NCT05257473

Brief Summary

This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)

Trial Health

58
Monitor

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
80

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Apr 2022

Longer than P75 for all trials

Geographic Reach
3 countries

11 active sites

Status
active not recruiting

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

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 25, 2022

Completed
1 month until next milestone

First Posted

Study publicly available on registry

February 25, 2022

Completed
2 months until next milestone

Study Start

First participant enrolled

April 13, 2022

Completed
4.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2026

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2026

Completed
Last Updated

June 8, 2025

Status Verified

June 1, 2025

Enrollment Period

4.1 years

First QC Date

January 25, 2022

Last Update Submit

June 6, 2025

Conditions

Becker Muscular DystrophyMuscular DystrophiesMuscular Dystrophy in ChildrenMuscular Dystrophy, Becker

Keywords

Clinical researchBMDBecker Muscular DystrophyEdgewise Therapeutics

Outcome Measures

Primary Outcomes (11)

  • 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.

    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.

    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.

    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.

    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.

    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.

    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.

    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.

    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.

    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.

    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.

    Through study completion, an average of 2 years

Other Outcomes (4)

  • To assess the natural history of MR measures of muscle quality in BMD

    Through study completion, an average of 2 years

  • MAGNETIC RESONANCE IMAGING TRANSVERSE RELAXATION TIME (T2)

    Through study completion, an average of 2 years

  • MAGNETIC RESONANCE SPECTROSCOPY TRANSVERSE RELAXATION TIME (T2)

    Through study completion, an average of 2 years

  • +1 more other outcomes

Eligibility Criteria

Age6 Years+
Sexmale(Gender-based eligibility)
Gender Eligibility DetailsBecker Muscular Dystrophy affects males.
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

Individuals age 6+ who are clinically affected with Becker Muscular Dystrophy

You may qualify if:

  • 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
  • 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:
  • \. Ambulatory, defined as able to walk 10 meters without assistive devices (orthotics allowed)

You may not qualify if:

  • 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.
  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (11)

University of California, Irvine

Orange, California, 92868, United States

Location

University of Colorado Anschutz Medical Campus

Aurora, Colorado, 80045, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

University of Auckland

Auckland, New Zealand

Location

John Walton Muscular Dystrophy Research Centre

Newcastle upon Tyne, NE1 4EP, United Kingdom

Location

Related Publications (1)

  • 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.

    PMID: 37591308DERIVED

Biospecimen

Retention: SAMPLES WITH DNA

Blood samples will be taken for biomarker development and retained for future research. No clinical diagnosis will be given to patients.

MeSH Terms

Conditions

Muscular Dystrophy, DuchenneMuscular Dystrophies

Condition Hierarchy (Ancestors)

Muscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Nicholas E. Johnson, MD

    Virginia Commonwealth University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
OTHER
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 25, 2022

First Posted

February 25, 2022

Study Start

April 13, 2022

Primary Completion

May 1, 2026

Study Completion

May 1, 2026

Last Updated

June 8, 2025

Record last verified: 2025-06

Data Sharing

IPD Sharing
Will not share

Locations

NZ(1)US(9)GB(1)