A Phase 2 interventional study of metformin HCl in Fanconi Anemia, sponsored by Boston Children's Hospital. Completed at 1 site in United States. Open to participants aged 6 Years to 35 Years. Per ClinicalTrials.gov, last updated 2022-11-29.
Sponsored by Boston Children's Hospital · Phase 2, Interventional, and Treatment
Among 68 studies in Fanconi Anemia with a site in United States — 14 open to participants now, 18 under way, none of them phase 3. Fanconi Anemia · United States.
Among the 68 studies in Fanconi Anemia with a site in United States, this one is the 28th largest by enrollment of the 48 interventional ones that give a count, is one of 5 that started in 2018, is one of 15 in phase 2, and was the 49th registered.
95 studies on the registry are indexed under Fanconi Anemia; 20 are open to participants now.
Browse Fanconi Anemia studies →14 of the 68 studies in Fanconi Anemia with a site in United States are open now. Recruiting first, the newest first:
Rockefeller University · Sites in United States
Ages 18+
University of Minnesota · Sites in United States
Ages 18+
National Cancer Institute (NCI) · Sites in United States
Ages 8–90
Masonic Cancer Center, University of Minnesota · Sites in United States
Ages 18–45 · Healthy volunteers
Porteus, Matthew, MD · Sites in United States
Ages 2+
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 15 Fanconi Anemia studies recruiting now, 8 (53%) set no upper age limit and 5 (33%) stop at 75 or younger; 11 take children under 18; 2 take healthy volunteers.
This study: 6 Years to 35 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
68 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 22 · Phase 2 26 · Phase 3 2
First in 1975; this study’s year is marked. 5 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 15 is below the median of 21 across 61 interventional studies indexed under Fanconi Anemia.
Median enrollment 21 across the 48 interventional studies, 67 across the 17 observational. Of the 47 that completed or were terminated, 17 (36%) have results posted.
This tab as a report to print or save as PDF → With an account.
3 of 68 have an industry lead sponsor. The 10 most frequent:
Boston Children's Hospital is the lead sponsor of 608 studies on the registry; 147 are open to participants now.
Of its 32 completed or terminated interventional studies of FDA-regulated products, 19 (59%) have results posted.
Boston Children's Hospital has 3 studies filed under Fanconi Anemia — fewer than ten, so there is no comparison to draw.
5 of the studies in Fanconi Anemia with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT02143830Children's Hospital Medical Center, CincinnatiHSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy | 2026-12 |
| NCT05973656Masonic Cancer Center, University of MinnesotaRole of Acetaldehyde in the Development of Oral Cancer | 2027-01-01 |
| NCT03579875Masonic Cancer Center, University of MinnesotaAlpha/Beta TCD HCT in Patients With Inherited BMF Disorders | 2027-01-01 |
| NCT03924401Emory UniversityAcute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant | 2027-03-01 |
| NCT00542373M.D. Anderson Cancer CenterWidefield Fluorescence and Reflectance Imaging Systems and Oral Tissue Samples in Monitoring Participants at Risk for Developing Oral Cancer | 2027-04-30 |
Add all 5 to your calendar (.ics) → With an account.
Among the 68 studies in Fanconi Anemia with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
This is a single institution, open-label, single arm pilot study of Metformin in patients with Fanconi Anemia (FA) and cytopenias with the primary endpoint of hematologic response. This study will also assess safety, tolerability, and the biologic effects of Metformin in patients with FA.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Participants must have a clinical diagnosis of Fanconi Anemia.
Participants must have normal organ function as defined below:
Age Maximum Serum Creatinine (mg/dL) Male Female
6 to \< 10 years 1 1 10 to \< 13 years 1.2 1.2 13 to \< 16 years 1.5 1.4
≥ 16 years 1.7 1.4
AND
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Receive metformin HCl
Drug: metformin HCl
Receive metformin HCl
Hematologic Response
Hematologic response is defined by specific increases in erythroid, platelet, and neutrophil counts. Erythroid response: Hemoglobin (Hgb) increase by \>1.5g/dL for non-transfusion dependent patients and for patients who are transfusion dependent: Transfusion independence Only transfusions given for Hgb ≤8g/dL or for symptoms will be counted in the response evaluation Platelet response: If initially \<20k/uL to start, then must increase to \>20k/UL with an absolute increase of 100%. If initially ≥20k/uL to start, then must have an absolute increase of \>30k/UL Neutrophil response: If initially \<500/uL to start, then must increase to \>500/uL with an absolute increase of \> 250/uL. If initially ≥500/uL to start, then must have an absolute increase of \>500/uL
Time frame: 6 months
| Milestone | Treatment Arm |
|---|---|
| Started | 14 |
| Completed | 12 |
| Not completed | 2 |
Hematologic response is defined by specific increases in erythroid, platelet, and neutrophil counts. Erythroid response: Hemoglobin (Hgb) increase by \>1.5g/dL for non-transfusion dependent patients and for patients who are transfusion dependent: Transfusion independence Only transfusions given for Hgb ≤8g/dL or for symptoms will be counted in the response evaluation Platelet response: If initially \<20k/uL to start, then must increase to \>20k/UL with an absolute increase of 100%. If initially ≥20k/uL to start, then must have an absolute increase of \>30k/UL Neutrophil response: If initially \<500/uL to start, then must increase to \>500/uL with an absolute increase of \> 250/uL. If initially ≥500/uL to start, then must have an absolute increase of \>500/uL
| Participants | Treatment Arm |
|---|---|
| Hematologic Response | 4 |
Collected over Study enrollment through 30 days after completion of treatment, up to 7 months. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Treatment Arm | 0/14 (0%) | 0/14 (0%) | 12/14 (85.7%) |
| Event | Treatment Arm |
|---|---|
| DiarrheaGastrointestinal disorders | 12/14 |
| Age, Categorical(Participants) | Treatment Arm |
|---|---|
| <=18 years | 11 |
| Between 18 and 65 years | 3 |
| >=65 years | 0 |
| Age, Continuous(years) | Treatment Arm |
|---|---|
| Mean | 9.4 (6.0 to 26.5) |
| Sex: Female, Male(Participants) | Treatment Arm |
|---|---|
| Female | 6 |
| Male | 8 |
| Race and Ethnicity Not Collected(Participants) | Treatment Arm |
|---|
| Region of Enrollment(Participants) | Treatment Arm |
|---|---|
| United States | 14 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Undecided
Ran from Mar 2018 to primary completion in Oct 2020, 30 months.
Results were first posted in Nov 2022, 2 years 1 month after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Nov 2022. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Metformin