Fetal Hemoglobin Induction Treatment Metformin

Sickle Cell Disease Clinical Trial

— FITMet  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02981329
• Other study ID #: H-38457 Metformin
• Status: Completed
• Phase: Early Phase 1
• Start date: March 2, 2017
• Est. completion date: July 20, 2020

Study information

• Verified date: October 2023
• Source: Baylor College of Medicine
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 37
• Est. completion date: July 20, 2020
• Est. primary completion date: July 20, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 10 Years to 60 Years

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Hemoglobin Disorder
• Hemoglobinopathies
• Sickle Cell Anemia
• Sickle Cell Disease

Intervention

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

Locations

Country	Name	City	State
United States	Texas Children's Hospital	Houston	Texas
United States	The University of Texas Health Science Center at Houston	Houston	Texas

Sponsors (2)

Lead Sponsor	Collaborator
Baylor College of Medicine	Pfizer

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	1 Year
Secondary	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.	1 Year
Secondary	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.	1 Year
Secondary	Variability of Hemoglobin Response	Evaluation of hematological variability of fetal hemoglobin induction will be assessed per subject per genetic analysis and summarized.	1 Year
Secondary	Evaluation of RNA Sequencing	Evaluate expression changes in paired samples collected before and on metformin through RNA sequencing.	1 Year