Clinical trial NCT05160415: A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy
Official title: A Phase 1b, Open-label Study of the Safety and Pharmacokinetics of EDG-5506 in Adults With Becker Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Becker Muscular Dystrophy
Keywords: Becker Muscular Dystrophy
Lead sponsor: Edgewise Therapeutics, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Medpace, Inc.
First posted: 2021-12-16
Enrollment: 12
Sex: MALE
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The ARCH study is an open-label, single-center, Phase 1b study of sevasemtem (EDG-5506) to assess the safety and pharmacokinetics (PK) of sevasemten in adults with Becker muscular dystrophy (BMD).

Sevasemten is an investigational product intended to protect and improve function of dystrophic muscle fibers.

Detailed description: This open-label study will evaluate the safety, tolerability, and pharmacokinetics (PK) of sevasemten in participants with BMD who completed the first-in-human study, EDG-5506-001, as well as additional (treatment-naïve) participants from outside the EDG-5506-001 study to meet the target sample size.

All participants will receive sevasemten. On-site visits will occur approximately monthly for the first 12 months, followed by every 3 months to assess safety and measures of function. This study will have a 24 month treatment period, followed by a 4 week follow-up period.

Eligibility criteria: Inclusion Criteria:

1. Participants who have completed Study EDG-5506-001.
2. Participants who were not from Study EDG-5506-001 must meet the following:

   1. Male sex at birth and aged 18 to 55 years inclusive at time of consent.
   2. Documented dystrophin mutation with phenotype consistent with BMD.
   3. Ambulatory at Screening (defined as ability to complete 100 meter \[m\] timed test, with or without assistance).
   4. Body weight ≥ 50 kg at the Screening visit.
   5. Body mass index (BMI) between 20 and 34 kg/m2 inclusive.

Exclusion Criteria:

1. Receipt of oral corticosteroids for \>5 days in the previous 6 months at a dose of \>5 mg equivalent per day. Lower oral doses or inhaled/intranasal steroids are permitted.
2. Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers.
3. Participation in any other investigational drug study or use of use of an investigational drug within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.
4. Medical history or other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory result or abnormality that may increase the risk of study participation or, in the Investigator's judgment, make the participant inappropriate for the study. Includes venous access that would be too difficult to facilitate repeated blood sampling.

Primary outcomes:
measure: Incidence of AEs in those treated with sevasemten
timeFrame: 25 Months
measure: Frequency of AEs in those treated with sevasemten
timeFrame: 25 Months
measure: Severity of AEs in those treated with sevasemten
timeFrame: 25 Months
Secondary outcomes:
measure: Incidence of treatment-emergent abnormal clinical chemistry test results
timeFrame: 24 Months
measure: Incidence of treatment-emergent abnormal hematology test results
timeFrame: 24 Months
measure: Incidence of treatment-emergent abnormal coagulation test results
timeFrame: 24 Months
measure: Incidence of treatment-emergent abnormal urinalysis test results
timeFrame: 24 Months
measure: Number of participants with changes in clinical chemistry
timeFrame: 24 Months
measure: Number of participants with changes in hematology
timeFrame: 24 Months
measure: Number of participants with changes in coagulation
timeFrame: 24 Months
measure: Number of participants with changes in urinalysis
timeFrame: 24 Months
measure: Number of participants with changes in vital signs
timeFrame: 24 Months
measure: Number of participants with changes in physical examination
timeFrame: 24 Months
measure: Number of participants with changes in ECG PR Interval
timeFrame: 24 Months
measure: Number of participants with changes in ECG QRS Interval
timeFrame: 24 Months
measure: Number of participants with changes in ECG QT Interval
timeFrame: 24 Months
measure: Number of participants with changes in ECG QTc Interval
timeFrame: 24 Months
measure: Number of participants with changes in FVC
timeFrame: 24 Months
measure: Number of participants with changes in FEV1
timeFrame: 24 Months
Other outcomes:
measure: Number of participants with changes in biomarkers of muscle fiber damage
timeFrame: 24 Months
Locations:
city: Atlanta
country: United States
facility: Rare Disease Research
state: Georgia
Study officials:
References: