RecruitingPhase 3ACTRN12624000781549

STRENGTH Study: Supplement Treatment Evaluation of L-carnitine for Muscle Fatigue and Weakness in Children with Neurofibromatosis Type 1

A Randomized, Double-Blind, Placebo Controlled, Phase 3 Study Assessing the Efficacy and Safety of L-carnitine Supplementation to Treat Muscle Fatigue and Weakness in Children with Neurofibromatosis Type 1


Sponsor

Royal North Shore Hospital

Enrollment

40 participants

Start Date

Sep 14, 2024

Study Type

Interventional

Conditions

Summary

The Strength Study is a Phase 3 clinical trial evaluating the efficacy and safety of L-carnitine in treating muscle fatigue and weakness in children with Neurofibromatosis Type 1 (NF1). Children with NF1 often experience reduced muscle mass, muscle weakness, and motor function issues, impacting their quality of life. NF1 deficiency can lead to the accumulation of intramyocellular lipids in muscles. L-carnitine helps transport fatty acids into mitochondria for energy production and has been used to treat disorders of fatty acid metabolism. This study will assess L-carnitine's effects over 12 to 25 weeks in children aged 8 to 12 with NF1, measuring improvements in muscle strength and activity. The primary measures will include Z-score changes on functional assessments, hand-dynamometry, and data from the GENEActiv Actigraph.


Eligibility

Sex: Both males and femalesMin Age: 8 YearssMax Age: 17 Yearss

Plain Language Summary

Simplified for easier understanding

Children with Neurofibromatosis Type 1 (NF1) — a genetic condition that affects the nervous system and causes tumours to grow on nerves — often experience muscle weakness and fatigue that impacts their daily activities and quality of life. There are currently very few treatments targeting this specific problem. This Phase 3 trial is testing whether a supplement called L-carnitine can improve muscle strength and physical activity in children with NF1. L-carnitine is a naturally occurring substance that helps the body produce energy from fat, and it has been used safely in other conditions involving muscle dysfunction. Participants will take the supplement for 12 to 25 weeks, and researchers will measure changes in muscle strength and daily activity levels. You may be eligible if your child is aged 8 to 17 years old, meets the diagnostic criteria for NF1, and has a history of muscle weakness. Children with seizures, significant skeletal abnormalities, or severe cognitive impairment would not be suitable. Your child must not have taken carnitine supplements in the past month.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

All participants will be prescribed a daily dose of L-carnitine equivalent to the nearest 500 mg dose of 50 mg/kg/day or placebo, given as a split dose to be taken at breakfast and at dinner. L-carnit

All participants will be prescribed a daily dose of L-carnitine equivalent to the nearest 500 mg dose of 50 mg/kg/day or placebo, given as a split dose to be taken at breakfast and at dinner. L-carnitine can either be given as hard capsules (500 mg) or as a powder for those seeking to avoid taking larger numbers of capsules. Participants will visit the study centres every 6 weeks during the intervention period for 12 weeks, then have a one-week washout. After Week 12 visit eligible patients will move on to the crossover study. This will be a further 12-week Treatment/Supplementation period. The purpose of study visits Week 6 and Week 12 is to undertake outcome assessments, complete patient and parent questionnaires and to provide more supplements to patients. Crossover period is when patients switch over between intervention arm and placebo arm. To monitor patient's adherence to the intervention a dosing diary will be provided to participants/guardians every study visits along with the IP tablets when they are dispensed. This will include date and time IP was taken by the patient and reasons if they were missed. Also, a robust process of counting leftover IP upon return will be done by the trial pharmacists and nurses and will be recorded on the patients' and pharmacy files.


Locations(1)

Royal North Shore Hospital - St Leonards

ACT, Australia

View Full Details on ANZCTR

For the most up-to-date information, visit the official listing.

Visit

ACTRN12624000781549


Related Trials