Actively Recruiting
Phase III Randomized Double-blind Placebo-controlled Trial of Metformin for Cognitive Recovery and White Matter Growth in Children and Adolescents Treated for Brain Tumors
Led by Donald Mabbott · Updated on 2026-05-01
140
Participants Needed
20
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating whether metformin can help improve brain growth and cognitive function in children and adolescents who have completed treatment for brain tumors. This Phase III multi-site randomized trial compares 16 weeks of oral metformin treatment to a placebo in participants aged 7 to 21 years and 11 months. The study focuses on survivors of medulloblastoma and other brain tumors treated with cranial radiation, aiming to address cognitive impairments caused by aggressive cancer therapies. Participants will be randomly assigned to receive either metformin or a matching placebo. The dosing starts at approximately 500mg/m2/day for one week and increases to 1000mg/m2/day for the following 15 weeks, with doses adjusted in half-tablet increments. The trial includes baseline assessments, a 16-week treatment period, and follow-up evaluations at week 17 and again at 24 weeks post-treatment to monitor cognitive and brain changes. During the study, participants will undergo cognitive testing measuring memory, working memory, processing speed, attention, and executive function. Brain imaging with diffusion MRI will assess white matter growth, especially in the corpus callosum and hippocampus. Parent or guardian reports on global health will also be collected. Safety, adherence, and long-term effects will be monitored throughout the trial, which may last up to 41 weeks from baseline to final follow-up.
CONDITIONS
Brief Title
Effect of Metformin on Behaviour and the Brain in Children Treated for a Brain Tumour
Who Can Participate
Eligibility Criteria
You may qualify if you...
- At least 3 weeks after completion of primary therapy for medulloblastoma, ependymoma, craniopharyngioma, germ cell tumours, or other brain tumours treated with cranial radiation as approved by the Study PI
- Aged 7 years to 21 years and 11 months at enrollment
- Native English or French speaker or have at least two years of schooling in English or French
- Able to swallow tablets whole, crushed, or via feeding tube and willing to follow study treatment
- Normal kidney function with estimated glomerular filtration rate (eGFR) greater than 75 ml/min/1.73m²
- Normal liver function with specified limits for AST, ALT, and bilirubin levels
- Informed consent and assent obtained from participant and/or legal guardian
You will not qualify if you...
- Full scale IQ score less than 60 or pro-rated IQ score below 60 at screening
- Known allergy or hypersensitivity to metformin hydrochloride
- Unstable or insulin-dependent (Type 1) diabetes
- History of hypoglycemia after age 2
- Diagnosed metabolic or lactic acidosis or bicarbonate level below 22 mmol/L
- History of renal disease or dysfunction before brain tumour diagnosis
- History of congestive heart failure requiring treatment within 2 years prior to study
- Currently participating in a cognitive rehabilitation study
- Current or planned treatment involving diuretics
- Current or planned use of certain medications that interact with metformin
- Pernicious anemia diagnosed at screening
- Current use of metformin hydrochloride
- Any condition that may interfere with study compliance or safety as judged by the Site PI
- Receiving palliative care
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 16 weeks
Participants receive either metformin or placebo daily for 16 weeks to test the effect on cognitive recovery and brain growth.
Visits at Baseline, Week 17 (Post-Intervention), and Week 41 (6 Month Follow-Up)
Duration - 24 weeks after treatment
Participants undergo assessment 24 weeks after treatment to monitor ongoing cognitive and brain changes.
1 visit at Week 41 (6 Month Follow-Up)
Trial Site Locations
Total: 20 locations
1
John Hunter Children's Hospital
New Lambton Heights, New South Wales, Australia, 2305
Actively Recruiting
2
Children's Hospital in Westmead
Westmead, New South Wales, Australia, 2145
Withdrawn
3
Women and Childen's Hospital
Adelaide, North Adelaide, Australia, SA 5006
Actively Recruiting
4
Monash Children's Hospital
Clayton, Victoria, Australia, 3168
Actively Recruiting
5
Royal Children's Hospital
Parkville, Victoria, Australia, 3052
Actively Recruiting
6
Perth Children's Hospital
Nedlands, Western Australia, Australia, 6009
Actively Recruiting
7
Alberta Children's Hospital
Calgary, Alberta, Canada, T3B 6A8
Actively Recruiting
8
Stollery Children's Hospital
Edmonton, Alberta, Canada, T6G 2B7
Actively Recruiting
9
Children's & Women's Health Centre of British Columbia
Vancouver, British Columbia, Canada, V6H 3V4
Actively Recruiting
10
Cancer Care Manitoba
Winnipeg, Manitoba, Canada, R3E 0V9
Actively Recruiting
11
Izaak Walton Killam (IWK) Health Centre
Halifax, Nova Scotia, Canada, B3K 6R8
Actively Recruiting
12
Hamilton Health Sciences - McMaster Children's Hospital
Hamilton, Ontario, Canada, L8S 4K1
Actively Recruiting
13
Children's Hospital, London Health Sciences Centre
London, Ontario, Canada, N6A 5W9
Actively Recruiting
14
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Actively Recruiting
15
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Actively Recruiting
16
CHU Sainte-Justine
Montreal, Quebec, Canada, H3T 1C5
Actively Recruiting
17
Montreal Children's Hospital
Montreal, Quebec, Canada, H4A 3J1
Actively Recruiting
18
CHU de Québec - Université Laval
Québec, Quebec, Canada, G1V 4G2
Actively Recruiting
19
CHU de Sherbrooke
Sherbrooke, Quebec, Canada, J1G 2E8
Actively Recruiting
20
Saskatchewan Health Authority
Saskatoon, Saskatchewan, Canada, S7N 0W8
Actively Recruiting
Research Team
L
Lauren Cole, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
SUPPORTIVE_CARE
Number of Arms
2
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