Fetal Hemoglobin Induction Treatment Metformin
The purpose of this study is to determine whether metformin is effective in the treatment for sickle cell anemia (SCA) and non-transfusion dependent thalassemia (NTDT).
- Sickle Cell Anemia
- Sickle Cell Disease
- Hemoglobin Disorder
- Hemoglobin Disease; Sickle-Cell, Thalassemia
- Eligible Ages
- Between 12 Years and 40 Years
- Eligible Genders
- Accepts Healthy Volunteers
- Confirmed diagnosis of sickle cell anemia or non-transfusion dependent thalassemia
- Age greater than or equal to 12 and less than or equal to 40 years of age.
- If on hydroxyurea, fetal hemoglobin less than 20% at a stable dose (mg/kg) determined by the primary hematology provider over at least four months.
- Creatinine less than or equal to 1.4mg/dL and estimated glomerular filtration rate greater than 45 ml/min/1.73 m2
- Liver function tests (specifically ALT and conjugated bilirubin) less than or equal to 3 times upper limits of normal
- Failure to meet inclusion criteria
- Simple or chronic red blood cell transfusion therapy in the last 3 months OR a HbA level greater than 5% in SCA patients
- Refusal to use medically effective birth control if female and sexually active.
- If on hydroxyurea, not at stable maximum tolerated dose for a minimum of 4 months (temporary exclusion).
- Creatinine greater than 1.4mg/dL
- Liver function tests (ALT and conjugated bilirubin) greater than 3 times upper limits of normal
- Early Phase 1
- Study Type
- Intervention Model
- Parallel Assignment
- Primary Purpose
- None (Open Label)
Group A: Hydroxyurea + Metformin
|Subjects who are currently taking Hydroxyurea as part of standard of care and have sickle cell anemia.||
Group B: Metformin
|Subjects who are not taking Hydroxyurea as part of standard of care and have non-transfusion dependent thalassemia or sickle cell anemia.||
- Baylor College of Medicine
Study ContactVivien Sheehan, MD
This is a dose escalation, pilot study for subjects with sickle cell anemia (SCA) disease and non-transfusion dependent thalassemia (NTDT) to determine if metformin has a beneficial effect on the treatment and quality of life of SCA and NTDT patients.