Fetal Hemoglobin Induction Treatment Metformin

Purpose

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

Conditions

  • Sickle Cell Anemia
  • Sickle Cell Disease
  • Hemoglobin Disorder

Eligibility

Eligible Ages
Between 10 Years and 60 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Early Phase 1
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Group A: Hydroxyurea + Metformin 		Subjects who are currently taking Hydroxyurea as part of standard of care and have sickle cell anemia.
  • Drug: Metformin
    Metfomin will be taken daily. The metformin dose will be increased during two time points per subject if protocol dose escalation criteria is met.
    Other names:
    • Glucophage
    • Riomet,
  • Behavioral: Questionnaires
    Questionnaires will be completed to assess the impact quality of life
Experimental
Group B: Metformin (Group B has closed to enrollment) 		Subjects who are not taking Hydroxyurea as part of standard of care and have sickle cell anemia.
  • Drug: Metformin
    Metfomin will be taken daily. The metformin dose will be increased during two time points per subject if protocol dose escalation criteria is met.
    Other names:
    • Glucophage
    • Riomet,
  • Behavioral: Questionnaires
    Questionnaires will be completed to assess the impact quality of life

More Details

Status
Completed
Sponsor
Baylor College of Medicine

Study Contact

Detailed 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.