Fetal Hemoglobin Induction Treatment Metformin (FITMet)

Baylor College of Medicine

Status and phase: Completed; Early Phase 1

Conditions: Hemoglobin Disorder; Sickle Cell Anemia; Sickle Cell Disease

Treatments: Behavioral: Questionnaires; Drug: Metformin

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02981329

H-38457 Metformin

Details and patient eligibility

About

The purpose of this study is to determine whether metformin is effective in the treatment for sickle cell anemia (SCA).

Full description

This is a dose escalation, pilot study for subjects with sickle cell anemia (SCA) disease to determine if metformin has a beneficial effect on the treatment and quality of life of SCA patients.

Enrollment: 37 patients

Sex: All

Ages: 10 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Confirmed diagnosis of sickle cell anemia
2. Age greater than or equal to 10 and less than or equal to 60 years of age.
3. 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.
4. Creatinine less than or equal to 1.4 mg/dL and estimated glomerular filtration rate greater than 45 ml/min/1.73 m2
5. Liver function tests (specifically ALT and conjugated bilirubin) less than or equal to 4 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 greater than 5% in SCA patients
3. Refusal to use medically effective birth control if female and sexually active.
4. If on hydroxyurea, not at stable dose of hydroxyurea for a minimum of 4 months (temporary exclusion).
5. Creatinine greater than 1.4mg/dL
6. Liver function tests (ALT and conjugated bilirubin) greater than 4 times upper limits of normal.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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37 participants in 2 patient groups

Group A: Hydroxyurea + Metformin

Experimental group

Description:

Subjects who are currently taking Hydroxyurea as part of standard of care and have sickle cell anemia.

Treatment:

Drug: Metformin

Behavioral: Questionnaires

Group B: Metformin (Group B has closed to enrollment)

Experimental group

Description:

Subjects who are not taking Hydroxyurea as part of standard of care and have sickle cell anemia.

Treatment:

Drug: Metformin

Behavioral: Questionnaires