Fetal Hemoglobin Induction Treatment Metformin

Early Phase 1

Completed

• Conditions: Hemoglobin Disorder; Sickle Cell Anemia; Sickle Cell Disease
• Interventions: Drug: Metformin; Behavioral: Questionnaires

• Registration Number: NCT02981329
• Lead Sponsor: Baylor College of Medicine

Brief Summary

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

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.

Study Timeline

• Study First Submitted: 2016-12-01
• Study First Submitted QC: 2016-12-02
• Study First Posted: 2016-12-05
• Study Start: 2017-03-02
• Primary Completion: 2020-07-20
• Study Completion: 2020-07-20
• Status Verified: 2023-10
• Last Update Submitted: 2023-10-11
• Last Update Posted: 2023-10-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 37

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Group A: Hydroxyurea + Metformin	Questionnaires	Subjects who are currently taking Hydroxyurea as part of standard of care and have sickle cell anemia.
Group B: Metformin (Group B has closed to enrollment)	Questionnaires	Subjects who are not taking Hydroxyurea as part of standard of care and have sickle cell anemia.
Group A: Hydroxyurea + Metformin	Metformin	Subjects who are currently taking Hydroxyurea as part of standard of care and have sickle cell anemia.
Group B: Metformin (Group B has closed to enrollment)	Metformin	Subjects who are not taking Hydroxyurea as part of standard of care and have sickle cell anemia.

Primary Outcome Measures

Name	Time	Method
Change in Fetal Hemoglobin (HbF) Percentage (SCA) or Change in Total Hemoglobin (Hb)	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.

Secondary Outcome Measures

Name	Time	Method
Variability of Hemoglobin Response	1 Year	Evaluation of hematological variability of fetal hemoglobin induction will be assessed per subject per genetic analysis and summarized.
Impact on Quality of Life	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.
Evaluation of RNA Sequencing	1 Year	Evaluate expression changes in paired samples collected before and on metformin through RNA sequencing.
Change in Laboratory Values	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.

Trial Locations

Locations (2)

Texas Children's Hospital; 🇺🇸 Houston, Texas, United States

The University of Texas Health Science Center at Houston; 🇺🇸 Houston, Texas, United States