Fetal Hemoglobin Induction Treatment Metformin

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.

Inclusion Criteria:

1. Confirmed diagnosis of sickle cell anemia or non-transfusion dependent thalassemia

2. Age greater than or equal to 12 and less than or equal to 40 years of age.

3. If on hydroxyurea, fetal hemoglobin less than 20% at maximum tolerated dose

4. Creatinine less than or equal to 1.4mg/dL and estimated glomerular filtration rate
greater than 45 ml/min/1.73 m2

5. Liver function tests less than or equal to 3 times upper limits of normal

Exclusion Criteria:

1. Failure to meet inclusion criteria

2. Simple or chronic red blood cell transfusion therapy in the last 3 months OR a HbA
level >5% in SCA patients

3. Pregnancy

4. Refusal to use medically effective birth control if female and sexually active.

5. If on hydroxyurea, not at stable maximum tolerated dose for a minimum of 4 months
(temporary exclusion).

6. Creatinine greater than 1.4mg/dL,

7. Liver function tests greater than 3x upper limits of normal