION, Whole-Body Irradiation
Roswell Park Cancer Institute
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Aplastic Anemia, B-Cell Non-Hodgkin Lymphoma, CD40 Ligand Deficiency, Chronic Granulomatous Disease, Chronic Leukemia in Remission, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Congenital Amegakaryocytic Thrombocytopenia, Congenital Neutropenia, Congenital Pure Red Cell Aplasia, Glanzmann Thrombasthenia, Immunodeficiency Syndrome, Myelodysplastic Syndrome, Myelofibrosis, Myeloproliferative Neoplasm, Paroxysmal Nocturnal Hemoglobinuria, Plasma Cell Myeloma, Polycythemia Vera, Recurrent Non-Hodgkin Lymphoma, Refractory Non-Hodgkin Lymphoma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndrome, Severe Aplastic Anemia, Shwachman-Diamond Syndrome, Sickle Cell Disease, T-Cell Non-Hodgkin Lymphoma, Thalassemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome
08/23
08/24
NCT04811716: Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy

Completed
2
24
Europe, RoW
Pozelimab, REGN3918, Cemdisiran, ALN-CC5
Regeneron Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
10/22
10/23
2021-006776-17: REDEEM - access to BCX9930

Ongoing
2
200
Europe
BCX9930, BCX9930, Tablet
BioCryst Pharmaceuticals Inc, BioCryst Pharmaceuticals Inc
Paroxysmal Nocturnal Hemoglobinuria, PNH, Diseases [C] - Immune System Diseases [C20]
 
 
NCT04170023: Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy

Terminated
2
29
Europe, Canada, RoW
ALXN2050, ACH-0145228
Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
04/23
03/24
NCT06050226: A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Recruiting
2
40
RoW
MY008211A tablets, MY008211A
Wuhan Createrna Science and Technology Co., Ltd
Paroxysmal Nocturnal Hemoglobinuria
01/24
07/24
HRS-5965-203, NCT06238544: Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria

Recruiting
2
24
RoW
HRS-5965 tablets
Chengdu Suncadia Medicine Co., Ltd.
Paroxysmal Nocturnal Hemoglobinuria
12/27
12/27
NCT06298955: Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria

Recruiting
2
25
Europe, RoW
OMS906 study drug
Omeros Corporation
Paroxysmal Nocturnal Hemoglobinuria
12/26
04/27
REDEEM-2, NCT05116787 / 2020-004403-14: BCX9930 for the Treatment of PNH in Subjects Not Receiving Other Complement Inhibitor Therapy

Terminated
2
12
RoW
BCX9930 monotherapy, Placebo
BioCryst Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria (PNH)
09/23
09/23
NCT04901936 / 2020-001350-21: A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Recruiting
2
12
Europe, US, RoW
Pegcetacoplan
Apellis Pharmaceuticals, Inc., Apellis Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria
04/24
10/24
CONSENTII, NCT03427060: Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms

Enrolling by invitation
2
6
US
Coversin
AKARI Therapeutics
Paroxysmal Nocturnal Hemoglobinuria (PNH)
06/24
06/24
NCT06412497: MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

Recruiting
2
60
US
Rituximab, Rabbit ATG, Thymoglobulin, Cyclophosphamide, Fludarabine, Total Body Irradiation, TBI, Cell Infusion, Post-Transplant G-CSF, Filgrastim, Tacrolimus, Mycophenolate Mofetil, MMF
Masonic Cancer Center, University of Minnesota
Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia, Acquired Pure Red Cell Aplasia, Paroxysmal Nocturnal Hemoglobinuria
05/35
05/36
HSK39297-201, NCT06561841: A Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）

Recruiting
2
45
RoW
HSK39297
Haisco Pharmaceutical Group Co., Ltd.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
02/25
03/25
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
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
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
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
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
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
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
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 Participants With Paroxysmal Nocturnal Hemoglobinuria

Active, not recruiting
2
28
Europe, RoW
BCX9930
BioCryst Pharmaceuticals, BioCryst Pharmaceuticals Inc
Paroxysmal Nocturnal Hemoglobinuria
02/26
02/26
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
COMPOSER, NCT03157635 / 2016-002128-10: Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria

Active, not recruiting
1/2
59
Europe, Japan, RoW
Crovalimab, Placebo
Hoffmann-La Roche, Chugai Pharmaceutical
Paroxysmal Hemoglobinuria, Nocturnal
01/26
01/26

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