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Completed

NCT Number: NCT02981329

Fetal Hemoglobin Induction Treatment Metformin

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

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Key information

Conditions

Sickle Cell Anemia Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobin Disorder Hemoglobinopathies Sickle Cell Disease

Age range

10 year–60 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Early Phase 1

Primary location

Texas Children's Hospital, Houston, Texas, United States

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

metformin

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.

Other names: Glucophage, Riomet,

Questionnaires

Behavioral

Questionnaires will be completed to assess the impact quality of life

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

Baylor College of Medicine

Other

Collaborators

  • Pfizer

Registry information

Official study title

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

Acronym: FITMet

Important dates

Study start
2017
Primary completion
2020
Study completion
2020
First posted
Dec 5, 2016
Registry last updated
Oct 12, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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