NCT04432584
A Study Evaluating The Safety, Pharmacokinetics, and Efficacy Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors treatment 3 recruiting NCT04747613
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria treatment 3 recruiting NCT06449001
Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis treatment 3 not_yet_recruiting NCT05131204
Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria treatment 3 terminated NCT05274633
Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria Not Available Not Available active_not_recruiting NCT06606314
Specified Drug-use Surveillance of Fabhalta Capsules No drug interventions Not Available Not Available not_yet_recruiting NCT05630001
Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. treatment 3 active_not_recruiting NCT05744921
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works. treatment 3 recruiting NCT06593938
A Study to Evaluate the Efficacy and Safety of Oral HRS-5965 in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy treatment 3 not_yet_recruiting NCT04679103
A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation) treatment 3 completed NCT04060264
Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria treatment 3 completed NCT05476887
To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 treatment 2 recruiting NCT03181633
A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) treatment 2 completed NCT01412047
Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies Study No drug interventions Not Available Not Available completed 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. treatment 2 recruiting NCT00731328
Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome No drug interventions treatment 2 completed NCT04463056
Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH treatment 3 completed NCT05828485
Effect of Food on Pharmacokinetics of MY008211A Tablets in Healthy Adult Subjects No drug interventions treatment 1 completed NCT03896152
Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria treatment 2 completed NCT00130000
Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria treatment 3 completed NCT06543459
To Evaluate the Effect of Single Oral Dose of MY008211A Tablets on QTc Interval in Healthy Subjects No drug interventions treatment 1 not_yet_recruiting NCT02179359
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies No drug interventions treatment Not Available recruiting NCT05889299
Study of the Safety and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria treatment 1 recruiting NCT03500549
Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) treatment 3 completed NCT00397813
Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders treatment 2 completed NCT01272817
Nonmyeloablative Allogeneic Transplant No drug interventions treatment Not Available completed NCT00122317
Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) treatment 3 completed NCT01760096
Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013) treatment 2 unknown_status NCT03157635
Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria treatment 1 / 2 active_not_recruiting NCT01364363
Unrelated Donor Stem Cell Transplantation No drug interventions treatment Not Available completed NCT05842486
Hematological Response in Patients With Paroxysmal Nocturnal Hemoglobinuria Treated With Anti-C5 Antibody: an External Control Arm Study for Iptacopan Use in Anti-C5 naïve Patients No drug interventions Not Available Not Available completed NCT03333486
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer treatment 2 active_not_recruiting NCT06051357
Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria No drug interventions treatment 2 active_not_recruiting NCT04079257
The Pharmacokinetics and Pharmacodynamics of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria No drug interventions Not Available Not Available completed NCT06411626
Home Reported Outcomes in PNH No drug interventions Not Available Not Available recruiting NCT06524726
The International PNH Interest Group PNH Registry No drug interventions Not Available Not Available recruiting NCT06050226
A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) No drug interventions treatment 2 recruiting NCT05133531
A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment treatment 3 recruiting NCT01529827
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies treatment 2 completed NCT01374360
Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry No drug interventions Not Available Not Available recruiting NCT06028594
Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Not Available Not Available available NCT04434092
A Phase III Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. treatment 3 active_not_recruiting NCT06238544
Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria No drug interventions treatment 2 recruiting NCT05886244
Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China treatment 3 active_not_recruiting NCT01194804
E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients treatment 2 completed NCT04654468
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition treatment 3 active_not_recruiting NCT06154512
A Real-world, Multi-center, Prospective, Observational Study for PNH in China No drug interventions Not Available Not Available recruiting NCT04671810
Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® Not Available Not Available completed NCT03866681
Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH treatment 4 unknown_status NCT06326814
A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults No drug interventions treatment 1 completed NCT06298955
Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria treatment 2 recruiting NCT03748823
Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab treatment 3 completed NCT04128943
Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria No drug interventions Not Available Not Available completed NCT00004464
Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria treatment 2 completed NCT04320602
Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab treatment 4 completed NCT01642979
Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria treatment 2 unknown_status NCT02588833
Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects. treatment 1 completed NCT04965597
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) treatment 2 recruiting NCT02605993
Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) treatment 2 completed NCT00856388
Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders treatment Not Available completed NCT03406507
