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Fetal Hemoglobin Induction Treatment Metformin (FITMet)

Primary Purpose

Sickle Cell Anemia, Sickle Cell Disease, Hemoglobin Disorder

Status

Completed

Phase

Early Phase 1

Locations

United States

Study Type

Interventional

Intervention

Metformin

Questionnaires

About this trial

This is an interventional treatment trial for Sickle Cell Anemia focused on measuring Metformin, Sickle Cell Anemia, Sickle Cell Disease, Hemoglobinopathies, Hemoglobin Disorder, Blood Disease, Hemoglobin Disease

Eligibility Criteria

10 Years - 60 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Confirmed diagnosis of sickle cell anemia
Age greater than or equal to 10 and less than or equal to 60 years of age.
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.
Creatinine less than or equal to 1.4 mg/dL and estimated glomerular filtration rate greater than 45 ml/min/1.73 m2
Liver function tests (specifically ALT and conjugated bilirubin) less than or equal to 4 times upper limits of normal.

Exclusion Criteria:

Failure to meet inclusion criteria
Simple or chronic red blood cell transfusion therapy in the last 3 months OR a HbA level greater than 5% in SCA patients
Refusal to use medically effective birth control if female and sexually active.
If on hydroxyurea, not at stable dose of hydroxyurea for a minimum of 4 months (temporary exclusion).
Creatinine greater than 1.4mg/dL
Liver function tests (ALT and conjugated bilirubin) greater than 4 times upper limits of normal.

Sites / Locations

Texas Children's Hospital
The University of Texas Health Science Center at Houston

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Group A: Hydroxyurea + Metformin

Group B: Metformin (Group B has closed to enrollment)

Arm Description

Subjects who are currently taking Hydroxyurea as part of standard of care and have sickle cell anemia.

Subjects who are not taking Hydroxyurea as part of standard of care and have sickle cell anemia.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.

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.

Variability of Hemoglobin Response

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

Evaluation of RNA Sequencing

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

Full Information

NCT ID

NCT02981329

First Posted

December 1, 2016

Last Updated

October 11, 2023

Sponsor

Baylor College of Medicine

Collaborators

Pfizer

1. Study Identification

Unique Protocol Identification Number

NCT02981329

Brief Title

Fetal Hemoglobin Induction Treatment Metformin

Acronym

FITMet

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

October 2023

Overall Recruitment Status

Completed

Study Start Date

March 2, 2017 (Actual)

Primary Completion Date

July 20, 2020 (Actual)

Study Completion Date

July 20, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Baylor College of Medicine

Collaborators

Pfizer

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Sickle Cell Anemia, Sickle Cell Disease, Hemoglobin Disorder

Keywords

Metformin, Sickle Cell Anemia, Sickle Cell Disease, Hemoglobinopathies, Hemoglobin Disorder, Blood Disease, Hemoglobin Disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Early Phase 1

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

37 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Group A: Hydroxyurea + Metformin

Arm Type

Experimental

Arm Description

Subjects who are currently taking Hydroxyurea as part of standard of care and have sickle cell anemia.

Arm Title

Group B: Metformin (Group B has closed to enrollment)

Arm Type

Experimental

Arm Description

Subjects who are not taking Hydroxyurea as part of standard of care and have sickle cell anemia.

Intervention Type

Drug

Intervention Name(s)

Metformin

Other Intervention Name(s)

Glucophage, Riomet,

Intervention Description

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

Intervention Type

Behavioral

Intervention Name(s)

Questionnaires

Intervention Description

Questionnaires will be completed to assess the impact quality of life

Primary Outcome Measure Information:

Title

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

Description

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 Outcome Measure Information:

Title

Change in Laboratory Values

Description

Time Frame

1 Year

Title

Impact on Quality of Life

Description

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

Title

Variability of Hemoglobin Response

Description

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

Time Frame

1 Year

Title

Evaluation of RNA Sequencing

Description

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

Time Frame

1 Year

10. Eligibility

Sex

All

Minimum Age & Unit of Time

10 Years

Maximum Age & Unit of Time

60 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Confirmed diagnosis of sickle cell anemia Age greater than or equal to 10 and less than or equal to 60 years of age. 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. Creatinine less than or equal to 1.4 mg/dL and estimated glomerular filtration rate greater than 45 ml/min/1.73 m2 Liver function tests (specifically ALT and conjugated bilirubin) less than or equal to 4 times upper limits of normal. Exclusion Criteria: Failure to meet inclusion criteria Simple or chronic red blood cell transfusion therapy in the last 3 months OR a HbA level greater than 5% in SCA patients Refusal to use medically effective birth control if female and sexually active. If on hydroxyurea, not at stable dose of hydroxyurea for a minimum of 4 months (temporary exclusion). Creatinine greater than 1.4mg/dL Liver function tests (ALT and conjugated bilirubin) greater than 4 times upper limits of normal.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Titilope Fasipe, MD

Organizational Affiliation

Baylor College of Medicine

Official's Role

Principal Investigator

Facility Information:

Facility Name

Texas Children's Hospital

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

Facility Name

The University of Texas Health Science Center at Houston

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

The leftover blood samples for genetic analysis will be banked indefinitely for future ancillary studies. Investigator who desires access to these samples must present their research idea prior to receiving access to the samples. The samples will be shared with researchers affiliated with Texas Children's Hospital, Baylor College of Medicine and/or other hematology collaborators for future studies associated with hematologic diseases and drugs used to treat such diseases. The recipient investigators are required to provide proof of IRB approval or exemption as per local IRB guidelines before the sample can be released for research purposes. After receipt of IRB approval, coded samples will be distributed to the recipient investigator.

Learn more about this trial

Fetal Hemoglobin Induction Treatment Metformin

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