Clinical trials: Congenital Hemolytic Anemia, active
Pyruvate Kinase Deficiency
Pyruvate Kinase Deficiency Global Longitudinal Registry
This study is an observational (ie, noninterventional), longitudinal, multicenter, global registry for patients with pyruvate kinase (PK) deficiency, a rare nonspherocytic hemolytic anemia.
- Trial name
- PEAK Registry (NCT03481738)
- Where
- 52 sites in United States, Canada and 14 more
- Volunteers
- 500 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Congenital Hemolytic Anemia, Sickle Cell Disease
Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
People with severe congenital anemias, such as sickle cell anemia and beta-thalassemia, have been cured with bone marrow transplantation (BMT).
- Trial name
- NCT00061568
- Testing
- Peripheral blood hematopoietic progenitor cell (PBPC) transplant, Alemtuzumab, Peripheral blood hematopoietic progenitor cell Apheresis
- Where
- United States
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency
A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders
Background: Pyrimidine and purine metabolism disorders (DPPMs) affect how the body metabolizes chemicals called pyrimidines and purines.
- Conditions
- AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of
- Trial name
- NCT06092346
- Where
- United States
- Volunteers
- 999 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Sickle Cell Disease, Sickle Cell Anaemia
Efficacy and Safety of SIL-8301 for Control of Hemolysis in a Uniform Sickle Cell Disease Endotype
SIL-8301 (senicapoc) is being developed for the chronic treatment of patients with sickle cell disease in both adults and children.
- Conditions
- Sickle Cell Disease, Sickle Cell Anaemia, Sickle Cell Anemia
- Trial name
- RESCUE (NCT07282210)
- Testing
- Senicapoc
- Where
- 4 sites in Canada
- Volunteers
- 105 people
- Sponsor
- Biossil Inc.
Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.
- Conditions
- Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
- Trial name
- NCT06363760
- Testing
- Safety and efficacy assessments
- Where
- 18 sites in United States, Canada
- Volunteers
- 54 people
- Sponsor
- Editas Medicine, Inc.
Sickle Cell Disease, Hemoglobinopathies
A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD).
- Trial name
- NCT04853576
- Testing
- EDIT-301
- Where
- 24 sites in United States, Canada
- Volunteers
- 45 people
- Sponsor
- Editas Medicine, Inc.
Β-thalassemia
Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.
This is a single-arm, open label, multi-center, single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia.
- Trial name
- NCT06655662
- Testing
- β-globin restored autologous hematopoietic stem cells
- Where
- 3 sites in China
- Volunteers
- 8 people
- Sponsor
- Shenzhen Hemogen
Thrombotic Thrombocytopenic Purpura, Congenital Thrombotic Thrombocytopenic Purpura
Genotype and Phenotype Correlation in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome)
Hereditary thrombotic thrombocytopenic purpura (Upshaw-Schulman syndrome) is a rare disorder characterized by thrombocytopenia as a result of platelet consumption, microangiopathic hemolytic anemia, occlusion of the…
- Conditions
- Thrombotic Thrombocytopenic Purpura, Congenital Thrombotic Thrombocytopenic Purpura, Familial Thrombotic Thrombocytopenic Purpura, Thrombotic Thrombocytopenic Purpura, Congenital
- Trial name
- TTP registry (NCT01257269)
- Testing
- Observation
- Where
- 7 sites in United States, Austria and 5 more
- Volunteers
- 450 people
- Sponsor
- Insel Gruppe AG, University Hospital Bern