Clinical trials: Paroxysmal Nocturnal Hemoglobinuria (PNH)
Extrahepatic Portal Vein Obstruction (EHPVO), Thrombophilia
Hematological Disorders in EHPVO Patients
This study focuses on patients who have a condition called extrahepatic portal vein obstruction (EHPVO), where a blood clot blocks the portal vein outside the liver.
- Conditions
- Extrahepatic Portal Vein Obstruction (EHPVO), Thrombophilia, Myeloproliferative Neoplasms (MPN), Antiphospholipid Syndrome (APS)
- Trial name
- EHPVO-HEM (NCT07108023)
- Testing
- No intervention (Observational Study)
- Volunteers
- 115 people
- Sponsor
- Rahab Nady
Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome
Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders
This clinical trial is studying how well giving fludarabine phosphate and melphalan together with total-body irradiation followed by donor stem cell transplant works in treating patients with hematologic cancer or bone marrow…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00856388
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 62 people
- Sponsor
- Roswell Park Cancer Institute
Paroxysmal Nocturnal Hemoglobinuria
Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
This program is designed to provide access to pozelimab and cemdisiran and document the long-term safety of pozelimab and cemdisiran combination therapy in patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- NCT06028594
- Testing
- Pozelimab, Cemdisiran
- Sponsor
- Regeneron Pharmaceuticals
PNH
Pegcetacoplan Long Term Safety and Efficacy Extension Study
This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.
- Trial name
- NCT03531255
- Testing
- Pegcetacoplan
- Where
- 62 sites in United States, Australia and 19 more
- Volunteers
- 137 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, open-label study to evaluate the long-term safety, tolerability and efficacy of HSK39297. Adult patients with PNH who had previously received and completed HSK39297 study treatment will be included.
- Trial name
- NCT06745622
- Testing
- HSK39297 tablets
- Where
- China
- Volunteers
- 47 people
- Sponsor
- Haisco Pharmaceutical Group Co., Ltd.
Paroxysmal Hemoglobinuria, Nocturnal
Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria
This is a Phase I/II, first-in-human study consisting of four sequential parts and an open-label extension (OLE). The safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single doses of crovalimab will be…
- Trial name
- COMPOSER (NCT03157635)
- Testing
- Crovalimab
- Where
- 15 sites in France, Germany and 5 more
- Volunteers
- 59 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Paroxysmal Nocturnal Hemoglobinuria
Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria®
This is prospective observational study of long-term pathogenic treatment of Elizaria® in patients with paroxysmal nocturnal hemoglobinuria.
- Trial name
- NAP (NCT04671810)
- Testing
- Elizaria®
- Where
- 8 sites in Russia
- Volunteers
- 44 people
- Sponsor
- AO GENERIUM
Paroxysmal Nocturnal Hemoglobinuria
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed…
- Trial name
- NCT04747613
- Testing
- Iptacopan
- Where
- 60 sites in United States, Brazil and 15 more
- Volunteers
- 208 people
- Sponsor
- Novartis Pharmaceuticals
Leukemia, Myelodysplastic Syndromes
Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome
RATIONALE: Giving low doses of chemotherapy and antithymocyte globulin before a donor stem cell transplant helps stop the growth of abnormal cells.
- Conditions
- Leukemia, Myelodysplastic Syndromes, Nonmalignant Neoplasm, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT00731328
- Testing
- nonmyeloablative allogeneic hematopoietic stem cell transplantation
- Where
- South Korea
- Volunteers
- 15 people
- Sponsor
- Asan Medical Center
Hemoglobinuria, Paroxysmal
Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry
This is an observational single-arm descriptive cohort study based on the secondary use of data collected on iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group…
- Trial name
- NCT06903234
- Testing
- Iptacopan
- Where
- Switzerland
- Volunteers
- 200 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
A Long-term Efficacy and Safety of NTQ5082 Capsules
NTQ5082 capsules are a small molecule CFB inhibitor. This study is a multicenter, open-label study evaluating the long-term efficacy and safety of NTQ5082 capsules in the treatment of patients with paroxysmal nocturnal…
- Trial name
- NCT07177872
- Testing
- NTQ5082 capsules 200 mg
- Where
- China
- Volunteers
- 78 people
- Sponsor
- Nanjing Chia-tai Tianqing Pharmaceutical
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study
The purpose of this study is to enable continued access to zilucoplan (RA101495) for patients with paroxysmal nocturnal hemoglobinuria (PNH) after they complete a zilucoplan clinical study.
- Trial name
- NCT03225287
- Testing
- Zilucoplan (RA101495)
- Where
- 12 sites in United States, Australia and 6 more
- Volunteers
- 19 people
- Sponsor
- Ra Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
A study designed to evaluate the safety of crovalimab with eculizumab in participants with PNH currently treated with complement inhibitors. This study will enroll approximately 190 participants.
- Trial name
- COMMODORE 1 (NCT04432584)
- Testing
- Crovalimab, Eculizumab
- Where
- 80 sites in United States, Belgium and 22 more
- Volunteers
- 190 people
- Sponsor
- Hoffmann-La Roche
Paroxysmal Nocturnal Hemoglobinuria
Study of the Safety and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria
The purpose of this study is to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
- Trial name
- NCT05889299
- Testing
- OMS906
- Where
- Ukraine
- Volunteers
- 15 people
- Sponsor
- Omeros Corporation
Paroxysmal Nocturnal Hemoglobinuria
A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, open-label study to evaluate the long-term safety and efficacy of XH-S003 capsules in PNH patients. About 26 PNH patients who had received and completed XH-S003 study treatment will be enrolled to take…
- Trial name
- XH-S003-II-102 (NCT07657052)
- Testing
- XH-S003 Capsule
- Where
- China
- Volunteers
- 26 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary purpose of this study is to evaluate the safety, tolerability, and efficacy of multiple intravenous (IV) doses of ravulizumab administered to complement inhibitor treatment-naïve participants with PNH.
- Trial name
- NCT02605993
- Testing
- Ravulizumab
- Where
- 16 sites in Canada, France and 5 more
- Volunteers
- 26 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
Efficacy and Safety of HRS-5965 in Patients With PNH Who Are Still Anemia After Anti-C5 Antibody Treatment
This trial is a multi-center, single-arm, open-label phase III clinical trial. A total of approximately 35 patients with paroxysmal nocturnal hemoglobinuria who remained anemic despite stable use of C5 complement inhibitor…
- Trial name
- NCT06715943
- Testing
- HRS-5965 capsule
- Where
- 2 sites in China
- Volunteers
- 39 people
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.
Paroxysmal Nocturnal Hemoglobinuria
Specified Drug-use Surveillance of Fabhalta Capsules
This is a multicenter, single-arm, non-interventional study (NIS) with a central registration system and an all-case surveillance system. The observation period is 48 weeks after the start of treatment with Fabhalta.
- Trial name
- NCT06606314
- Where
- 91 sites in Japan
- Volunteers
- 132 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
The objective of this study was to assess the long-term safety and efficacy of eculizumab in hemolytic PNH patients who completed the 4-week screening and 12-week treatment period of the C07-001 study.
- Trial name
- NCT01194804
- Testing
- Eculizumab
- Volunteers
- 27 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment
The main purpose of this study is to evaluate the efficacy of MY008211A in PNH patients with residual anemia despite treatment with anti-C5 antibody.
- Trial name
- NCT06932471
- Testing
- MY008211A tablets
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Wuhan Createrna Science and Technology Co., Ltd