Becker Muscular Dystrophy

Becker muscular dystrophy (BMD) is a genetic disorder causing progressive muscle weakness, but with a later onset and slower progression than Duchenne Muscular Dystrophy. People with BMD often remain ambulatory into adulthood and can have a near-normal life expectancy with proper care.

  • Cause: Mutation in the DMD gene, resulting in reduced or abnormal dystrophin; inherited in an X-linked recessive pattern, mainly affecting males.

  • Features: Muscle weakness beginning in adolescence or early adulthood, especially in hips, thighs, and shoulders; may include heart involvement (cardiomyopathy).

  • Diagnosis: Based on symptoms, creatine kinase levels, genetic testing, and sometimes muscle biopsy.

  • Treatment: No cure; management includes physical therapy, heart and respiratory monitoring, and medications to support cardiac function.

Active Trials

  • TITLE: A Study of EDG-5506 in Adult and Adolescent Males With Becker Muscular Dystrophy

    SPONSOR: Edgewise Therapeutics, Inc.

    INDICATION: Becker Muscular Dystrophy

    PROTOCOL: EDG-5506-201

    PHASE: 2

    DESCRIPTION: The CANYON study is a double-blind, placebo-controlled, Phase 2 study of EDG-5506 to assess the safety, biomarkers, and pharmacokinetics (PK) of EDG-5506 in adults and adolescents with Becker muscular dystrophy (BMD). EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers.

    STATUS: Active

    RECRUITING PATIENTS: No

    RDR LOCATION: Georgia, North Carolina

  • TITLE: A Study to Asses the Long-term Effects of EDG-5506 on Safety, Biomarkers, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy

    SPONSOR: Edgewise Therapeutics, Inc.

    INDICATION: Becker Muscular Dystrophy

    PROTOCOL: EDG-5506-203

    PHASE: 2 OL

    DESCRIPTION: The MESA study is a open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of EDG-5506. EDG-5506-203 MESA will provide continued access to EDG-5506 treatment to participants with Becker muscular dystrophy who were previously enrolled in EDG-5506-002 ARCH, completed EDG-5506-201 CANYON and GRAND CANYON, or completed EDG-5506-202 DUNE.

    STATUS: Active

    RECRUITING PATIENTS: No (rollover patients from EDG-5506 studies)

    RDR LOCATION: Georgia, North Carolina

Past Trials

  • TITLE: CureDuchenne Link®: A Resource to Support Research Studies in Duchenne and Becker Muscular Dystrophy (DMD/BMD).

    SPONSOR: CureDuchenne

    INDICATION: Duchenne Muscular Dystrophy / Becker Muscular Dystrophy

    PROTOCOL: CD-2021-01

    PHASE: NA

    DESCRIPTION: CureDuchenne link is a data hub comprised of integrated biospecimens, clinical data, and self- and/or caregiver-reported information from participants. Anyone over 4 weeks old who has been diagnosed with DMD or BMD or who is a carrier of DMD or BMD can join. Parents or legal guardians can sign up their child(ren).

    STATUS: Closed

    RECRUITING PATIENTS: No

    RDR LOCATION: Georgia, North Carolina

  • TITLE: A Phase 1b, Open-label Study of the Safety and Pharmacokinetics of EDG-5506 in Adults with Becker Muscular Dystrophy

    SPONSOR: Edgewise Therapeutics, Inc.

    INDICATION: Becker Muscular Dystrophy

    PROTOCOL: EDG-5506-002

    PHASE: 1b

    DESCRIPTION: EDG-5506 selectively modulates fast muscle myosin to reduce muscle stress caused by the absence of dystrophin. By protecting fast muscle fibers, EDG-5506 can potentially limit muscle breakdown and disease progression in Becker muscular dystrophy (BMD). This open-label study will evaluate the safety and pharmacokinetics (PK) of EDG-5506 in participants with BMD.

    STATUS: Completed

    RECRUITING PATIENTS: No

    RDR LOCATION: Georgia