A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults

• Status: Recruiting
• Trial end date: 2021-10-01
• Target enrollment: 0
• Participant gender: All

Summary

EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers. This Phase 1 study of EDG-5506 will assess the safety, tolerability, and pharmacokinetics (PK) and of EDG-5506 in adult healthy volunteers and in adults with Becker muscular dystrophy (BMD).

• Phase: Phase 1
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Edgewise Therapeutics, Inc.
• Collaborator: Worldwide Clinical Trials

Criteria

Inclusion Criteria:

• Male or female (HV only) adults aged 18 to 55 years, inclusive at the time of consent.

• For the healthy volunteer (HV) cohorts, good general health, with no significant medical history, no clinically significant abnormalities on physical examination at Screening and before administration of the initial dose of study drug.

• BMD patients must have medical records documenting pathological dystrophin mutation consistent with a diagnosis of BMD.

• BMD patients must have an ability to ambulate and an Eastern Cooperative Oncology Group (ECOG) score of 0, 1 or 2.

• BMD patients must have a Baseline creatine kinase (CK) ≥ 1200 U/L.

• BMD patients must meet New York Heart Association (NYHA) Class 1 or 2 designation

• Body weight greater than or equal to 50 kg at the Screening visit.

• Body mass index between 18 and 30 kg/m2, inclusive.

• Screening or pre-dose forced vital capacity percent predicted >80% (Healthy and BMD patients).

• Female subjects must be of non-childbearing potential. They must be either surgically sterile (hysterectomy, bilateral tubal ligation, salpingectomy, and/or bilateral oophorectomy at least 26 weeks before the Screening Visit) or post-menopausal, defined as spontaneous amenorrhea for at least 2 years, with follicle-stimulating hormone in the post-menopausal range at Screening, based on the central laboratory's ranges.

• Male subjects with female partners must use a medically accepted contraceptive regimen from the first dose of study drug and for 90 days after the last administration of study drug. In addition, male subjects must not donate sperm for 6 months following his last day of dosing. Medically accepted contraceptive methods are defined as those with 90% or greater efficacy.

• Is currently a non-smoker and must not have used any tobacco products within 3 months prior to the Screening visit.

• Able and willing to attend the necessary visits at the study center.

Exclusion Criteria:

• History of, or physical exam findings indicating clinically significant endocrine, neurological, gastrointestinal, cardiovascular, hematological, hepatic, immunological, renal, respiratory, or genitourinary abnormalities or diseases that, in the opinion of the investigator, would render the subject being unsuitable for the study.

• History of neoplastic disease (with the exception of adequately treated non-melanomatous skin carcinoma and treated cervical carcinoma in-situ).

• Fever (body temperature >38°C [>100°F]) or symptomatic viral/bacterial infection or use of antibiotics or antivirals within 2 weeks prior to Screening.

• Hospitalization or treatment of infection with parenteral antibiotics or antivirals within 45 days of Screening visit, and between Screening and admission to site.

• Pregnant or lactating females.

• Subject is unable to refrain from strenuous exercise for 3 days prior to check-in and during study.

• Have participated in any other investigational drug study within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.