NCT02948244

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
11

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Oct 2017

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 26, 2016

Completed
2 days until next milestone

First Posted

Study publicly available on registry

October 28, 2016

Completed
1 year until next milestone

Study Start

First participant enrolled

October 31, 2017

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 31, 2021

Completed
1.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2022

Completed
Last Updated

February 8, 2023

Status Verified

February 1, 2023

Enrollment Period

3.3 years

First QC Date

October 26, 2016

Last Update Submit

February 4, 2023

Conditions

Facio-Scapulo-Humeral DystrophyFSHD2FSHD1

Keywords

fshdcreatine monohydratechildrenpediatricsFacio-Scapulo-Humeral Dystrophy

Outcome Measures

Primary Outcomes (1)

  • Motor Function Measure for Neuromuscular disease

    Composite functional outcome measure

    3 months

Secondary Outcomes (13)

  • Muscle Magnetic Resonance Imaging

    3 months

  • Muscle Ultrasound Scan

    3 months

  • Performance of the Upper Limb Measure

    3 months

  • ACTIVILIM

    3 Months

  • PedsQL Neuromuscular

    3 months

  • +8 more secondary outcomes

Study Arms (2)

Group A - Active/Placebo

ACTIVE COMPARATOR

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

Dietary Supplement: Creatine MonohydrateDietary Supplement: Placebo

Group B - Placebo/Active

ACTIVE COMPARATOR

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

Dietary Supplement: Creatine MonohydrateDietary Supplement: Placebo

Interventions

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.

Group A - Active/PlaceboGroup B - Placebo/Active
PlaceboDIETARY_SUPPLEMENT

Placebo

Group A - Active/PlaceboGroup B - Placebo/Active

Eligibility Criteria

Age5 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The Royal Children's Hospital

Melbourne, Victoria, 3052, Australia

Location

MeSH Terms

Conditions

Muscular Dystrophy, Facioscapulohumeral

Interventions

Creatine

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

GuanidinesAmidinesOrganic ChemicalsAmino AcidsAmino Acids, Peptides, and Proteins

Study Officials

  • 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

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 26, 2016

First Posted

October 28, 2016

Study Start

October 31, 2017

Primary Completion

January 31, 2021

Study Completion

July 1, 2022

Last Updated

February 8, 2023

Record last verified: 2023-02

Data Sharing

IPD Sharing
Will not share

Locations

AU(1)