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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05257473




Registration number
NCT05257473
Ethics application status
Date submitted
25/01/2022
Date registered
25/02/2022
Date last updated
1/05/2024

Titles & IDs
Public title
Defining Endpoints in Becker Muscular Dystrophy
Scientific title
Defining Endpoints in Becker Muscular Dystrophy
Secondary ID [1] 0 0
GRASP-BMD
Secondary ID [2] 0 0
HM20023412
Universal Trial Number (UTN)
Trial acronym
GRASP-01-002
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Becker Muscular Dystrophy 0 0
Muscular Dystrophies 0 0
Muscular Dystrophy in Children 0 0
Muscular Dystrophy, Becker 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
To assess the natural history of measures of muscle function in BMD
Timepoint [1] 0 0
Through study completion, an average of 2 years
Primary outcome [2] 0 0
4-Stair Climb
Timepoint [2] 0 0
Through study completion, an average of 2 years
Primary outcome [3] 0 0
100-Meter Timed Test
Timepoint [3] 0 0
Through study completion, an average of 2 years
Primary outcome [4] 0 0
PERFORMANCE OF UPPER LIMB 2.0 (PUL)
Timepoint [4] 0 0
Through study completion, an average of 2 years
Primary outcome [5] 0 0
HAND HELD DYNAMOMETRY (HHD) AND GRIP
Timepoint [5] 0 0
Through study completion, an average of 2 years
Primary outcome [6] 0 0
TIMED UP-AND-GO (TUG)
Timepoint [6] 0 0
Through study completion, an average of 2 years
Primary outcome [7] 0 0
Measures of Pulmonary Function (Seated and supine FVC)
Timepoint [7] 0 0
Through study completion, an average of 2 years
Primary outcome [8] 0 0
Measures of Pulmonary Function (MEP and MIP)
Timepoint [8] 0 0
Through study completion, an average of 2 years
Primary outcome [9] 0 0
Measures of Pulmonary Function (other)
Timepoint [9] 0 0
Through study completion, an average of 2 years
Primary outcome [10] 0 0
Measure of ejection fraction (ECHO)
Timepoint [10] 0 0
Through study completion, an average of 2 years
Primary outcome [11] 0 0
Measure of systolic and diastolic function (ECHO)
Timepoint [11] 0 0
Through study completion, an average of 2 years

Eligibility
Key inclusion criteria
For ages 6-12

1. Clinically affected (defined as weakness on bedside evaluation in a pattern consistent
with BMD)

2. Genetic confirmation of an in-frame dystrophin mutation

3. Ambulatory

4. Willing and able to give informed consent and follow all procedures and requirements

For ages 13 and older

1. Clinically affected (defined as weakness on bedside evaluation in a pattern consistent
with BMD)

2. Genetic confirmation of a dystrophin mutation

3. Willing and able to give informed consent and follow all procedures and requirements

For participants in the MRI substudy:

1. Ambulatory, defined as able to walk 10 meters without assistive devices (orthotics
allowed)
Minimum age
6 Years
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
For ages 6-12

1. Out of frame dystrophin mutation

2. Use of chronic corticosteroids at baseline, defined as greater than 6 months of
chronic use, will be limited to 20% of the overall population

3. Non-ambulatory, defined as the inability to walk 10 meters without assistive device
(excluding orthotics)

4. >16 hours of ventilatory support

5. Any other illness that would interfere with the ability to undergo safe testing or
would interfere with interpretation of the results in the opinion of the site
investigator.

6. Under the age of 6 at time of enrollment

7. For MR Cohort: Have contraindications to MRI or MRS (e.g., non-MR compatible implanted
medical devices or severe claustrophobia)

For ages 13 and older

1. Loss of ambulation prior to age 16

2. Use of chronic corticosteroids, defined as greater than 6 months of chronic use, will
be limited to 20% of the overall population

3. Less than 30% of the overall population will be non-ambulatory, defined as the
inability to walk 10 meters without assistive device (excluding orthotics)

4. >16 hours of ventilatory support

5. Subjects aged 13-16 only: time to rise >10 seconds

6. For MR Cohort: Have contraindications to MRI or MRS (e.g., non-MR compatible implanted
medical devices or severe claustrophobia)

Study design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
13/04/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/05/2026
Actual
Sample size
Target
80
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Iowa
Country [4] 0 0
United States of America
State/province [4] 0 0
Kansas
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
Missouri
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Virginia
Country [10] 0 0
New Zealand
State/province [10] 0 0
Auckland
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Newcastle Upon Tyne

Funding & Sponsors
Primary sponsor type
Other
Name
Virginia Commonwealth University
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
Edgewise Therapeutics, Inc.
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
This is a 24-month, observational study of 50 participants with Becker muscular dystrophy
(BMD)
Trial website
https://clinicaltrials.gov/ct2/show/NCT05257473
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Nicholas E. Johnson, MD
Address 0 0
Virginia Commonwealth University
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Ruby Langeslay
Address 0 0
Country 0 0
Phone 0 0
804-828-8481
Fax 0 0
Email 0 0
ruby.langeslay@vcuhealth.org
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05257473