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Trial record 2 of 33 for:    FSHD

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

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ClinicalTrials.gov Identifier: NCT02948244
Recruitment Status : Recruiting
First Posted : October 28, 2016
Last Update Posted : November 17, 2017
Sponsor:
Information provided by (Responsible Party):
Murdoch Childrens Research Institute

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 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 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
Estimated Primary Completion Date : November 2018
Estimated Study Completion Date : December 2018


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




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)



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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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02948244


Contacts
Contact: Ian R Woodcock, MBBS +61 3 9345 5661 ian.woodcock@rch.org.au
Contact: Monique M Ryan +61 3 9345 5661 neurology.department@rch.org.au

Locations
Australia, Victoria
The Royal Children's Hospital Recruiting
Melbourne, Victoria, Australia, 3052
Contact: Ian R Woodcock, MBBS, MSc    +61 (0) 3 9345 5661    ian.woodcock@rch.org.au   
Contact: Daniella Villano, BN    +61 (0) 3 9345 5661    daniella.villano@rch.org.au   
Principal Investigator: Ian R Woodcock, MBBS, MSc         
Sub-Investigator: Katy de Valle, BSc Physio         
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

Responsible Party: Murdoch Childrens Research Institute
ClinicalTrials.gov Identifier: NCT02948244     History of Changes
Other Study ID Numbers: 36298
First Posted: October 28, 2016    Key Record Dates
Last Update Posted: November 17, 2017
Last Verified: November 2017
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