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

NCT ID: NCT02948244

Last Updated: 2023-02-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-10-31
• Study Completion Date: 2022-07-01

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.

Conditions

Facio-Scapulo-Humeral Dystrophy; FSHD2; FSHD1

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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.

Group Type: ACTIVE_COMPARATOR

Creatine Monohydrate

Intervention Type: DIETARY_SUPPLEMENT

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.

Placebo

Intervention Type: DIETARY_SUPPLEMENT

Placebo

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.

Group Type: ACTIVE_COMPARATOR

Creatine Monohydrate

Intervention Type: DIETARY_SUPPLEMENT

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.

Placebo

Intervention Type: DIETARY_SUPPLEMENT

Placebo

Interventions

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.

Intervention Type: DIETARY_SUPPLEMENT

Placebo

Placebo

Intervention Type: DIETARY_SUPPLEMENT

Eligibility 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.

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Murdoch Childrens Research Institute

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Ian R Woodcock, MBBS

Role: PRINCIPAL_INVESTIGATOR

Murdoch Children Research Institute/Royal Children Hospital, Melbourne

Monique M Ryan

Role: PRINCIPAL_INVESTIGATOR

Murdoch Children Research Institute/Royal Children Hospital, Melbourne

Locations

The Royal Children's Hospital

Melbourne, Victoria, Australia

Countries

Australia

Other Identifiers

36298

Identifier Type: -

Identifier Source: org_study_id