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Cerebral PalsyPhase 2NCT03710343

Metformin for Motor and Cognitive Improvement in Children With Cerebral Palsy: A Feasibility Study

A Phase 2 study testing Metformin in people with Cerebral Palsy, run by Holland Bloorview Kids Rehabilitation Hospital at 2 sites in Canada.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The study design is a single-subject ABA clinical trial that is investigating the feasibility including adherence, safety and tolerability of metformin in children aged 5 to 18 years with cerebral palsy (CP). ABA refers to Phase A1 with no metformin, Phase B with metformin, and Phase A2 with no metformin. Read more about this study

Who is it for?
Ages 5 to 18 years
Conditions the study is for
Cerebral Palsy
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
3 participants
Where
2 sitesCanada
Study dates
14 Nov 2023to 24 Apr 2025
Phase
Phase 2
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

  • Physician diagnosis of cerebral palsy defined "as a group of permanent disorders of the development of movement and posture causing activity limitation that is attributed to non-progressive disturbances that occurred in the developing fetal or infant brain."
  • Evidence of WMI or GMI pattern on prior clinical neuro-imaging scanning (MRI)
  • No history of hypoglycemia after 2 years of age
  • No aspiration pneumonias in the last year requiring hospitalization
  • No lower extremity orthopedic surgery in the last six months prior to trial entry
  • No acute or chronic metabolic acidosis and/or lactic acidosis over the lifespan, including a lactate level greater than 2.4 mmol/L at the screening visit.
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  • No history of renal disease
  • Age 5 to 18 years, 11 months at the time of enrollment
  • Either declare English as their native language or have had at least two years of schooling in English at the time of their baseline assessment
  • Gross Motor Function Classification System Level of II - V at the time of enrollment
  • Ability to communicate (verbal or non-verbal) pain or discomfort
  • With the exception of physiotherapy, no participation in active gross motor rehabilitation treatment (e.g. receiving lower extremity botulinum toxin injections, engaged in robotic walking therapy) up to 4 months prior to trial entry period and willingness to forgo introducing any new CP treatments during the 16 week trial period
  • Able to consume whole or crushed tablets swallowed orally or through a gastrostomy tube
  • Ability to understand and follow single step instructions/commands (i.e. blinking eyes, opening mouth, and moving head side to side).
  • Meet criteria for normal organ function requirements as described below:
  • Normal renal function defined as: Estimated glomerular filtration rate (eGFR) > 75ml/min/1.73m2
  • eGFR is calculated using the Schwartz formula: eGFR (mL/min/1.73 m²) = (0.41 × Height in cm) / Creatinine in mg/dL [29, 30]
  • Normal liver function defined as:
  • Total bilirubin < upper limit of normal (ULN) for age
  • SGOT (AST) or SGPT (ALT) < upper limit of normal (ULN) for age
  • Maximum AST Level (U/L) Male Female <12 years <47 <47
  • ≥ 12 years <35 <30
  • Maximum ALT Level (U/L)
  • Male Female All Ages <50 <36
  • Maximum Total Billirubin Level ( μmol/L) Male Female
  • All Ages <20 <20
  • Informed consent (and assent, where applicable) will be obtained from the participants by study team members authorized to consent for this study

Who cannot join

  • Participants who meet any of the following criteria will not be eligible to take part in the trial:
  • No prior clinically ordered neuro-imaging to allow determination of WMI or GMI
  • Have a known hypersensitivity to metformin hydrochloride or any of the excipients
  • Have Diabetes (Type I or II)
  • Have taken oral metformin previously
  • Have been part of another clinical intervention study within the past 3 months prior to study entry
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  • Require sedation for blood tests
  • Treatment or planned treatment involving diuretics
  • Current or planned treatment with cationic drugs excreted by the kidneys (e.g. amiloride, cimetidine, digoxin, morphine, nifedipine, procainamide, quinidine, quinine, ranitidine, triamterene, trimethoprim and vancomycin).
  • Current or planned treatment with concomitant medications with potential unacceptable interaction with metformin including topiramate, lamotrigine, levetiracetam, beta blockers, ACE inhibitors, glycopyrrolate, and carbonic anhydrase inhibitors, or at the discretion of the delegated study physician for medications with potential interactions such as sertraline, lansoprazole and omeprazole.
  • Receiving deep brain stimulation or intrathecal baclofen
  • Dosage of oral baclofen and benzodiazepines stabilized for less than 2 months prior to study entry, and/or planning to change the dosage over the treatment period (if applicable)
  • Females who are pregnant, nursing, or planning a pregnancy during the study
  • Pernicious anemia (according to results of the screening visit blood draw)
  • Weight for age percentile less than 5%
  • Uncontrolled seizures with or without medication (defined by a seizure lasting longer than 10 minutes in duration within six months prior to study entry or change in seizure medication due to poor seizure control in the 3 months prior to trial entry).
  • History of congestive heart failure (including the use of diuretics) requiring pharmacologic treatment within two years prior to study entry

Trial locations and contacts

2 sites in 1 country.

