Clinical trials: PNH - Paroxysmal Nocturnal Hemoglobinuria
Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML)
Haploidentical Stem Cell Transplant for Treatment Refractory Hematological Malignancies
Relapsed disease is the most common cause of death in children with hematological malignancies. Patients who fail high-intensity conventional chemotherapeutic regimens or relapse after stem cell transplantation have a poor…
- Conditions
- Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML), Secondary AML, Myelodysplastic Syndrome (MDS)
- Trial name
- NCT00145613
- Testing
- Stem Cell Transplantation, Miltenyi Biotec CliniMACS, Systemic chemotherapy and antibodies
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- St. Jude Children's Research Hospital
Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria
Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF
This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell…
- Conditions
- Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Inherited Bone Marrow Failure Syndromes
- Trial name
- NCT03047746
- Testing
- CliniMACs
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Children's Hospital of Philadelphia
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
Paroxysmal Nocturnal Hemoglobinuria
Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy
This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 administered intravenously to adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
- Trial name
- NCT05646524
- Testing
- NM8074
- Volunteers
- 12 people
- Sponsor
- NovelMed Therapeutics
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
The International PNH Interest Group PNH Registry
The aim of this International PNH Interest Group (IPIG) registry is to develop an international database to prospectively collect data on patients with PNH covering clinical outcomes, patient reported outcomes (PROs), and…
- Trial name
- NCT06524726
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- International PNH Interest Group
Paroxysmal Nocturnal Hemoglobinuria, PNH
To Evaluate Safety, Tolerability, PK/PD & Food Effect of Single/Multiple Oral TUL321 Capsules in Healthy Participants.
Phase I Study Evaluating Safety, Tolerability, PK and PD of Single and Multiple Doses of TUL321 Capsule in Healthy Participants and Food Effect on PK
- Trial name
- NCT07760467
- Testing
- Part1: TUL321, Part2: TUL321
- Where
- China
- Volunteers
- 94 people, healthy volunteers welcome
- Sponsor
- Zhuhai United Laboratories Co., Ltd.
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This phase II trial studies how well giving fludarabine phosphate, melphalan, and low-dose total-body irradiation (TBI) followed by donor peripheral blood stem cell transplant (PBSCT) works in treating patients with hematologic…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01529827
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- Roswell Park Cancer Institute
Paroxysmal Nocturnal Hemoglobinuria, C3 Glomerulopathy
A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy
This is a post-marketing surveillance study conducted as part of the Risk Management Plan (RMP) for South Korea, to evaluate the safety and effectiveness of iptacopan in real-world clinical settings for the treatment of either…
- Trial name
- NCT07416162
- Where
- 3 sites in South Korea
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
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，IgA Nephropathy
Comparison of HRS-5965 Capsules and Tablets in Healthy Subjects
The study is designed as a single-center, randomized, open-label, cross-over phase I clinical trial with the intention of recruiting 16 healthy subjects.
- Trial name
- NCT06490991
- Testing
- HRS-5965 tablets；HRS-5965 capsules
- Where
- China
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.
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 (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)
Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment
This study was a multi-center, randomized, open-label, active comparator-controlled, parallel group study. The purpose of this Phase 3 study in PNH patients presenting with residual anemia despite treatment with anti-C5 antibody…
- Trial name
- APPLY-PNH (NCT04558918)
- Testing
- LNP023, Eculizumab, Ravulizumab
- Where
- 39 sites in United States, Brazil and 10 more
- Volunteers
- 97 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Haemoglobinuria
Real-life Treatment Outcomes of Ravulizumab in PNH
The PNH-RECORD study, a Polish multicenter observational (non-interventional), open-label, retrospective with prospective follow-up.
- Trial name
- PNH-RECORD (NCT06633536)
- Testing
- Ravulizumab
- Where
- 11 sites in Poland
- Volunteers
- 64 people
- Sponsor
- AstraZeneca
IgA Nephropathy, Paroxysmal Nocturnal Hemoglobinuria
A Clinical Study to Evaluate the Effect of Food on the Pharmacokinetic Profiles of HRS-5965 Capsules in Healthy Subjects
The study is being conducted to evaluate the effect of food on pharmacokinetics of HRS-5965 capsules after oral administration.
- Trial name
- NCT07035665
- Testing
- HRS-5965 Capsules
- Where
- China
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.
Allogeneic Stem Cell Transplant, Leukemia
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
This is a phase II, single-center study to evaluate the efficacy of a novel cytoreductive regimen followed by CD34+E- selected T cell depleted allogeneic stem cell (or soybean agglutinated and E-rosetted BM) transplant as…
- Conditions
- Allogeneic Stem Cell Transplant, Leukemia, Non-Hodgkins, Lymphoblastic Lymphoma
- Trial name
- NCT00587054
- Testing
- cytoreductive regimen followed by a CD34+E- selected allogeneic stem cell transplant
- Where
- United States
- Volunteers
- 129 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
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
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.
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH
This is a randomized, double-blind, active-controlled phase 3 study of ABP 959 in participants with paroxysmal nocturnal hemoglobinuria.
- Trial name
- DAHLIA (NCT03818607)
- Testing
- ABP 959, Eculizumab
- Where
- 24 sites in United States, Czechia and 12 more
- Volunteers
- 42 people
- Sponsor
- Amgen