Fetal Hemoglobin Induction Treatment Metformin

NCT02981329 | Completed Early Phase 1 DMC FDA Drug

• 1 other identifier: H-38457 Metformin
• Study Type: interventional
• Target: 37
• Locations: 1 country
• Sites: 2

Brief Summary

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

Conditions

Sickle Cell Anemia; Sickle Cell Disease; Hemoglobin Disorder

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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.

  1 Year

Secondary Outcomes (4)

• Change in Laboratory Values

  1 Year

• Impact on Quality of Life

  1 Year

• Variability of Hemoglobin Response

  1 Year

• Evaluation of RNA Sequencing

  1 Year

Study Arms (2)

Group A: Hydroxyurea + Metformin

EXPERIMENTAL

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

Drug: Metformin; Behavioral: Questionnaires

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

EXPERIMENTAL

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

Drug: Metformin; Behavioral: Questionnaires

Interventions

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.

Also known as: Glucophage, Riomet,

Group A: Hydroxyurea + Metformin; Group B: Metformin (Group B has closed to enrollment)

Questionnaires BEHAVIORAL

Questionnaires will be completed to assess the impact quality of life

Group A: Hydroxyurea + Metformin; Group B: Metformin (Group B has closed to enrollment)

Eligibility Criteria

• Age: 10 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• Creatinine greater than 1.4mg/dL
• Liver function tests (ALT and conjugated bilirubin) greater than 4 times upper limits of normal.

Sponsors & Collaborators

• Baylor College of Medicine: lead
• Pfizer: collaborator

Study Sites (2)

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

The University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Hemoglobinopathies; Hematologic Diseases

Interventions

Metformin; Surveys and Questionnaires

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemic and Lymphatic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Biguanides; Guanidines; Amidines; Organic Chemicals; Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Titilope Fasipe, MD

  Baylor College of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: December 1, 2016
• First Posted: December 5, 2016
• Study Start: March 2, 2017
• Primary Completion: July 20, 2020
• Study Completion: July 20, 2020
• Last Updated: October 12, 2023

Record last verified: 2023-10

Data Sharing

• IPD Sharing: Will share

Locations

US (2)