Clinical trials: PNH, active
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Background: Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia.
- Trial name
- NCT01174108
- Testing
- Miltenyi CD34 Reagent System, Donor derived G-CSF mobilized PBC
- Where
- 2 sites in United States
- Volunteers
- 120 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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 (PNH), Persistent Anemia
A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor
This study is researching an experimental drug called ALN-CFB. The study is focused on people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are currently taking a complement component C5 inhibitor ("C5-inhibitor") and…
- Trial name
- NCT07187401
- Testing
- ALN-CFB
- Where
- 8 sites in Canada, South Korea and 1 more
- Volunteers
- 24 people
- Sponsor
- Regeneron Pharmaceuticals
PNH - Paroxysmal Nocturnal Hemoglobinuria
A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a multicenter, open-label, Phase Ib clinical trial designed to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple doses of the humanized monoclonal antibody EA5 in adult patients with…
- Trial name
- NCT07256301
- Testing
- EA5
- Where
- 2 sites in China
- Volunteers
- 24 people
- Sponsor
- Shanghai Lanyi Therapeutics Co., Ltd.
PNH - Paroxysmal Nocturnal Hemoglobinuria
A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a multicenter, randomized, single-blind Phase II trial to evaluate the efficacy and safety of XH-S003 capsules in PNH patients.
- Trial name
- XH-S003-II-101 (NCT06978699)
- Testing
- XH-S003 Capsule
- Where
- China
- Volunteers
- 24 people
- Sponsor
- S-INFINITY Pharmaceuticals Co., Ltd
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH, showing signs of active hemolysis.
- Trial name
- NCT06134414
- Testing
- MY008211A tablets
- Volunteers
- 40 people
- Sponsor
- Wuhan Createrna Science and Technology Co., Ltd
Ultomiris-exposed Pregnant/ Postpartum, Pregnancy
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of…
- Conditions
- Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS)
- Trial name
- NCT06312644
- Testing
- Ultomiris
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 75 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
The first-in-human Phase 1/Phase 2a study described herein will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-038 in both healthy participants (HP) and in patients with paroxysmal…
- Trial name
- PNH (NCT05876312)
- Testing
- ADX-038
- Where
- 4 sites in Australia, United Kingdom
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- ADARx Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
SLN12140 in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
The goal of this clinical trial is to learn if drug SLN12140 works to treat Complement Inhibitor-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria in adults.
- Trial name
- NCT07387302
- Testing
- SLN12140
- Volunteers
- 10 people
- Sponsor
- Linno Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal…
- Trial name
- NCT04901936
- Testing
- Pegcetacoplan
- Where
- 12 sites in United States, Czechia and 7 more
- Volunteers
- 12 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
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
Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria
This was a phase 1b/2,open label, multi-center study to assess efficacy and safety of HS-10542 in adulte patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis.
- Trial name
- NCT07470762
- Testing
- HS-10542
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Jiangsu Hansoh Pharmaceutical Co., Ltd.
Paroxysmal Nocturnal Hemoglobinuria
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.
- Trial name
- NCT05389449
- Testing
- Danicopan
- Where
- 45 sites in United States, Brazil and 13 more
- Volunteers
- 80 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
PNH - Paroxysmal Nocturnal Hemoglobinuria
Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The proposed study, NM8074-PNH-101, is a phase II, open-label, multi-dose, unicenter trial to evaluate the safety and efficacy of NM8074 in Soliris-treated PNH subjects.
- Trial name
- NCT05731050
- Testing
- NM8074
- Volunteers
- 6 people
- Sponsor
- NovelMed Therapeutics
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders
RATIONALE: Giving chemotherapy and total-body irradiation before a donor peripheral stem cell transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00544115
- Testing
- busulfan, cyclophosphamide, cyclosporine
- Volunteers
- 260 people
- Sponsor
- City of Hope Medical Center
Paroxysmal Nocturnal Hemoglobinuria, PNH
A Multicenter, Randomized, Open-Label, Positive Controlled Phase III Study to Evaluate the Efficacy and Safety of VSA012 Injection in Participants With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy
A study of the efficacy and safety of VSA012 Injection compared to eculizumab for 24 weeks in patients with PNH.
- Trial name
- NCT07816237
- Testing
- VSA012, Eculizumab (Soliris®)
- Where
- 14 sites in China
- Volunteers
- 70 people
- Sponsor
- Bisirna Therapeutics (Suzhou) Co., Ltd.
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
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 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
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