Clinical trials: Paroxysmal Nocturnal Hemoglobinuria, PNH, open to volunteers
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.
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 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 Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
This is an single arm, open label designed phase III study to evaluate the efficacy and safety of oral FXS6837 capsules in PNH patients with residual anemia despite the stable treatment with anti-C5 antibody.
- Trial name
- NCT07914894
- Testing
- FXS6837 Capsule
- Where
- 2 sites in China
- Volunteers
- 20 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Paroxysmal Nocturnal Hemoglobinuria
Global PNH Patient Registry
The primary aim of the Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry is to conduct a prospectively-planned and efficient natural history study that will result in a more comprehensive understanding of the…
- Trial name
- NCT05755867
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Aplastic Anemia and MDS International Foundation
Paroxysmal Nocturnal Hemoglobinuria
A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently
This study is researching a treatment combination with two experimental drugs called pozelimab and cemdisiran referred to as "study drugs".
- Trial name
- NCT07154745
- Testing
- Pozelimab, Cemdisiran
- Where
- 20 sites in Brazil, Canada and 5 more
- Volunteers
- 35 people
- Sponsor
- Regeneron Pharmaceuticals
Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia
MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.
A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal…
- Conditions
- Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia, Acquired Pure Red Cell Aplasia, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT06412497
- Testing
- Rituximab, Rabbit ATG, Cyclophosphamide
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Paroxysmal Nocturnal Hemoglobinuria, PNH
Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal…
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis
- Trial name
- NCT06449001
- Testing
- Danicopan
- Where
- 4 sites in Canada, France and 1 more
- Volunteers
- 6 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment
The study is being conducted to compare the pharmacokinetics, safety, and pharmacodynamics of HSK39297 in subjects with mild to moderate hepatic impairment and normal hepatic function
- Trial name
- NCT07152288
- Testing
- HSK39297 tablets
- Where
- China
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Haisco Pharmaceutical Group Co., Ltd.
Inherited Bone Marrow Failure Syndrome, Fanconi Anemia
Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data
A non-profit, translational, prospective and retrospective study. Patients will be enrolled according to the procedures described in the protocol, and samples will be processed and analyzed as specified in the protocol.
- Conditions
- Inherited Bone Marrow Failure Syndrome, Fanconi Anemia, Dyskeratosis Congenita, Telomere Biology Disorder
- Trial name
- 2023-12377081 (NCT07877727)
- Where
- 4 sites in Italy
- Volunteers
- 250 people
- Sponsor
- Istituto Giannina Gaslini
PNH - Paroxysmal Nocturnal Hemoglobinuria
Study of SGB-9768 in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, randomized, open-Label Phase II study to evaluate the efficacy and safety of multiple doses of SGB-9768 in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- NCT07796256
- Testing
- SGB-9768
- Where
- 2 sites in China
- Volunteers
- 24 people
- Sponsor
- Suzhou Sanegene Bio Inc.
Multiple Myeloma, Acute Myeloid Leukemia
National Longitudinal Cohort of Hematological Diseases
Background Hematological diseases are disorders of the blood and hematopoietic organs. The current hematological cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China.
- Conditions
- Multiple Myeloma, Acute Myeloid Leukemia, Hemophilia, Hemophilia A
- Trial name
- NICHE (NCT04645199)
- Where
- China
- Volunteers
- 2,300 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Paroxysmal Nocturnal Hemoglobinuria
Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris
This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 in PNH patients undergoing complement-inhibitor therapy with Soliris.
- Trial name
- NCT05646563
- Testing
- NM8074, Soliris
- Volunteers
- 12 people
- Sponsor
- NovelMed Therapeutics
Paroxysmal Nocturnal Hemoglobinuria, PNH, Complement-mediated Kidney Disease
A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants
This study is a first-in-human (FIH) trial of CMS-D017 conducted in healthy Chinese adult participants, consisting of two parts: Part 1-a single ascending dose (SAD) study (referred to as Part 1 SAD), and Part 2-a multiple…
- Trial name
- NCT07462780
- Testing
- CMS-D017 Capsule
- Where
- China
- Volunteers
- 88 people, healthy volunteers welcome
- Sponsor
- Shenzhen Kangzhe Biotechnology Co., Ltd.
Paroxysmal Nocturnal Hemoglobinuria
A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China
The implementation of new standards for the management of PNH and the use of iptacopan in patients with PNH are expected to change the treatment landscape and improve the overall prognosis of patients.
- Trial name
- PNH-NIS (NCT06931691)
- Testing
- LNP023
- Where
- 14 sites in China
- Volunteers
- 80 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria, PNH
Efficacy and Safety of Lanoracopan in the Treatment of PNH
This study aimed to evaluate the efficacy and safety of Lanoracopan Hydrochloride in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Trial name
- NCT07788105
- Testing
- Lanoracopan Hydrochloride
- Volunteers
- 90 people
- Sponsor
- Peking Union Medical College Hospital
Bone Marrow Failure Disorders, VEXAS Syndrome
Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study
STUDY DESCRIPTION: Bone marrow failure can result from immune mediated attack on stem cells or from inherited genetic defects such as telomere biology disorders or Fanconi anemia.
- Conditions
- Bone Marrow Failure Disorders, VEXAS Syndrome, Hemoglobinurea, Paroxysmal, Myelodysplastic Syndromes
- Trial name
- NCT07102849
- Where
- United States
- Volunteers
- 1,400 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Paroxysmal Nocturnal Hemoglobinuria
Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is an extension study for patients who have completed a prior P10-LP005-02 clinical study. The aim of this study is to evaluate the long-term safety, efficacy, and pharmacokinetics of LP-005 injection in adult patients with…
- Trial name
- NCT07266155
- Testing
- LP-005 Injection
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Longbio Pharma
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Background: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems.
- Conditions
- Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS), Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Trial name
- NCT03520647
- Testing
- Cyclophosphamide, Peripheral Blood Stem Cells
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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.