Clinical trials: PNH
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
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
Preeclampsia, Severe Preeclampsia
Complement Regulation to Undo Systemic Harm in Preeclampsia
This is a Phase II, single arm, open-label study to determine if treatment with eculizumab prolongs pregnancy compared to historical controls in women with preeclampsia between 23-30 weeks gestation.
- Conditions
- Preeclampsia, Severe Preeclampsia, Eculizumab, HELLP
- Trial name
- CRUSH (NCT04725812)
- Testing
- Eculizumab
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Cedars-Sinai Medical Center
Paroxysmal Nocturnal Hemoglobinuria
Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab
The primary purpose of this study is to assess the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab in participants who are prescribed and are receiving a higher than approved dose of eculizumab to treat…
- Trial name
- NCT04320602
- Testing
- Eculizumab, Ravulizumab
- Where
- 4 sites in United Kingdom
- Volunteers
- 18 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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
Bone Marrow Failure Syndromes, Anemia, Aplastic
Identifying Characteristics of Bone Marrow Failure Syndromes
Bone marrow failure syndromes (BMFS) are rare disorders characterized by dysfunctional hematopoietic stem cells, which give rise to all red and white blood cells.
- Conditions
- Bone Marrow Failure Syndromes, Anemia, Aplastic, Myelodysplastic Syndromes, Hemoglobinuria, Paroxysmal
- Trial name
- NCT00315419
- Where
- 4 sites in United States
- Volunteers
- 450 people
- Sponsor
- Office of Rare Diseases (ORD)
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
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.
- Conditions
- Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT02179359
- Testing
- Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
- Where
- United States
- Volunteers
- 38 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.
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
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
Hemoglobinuria, Paroxysmal
Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary objective of this study was to evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) parameter estimates of eculizumab to confirm the dose regimens for pediatric and adolescent participants with PNH.
- Trial name
- NCT00867932
- Testing
- Eculizumab
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Alexion Pharmaceuticals, 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
Dyskeratosis Congenita, Fanconi Anemia
Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment
RATIONALE: Studying biopsy, bone marrow, and blood samples from patients with cytopenia that did not respond to treatment may help doctors learn more about the disease and plan the best treatment.
- Conditions
- Dyskeratosis Congenita, Fanconi Anemia, Myelodysplastic Syndromes, Pearson Marrow-pancreas Syndrome
- Trial name
- NCT00499070
- Testing
- polymerase chain reaction, flow cytometry, immunologic technique
- Where
- 10 sites in Austria, Belgium and 8 more
- Volunteers
- 119 people
- Sponsor
- University Hospital Freiburg
Paroxysmal Nocturnal Hemoglobinuria
A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria
The purpose of this study was to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and efficacy of ravulizumab in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- NCT03406507
- Testing
- Ravulizumab
- Where
- 9 sites in United States, France and 4 more
- Volunteers
- 13 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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)
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)
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
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
Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
A study to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH.
- Trial name
- NCT01192399
- Testing
- Eculizumab
- Volunteers
- 29 people
- Sponsor
- Alexion Pharmaceuticals, Inc.