Fetal Hemoglobin Induction Treatment Metformin

Early Phase 1CompletedNCT02981329

Baylor College of Medicine37 enrolled

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Sickle Cell Anemia Sickle Cell Disease Hemoglobin Disorder

Interventions

MetforminDRUG

Metfomin will be taken daily. The metformin dose will be increased during two time points per subject if protocol dose escalation criteria is met.

QuestionnairesBEHAVIORAL

Questionnaires will be completed to assess the impact quality of life

Eligibility

Sex: ALLMin age: 10 YearsMax age: 60 Years

Medical Language ↔ Plain English

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.

Locations (2)

Texas Children's Hospital

Houston, Texas, United States

The University of Texas Health Science Center at Houston

Houston, Texas, United States

Outcomes

Primary Outcomes

Change in Fetal Hemoglobin (HbF) Percentage (SCA) or Change in Total Hemoglobin (Hb)

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

Time frame: 1 Year

Secondary Outcomes

Change in Laboratory Values

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.

Time frame: 1 Year

Impact on Quality of Life

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.

Time frame: 1 Year

Variability of Hemoglobin Response

Evaluation of hematological variability of fetal hemoglobin induction will be assessed per subject per genetic analysis and summarized.

Time frame: 1 Year

Evaluation of RNA Sequencing

Evaluate expression changes in paired samples collected before and on metformin through RNA sequencing.

Time frame: 1 Year

Data from ClinicalTrials.gov

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