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

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ClinicalTrials.gov Identifier: NCT04585464
Recruitment Status : Recruiting
First Posted : October 14, 2020
Last Update Posted : October 14, 2020
Sponsor:
Collaborator:
Worldwide Clinical Trials
Information provided by (Responsible Party):
Edgewise Therapeutics, Inc.

Brief 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).

Condition or disease Intervention/treatment Phase
Healthy Volunteer Becker Muscular Dystrophy Drug: EDG-5506 Drug: Placebo Phase 1

Detailed Description:
Enrolled participants in this study will receive a single oral dose or multiple oral doses of EDG-5506 or a placebo. Blood and urine samples will be collected to measure how EDG-5506 is processed by the body and how the body responds when exposed to EDG-5506. Participants in the single ascending dose part of the study will remain in the clinic for 7 days with a 14-day follow-up period. Participants in the food effect study would come back to clinic after the their 21-day participation in the single ascending dose part of the study and stay in clinic for an additional 7 days followed by another 14-day follow-up period. Participants in the multiple ascending dose part of the study will remain in the clinic for 16 days with a 13-day follow-up period. Safety, tolerability, and pharmacokinetics of EDG-5506 will be assessed in healthy volunteers prior to enrolling participants with Becker muscular dystrophy.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 152 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1, Randomized, Placebo-Controlled, Double-blind, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, PK and Food Effect of EDG-5506 in Adult Healthy Volunteers and Adults With Becker Muscular Dystrophy
Estimated Study Start Date : October 12, 2020
Estimated Primary Completion Date : October 2021
Estimated Study Completion Date : October 2021


Arm Intervention/treatment
Experimental: Healthy Volunteer: Single Ascending Dose

Single oral ascending dose in healthy volunteers

Interventions:

Drug: EDG-5506 Drug: Placebo

Drug: EDG-5506
EDG-5505 is administered orally as a single dose or once daily

Drug: Placebo
Placebo is administered orally as a single dose or once daily

Experimental: Healthy Volunteer: Multiple Ascending Dose

Multiple oral ascending doses in healthy volunteers

Interventions:

Drug: EDG-5506 Drug: Placebo

Drug: EDG-5506
EDG-5505 is administered orally as a single dose or once daily

Drug: Placebo
Placebo is administered orally as a single dose or once daily

Experimental: Healthy Volunteer: Food Effect

Crossover food effect (fed versus fasted) single oral dose in healthy volunteers

Interventions:

Drug: EDG-5506 Drug: Placebo

Drug: EDG-5506
EDG-5505 is administered orally as a single dose or once daily

Drug: Placebo
Placebo is administered orally as a single dose or once daily

Experimental: Becker Muscular Dystrophy: Multiple Ascending Dose

Multiple oral ascending doses in adults with Becker muscular dystrophy

Interventions:

Drug: EDG-5506 Drug: Placebo

Drug: EDG-5506
EDG-5505 is administered orally as a single dose or once daily

Drug: Placebo
Placebo is administered orally as a single dose or once daily




Primary Outcome Measures :
  1. Incidence, frequency, severity and dose-relationship of adverse events [ Time Frame: Up to 30 days of monitoring ]
  2. Incidence of abnormal laboratory test results (clinical chemistry, hematology, urinalysis, coagulation) [ Time Frame: Up to 30 days of monitoring ]
  3. Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG) [ Time Frame: Up to 30 days of monitoring ]
  4. Incidence of abnormal vital signs [ Time Frame: Up to 30 days of monitoring ]
  5. Incidence of abnormal physical exam findings [ Time Frame: Up to 30 days of monitoring ]

Secondary Outcome Measures :
  1. Plasma maximum measured drug concentration (Cmax) [ Time Frame: Up to 30 days of testing ]
  2. Time of maximum concentration (Tmax) [ Time Frame: Up to 30 days of testing ]
  3. Area under the concentration-time curve (AUC) [ Time Frame: Up to 30 days of testing ]
  4. Plasma half-life (T½) [ Time Frame: Up to 30 days of testing ]
  5. Renal clearance (CLR) [ Time Frame: Up to 30 days of testing ]
  6. Drug excreted unchanged in urine (Amt0-24) [ Time Frame: Up to 30 days of testing ]
  7. Fraction excreted in urine (Fe) [ Time Frame: Up to 30 days of testing ]


Information from the National Library of Medicine

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Ages Eligible for Study:   19 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Healthy male and female (non-childbearing potential) volunteers and adult male Becker muscular dystrophy (BMD) participants
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • Male or female (HV only) adults aged 19 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.
  • Subjects who are unwilling to stay indoors, or if outdoors, unwilling to use a sunscreen on all areas of skin exposed to sunlight after their first dose, during their treatment period and for 2 days after their last dose of study drug.
  • Have participated in any other investigational drug study within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04585464


Locations
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United States, Texas
Worldwide Clinical Trials Recruiting
San Antonio, Texas, United States, 78217
Contact: Andrea Velasquez    210-635-1515    andrea.velasquez@worldwide.com   
Sponsors and Collaborators
Edgewise Therapeutics, Inc.
Worldwide Clinical Trials
Investigators
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Study Director: Kirsten Gruis, MD Edgewise Therapeutics, Inc.
Additional Information:
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Responsible Party: Edgewise Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04585464    
Other Study ID Numbers: EDG-5506-001
First Posted: October 14, 2020    Key Record Dates
Last Update Posted: October 14, 2020
Last Verified: October 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Edgewise Therapeutics, Inc.:
Healthy Volunteers
Becker Muscular Dystrophy
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked