Fanconi AnemiaPhase 3NCT06519786
Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi Anemia
A Phase 3 study testing Metformin in people with Fanconi Anemia, run by Ain Shams University at 2 sites in Egypt.
Trial summary
Prospective interventional open-label non-randomized controlled trial to assess safety and efficacy of metformin in treating cytopenia in children and adolescents with Fanconi Anemia. Read more about this study
- Who is it for?
- Ages 5 to 18 years
- Conditions the study is for
- Fanconi Anemia
- What is being tested?
- Metformin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 2 sitesEgypt
- Study dates
- 5 Oct 2022to Mar 2025
- Phase
- Phase 3
- 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 5 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age: 5 to 18 years
- Patients who are diagnosed with Fanconi anemia based on clinical features and confirmed by increased chromosomal breakage on diepoxybutane (DEB) stress testing.
- Presence of cytopenia (at least one of the following: hemoglobin (Hb) < 10 g/dL, platelet count < 100 x 109/L, or an absolute neutrophil count (ANC) < 1.0 x 109/L
- Patients receiving other therapies e.g., androgens are eligible for enrollment after a one-month washout period before the start of metformin.
Who cannot join
- Patients who underwent bone marrow transplantation.
- Patients with evidence of myelodysplasia, leukemia, or other concurrent malignancy.
- Patients who have a history of allergic reactions to metformin or similar compounds.
- Patients with a history of symptomatic hypoglycemia over the past year or hypoglycemia < 50 mg/dL on screening and baseline laboratory assessments.
- Patients with type 1 diabetes mellitus.
- Patients with vitamin B12 deficiency.
- Patients with Glucose-6-Phosphate Dehydrogenase deficiency.
- Patients with abnormal Kidney function tests including serum creatinine, elevated liver function tests including live enzymes (ALT or AST > 135 U/L, total bilirubin > 1.5 x upper limit of normal for age, and/or patients with metabolic acidosis (bicarbonate < 17 meq/L on venous blood gases).
Trial locations and contacts
2 sites in 1 country.
Egypt2 sites, 2 recruiting
- Recruiting
University of Alexandria
Alexandria
- Recruiting
Ain Shams University
Cairo, 11566
Sara M Makkeyah, MD, 01140105222, smakkeyah@med.asu.edu.eg
About this study
Prospective interventional open-label non-randomized controlled trial to assess safety and efficacy of metformin in treating cytopenia in children and adolescents with Fanconi Anemia.
Read the detailed description
Fanconi anemia (FA) is a genetic disease characterized by bone marrow failure, cancer susceptibility, and developmental abnormalities. Allogeneic hematopoietic stem cell transplantation offers curative therapy for hematologic complications of FA.
Oxymetholone is commonly used in the management of FA as it improves blood counts, red cells, and platelets. However, its use is limited by its high toxicity profile.
Metformin is a potential agent that reduces levels of both chromosomal radials and breaks in FA cells and increases the size of the hematopoietic stem cell compartment thus reducing cytopenia in patients with FA.
- Conditions
- Fanconi Anemia
- Being tested
- Metformin
- Medical subject headings
- Fanconi Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MetforminDrug
Also called Glucophage
Patients will receive metformin immediate-release tablets orally for 24 weeks. Starting dose will be 500 mg once daily for all patients and the dose will be increased by 500 mg weekly until the goal dose is achieved (500 mg twice daily for patients < 10 years of age, and 1000 mg twice daily for patients 10 years or older)
| Study group | Role | What they receive |
|---|---|---|
| Metformin group | Experimental | Patients will receive metformin immediate-release tablets orally for 24 weeks. Starting dose will be 500 mg once daily for all patients and the dose will be increased by 500 mg weekly until the goal dose is achieved (500 mg twice daily for patients < 10 years of age, and 1000 mg twice daily for patients 10 years or older). Receives: Drug: Metformin |
| Other treatment group | No intervention | Patients will receive supportive treatment as indicated |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hematologic response (erythroid)
Erythroid response (pretreatment, < 11 g/dL): Hgb increase by > 1.5 g/dL or Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk.
Measured: 24 weeks
Hematologic response (platelets)
Platelet response (pretreatment, < 100x10e9/L): Absolute increase of > 30 x 10e9/L for patients starting with > 20 x 10e9/L platelets or Increase from < 20 x 10e9/L to > 20 x10e9/L and by at least 100%
Measured: 24 weeks
Hematologic response (Neutrophil count)
Neutrophil response (pretreatment, < 1.0 x 10e9/L): At least 100% increase and an absolute increase > 0.5 x 10e9/L
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jul 2024
- Study start5 Oct 2022
- Primary completionOct 2024 (estimated)
- Study completionMar 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fanconi AnemiaStudies of the same condition, open and under way.
