Clinical Trial: Fetal Hemoglobin Induction Treatment Metformin

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Use of Metformin as a Fetal Hemoglobin Inducer in Patients With Hemoglobinopathies

Brief Summary: 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).

Detailed Summary:

This is a single center, 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 of SCA and NTDT patients.

In addition the following items will also occur:

Data Collection will occur from subject's medical records in regards to their SCA or NTDT medical history.

Subjects will be asked to completed questionnaires to access increase or decrease of quality of life.

Biological samples (blood and urine) will be collected throughout the study to access the effects the study drug has on SCA and NTDT disease treatment, general health, and genetic analysis.


Sponsor: Baylor College of Medicine

Current Primary Outcome: Change in Fetal Hemoglobin (HbF) Percentage (SCA) or Change in Total Hemoglobin (Hb) (NTDT) [ Time Frame: 1 Year ]

Change in HbF percentage (%) or total Hb will be assessed by comparing baseline values to on treatment values per subject and will be summarized.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change in Laboratory Values [ Time Frame: 1 Year ]
    Evaluation and percentage of change in numeric values of total blood count, liver function, HbF levels, whole blood viscosity, and percent dense red blood cells will be evaluated per subject over the duration of the study and summarized.
  • Impact on Quality of Life [ Time Frame: 1 Year ]
    Evaluation of subject's change in quality of life will be assessed per subject per study questionnaire(s) over the duration of the study and summarized.
  • Variability of Hemoglobin Response [ Time Frame: 1 Year ]
    Evaluation of hematological variability of fetal hemoglobin induction will be assessed per subject per genetic analysis and summarized.


Original Secondary Outcome: Same as current

Information By: Baylor College of Medicine

Dates:
Date Received: December 1, 2016
Date Started: November 2016
Date Completion: November 2022
Last Updated: December 2, 2016
Last Verified: November 2016