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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 : August 26, 2019
Sponsor:
Information provided by (Responsible Party):
Murdoch Childrens Research Institute

Tracking Information
First Submitted Date  ICMJE October 26, 2016
First Posted Date  ICMJE October 28, 2016
Last Update Posted Date August 26, 2019
Actual Study Start Date  ICMJE October 31, 2017
Estimated Primary Completion Date December 31, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 27, 2016)
Motor Function Measure for Neuromuscular disease [ Time Frame: 3 months ]
Composite functional outcome measure
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02948244 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 13, 2017)
  • Muscle Magnetic Resonance Imaging [ Time Frame: 3 months ]
  • Muscle Ultrasound Scan [ Time Frame: 3 months ]
  • Performance of the Upper Limb Measure [ Time Frame: 3 months ]
  • ACTIVILIM [ Time Frame: 3 Months ]
  • PedsQL Neuromuscular [ Time Frame: 3 months ]
  • FSH-COM [ Time Frame: 3 months ]
    FSHD specific composite measure
  • FSH-Health Index (Pediatric Version) [ Time Frame: 3 months ]
    Patient reported outcome measure specific for patients with FSHD.
  • Six Minute Walk Test [ Time Frame: 3 months ]
  • FSHD Severity Score [ Time Frame: 3 months ]
  • Quantitative muscle strength testing [ Time Frame: 3 months ]
  • GPX3 Level [ Time Frame: 3 months ]
    Possible biomarker of disease severity in FSHD
  • Step Counter [ Time Frame: 3 months ]
    Physical activity measure
  • Laboratory safety monitoring [ Time Frame: 3 months ]
    bloods and urine safety testing (urea and electrolytes, urine plasma creatine:creatinine ratios)
Original Secondary Outcome Measures  ICMJE
 (submitted: October 27, 2016)
  • Muscle Magnetic Resonance Imaging [ Time Frame: 3 months ]
  • Muscle Ultrasound Scan [ Time Frame: 3 months ]
  • Dual-Energy X-ray Absorptiometry Scan [ Time Frame: 3 months ]
  • Performance of the Upper Limb Measure [ Time Frame: 3 months ]
  • ACTIVILIM [ Time Frame: 3 Months ]
  • PedsQL Neuromuscular [ Time Frame: 3 months ]
  • Six Minute Walk Test [ Time Frame: 3 months ]
  • FSH-COM [ Time Frame: 3 months ]
    FSHD specific composite measure
  • FSHD Severity Score [ Time Frame: 3 months ]
  • Quantitative muscle strength testing [ Time Frame: 3 months ]
  • GPX3 Level [ Time Frame: 3 months ]
    Possible biomarker of disease severity in FSHD
  • Step Counter [ Time Frame: 3 months ]
    Physical activity measure
  • Laboratory safety monitoring [ Time Frame: 3 months ]
    bloods and urine safety testing (urea and electrolytes, urine plasma creatine:creatinine ratios)
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Effect of Creatine Monohydrate on Functional Muscle Strength in Children With FSHD
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Facio-Scapulo-Humeral Dystrophy
  • FSHD2
  • FSHD1
Intervention  ICMJE
  • 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
Study Arms  ICMJE
  • 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.
    Interventions:
    • Dietary Supplement: Creatine Monohydrate
    • Dietary Supplement: 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.
    Interventions:
    • Dietary Supplement: Creatine Monohydrate
    • Dietary Supplement: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 27, 2016)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 1, 2020
Estimated Primary Completion Date December 31, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
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
Listed Location Countries  ICMJE Australia
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02948244
Other Study ID Numbers  ICMJE 36298
Has Data Monitoring Committee No
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Murdoch Childrens Research Institute
Study Sponsor  ICMJE Murdoch Childrens Research Institute
Collaborators  ICMJE Not Provided
Investigators  ICMJE
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
PRS Account Murdoch Childrens Research Institute
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP