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Facio-Scapulo-Humeral DystrophyNCT02948244

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

A study testing Creatine Monohydrate in people with Facio-Scapulo-Humeral Dystrophy and FSHD2, run by Murdoch Childrens Research Institute at 1 site in Australia.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 5 to 18 years
Conditions the study is for
Facio-Scapulo-Humeral Dystrophy, FSHD2, FSHD1
What is being tested?
Creatine Monohydrate
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
11 participants
Where
1 siteAustralia
Study dates
31 Oct 2017to 1 Jul 2022
Phase
Interventional
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 5 to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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.

Who cannot join

  • 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 locations and contacts

1 site in 1 country.

Australia1 site

  • The Royal Children's Hospital

    Melbourne, Victoria, 3052

Other trials in Australia

About this study

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

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.

Being tested
Creatine Monohydrate, Placebo
Keywords
fshd, creatine monohydrate, children, pediatrics, Facio-Scapulo-Humeral Dystrophy
Medical subject headings
Muscular Dystrophy, Facioscapulohumeral

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Creatine MonohydrateDietary 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.

  • PlaceboDietary supplement

    Placebo

Study groups in NCT02948244
Study groupRoleWhat they receive
Group A - Active/PlaceboActive comparatorParticipants will receive 3 months of creatine monohydrate followed by a 6 week washout period followed by 3 months of placebo.
Receives: Dietary Supplement: Creatine Monohydrate; Dietary Supplement: Placebo
Group B - Placebo/ActiveActive comparatorParticipants will receive 3 months of placebo followed by a 6 week washout period followed by 3 months of creatine monohydrate.
Receives: Dietary Supplement: Creatine Monohydrate; Dietary Supplement: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Motor Function Measure for Neuromuscular disease

    Composite functional outcome measure

    Measured: 3 months

Secondary outcomes (13)

  1. Muscle Magnetic Resonance Imaging

    Measured: 3 months

  2. Muscle Ultrasound Scan

    Measured: 3 months

  3. Performance of the Upper Limb Measure

    Measured: 3 months

  4. ACTIVILIM

    Measured: 3 Months

  5. PedsQL Neuromuscular

    Measured: 3 months

  6. FSH-COM

    FSHD specific composite measure

    Measured: 3 months

  7. FSH-Health Index (Pediatric Version)

    Patient reported outcome measure specific for patients with FSHD.

    Measured: 3 months

  8. Six Minute Walk Test

    Measured: 3 months

  9. FSHD Severity Score

    Measured: 3 months

  10. Quantitative muscle strength testing

    Measured: 3 months

  11. GPX3 Level

    Possible biomarker of disease severity in FSHD

    Measured: 3 months

  12. Step Counter

    Physical activity measure

    Measured: 3 months

  13. Laboratory safety monitoring

    bloods and urine safety testing (urea and electrolytes, urine plasma creatine:creatinine ratios)

    Measured: 3 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Oct 2016 (estimated)
  2. Study start31 Oct 2017
  3. Primary completion31 Jan 2021
  4. Study completion1 Jul 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase N/A
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
11 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02948244
Sponsor's protocol ID
36298
Status last verified
Feb 2023
First submitted
26 Oct 2016
Last updated
8 Feb 2023
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Woodcock IR, de Valle K, Cairns A, Davidson ZE, Kean M, Varma N, Grobler A, Metz D, Carroll K, Dilek N, Heatwole C, Ryan MM, Delatycki MB, Yiu EM. Effect of creatine monohydrate on motor function in children with facioscapulohumeral muscular dystrophy: A multicenter, randomized, double-blind placebo-controlled crossover trial. Pharmacotherapy. 2025 Jun;45(6):341-351. doi: 10.1002/phar.70025. Epub 2025 May 14.

    PubMed 40366059

Questions about this trial

What is this clinical trial studying?

A study testing Creatine Monohydrate in people with Facio-Scapulo-Humeral Dystrophy and FSHD2, run by Murdoch Childrens Research Institute at 1 site in Australia.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 8 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Motor Function Measure for Neuromuscular disease (time frame: 3 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02948244, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Feb 2023.

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