A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria treatment 3 completed NCT04888507
Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy treatment 2 completed NCT03818607
A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH treatment 3 completed NCT05642585
A Study of Single-dose MY008211A in Healthy Adults No drug interventions treatment 1 completed NCT06100900
Dose Escalation of BCX10013 in Subjects With Paroxysmal Nocturnal Hemoglobinuria treatment 1 recruiting NCT00143559
Stem Cell Transplantation as Immunotherapy for Hematologic Malignancies No drug interventions treatment 2 completed NCT04162470
REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability. treatment 3 terminated NCT01192399
Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients treatment 2 completed NCT03829449
rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study treatment 3 terminated NCT06578949
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) treatment 3 not_yet_recruiting NCT05389449
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH treatment 3 active_not_recruiting NCT05490017
To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104 treatment 1 completed NCT04330534
First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH No drug interventions treatment 1 / 2 completed NCT05646524
Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy No drug interventions treatment 2 not_yet_recruiting NCT04811716
Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy treatment 2 completed NCT04058158
A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria treatment 3 completed NCT05646563
Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris treatment 2 not_yet_recruiting NCT04469465
Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA) treatment 3 completed NCT03329365
Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS No drug interventions Not Available Not Available active_not_recruiting NCT00012376
Chemotherapy Plus Sargramostim in Treating Patients With Refractory Myeloid Cancer treatment 1 completed NCT01294891
Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease No drug interventions Not Available Not Available completed NCT05755867
Global PNH Patient Registry No drug interventions Not Available Not Available recruiting NCT05972967
Safety and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria Patients With a Sub-optimal Response to Ravulizumab treatment 2 recruiting NCT04085601
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH treatment 3 completed NCT05776472
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Available Not Available recruiting NCT05828472
A Study of Multiple Ascending Doses MY008211A in Healthy Adults No drug interventions treatment 1 completed NCT04702568
A Long Term Safety Study of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) No drug interventions treatment 2 terminated NCT03047746
Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF No drug interventions treatment Not Available recruiting NCT05741346
Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria No drug interventions treatment 2 active_not_recruiting NCT05982938
Danicopan Early Access Program Not Available Not Available available NCT04558918
Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment treatment 3 completed NCT03053102
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) treatment 2 completed NCT00004143
Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes treatment 2 completed NCT03225287
Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study treatment 2 terminated NCT05116787
BCX9930 for the Treatment of PNH in Subjects Not Receiving Other Complement Inhibitor Therapy No drug interventions treatment 2 terminated NCT02352493
A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH No drug interventions treatment 1 / 2 completed NCT03472885
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab treatment 2 completed NCT04901936
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) treatment 2 recruiting NCT00145613
Haploidentical Stem Cell Transplant for Treatment Refractory Hematological Malignancies No drug interventions treatment 2 completed NCT06561841
A Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH） No drug interventions treatment 2 recruiting NCT04820530
Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy treatment 3 completed NCT03588026
Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576 No drug interventions treatment 3 completed NCT01335165
Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) No drug interventions treatment 1 terminated NCT06312644
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy Not Available Not Available recruiting NCT06294301
A Study of Single and Multiple Doses of LP-005 in Healthy Adult Participants No drug interventions treatment 1 recruiting NCT05876312
Safety, Tolerability, PK and PD of ADX-038 in HV and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients No drug interventions treatment 1 recruiting NCT06134414
Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) No drug interventions treatment 2 not_yet_recruiting NCT00587054
Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic Disorders No drug interventions treatment 2 completed 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 treatment 2 recruiting NCT03030183
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab treatment 2 completed NCT02534909
Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria treatment 2 completed NCT00566696
Mismatched Family Member Donor Transplantation for Children and Young Adults With High Risk Hematological Malignancies treatment 2 completed NCT02946463
ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) treatment 3 completed NCT02264639
A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH treatment 1 completed NCT03946748
Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) treatment 2 completed NCT05116774
BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy treatment 2 terminated NCT03078582
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients treatment 2 completed NCT03427060
Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms treatment 2 enrolling_by_invitation NCT03056040
ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab treatment 3 completed NCT05222412
Managed Access Programs for LNP023, Iptacopan Not Available Not Available available NCT04170023
Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy treatment 2 terminated NCT00186823
Haploidentical Stem Cell Transplantation for Patients With Hematologic Malignancies No drug interventions treatment 3 completed