Sickle Cell AnemiaEarly Phase 1FITMetNCT02981329
Fetal Hemoglobin Induction Treatment Metformin
A Early Phase 1 study testing Metformin and Questionnaires in people with Sickle Cell Anemia and Sickle Cell Disease, run by Baylor College of Medicine at 2 sites in United States.
Trial summary
The purpose of this study is to determine whether metformin is effective in the treatment for sickle cell anemia (SCA). Read more about this study
- Who is it for?
- Ages 10 to 60 years
- Conditions the study is for
- Sickle Cell Anemia, Sickle Cell Disease, Hemoglobin Disorder
- What is being tested?
- Metformin and Questionnaires
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 37 participants
- Where
- 2 sitesUnited States
- Study dates
- 2 Mar 2017to 20 Jul 2020
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Texas Children's Hospital
Houston, Texas, 77030
The University of Texas Health Science Center at Houston
Houston, Texas, 77030
About this study
Official title: Use of Metformin as a Fetal Hemoglobin Inducer in Patients With Hemoglobinopathies
The purpose of this study is to determine whether metformin is effective in the treatment for sickle cell anemia (SCA).
Read the 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.
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Hemoglobin Disorder
- Being tested
- Metformin, Questionnaires
- Keywords
- Metformin, Sickle Cell Anemia, Sickle Cell Disease, Hemoglobinopathies, Hemoglobin Disorder, Blood Disease, Hemoglobin Disease
- Medical subject headings
- Anemia, Sickle Cell, Hemoglobinopathies, Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MetforminDrug
Also called Glucophage, Riomet,
Metfomin will be taken daily. The metformin dose will be increased during two time points per subject if protocol dose escalation criteria is met.
QuestionnairesBehavioural
Questionnaires will be completed to assess the impact quality of life
| Study group | Role | What they receive |
|---|---|---|
| Group A: Hydroxyurea + Metformin | Experimental | Subjects who are currently taking Hydroxyurea as part of standard of care and have sickle cell anemia. Receives: 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. Receives: Drug: Metformin; Behavioral: Questionnaires |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 1 Year
Secondary outcomes (4)
Change in Laboratory Values
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.
Measured: 1 Year
Impact on Quality of Life
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.
Measured: 1 Year
Variability of Hemoglobin Response
Evaluation of hematological variability of fetal hemoglobin induction will be assessed per subject per genetic analysis and summarized.
Measured: 1 Year
Evaluation of RNA Sequencing
Evaluate expression changes in paired samples collected before and on metformin through RNA sequencing.
Measured: 1 Year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Dec 2016 (estimated)
- Study start2 Mar 2017
- Primary completion20 Jul 2020
- Study completion20 Jul 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell AnemiaStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer
- Responsible party
- Principal investigator, Titilope Fasipe, Baylor College of Medicine
- Investigators
- Titilope Fasipe, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02981329
- Sponsor's protocol ID
- H-38457 Metformin
- Acronym
- FITMet
- Status last verified
- Oct 2023
- First submitted
- 1 Dec 2016
- Last updated
- 12 Oct 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Use of Metformin as a Fetal Hemoglobin Inducer in Patients With Hemoglobinopathies
- ClinicalTrials.gov ID
- NCT02981329
- Acronym / sponsor ID
- FITMet / H-38457 Metformin
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Hemoglobin Disorder
- Keywords
- Metformin, Sickle Cell Anemia, Sickle Cell Disease, Hemoglobinopathies, Hemoglobin Disorder, Blood Disease, Hemoglobin Disease
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- Pfizer
- Study start
- 2 Mar 2017 (actual)
- Primary completion
- 20 Jul 2020 (actual)
- Study completion
- 20 Jul 2020 (actual)
- First posted
- 5 Dec 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine whether metformin is effective in the treatment for sickle cell anemia (SCA).
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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A: Hydroxyurea + Metformin | Experimental | Drug: Metformin, Behavioral: QuestionnairesSubjects who are currently taking Hydroxyurea as part of standard of care and have sickle cell anemia. |
| Group B: Metformin (Group B has closed to enrollment) | Experimental | Drug: Metformin, Behavioral: QuestionnairesSubjects who are not taking Hydroxyurea as part of standard of care and have sickle cell anemia. |
Who may qualify
- Ages
- 10 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MetforminAlso: Glucophage, Riomet,Metfomin will be taken daily. The metformin dose will be increased during two time points per subject if protocol dose escalation criteria is met. | Drug | Group A: Hydroxyurea + Metformin, Group B: Metformin (Group B has closed to enrollment) |
| QuestionnairesQuestionnaires will be completed to assess the impact quality of life | Behavioural | Group A: Hydroxyurea + Metformin, Group B: Metformin (Group B has closed to enrollment) |
Primary outcome
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.
4 secondary outcomes
Change in Laboratory Values
Time frame: 1 Year
Impact on Quality of Life
Time frame: 1 Year
Variability of Hemoglobin Response
Time frame: 1 Year
Evaluation of RNA Sequencing
Time frame: 1 Year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Oct 2023.
- Individual participant dataSharing planned: Yes. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Titilope Fasipe, MD principal investigator
Baylor College of Medicine
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Metformin and Questionnaires in people with Sickle Cell Anemia and Sickle Cell Disease, run by Baylor College of Medicine at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Fetal Hemoglobin (HbF) Percentage (SCA) or Change in Total Hemoglobin (Hb) (time frame: 1 Year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Oct 2023; AzTrials last refreshed its copy on 6 Nov 2026.