Clinical trials: Red-Cell Aplasia, Pure
Sickle Cell Disease, Thalassemia
Multi-Center Study of Iron Overload: Pilot Study
The purpose of this study is to initiate pilot studies to demonstrate that a sufficient number of iron-overloaded thalassemia, SCD and DBA populations with similar duration of chronic transfusion, and age at start of transfusions…
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- MCSIO (NCT01114776)
- Where
- 3 sites in United States, Germany and 1 more
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Chronic Kidney Disease, End-Stage Renal Failure
Cohort Study to Determine the Long-Term Safety and Efficacy of Biogeneric Epoetin Treatment for Renal Anemia
The purpose of this study is to establish the long-term safety and efficacy of Biogeneric Epoetin, the attainability of therapeutic target for anaemia management, and the impact of Epoetin treatment on long-term health outcome…
- Conditions
- Chronic Kidney Disease, End-Stage Renal Failure, Anaemia
- Trial name
- NCT00238043
- Testing
- Biogeneric Epoetin
- Where
- 14 sites in Malaysia
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Ministry of Health, Malaysia
Pure Red Cell Aplasia
Sirolimus Treatment for Refractory PRCA
Pure red cell aplasia (PRCA) is a kind of anemia characterized by severe reticulocytopenia and obvious bone marrow erythroblastic cells decreased.
- Trial name
- NCT03364764
- Testing
- Sirolimus
- Where
- China
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Bing Han