A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy

Korábbi kezelés

• 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.
• Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers.
• 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.

Más betegség

• Positive test for hepatitis C antibody (unless negative HCV PCR), hepatitis B surface antigen, or human immunodeficiency virus (HIV) antibody at screening.
• History of substance abuse or dependency.

Laborértékek

• Cardiac echocardiogram ejection fraction <45% or New York Heart Association (NYHA) Class III or Class IV.
• Moderate or severe renal or hepatic impairment (eGFR <60 mL/min/1.73 m2).
• 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.

Egyéb

• Any clinically significant changes during or following the completion of Study EDG 5506-001 that would affect the potential safety of the participant to receive EDG 5506.
• Baseline 12-lead electrocardiogram (ECG) that demonstrates clinically relevant abnormalities that may affect participant safety or interpretation of study results.
• Forced vital capacity (FVC) predicted <65% or using daytime (mechanical or noninvasive) ventilatory support.
• Participants who are unlikely to comply with the study protocol or, in the opinion of the Investigator, would not be a suitable candidate for participation in the study.

• Participants who have completed Study EDG-5506-001.
• Participants who were not from Study EDG-5506-001 must meet the following:
  • Male sex at birth and aged 18 to 55 years inclusive at time of consent.
  • Documented dystrophin mutation with phenotype consistent with BMD.
  • Ambulatory at Screening (defined as ability to complete 100 meter [m] timed test, with or without assistance).
  • Body weight ≥ 50 kg at the Screening visit.
  • Body mass index (BMI) between 20 and 34 kg/m2 inclusive.
• Female sexual partners of male participants must use highly effective contraception (<1% failure rate per year) through 6 months after last dose.
• Capable of giving signed informed consent.