Clinical trials: PNH
Paroxysmal Nocturnal Hemoglobinuria
First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH
This is a 3-part Phase 1 dose-ranging study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single (Part 1) and multiple (Part 2) ascending doses of BCX9930 in healthy subjects and in subjects with…
- Trial name
- NCT04330534
- Testing
- BCX9930
- Where
- 4 sites in Austria, South Africa and 1 more
- Volunteers
- 168 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
A Study of Single-dose MY008211A in Healthy Adults
The trial is the first human trial. The safety, tolerability, PK and PD of MY008211A Tablets will be evaluated in healthy subjects.
- Trial name
- NCT05642585
- Testing
- MY008211A tablets
- Where
- China
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- Wuhan Createrna Science and Technology Co., Ltd
PNH
A Phase III Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy
The purpose of this study is to evaluate the efficacy and safety of HSK39297 tablets compared to eculizumab in Patients with PNH who Are Naive to Complement Inhibitor Therapy.
- Trial name
- NCT06799546
- Testing
- HSK39297 tablets, Eculizumab Injection
- Where
- 2 sites in China
- Volunteers
- 73 people
- Sponsor
- Haisco Pharmaceutical Group 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
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)
Study on Mass Balance of [14C]HSK39297 in Healthy Chinese Adult Male Subjects
To evaluate the absorption, metabolism and excretion in healthy Chinese male subjects after a single oral dose of [14C]HSK39297
- Trial name
- NCT06852092
- Testing
- Carbon-14 labeled HSK39297
- Where
- China
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Haisco Pharmaceutical Group Co., Ltd.
Paroxysmal Nocturnal Haemoglobinuria (PNH)
Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy
The main purpose of this study is to evaluate the efficacy of MY008211A compared to eculizumab in PNH patients.
- Trial name
- NCT06932744
- Testing
- MY008211A tablets, Eculizumab Injection
- Where
- 2 sites in China
- Volunteers
- 66 people
- Sponsor
- Wuhan Createrna Science and Technology Co., Ltd
Paroxysmal Nocturnal Hemoglobinuria
A Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients
NTQ5082 capsule is a small molecule CFB factor inhibitor. This study is a multicenter, randomized, open label phase II clinical trial aimed at evaluating the efficacy, safety, and PK/PD characteristics of NTQ5082 capsules in the…
- Trial name
- NCT06764303
- Testing
- NTQ5082 100mg qd, NTQ5082 200mg qd
- Where
- 2 sites in China
- Volunteers
- 24 people
- Sponsor
- Nanjing Chia-tai Tianqing Pharmaceutical
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
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
Hemoglobinuria, Paroxysmal
Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria
This study will examine the safety and effectiveness of the experimental drug eculizumab in treating patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare disorder of red blood cells that leads to premature destruction…
- Trial name
- NCT00098280
- Testing
- Eculizumab
- Where
- United States
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
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
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
Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, single-arm, open-label study. Patients with Paroxysmal Nocturnal Hemoglobinuria who had previously received and completed the HRS-5965 study well included.
- Trial name
- NCT06238544
- Testing
- HRS-5965 tablets, HRS-5965 Capsules
- Where
- 2 sites in China
- Volunteers
- 132 people
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.
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 Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition
This study will enroll participants aged 12 years or older with a body weight ≥ 40 kilograms (kg) diagnosed with PNH who have not been previously treated with complement inhibitor therapy.
- Trial name
- COMMODORE 3 (NCT04654468)
- Testing
- Crovalimab
- Where
- 5 sites in China
- Volunteers
- 51 people
- Sponsor
- Hoffmann-La Roche
Paroxysmal Nocturnal Hemoglobinuria (PNH)
ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
The primary purpose of this study was to assess the noninferiority of ravulizumab compared to eculizumab in adult participants with PNH who were clinically stable after having been treated with eculizumab for at least 6 months.
- Trial name
- NCT03056040
- Testing
- Ravulizumab, Eculizumab
- Where
- 51 sites in United States, Australia and 9 more
- Volunteers
- 202 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH.
An open label study to evaluate the efficacy and safety of FXS6837 capsules compared to eculizumab in naive PNH patients. About 90 PNH patients who are naive to complement inhibitor therapies will be enrolled to take FXS6837…
- Trial name
- NCT07747090
- Testing
- FXS6837 Capsule, Eculizumab infusion
- Where
- China
- Volunteers
- 90 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Danicopan Early Access Program
This is an EAP, designed to provide early access to danicopan for participants with PNH experiencing clinically significant EVH who have failed, not tolerated, or are unable to receive other approved treatments; in the Treating…
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis
- Trial name
- NCT05982938
- Testing
- Danicopan
- Where
- 15 sites in Italy
- Sponsor
- Alexion Pharmaceuticals, Inc.