Canada2 sites

  • Holland Bloorview Kids Rehabilitation Hospital

    Toronto, Ontario, M4G1R8

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8

Other trials in Canada

About this study

The study design is a single-subject ABA clinical trial that is investigating the feasibility including adherence, safety and tolerability of metformin in children aged 5 to 18 years with cerebral palsy (CP). ABA refers to Phase A1 with no metformin, Phase B with metformin, and Phase A2 with no metformin. Secondarily, the study is exploring whether metformin has possible health benefits for improving motor function and cognition.

Read the detailed description

The trial is designed as a 48 week limited institution two-site single case ABA study feasibility trial. ABA refers to Phase A1 with no metformin, Phase B with metformin, and Phase A2 with no metformin. Primary endpoints are feasibility, including recruitment, adherence to study medication and outcome measure completion, safety and tolerability of metformin. Key secondary endpoints are gross motor function and sustainability of intervention. Exploratory endpoints are cognitive and MRI measures as well as qualitative information regarding barriers to participation.

All participants will receive the study drug (metformin) during the 16 week intervention period.

This study will be done at two different locations in Toronto 1) Holland Bloorview Kids Rehabilitation Hospital and 2) the Hospital for Sick Children (SickKids). Each participant will be asked to go to both hospitals to do different tests and assessments for the study. Ten participants with physician diagnosis of CP age 5 to 18 with evidence of white matter imaging (WMI) or Grey Matter Imaging (GMI), and gross motor function classification system (GMFCS) levels II-V will be recruited for participation. MRI, cognitive testing and focus groups will be conducted at SickKids where paediatric protocols and processes have been developed.

Conditions
Cerebral Palsy
Being tested
Metformin
Keywords
Cerebral Palsy, Pediatric, Motor Function, GMFCS, Cognitive Function, Metformin
Medical subject headings
Cerebral Palsy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MetforminDrug

    Also called Sandoz Metformin FC

    Single case ABA study design. Phase B (intervention period) with metformin administered orally (or by gastrostomy tube) for 16 weeks.

Study groups in NCT03710343
Study groupRoleWhat they receive
MetforminExperimentalMetformin oral tablet will be taken by mouth or through a gastrostomy tube, once or twice a day for 16 weeks.
Receives: Drug: Metformin

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The first indicator of feasibility will be assessed

    Whether 50% of identified eligible potential participants are consented.

    Measured: 1.25 years

  2. The second indicator of feasibility will be assessed

    Whether 80% of the study medication (metformin) was taken by all enrolled participants

    Measured: 1.25 years

  3. The third indicator of feasibility will be assessed

    Whether the GMFM-66 was performed for 80% of participants at all time points: pre-pre intervention , pre-intervention, post-intervention and at follow up (4 months post-intervention).

    Measured: 1.25 years

  4. The fourth indicator of feasibility will be assessed

    Whether an MRI was performed for 70% of participants at pre-intervention (i.e. beginning of the 16 week-intervention) and post-intervention (i.e. at the end of the 16-week intervention).

    Measured: 1.25 years

  5. Tolerability and safety

    Tolerability and safety of metformin will be evaluated with adverse event reporting and by semi-structured interviews of participants' perceptions of the study procedures known as the Safety Monitoring Uniform Research Form (SMURF) during the 16-week intervention period at all 7 safety visits.

    Measured: 1.25 years

Secondary outcomes (2)

  1. Gross Motor Function Measure-66

    Gross motor function as assessed by the Gross Motor Function Measure-66 (GMFM-66).

    Measured: Change in Gross Motor Function Measure-66 (GMFM-66) from pre-pre Intervention (visit 1) to baseline/pre-Intervention (visit 2) to week 16 of Intervention (visit 9) to 48 weeks

  2. Change in spasticity as measured by the Modified Tardieu Scale from baseline/pre-intervention (visit 2) to 48 weeks

    Measured by the Modified Tardieu Scale (MTS), which consists of performing a passive muscle stretch at two velocities, slow and fast. The rater measures the angle of the spastic catch in the fast stretch (defined as R1) and then measures the passive range of motion during the slow stretch (defined as R2) in the ankle plantar flexors and knee flexors bilaterally. The difference between R2 and R1 will be the measure of the dynamic component of spasticity. Results are presented on a scale ranging from 0-4 where decreased scores indicate less resistance, which is considered to be a better outcome.

    Measured: Change in Modified Tardieu Scale (MTS) measure from baseline/pre-intervention (visit 2) to week 16 of Intervention (visit 9) to 48 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Oct 2018
  2. Study start14 Nov 2023
  3. Primary completion11 Apr 2025
  4. Study completion24 Apr 2025

Resources

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
The trial is designed as a 48 week limited institution two-site single case ABA study feasibility trial.
Masking
None (open label)
Enrolment
3 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03710343
Sponsor's protocol ID
MET-09-2017
Status last verified
Jul 2025
First submitted
11 Jul 2018
Last updated
2 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Metformin in people with Cerebral Palsy, run by Holland Bloorview Kids Rehabilitation Hospital at 2 sites in Canada.

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 27 inclusion and 17 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: finished, as of the sponsor's last update on 2 Mar 2026. 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 Canada. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The first indicator of feasibility will be assessed (time frame: 1.25 years). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03710343.

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 2 Mar 2026.

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