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Fetal Hemoglobin Induction Treatment Metformin (FITMet)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02981329
Recruitment Status : Completed
First Posted : December 5, 2016
Last Update Posted : February 21, 2023
Sponsor:
Collaborator:
Pfizer
Information provided by (Responsible Party):
Titilope Fasipe, Baylor College of Medicine

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

Condition or disease Intervention/treatment Phase
Sickle Cell Anemia Sickle Cell Disease Hemoglobin Disorder Drug: Metformin Behavioral: Questionnaires Early Phase 1

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 37 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Use of Metformin as a Fetal Hemoglobin Inducer in Patients With Hemoglobinopathies
Actual Study Start Date : March 2, 2017
Actual Primary Completion Date : July 20, 2020
Actual Study Completion Date : July 20, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Arms and Interventions
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Arm Intervention/treatment
Experimental: Group A: Hydroxyurea + Metformin
Subjects who are currently taking Hydroxyurea as part of standard of care and have sickle cell anemia.
 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.
Other Names:
  • Glucophage
  • Riomet,

Behavioral: Questionnaires
Questionnaires will be completed to assess the impact quality of life
Experimental: 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.
 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.
Other Names:
  • Glucophage
  • Riomet,

Behavioral: Questionnaires
Questionnaires will be completed to assess the impact quality of life


Outcome Measures
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Primary Outcome Measures :
  1. Change in Fetal Hemoglobin (HbF) Percentage (SCA) or Change in Total Hemoglobin (Hb) [ Time Frame: 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 :
  1. Change in Laboratory Values [ Time Frame: 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.

  2. Impact on Quality of Life [ Time Frame: 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.

  3. Variability of Hemoglobin Response [ Time Frame: 1 Year ]
    Evaluation of hematological variability of fetal hemoglobin induction will be assessed per subject per genetic analysis and summarized.

  4. Evaluation of RNA Sequencing [ Time Frame: 1 Year ]
    Evaluate expression changes in paired samples collected before and on metformin through RNA sequencing.


Eligibility Criteria
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Ages Eligible for Study:   10 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02981329


Locations
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United States, Texas
Texas Children's Hospital
Houston, Texas, United States, 77030
The University of Texas Health Science Center at Houston
Houston, Texas, United States, 77030
Sponsors and Collaborators
Baylor College of Medicine
Pfizer
Investigators
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Principal Investigator: Titilope Fasipe, MD Baylor College of Medicine
More Information
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Responsible Party: Titilope Fasipe, Assistant Professor, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT02981329    
Other Study ID Numbers: H-38457 Metformin
First Posted: December 5, 2016    Key Record Dates
Last Update Posted: February 21, 2023
Last Verified: February 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Titilope Fasipe, Baylor College of Medicine:
Metformin
Sickle Cell Anemia
Sickle Cell Disease
Hemoglobinopathies
 Hemoglobin Disorder
Blood Disease
Hemoglobin Disease
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Hemoglobinopathies
Anemia
Hematologic Diseases
Anemia, Hemolytic, Congenital
 Anemia, Hemolytic
Genetic Diseases, Inborn
Metformin
Hypoglycemic Agents
Physiological Effects of Drugs