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

Murdoch Childrens Research Institute

Status: Completed

Conditions: Facio-Scapulo-Humeral Dystrophy; FSHD1; FSHD2

Treatments: Dietary Supplement: Placebo; Dietary Supplement: Creatine Monohydrate

Study type: Interventional

Funder types: Other

Identifiers

NCT02948244

36298

Details and patient eligibility

About

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.

Enrollment: 11 patients

Sex: All

Ages: 5 to 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

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11 participants in 2 patient groups

Group A - Active/Placebo

Active Comparator group

Description:

Participants will receive 3 months of creatine monohydrate followed by a 6 week washout period followed by 3 months of placebo.

Treatment:

Dietary Supplement: Placebo

Dietary Supplement: Creatine Monohydrate

Group B - Placebo/Active

Active Comparator group

Description:

Participants will receive 3 months of placebo followed by a 6 week washout period followed by 3 months of creatine monohydrate.

Treatment:

Dietary Supplement: Placebo

Dietary Supplement: Creatine Monohydrate