Effect of Creatine Monohydrate on Functional Muscle Strength in Children With FSHD

• ClinicalTrials.gov Identifier: NCT02948244
• Recruitment Status: Completed
• First Posted: October 28, 2016
• Last Update Posted: February 8, 2023
• Sponsor: Murdoch Childrens Research Institute
• Information provided by (Responsible Party): Murdoch Childrens Research Institute

Study Description

Brief Summary:

This multi-centre, randomised, double-blind, placebo-controlled crossover trial will compare changes in strength-related motor function following treatment with creatine monohydrate to treatment with placebo, as measured by the Motor Function Measure, from baseline to 12 weeks. Eligible subjects will undergo baseline assessments then will be randomised to either creatine monohydrate therapy or placebo for three months, followed by a six week wash-out period, then crossover to a further three months of therapy with either placebo or creatine. Subjects will undergo clinical assessments and study safety assessments at the beginning and end of each treatment period. The study will begin recruitment in early 2017.

Condition or disease	Intervention/treatment	Phase
Facio-Scapulo-Humeral Dystrophy; FSHD2; FSHD1	Dietary Supplement: Creatine Monohydrate; Dietary Supplement: Placebo	Not Applicable

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 11 participants
• Allocation: Randomized
• Intervention Model: Crossover Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: Effect of Creatine Monohydrate on Functional Muscle Strength and Muscle Mass in Children With FSHD: a Multi-centre, Randomised, Double-blind Placebo-controlled Crossover Trial
• Actual Study Start Date: October 31, 2017
• Actual Primary Completion Date: January 31, 2021
• Actual Study Completion Date: July 1, 2022

Arms and Interventions

Arm	Intervention/treatment
Active Comparator: Group A - Active/Placebo; Participants will receive 3 months of creatine monohydrate followed by a 6 week washout period followed by 3 months of placebo.	Dietary Supplement: Creatine Monohydrate; Synthetically produced dietary supplement Creatine Monohydrate will be used in powder form reconstituted to a drink. The dosage will be 100mg/kg/day up to a maximum of 10 grams daily.; Dietary Supplement: Placebo; Placebo
Active Comparator: Group B - Placebo/Active; Participants will receive 3 months of placebo followed by a 6 week washout period followed by 3 months of creatine monohydrate.	Dietary Supplement: Creatine Monohydrate; Synthetically produced dietary supplement Creatine Monohydrate will be used in powder form reconstituted to a drink. The dosage will be 100mg/kg/day up to a maximum of 10 grams daily.; Dietary Supplement: Placebo; Placebo

Outcome Measures

Primary Outcome Measures :

1. Motor Function Measure for Neuromuscular disease [ Time Frame: 3 months ]
   Composite functional outcome measure

Secondary Outcome Measures :

1. Muscle Magnetic Resonance Imaging [ Time Frame: 3 months ]
2. Muscle Ultrasound Scan [ Time Frame: 3 months ]
3. Performance of the Upper Limb Measure [ Time Frame: 3 months ]
4. ACTIVILIM [ Time Frame: 3 Months ]
5. PedsQL Neuromuscular [ Time Frame: 3 months ]
6. FSH-COM [ Time Frame: 3 months ]
   FSHD specific composite measure
7. FSH-Health Index (Pediatric Version) [ Time Frame: 3 months ]
   Patient reported outcome measure specific for patients with FSHD.
8. Six Minute Walk Test [ Time Frame: 3 months ]
9. FSHD Severity Score [ Time Frame: 3 months ]
10. Quantitative muscle strength testing [ Time Frame: 3 months ]
11. GPX3 Level [ Time Frame: 3 months ]
    Possible biomarker of disease severity in FSHD
12. Step Counter [ Time Frame: 3 months ]
    Physical activity measure
13. Laboratory safety monitoring [ Time Frame: 3 months ]
    bloods and urine safety testing (urea and electrolytes, urine plasma creatine:creatinine ratios)

Eligibility Criteria

• Ages Eligible for Study: 5 Years to 18 Years (Child, Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Is between the ages of 5 and 18 years inclusive at the time of randomisation;
• Has a confirmed genetic diagnosis of Facioscapulohumeral Muscular Dystrophy (FSHD) types 1 or 2;
• Has a legally acceptable representative capable of understanding the informed consent document and providing consent on the participant's behalf.

Exclusion Criteria:

• Has clinically significant elevation in plasma creatinine level or unexplained hypertension at screening;
• Has a prior diagnosis of chronic renal failure;
• Has a known hypersensitivity to creatine monohydrate of maltodextrin placebo;
• Patients already taking any medications to increase muscle bulk or strength or concomitant use of regular sodium valproate, corticosteroids of alpha agonists such as salbutamol.

Contacts and Locations

Locations

Australia, Victoria

The Royal Children's Hospital

Melbourne, Victoria, Australia, 3052

Sponsors and Collaborators

Murdoch Childrens Research Institute

Investigators

• Principal Investigator: Ian R Woodcock, MBBS; Murdoch Children Research Institute/Royal Children Hospital, Melbourne
• Principal Investigator: Monique M Ryan; Murdoch Children Research Institute/Royal Children Hospital, Melbourne

More Information

• Responsible Party: Murdoch Childrens Research Institute
• ClinicalTrials.gov Identifier: NCT02948244
• Other Study ID Numbers: 36298
• First Posted: October 28, 2016
• Last Update Posted: February 8, 2023
• Last Verified: February 2023

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: No

Keywords provided by Murdoch Childrens Research Institute:

fshd; creatine monohydrate; children; pediatrics; Facio-Scapulo-Humeral Dystrophy

Additional relevant MeSH terms:

Muscular Dystrophy, Facioscapulohumeral; Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn