Becker Muscular DystrophyGRASP-01-002NCT05257473
Defining Endpoints in Becker Muscular Dystrophy
An observational study of Becker Muscular Dystrophy and Muscular Dystrophies, run by Virginia Commonwealth University at 11 sites in 3 countries.
Trial summary
This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD) Read more about this study
- Who is it for?
- Ages 6 years and older, men only
- Conditions the study is for
- Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children
- How many people
- 80 participants
- Where
- 11 sitesin 3 countries
- Study dates
- 13 Apr 2022to 30 Sep 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 6 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals age 6+ who are clinically affected with Becker Muscular Dystrophy
Who may be able to join
- For ages 6-12
- Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with BMD)
- Genetic confirmation of an in-frame dystrophin mutation
- Ambulatory
- Willing and able to give informed consent and follow all procedures and requirements
- For ages 13 and older
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- Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with BMD)
- Genetic confirmation of a dystrophin mutation
- 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)
Who cannot join
- For ages 6-12
- Out of frame dystrophin mutation
- Use of chronic corticosteroids at baseline, defined as greater than 6 months of chronic use, will be limited to 20% of the overall population
- Non-ambulatory, defined as the inability to walk 10 meters without assistive device (excluding orthotics)
- >16 hours of ventilatory support
- 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.
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- Under the age of 6 at time of enrollment
- 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
- Loss of ambulation prior to age 16
- Use of chronic corticosteroids, defined as greater than 6 months of chronic use, will be limited to 20% of the overall population
- Less than 30% of the overall population will be non-ambulatory, defined as the inability to walk 10 meters without assistive device (excluding orthotics)
- >16 hours of ventilatory support
- Subjects aged 13-16 only: time to rise >10 seconds
- For MR Cohort: Have contraindications to MRI or MRS (e.g., non-MR compatible implanted medical devices or severe claustrophobia)
Trial locations and contacts
11 sites in 3 countries.
11 sites in 3 countries
United States9 sites
University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Iowa
Iowa City, Iowa, 52242
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of Minnesota
Minneapolis, Minnesota, 55455
University of California, Irvine
Orange, California, 92868
Virginia Commonwealth University
Richmond, Virginia, 23298
Washington University School of Medicine
St Louis, Missouri, 63110
United Kingdom1 site
John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne, NE1 4EP
About this study
This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)
Read the detailed description
Becker Muscular Dystrophy (BMD) is most frequently due to in-frame mutations in the dystrophin gene that are associated with reduced levels of frequently shortened dystrophin, though other mutations may be related to the Becker phenotype. There is wide variation in the age of onset and degree of progression, ranging from childhood to late adulthood. The more severe form of dystrophinopathy, Duchenne muscular dystrophy, has a more characteristic rate of progression and overall natural history. The wide variation in severity of progression has led to challenges in the design and conduct of approaching therapeutic trials. There is a need for a more rigorous natural history study to assist in the design of these promising therapeutic trials.
- Conditions
- Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
- Keywords
- Clinical research, BMD, Becker Muscular Dystrophy, Edgewise Therapeutics
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the natural history of measures of muscle function in BMD
North Star Assessment for LGMD (NSAD: The NSAD is a functional scale specifically designed to measure motor performance in individuals with LGMD and is being evaluated in BMD due to the similar limb-girdle pattern of weakness. It consists of 29 items that are considered clinically relevant items from the adapted North Star Ambulatory Assessment and the Motor Function Measure 20 with a maximum score of 54 and higher scores indicate higher functional abilities.
Measured: Through study completion, an average of 2 years
4-Stair Climb
Participants will perform the 4-stair climb with instructions to ascend 4 steps as quickly and as safely possible, using handrails if needed.
Measured: Through study completion, an average of 2 years
100-Meter Timed Test
The participant will be asked to complete 4 laps around 2 cones set 25 meters apart as quickly as safely possible, running if able and the time in seconds is recorded.
Measured: Through study completion, an average of 2 years
PERFORMANCE OF UPPER LIMB 2.0 (PUL)
The PUL is a tool designed for assessing upper limb function in persons with neuromuscular disorders.
Measured: Through study completion, an average of 2 years
HAND HELD DYNAMOMETRY (HHD) AND GRIP
Hand held dynamometry using the MicroFET2 myometer will be utilized to capture isometric strength in target muscle groups. Maximum strength in kilograms will be reported for each muscle group provided a continuous scale variable for analysis. CITEC myometer will be used to measure the and Grip of the subject. These pinch and grip techniques will also capture the maximum strength in newtons for the muscle groups involved.
Measured: Through study completion, an average of 2 years
TIMED UP-AND-GO (TUG)
The TUG will be administered using the appropriate stable seating surface (i.e., cube chair or straight back chair) to achieve 90 degree of both hip and knee flexion when participant is seated with both feet flat on the floor to start. The test should be performed barefoot. The fastest time to stand from the chair, walk 3 meters, and return to seated, will be recorded.
Measured: Through study completion, an average of 2 years
Measures of Pulmonary Function (Seated and supine FVC)
Spirometry will be performed in a sitting and supine position using standardized equipment. Forced vital capacity (FVC) sitting and supine.
Measured: Through study completion, an average of 2 years
Measures of Pulmonary Function (MEP and MIP)
Sitting maximal expiratory and inspiratory pressures (MEP and MIP) will be assessed.
Measured: Through study completion, an average of 2 years
Measures of Pulmonary Function (other)
Use of nocturnal or daytime positive pressure ventilation (PPV) (e.g., BiPAP or CPAP) will be recorded.
Measured: Through study completion, an average of 2 years
Measure of ejection fraction (ECHO)
A transthoracic echocardiogram (ECHO) will be performed. Measures of ejection fraction will be recorded.
Measured: Through study completion, an average of 2 years
Measure of systolic and diastolic function (ECHO)
A transthoracic echocardiogram (ECHO) will be performed. Measures of presence of systolic and diastolic function will be recorded.
Measured: Through study completion, an average of 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2022
- Study start13 Apr 2022
- Primary completion30 Sep 2026 (estimated)
- Study completion30 Sep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Becker Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Virginia Commonwealth University
- Sponsor type
- University, hospital or other
- Collaborators
- Edgewise Therapeutics, Inc.
- Responsible party
- Sponsor
- Investigators
- Nicholas E. Johnson, MD, Principal investigator, Virginia Commonwealth University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05257473
- Sponsor's protocol ID
- HM20023412
- Other IDs
- GRASP-BMD
- Acronym
- GRASP-01-002
- Status last verified
- Sep 2026
- First submitted
- 25 Jan 2022
- Last updated
- 3 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Straub V, Guglieri M. An update on Becker muscular dystrophy. Curr Opin Neurol. 2023 Oct 1;36(5):450-454. doi: 10.1097/WCO.0000000000001191. Epub 2023 Aug 21.
PubMed 37591308
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Defining Endpoints in Becker Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT05257473
- Acronym / sponsor ID
- GRASP-01-002 / HM20023412
- Conditions
- Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
- Keywords
- Clinical research, BMD, Becker Muscular Dystrophy, Edgewise Therapeutics
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Virginia Commonwealth UniversityUniversity, hospital or other
- Collaborators
- Edgewise Therapeutics, Inc.
- Study start
- 13 Apr 2022 (actual)
- Primary completion
- 30 Sep 2026 (estimated)
- Study completion
- 30 Sep 2026 (estimated)
- First posted
- 25 Feb 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)
Detailed description
Becker Muscular Dystrophy (BMD) is most frequently due to in-frame mutations in the dystrophin gene that are associated with reduced levels of frequently shortened dystrophin, though other mutations may be related to the Becker phenotype. There is wide variation in the age of onset and degree of progression, ranging from childhood to late adulthood. The more severe form of dystrophinopathy, Duchenne muscular dystrophy, has a more characteristic rate of progression and overall natural history. The wide variation in severity of progression has led to challenges in the design and conduct of approaching therapeutic trials. There is a need for a more rigorous natural history study to assist in the design of these promising therapeutic trials.
Study design
- Observational model
- Cohort
- Time perspective
- Other
Who may qualify
- Ages
- 6 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Individuals age 6+ who are clinically affected with Becker Muscular Dystrophy
Key inclusion criteria (11 listed)
- For ages 6-12
- Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with BMD)
- Genetic confirmation of an in-frame dystrophin mutation
- Ambulatory
- Willing and able to give informed consent and follow all procedures and requirements
Key exclusion criteria (15 listed)
- For ages 6-12
- Out of frame dystrophin mutation
- Use of chronic corticosteroids at baseline, defined as greater than 6 months of chronic use, will be limited to 20% of the overall population
- Non-ambulatory, defined as the inability to walk 10 meters without assistive device (excluding orthotics)
- >16 hours of ventilatory support
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To assess the natural history of measures of muscle function in BMD
Time frame: Through study completion, an average of 2 years
North Star Assessment for LGMD (NSAD: The NSAD is a functional scale specifically designed to measure motor performance in individuals with LGMD and is being evaluated in BMD due to the similar limb-girdle pattern of weakness. It consists of 29 items that are considered clinically relevant items from the adapted North Star Ambulatory Assessment and the Motor Function Measure 20 with a maximum score of 54 and higher scores indicate higher functional abilities.
4-Stair Climb
Time frame: Through study completion, an average of 2 years
Participants will perform the 4-stair climb with instructions to ascend 4 steps as quickly and as safely possible, using handrails if needed.
100-Meter Timed Test
Time frame: Through study completion, an average of 2 years
The participant will be asked to complete 4 laps around 2 cones set 25 meters apart as quickly as safely possible, running if able and the time in seconds is recorded.
PERFORMANCE OF UPPER LIMB 2.0 (PUL)
Time frame: Through study completion, an average of 2 years
The PUL is a tool designed for assessing upper limb function in persons with neuromuscular disorders.
HAND HELD DYNAMOMETRY (HHD) AND GRIP
Time frame: Through study completion, an average of 2 years
Hand held dynamometry using the MicroFET2 myometer will be utilized to capture isometric strength in target muscle groups. Maximum strength in kilograms will be reported for each muscle group provided a continuous scale variable for analysis. CITEC myometer will be used to measure the and Grip of the subject. These pinch and grip techniques will also capture the maximum strength in newtons for the muscle groups involved.
TIMED UP-AND-GO (TUG)
Time frame: Through study completion, an average of 2 years
The TUG will be administered using the appropriate stable seating surface (i.e., cube chair or straight back chair) to achieve 90 degree of both hip and knee flexion when participant is seated with both feet flat on the floor to start. The test should be performed barefoot. The fastest time to stand from the chair, walk 3 meters, and return to seated, will be recorded.
Measures of Pulmonary Function (Seated and supine FVC)
Time frame: Through study completion, an average of 2 years
Spirometry will be performed in a sitting and supine position using standardized equipment. Forced vital capacity (FVC) sitting and supine.
Measures of Pulmonary Function (MEP and MIP)
Time frame: Through study completion, an average of 2 years
Sitting maximal expiratory and inspiratory pressures (MEP and MIP) will be assessed.
Measures of Pulmonary Function (other)
Time frame: Through study completion, an average of 2 years
Use of nocturnal or daytime positive pressure ventilation (PPV) (e.g., BiPAP or CPAP) will be recorded.
Measure of ejection fraction (ECHO)
Time frame: Through study completion, an average of 2 years
A transthoracic echocardiogram (ECHO) will be performed. Measures of ejection fraction will be recorded.
Measure of systolic and diastolic function (ECHO)
Time frame: Through study completion, an average of 2 years
A transthoracic echocardiogram (ECHO) will be performed. Measures of presence of systolic and diastolic function will be recorded.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2026.
- Derived from this study: PubMed 37591308Straub V, Guglieri M. An update on Becker muscular dystrophy. Curr Opin Neurol. 2023 Oct 1;36(5):450-454. doi: 10.1097/WCO.0000000000001191. Epub 2023 Aug 21.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Nicholas E. Johnson, MD principal investigator
Virginia Commonwealth University
11 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Becker Muscular Dystrophy and Muscular Dystrophies, run by Virginia Commonwealth University at 11 sites in 3 countries.
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 11 inclusion and 15 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: under way, not looking for participants, as of the sponsor's last update on 3 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, New Zealand, United Kingdom. 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?
This study is still under way. Results are not available in the source record yet.
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?
To assess the natural history of measures of muscle function in BMD (time frame: Through study completion, an average of 2 years). The study lists 11 primary outcomes in all. 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 NCT05257473, 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 3 Sep 2026.