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Trial Outcomes & Findings for Pilot Study of Metformin for Patients With Fanconi Anemia (NCT NCT03398824)

NCT ID: NCT03398824

Last Updated: 2022-11-29

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

6 months

Results posted on

2022-11-29

Participant Flow

Participant milestones

Participant milestones
Measure
Treatment Arm
Receive metformin HCl metformin HCl: Receive metformin HCl
Overall Study
STARTED
14
Overall Study
COMPLETED
12
Overall Study
NOT COMPLETED
2

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Treatment Arm
n=14 Participants
Receive metformin HCl metformin HCl: Receive metformin HCl
Age, Categorical
<=18 years
11 Participants
n=14 Participants
Age, Categorical
Between 18 and 65 years
3 Participants
n=14 Participants
Age, Categorical
>=65 years
0 Participants
n=14 Participants
Age, Continuous
9.4 years
n=14 Participants
Sex: Female, Male
Female
6 Participants
n=14 Participants
Sex: Female, Male
Male
8 Participants
n=14 Participants
Region of Enrollment
United States
14 Participants
n=14 Participants

PRIMARY outcome

Timeframe: 6 months

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

Outcome measures

Outcome measures
Measure
Treatment Arm
n=14 Participants
Receive metformin HCl metformin HCl: Receive metformin HCl
Hematologic Response
4 Participants

Adverse Events

Treatment Arm

Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Treatment Arm
n=14 participants at risk
Receive metformin HCl metformin HCl: Receive metformin HCl
Gastrointestinal disorders
Diarrhea
85.7%
12/14 • Study enrollment through 30 days after completion of treatment, up to 7 months

Additional Information

Akiko Shimamura

Boston Children's Hospital

Phone: 617-919-6946

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place