What side effects are associated with this type of intervention?

Common treatments for cerebral palsy include physical and occupational therapy, medications to reduce spasticity and dystonia, and orthopedic interventions. Medications such as baclofen and botulinum toxin can cause side effects like drowsiness, weakness, and injection site reactions. Orthopedic surgeries may lead to complications such as infections or issues with bone healing. Metformin, being studied for cerebral palsy, is generally well-tolerated but can cause gastrointestinal issues like nausea and diarrhea. It is important to monitor for these side effects and discuss them with a healthcare provider.

What prior FDA approvals exist?

There are no specific FDA-approved drugs for the treatment of cerebral palsy that are similar to metformin, which reduces hepatic glucose production and improves insulin sensitivity. Treatments for cerebral palsy primarily focus on managing symptoms such as spasticity and dystonia, using medications like baclofen, diazepam, and dantrolene. These drugs do not share the same mechanism of action as metformin.

What other types of research exist?

Promising alternatives to Metformin for improving insulin sensitivity and reducing hepatic glucose production in Cerebral Palsy patients may include GLP-1 agonists (such as liraglutide and exenatide) and thiazolidinediones. However, their specific efficacy and safety in CP patients need further investigation.

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Anti-diabetic drug

Metformin for Cerebral Palsy

Phase 2

Waitlist Available

Led By Darcy Fehlings, MD, MSc

Research Sponsored by Holland Bloorview Kids Rehabilitation Hospital

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

No history of hypoglycemia after 2 years of age

Age 5 to 18 years, 11 months at the time of enrollment

Must not have

Pernicious anemia

No prior clinically ordered neuro-imaging to allow determination of WMI or GMI

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1.25 years

Awards & highlights

All Individual Drugs Already Approved

Approved for 20 Other Conditions

No Placebo-Only Group

Summary

This trial is testing a diabetes medication in children with cerebral palsy to see if it can help them move and think better by improving how their bodies use energy.

Who is the study for?

Children aged 5-18 with cerebral palsy who can follow simple instructions, have no history of renal disease or severe lung infections, and haven't had recent orthopedic surgery. They must not be on certain other medications, have uncontrolled seizures, heart failure, diabetes, or a known allergy to metformin.

What is being tested?

The trial is testing the safety and how well children stick to taking metformin. It's also looking at whether metformin can improve movement and thinking skills in kids with cerebral palsy. The study has periods without the drug followed by a period with it.

What are the potential side effects?

Metformin may cause stomach upset like diarrhea or nausea, potential for low blood sugar levels (hypoglycemia), vitamin B12 deficiency over long-term use which could lead to anemia or nerve problems.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I haven't had low blood sugar episodes since turning 2 years old.

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I am between 5 and 18 years old.

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English is my native language or I've studied in English for at least two years.

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I have been diagnosed with cerebral palsy, affecting my movement and posture.

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I haven't been hospitalized for lung infections caused by inhaling food or liquid in the past year.

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I have never had kidney disease.

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My mobility is moderately to severely limited.

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My kidney and liver are functioning normally.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have pernicious anemia.

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I haven't had brain scans to check for white or gray matter issues.

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I need sedation to undergo blood tests.

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I am currently taking or planning to take water pills.

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I am taking or will take drugs that are cleared by the kidneys.

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I am not pregnant, nursing, or planning to become pregnant during the study.

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My weight is in the bottom 5% for my age.

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I experience seizures that medication does not control.

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I have diabetes (Type I or II).

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1.25 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1.25 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1.25 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

The first indicator of feasibility will be assessed

The fourth indicator of feasibility will be assessed

The second indicator of feasibility will be assessed

+2 more

Secondary study objectives

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

Gross Motor Function Measure-66

Other study objectives

Neuropsychological Tests

Changes in the tissue structure cortical-spinal tract and other relevant white matter tracts or grey matter tracts as measured by Diffusion Kurtosis Imaging (DKI).

Children's Auditory Verbal Learning Test-2 [CAVLT-2] or Rey Auditory Verbal Learning Test [RAVLT] (depending on age of participant)

+2 more

Side effects data

From 2015 Phase 4 trial • 156 Patients • NCT02002221

13%

Nasopharyngitis

10%

Hyperhidrosis

Hunger

Tremor

Asthenia

Hypoglycaemia

Femoral neck fracture

Squamous cell carcinoma of the tongue

100%

80%

60%

40%

20%

Study treatment Arm

Vildagliptin (LAF237)

Placebo

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 20 Other Conditions

This treatment demonstrated efficacy for 20 other conditions.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: MetforminExperimental Treatment1 Intervention

Metformin oral tablet will be taken by mouth or through a gastrostomy tube, once or twice a day for 16 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Metformin

FDA approved

0 / 17Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Cerebral Palsy (CP) include physical and occupational therapy, which aim to improve motor function and independence by enhancing muscle strength, coordination, and flexibility. Medications such as botulinum toxin and baclofen reduce spasticity and dystonia by inhibiting nerve signals that cause muscle contractions. Orthopedic interventions, including surgeries and bracing, correct deformities and improve mobility. Emerging treatments like metformin, which reduces hepatic glucose production and improves insulin sensitivity, are being studied for their potential to enhance motor and cognitive functions. These treatments are crucial for CP patients as they address the diverse symptoms and improve overall quality of life.

Short-term causal effects of common treatments in ambulatory children and young adults with cerebral palsy: three machine learning estimates.Trends in publications about cerebral palsy 1990 to 2020.The forgotten sixth sense in cerebral palsy: do we have enough evidence for proprioceptive treatment?