Fetal Hemoglobin Induction Treatment Metformin

NCT ID: NCT02981329

Last Updated: 2023-10-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

EARLY_PHASE1

Total Enrollment

37 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-03-02

Study Completion Date

2020-07-20

Brief Summary

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

Detailed Description

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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.

Conditions

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Sickle Cell Anemia Sickle Cell Disease Hemoglobin Disorder

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Group A: Hydroxyurea + Metformin

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

Group Type EXPERIMENTAL

Metformin

Intervention Type DRUG

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

Questionnaires

Intervention Type BEHAVIORAL

Questionnaires will be completed to assess the impact quality of life

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

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

Group Type EXPERIMENTAL

Metformin

Intervention Type DRUG

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

Questionnaires

Intervention Type BEHAVIORAL

Questionnaires will be completed to assess the impact quality of life

Interventions

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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.

Intervention Type DRUG

Questionnaires

Questionnaires will be completed to assess the impact quality of life

Intervention Type BEHAVIORAL

Other Intervention Names

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Glucophage Riomet,

Eligibility Criteria

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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.

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.
Minimum Eligible Age

10 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Pfizer

INDUSTRY

Sponsor Role collaborator

Baylor College of Medicine

OTHER

Sponsor Role lead

Responsible Party

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Titilope Fasipe

Assistant Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Titilope Fasipe, MD

Role: PRINCIPAL_INVESTIGATOR

Baylor College of Medicine

Locations

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Texas Children's Hospital

Houston, Texas, United States

Site Status

The University of Texas Health Science Center at Houston

Houston, Texas, United States

Site Status

Countries

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United States

Other Identifiers

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H-38457 Metformin

Identifier Type: -

Identifier Source: org_study_id

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