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NCT01981525 · ClinicalTrials.gov registry record · Phase 1
A Pilot Study of Metformin in Patients With a Diagnosis of Li-Fraumeni Syndrome
A Phase 1 study, sponsored by National Cancer Institute (NCI).
- Completed
- Registry status
- Phase 1
- Development phase
- 26
- Enrollment target
NCT01981525: Completed Phase 1 study, sponsored by National Cancer Institute (NCI).
NCT01981525 is a Phase 1 study that has completed, run by National Cancer Institute (NCI). The registered enrollment target is 26 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (57% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01981525, a Phase 1 study, has completed, sponsored by National Cancer Institute (NCI).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 26 participants
- Enrollment target
Study Summary
Background: * Li Fraumeni Syndrome (LFS) is a highly penetrant, autosomal dominant cancer predisposition disorder. Four main cancer types including sarcoma, adrenocortical carcinoma, breast cancer, and malignant brain tumors commonly characterize LFS but the syndrome can include other cancers. * Metformin is an oral biguanide drug that is approved by the FDA for the treatment of type II diabetes. Metformin has been associated with reduced cancer risk in several epidemiologic studies and reduced cancer mortality in patients with type 2 diabetes. * Metformin decreases circulating insulin and IGF1, and promotes glucose uptake in skeletal muscle and inhibits gluconeogenesis in the liver. Elevations in circulating insulin and IGF1 levels have been associated with increased cancer risk. * Preclinical research in animal models shows that metformin may be more toxic in cancer cells that have lost p53 function. * Lifetime risk of cancer in LFS patients with germline TP53 mutations is estimated to be up to 70% by age 60, with women having excess lifetime cancer risk (up to 100%) compared to men (up to 80%). There are currently no approved chemopreventive agents for patients with LFS. * Metformin has been shown to be safe and tolerable in diabetic and non-diabetics, and may be an ideal candidate for chemoprevention of cancer in this population. Objectives: * Determine the tolerability of oral daily metformin in patients with LFS caused by germline TP53 mutations. * Determine if 8 weeks of daily metformin administration has any effect on circulating IGF-1, insulin, and IGFBP3 Eligibility: * Must have a germline TP53 mutation and provide documentation of testing. * Must have adequate organ function. * Age greater than or equal to 18 years. Design: * This is a pilot study to assess the tolerability of daily oral metformin administration in patients with LFS caused by germline TP53 mutations and to study the effect of metformin on biomarker levels in these subjects. * In th
Primary Outcome
toxicity assessment by CTCAE ver 4.0
Interventions
- DRUG Metformin
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 26 participants |
| Start Date | 2014-01-27 |
| Est. Completion | 2020-12-11 |
| Phase | Phase 1 |
What the finished NCT01981525 record still lists
NCT01981525 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 26 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (57% lower).
The record links to 0 conditions, and to 1 intervention - of which Metformin is the first listed.
NCT01981525 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01981525 about?
NCT01981525 is a clinical study titled "A Pilot Study of Metformin in Patients With a Diagnosis of Li-Fraumeni Syndrome". Background: * Li Fraumeni Syndrome (LFS) is a highly penetrant, autosomal dominant cancer predisposition disorder. Four main cancer types including sarcoma, adrenocortical carcinoma, breast cancer, and malignant brain tumors commonly characterize LFS but the syndrome can include other cancers. * Me...
What is the current status of trial NCT01981525?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 26 participants. The study started on 2014-01-27. Estimated completion is 2020-12-11.
What interventions are being tested in trial NCT01981525?
The interventions under investigation include: Metformin (DRUG).
Who is sponsoring clinical trial NCT01981525?
This trial is sponsored by National Cancer Institute (NCI), which has 3,257 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01981525's enrollment target sits among peer trials
26 1482nd of 2000 higher than 506 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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