- More trials run by Ain Shams UniversityThe 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
- Phase 3
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ain Shams University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06519786
- Sponsor's protocol ID
- FMASU MD 274/2022
- Status last verified
- Dec 2023
- First submitted
- 27 Dec 2023
- Last updated
- 25 Jul 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi Anemia
- ClinicalTrials.gov ID
- NCT06519786
- Acronym / sponsor ID
- FMASU MD 274/2022
- Conditions
- Fanconi Anemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Ain Shams UniversityUniversity, hospital or other
- Study start
- 5 Oct 2022 (actual)
- Primary completion
- Oct 2024 (estimated)
- Study completion
- Mar 2025 (estimated)
- First posted
- 25 Jul 2024 (actual)
- 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.
Prospective interventional open-label non-randomized controlled trial to assess safety and efficacy of metformin in treating cytopenia in children and adolescents with Fanconi Anemia.
Detailed description
Fanconi anemia (FA) is a genetic disease characterized by bone marrow failure, cancer susceptibility, and developmental abnormalities. Allogeneic hematopoietic stem cell transplantation offers curative therapy for hematologic complications of FA.
Oxymetholone is commonly used in the management of FA as it improves blood counts, red cells, and platelets. However, its use is limited by its high toxicity profile.
Metformin is a potential agent that reduces levels of both chromosomal radials and breaks in FA cells and increases the size of the hematopoietic stem cell compartment thus reducing cytopenia in patients with FA.
Study design
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Metformin group | Experimental | Drug: MetforminPatients will receive metformin immediate-release tablets orally for 24 weeks. Starting dose will be 500 mg once daily for all patients and the dose will be increased by 500 mg weekly until the goal dose is achieved (500 mg twice daily for patients < 10 years of age, and 1000 mg twice daily for patients 10 years or older). |
| Other treatment group | No intervention | –Patients will receive supportive treatment as indicated |
Who may qualify
- Ages
- 5 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Age: 5 to 18 years
- Patients who are diagnosed with Fanconi anemia based on clinical features and confirmed by increased chromosomal breakage on diepoxybutane (DEB) stress testing.
- Presence of cytopenia (at least one of the following: hemoglobin (Hb) < 10 g/dL, platelet count < 100 x 109/L, or an absolute neutrophil count (ANC) < 1.0 x 109/L
- Patients receiving other therapies e.g., androgens are eligible for enrollment after a one-month washout period before the start of metformin.
Key exclusion criteria (8 listed)
- Patients who underwent bone marrow transplantation.
- Patients with evidence of myelodysplasia, leukemia, or other concurrent malignancy.
- Patients who have a history of allergic reactions to metformin or similar compounds.
- Patients with a history of symptomatic hypoglycemia over the past year or hypoglycemia < 50 mg/dL on screening and baseline laboratory assessments.
- Patients with type 1 diabetes mellitus.
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: GlucophagePatients will receive metformin immediate-release tablets orally for 24 weeks. Starting dose will be 500 mg once daily for all patients and the dose will be increased by 500 mg weekly until the goal dose is achieved (500 mg twice daily for patients < 10 years of age, and 1000 mg twice daily for patients 10 years or older) | Drug | Metformin group |
Primary outcome
Hematologic response (erythroid)
Time frame: 24 weeks
Erythroid response (pretreatment, < 11 g/dL): Hgb increase by > 1.5 g/dL or Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk.
Hematologic response (platelets)
Time frame: 24 weeks
Platelet response (pretreatment, < 100x10e9/L): Absolute increase of > 30 x 10e9/L for patients starting with > 20 x 10e9/L platelets or Increase from < 20 x 10e9/L to > 20 x10e9/L and by at least 100%
Hematologic response (Neutrophil count)
Time frame: 24 weeks
Neutrophil response (pretreatment, < 1.0 x 10e9/L): At least 100% increase and an absolute increase > 0.5 x 10e9/L
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2024.
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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.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Metformin in people with Fanconi Anemia, run by Ain Shams University at 2 sites in Egypt.
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 4 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 25 Jul 2024. 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 Egypt. 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?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Sara M Makkeyah, MD (+201140105222, smakkeyah@med.asu.edu.eg), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hematologic response (erythroid) (time frame: 24 weeks). The study lists 3 primary outcomes in all. 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 25 Jul 2024.