We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04585464
Recruitment Status : Completed
First Posted : October 14, 2020
Last Update Posted : June 28, 2022
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 42-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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 127 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, and PK of EDG-5506 in Adult Healthy Volunteers and Adults With Becker Muscular Dystrophy
Actual Study Start Date : October 12, 2020
Actual Primary Completion Date : November 15, 2021
Actual Study Completion Date : December 27, 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-5506 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-5506 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-5506 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 42 days of monitoring ]
  2. Incidence of abnormal laboratory test results (clinical chemistry, hematology, urinalysis, coagulation) [ Time Frame: Up to 42 days of monitoring ]
  3. Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG) [ Time Frame: Up to 42 days of monitoring ]
  4. Incidence of abnormal vital signs [ Time Frame: Up to 42 days of monitoring ]
  5. Incidence of abnormal physical exam findings [ Time Frame: Up to 42 days of monitoring ]

Secondary Outcome Measures :
  1. Plasma maximum measured drug concentration (Cmax) [ Time Frame: Up to 42 days of testing ]
  2. Time of maximum concentration (Tmax) [ Time Frame: Up to 42 days of testing ]
  3. Area under the concentration-time curve (AUC) [ Time Frame: Up to 42 days of testing ]
  4. Plasma half-life (T½) [ Time Frame: Up to 42 days of testing ]
  5. Renal clearance (CLR) [ Time Frame: Up to 42 days of testing ]
  6. Drug excreted unchanged in urine (Amt0-24) [ Time Frame: Up to 42 days of testing ]
  7. Fraction excreted in urine (Fe) [ Time Frame: Up to 42 days of testing ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Healthy volunteers: Male and non-childbearing potential females. Male Becker muscular dystrophy (BMD) participants
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • For all potential participants (Healthy volunteers and BMD): Male or for HV: female. For all: adults aged 18 to 55 years at time of consent.
  • For HVs: Good general health, with no significant medical history, no clinically significant abnormalities on physical exam
  • For BMD: Diagnosis of BMD based on documentation of mutation(s) in the dystrophin gene and BMD phenotype
  • For BMD: Ability to ambulate
  • For all: Weight greater than or equal to 50 kg and BMI less than 33 kg/m2
  • For HV: Females must be of non-childbearing potential.
  • For all: Males with female partners must use a medically accepted contraceptive regimen from first dose through 90 days after the last dose
  • For all: Non-smoker and must not have used any tobacco products within 3 months prior to the Screening visit.
  • For all: Able and willing to attend the necessary visits at the study center.

Exclusion Criteria:

  • For all: 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.
  • For all: Unable to refrain from strenuous exercise for 3 days prior to check-in and during study.
  • For all: Participation 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
Layout table for location information
United States, Texas
Worldwide Clinical Trials
San Antonio, Texas, United States, 78217
Sponsors and Collaborators
Edgewise Therapeutics, Inc.
Worldwide Clinical Trials
Investigators
Layout table for investigator information
Study Director: Medical Director Edgewise Therapeutics, Inc.
Additional Information:
Layout table for additonal information
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: June 28, 2022
Last Verified: June 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
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:
Layout table for MeSH terms
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked