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Fetal Hemoglobin Induction Treatment Metformin (FITMet)

This study is currently recruiting participants.
Verified December 2017 by Vivien Sheehan, Baylor College of Medicine
Sponsor:
ClinicalTrials.gov Identifier:
NCT02981329
First Posted: December 5, 2016
Last Update Posted: December 15, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
Pfizer
Information provided by (Responsible Party):
Vivien Sheehan, Baylor College of Medicine
  Purpose
The purpose of this study is to determine whether metformin is effective in the treatment for sickle cell anemia (SCA) and non-transfusion dependent thalassemia (NTDT).

Condition Intervention Phase
Sickle Cell Anemia Sickle Cell Disease Hemoglobin Disorder Hemoglobin Disease; Sickle-Cell, Thalassemia Drug: Metformin Early Phase 1

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Vivien Sheehan, Baylor College of Medicine:

Primary Outcome Measures:
  • Change in Fetal Hemoglobin (HbF) Percentage (SCA) or Change in Total Hemoglobin (Hb) (NTDT) [ 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:
  • 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.

  • 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.

  • 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.


Estimated Enrollment: 64
Actual Study Start Date: March 2, 2017
Estimated Study Completion Date: November 2022
Estimated Primary Completion Date: November 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Group A: Hydroxyurea + Metformin
Subjects who are currently taking Hydroxyurea as part of standard of care and have sickle cell anemia.
Drug: Metformin

Subjects will be started at a dose of 500 milligrams/day of metformin taken twice a day. After one week dose, the dose will be increased to 750 milligrams/day taken twice a day (total dose 1500 mg/day).

After 3 months on metformin at 1500 milligrams/day, the dose will be increased by 500 milligrams per day per week until a maximum dose of 2500 milligrams/day.

Experimental: Group B: Metformin
Subjects who are not taking Hydroxyurea as part of standard of care and have non-transfusion dependent thalassemia or sickle cell anemia.
Drug: Metformin

Subjects will be started at a dose of 500 milligrams/day of metformin taken twice a day. After one week dose, the dose will be increased to 750 milligrams/day taken twice a day (total dose 1500 mg/day).

After 3 months on metformin at 1500 milligrams/day, the dose will be increased by 500 milligrams per day per week until a maximum dose of 2500 milligrams/day.


Detailed Description:

This is a dose escalation, pilot study for subjects with sickle cell anemia (SCA) disease and non-transfusion dependent thalassemia (NTDT) to determine if metformin has a beneficial effect on the treatment of SCA and NTDT patients.

In addition the following items will also occur:

Data Collection will occur from subject's medical records in regards to their SCA or NTDT medical history.

Subjects will be asked to completed questionnaires to access increase or decrease of quality of life.

Biological samples (blood and urine) will be collected throughout the study to access the effects the study drug has on SCA and NTDT disease treatment, general health, and genetic analysis.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years to 40 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Confirmed diagnosis of sickle cell anemia or non-transfusion dependent thalassemia
  2. Age greater than or equal to 12 and less than or equal to 40 years of age.
  3. If on hydroxyurea, fetal hemoglobin less than 20% at maximum tolerated dose
  4. Creatinine less than or equal to 1.4mg/dL and estimated glomerular filtration rate greater than 45 ml/min/1.73 m2
  5. Liver function tests less than or equal to 3 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 >5% in SCA patients
  3. Pregnancy
  4. Refusal to use medically effective birth control if female and sexually active.
  5. If on hydroxyurea, not at stable maximum tolerated dose for a minimum of 4 months (temporary exclusion).
  6. Creatinine greater than 1.4mg/dL,
  7. Liver function tests greater than 3x upper limits of normal
  Contacts and Locations
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


Contacts
Contact: Vivien Sheehan, MD 832-824-4459 vxsheeha@texaschildrens.org
Contact: Bodgan Dinu brdinu@texaschildrens.org

Locations
United States, Texas
Texas Children's Hospital Recruiting
Houston, Texas, United States, 77030
Contact: Vivien Sheehan, MD    832-824-4459    vxsheeha@texaschildrens.org   
Contact: Bodgan Dinu       brdinu@texaschildrens.org   
Sponsors and Collaborators
Baylor College of Medicine
Pfizer
Investigators
Principal Investigator: Vivien Sheehan, MD Texas Children's Hospital
  More Information

Responsible Party: Vivien Sheehan, Assistant Professor, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT02981329     History of Changes
Other Study ID Numbers: H-38457 Metformin
First Submitted: December 1, 2016
First Posted: December 5, 2016
Last Update Posted: December 15, 2017
Last Verified: December 2017
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.

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Vivien Sheehan, Baylor College of Medicine:
Metformin
Sickle Cell Anemia
Sickle Cell Disease
Hemoglobinopathies
Hemoglobin Disorder
Blood Disease
Hemoglobin Disease; Sickle-Cell, Thalassemia
Hemoglobin Disease

Additional relevant MeSH terms:
Anemia, Sickle Cell
Thalassemia
Hemoglobinopathies
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Genetic Diseases, Inborn
Metformin
Hydroxyurea
Hypoglycemic Agents
Physiological Effects of Drugs
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors