Clinical trials: Hemoglobinuria, Paroxysmal Nocturnal (PNH)
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.
Hemoglobinuria, Paroxysmal
Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab
The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH
- Trial name
- NCT00122304
- Testing
- eculizumab
- Where
- 48 sites in United States, Australia and 11 more
- Volunteers
- 85 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data
This is a noninterventional registry-based cohort study utilizing data collected on danicopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry.
- Trial name
- NCT07413250
- Testing
- Danicopan, Soliris/Ultomiris
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study
Long term management of patients with complement related diseases including Paroxysmal Nocturnal Haemoglobinuria and Atypical Haemolytic Uraemic Syndrome
- Trial name
- CONSERVE (NCT03829449)
- Testing
- rVA576
- Where
- Poland
- Volunteers
- 15 people
- Sponsor
- AKARI Therapeutics
Paroxysmal Nocturnal Hemoglobinuria
To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104
The purpose of this study is to evaluate the safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in complement inhibitor-naïve participants with PNH.
- Trial name
- NCT05476887
- Testing
- KP104
- Where
- 4 sites in China
- Volunteers
- 35 people
- Sponsor
- Kira Pharmacenticals (US), LLC.
Paroxysmal Nocturnal Hemoglobinuria
Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria
This study was designed to provide continued access to BCX9930 for participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) who had benefited from treatment with BCX9930 in another BioCryst-sponsored study for PNH who, in the…
- Trial name
- NCT05741346
- Testing
- BCX9930
- Where
- 11 sites in France, Hungary and 5 more
- Volunteers
- 28 people
- Sponsor
- BioCryst Pharmaceuticals
C3 Glomerulopathy (C3G), Paroxysmal Nocturnal Hemoglobinuria (PNH)
Managed Access Programs for LNP023, Iptacopan
The purpose of this registration form is to list all Managed Access Programs (MAPs) related to LNP023, Iptacopan
- Trial name
- NCT05222412
- Testing
- Iptacopan
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH
This study will be the initial exploration of pegcetacoplan in patients with PNH. The assessments of the safety, tolerability, PK, and PD following administration of single and multiples doses of pegcetacoplan will guide…
- Trial name
- NCT02264639
- Testing
- Pegcetacoplan
- Where
- 7 sites in United States
- Volunteers
- 9 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Chronic Myelomonocytic Leukemia
Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders
This phase II trial studies the side effects and best dose of total-body irradiation when given together with fludarabine phosphate followed by a donor peripheral stem cell transplant in treating patients with myelodysplastic…
- Conditions
- Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndrome, Essential Thrombocythemia
- Trial name
- NCT00397813
- Testing
- Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- 3 sites in United States
- Volunteers
- 77 people
- Sponsor
- Fred Hutchinson Cancer Center
Paroxysmal Nocturnal Hemoglobinuria (PNH)
BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy
The purpose of this study was to determine the efficacy and safety of BCX9930 monotherapy for the treatment of adult participants with PNH not currently receiving complement inhibitor therapy.
- Trial name
- REDEEM-2 (NCT05116787)
- Testing
- BCX9930 monotherapy
- Where
- 5 sites in Malaysia, South Africa and 1 more
- Volunteers
- 12 people
- Sponsor
- BioCryst Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria
This clinical study is a randomized, open-label, international, multi-center, comparative study of efficacy and safety of BCD-148 and Soliris® in PNH patients.
- Trial name
- NCT04060264
- Testing
- BCD-148, Soliris
- Where
- Russia
- Volunteers
- 28 people
- Sponsor
- Biocad
Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria (PNH), Level of CD59 on Red and White Blood Cells in Bone Marrow Failure Syndromes
Examine red and white blood cells of PNH patients with bone marrow failure syndromes.
- Trial name
- EXPLORE (NCT01192425)
- Volunteers
- 5,580 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary objective of the study is to demonstrate a reduction in intravascular hemolysis by REGN3918 over 26 weeks of treatment in patients with active PNH who are treatment-naive to complement inhibitor therapy or have not…
- Trial name
- NCT03946748
- Testing
- REGN3918
- Where
- 12 sites in Hong Kong, Hungary and 3 more
- Volunteers
- 24 people
- Sponsor
- Regeneron Pharmaceuticals
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
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, single-arm, open-label study to characterize long-term safety and tolerability of MY008211A tablets and to provide access to MY008211A tablets to patients with PNH who have completed Phase 2 or 3 studies…
- Trial name
- NCT06933914
- Testing
- MY008211A tablets
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Wuhan Createrna Science and Technology Co., Ltd
Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Study of Single and Multiple Doses of LP-005 in Healthy Adult Participants
The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of LP-005 in healthy volunteers.
- Trial name
- NCT06294301
- Testing
- LP-005 Dose 1 (Single), LP-005 Dose 2 (Single), LP-005 Dose 3 (Single)
- Where
- China
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- Longbio Pharma
Paroxysmal Nocturnal Hemoglobinuria, PNH
Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies Study
How does long term treatment with Soliris affect HAHA in PNH patients?
- Trial name
- NCT01412047
- Where
- 14 sites in United States, Australia and 8 more
- Volunteers
- 75 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Bone Marrow Failure Syndromes, Anemia, Aplastic
Identifying Characteristics of Bone Marrow Failure Syndromes
Bone marrow failure syndromes (BMFS) are rare disorders characterized by dysfunctional hematopoietic stem cells, which give rise to all red and white blood cells.
- Conditions
- Bone Marrow Failure Syndromes, Anemia, Aplastic, Myelodysplastic Syndromes, Hemoglobinuria, Paroxysmal
- Trial name
- NCT00315419
- Where
- 4 sites in United States
- Volunteers
- 450 people
- Sponsor
- Office of Rare Diseases (ORD)
Paroxysmal Nocturnal Hemoglobinuria, PNH
Long-term Safety of Danicopan: IPIG Registry-based Cohort Study
This is a noninterventional cohort study using primary and secondary data from the International PNH Interest Group (IPIG) PNH registry.
- Trial name
- NCT07413679
- Testing
- Danicopan
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.
The objectives of the study are to assess the safety, tolerability, preliminary efficacy and PK of multiple subcutaneous (SC) doses of pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who have not received…
- Trial name
- PADDOCK (NCT02588833)
- Testing
- Pegcetacoplan
- Where
- 7 sites in Hong Kong, Malaysia and 2 more
- Volunteers
- 23 people
- Sponsor
- Apellis Pharmaceuticals, Inc.