Purpose

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).

Conditions

Eligibility

Eligible Ages
Between 12 Years and 40 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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 a stable dose (mg/kg) determined by the primary hematology provider over at least four months.
  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 (specifically ALT and conjugated bilirubin) 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 greater than 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 (ALT and conjugated bilirubin) greater than 3 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.
  • Behavioral: Questionnaires
    Questionnaires will be completed to assess the impact quality of life
Experimental
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.
  • 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.
  • Behavioral: Questionnaires
    Questionnaires will be completed to assess the impact quality of life

Recruiting Locations

The University of Texas Health Science Center at Houston
Houston, Texas 77030
Contact:
Harinder Juneja, MD

More Details

Status
Recruiting
Sponsor
Baylor College of Medicine

Study Contact

Vivien Sheehan, MD
832-824-4459
vxsheeha@texaschildrens.org

Detailed Description

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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.