Clinical trials: Sickle Cell Thalassemia
Sickle Cell Disease, Thalassemia
Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere
Primary Objective 1. Define the pharmacokinetics of liquid-formulated HU in infants (9 months to <2 years) 2. Assess the relative bioavailability of HU "sprinkles" compared to capsules in children and adolescents (≥2 to 18…
- Trial name
- NCT03825341
- Testing
- Hydroxyurea, Hydroxyurea Oral Capsule
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- St. Jude Children's Research Hospital
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Sickle Cell Anemia, Thalassemia
L-Glutamine Therapy for Sickle Cell Anemia
The primary purpose is to evaluate the effect of L-glutamine therapy on exercise endurance and breath by breath exercise response of sickle cell anemia patients The secondary purpose is to assess the effect of L-glutamine on…
- Trial name
- NCT00586209
- Testing
- L-Glutamine
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Hemoglobinopathies, Sickle Cell Disease
Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies
The purpose of this study is to determine if treatment with reduced-dose busulfex, fludarabine and alemtuzumab (CAMPATH) followed by sten cell infusion will allow for donor stem cells to grow in patients with hemoglobinopathies…
- Conditions
- Hemoglobinopathies, Sickle Cell Disease, Thalassemia
- Trial name
- NCT00153985
- Testing
- Busulfex, Fludarabine, Alemtuzumab
- Where
- 6 sites in United States
- Volunteers
- 2 people
- Sponsor
- Dana-Farber Cancer Institute