Clinical trials: Hemoglobinuria, Paroxysmal Nocturnal (PNH)
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.
Hemoglobinuria, Paroxysmal
Multicenter Retrospective Analysis About the Clinical Characteristics of Korean Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
Paroxysmal nocturnal hemoglobinuria (PNH) is a hematopoietic stem cell disorder in which unregulated activation of the complement system leads to significant ischemic morbidities with shortened lifespan.
- Trial name
- NCT01224483
- Where
- South Korea
- Volunteers
- 350 people
- Sponsor
- Samsung Medical Center
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)
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.
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
To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104
The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in healthy volunteers.
- Trial name
- NCT05490017
- Testing
- KP104
- Where
- Australia
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Kira Pharmacenticals (US), LLC.
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.
Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with…
- Conditions
- Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia
- Trial name
- NCT04965597
- Testing
- Treosulfan, Fludarabine Phosphate, Tacrolimus
- Where
- 24 sites in United States
- Volunteers
- 40 people
- Sponsor
- Fred Hutchinson Cancer Center
Paroxysmal Nocturnal Hemoglobinuria
Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH
Classical paroxysmal nocturnal hemoglobinuria(PNH) is mainly characterized by hemolysis and thrombosis, which reduced patients ' quality of life(QoL) greatly and even lead to death.
- Trial name
- NCT03866681
- Testing
- sirolimus
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Peking Union Medical College Hospital
Paroxysmal Nocturnal Hemoglobinuria
Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, randomized, open-label phase II clinical trial. A total of 24 patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included.
- Trial name
- NCT06051357
- Testing
- HRS-5965 tablets
- Where
- 2 sites in China
- Volunteers
- 26 people
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.
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.
Paroxysmal Nocturnal Hemoglobinuria
Safety and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria Patients With a Sub-optimal Response to Ravulizumab
The purpose of this study is to assess the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of OMS906 for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in patients who have a…
- Trial name
- NCT05972967
- Testing
- OMS906
- Where
- 5 sites in Germany, Greece and 2 more
- Volunteers
- 12 people
- Sponsor
- Omeros Corporation
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
Leukemia, Acute Lymphocytic (ALL), Leukemia, Myeloid, Acute(AML)
Haploidentical Stem Cell Transplantation for Patients With Hematologic Malignancies
Blood and marrow stem cell transplant has improved the outcome for patients with high-risk hematologic malignancies. However, most patients do not have an appropriate HLA (immune type) matched sibling donor available and/or are…
- Conditions
- Leukemia, Acute Lymphocytic (ALL), Leukemia, Myeloid, Acute(AML), Leukemia, Myeloid, Chronic(CML), Juvenile Myelomonocytic Leukemia(JMML)
- Trial name
- NCT00186823
- Testing
- Miltenyi Biotec CliniMACS, Stem Cell Transplantation, TBI, systemic chemotherapy and antibodies as follows:
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- St. Jude Children's Research Hospital
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)
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The purpose of this study was to determine the safety and efficacy of ACH-0144471 (also known as danicopan and ALXN2040) in currently untreated participants with PNH.
- Trial name
- NCT03053102
- Testing
- Danicopan
- Where
- 5 sites in Italy, New Zealand and 2 more
- Volunteers
- 10 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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