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Trial record 1 of 6 for:    edg-5506
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A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy (ARCH)

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: NCT05160415
Recruitment Status : Active, not recruiting
First Posted : December 16, 2021
Last Update Posted : November 3, 2022
Sponsor:
Collaborator:
Medpace, Inc.
Information provided by (Responsible Party):
Edgewise Therapeutics, Inc.

Study Description
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Brief Summary:

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

EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers.


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

Detailed Description:

This open-label study will evaluate the safety, tolerability, and pharmacokinetics (PK) of EDG-5506 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 EDG-5506. 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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1b, Open-label Study of the Safety and Pharmacokinetics of EDG-5506 in Adults With Becker Muscular Dystrophy
Actual Study Start Date : December 28, 2021
Estimated Primary Completion Date : April 2024
Estimated Study Completion Date : April 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Treatment
Drug: EDG-5506
 Drug: EDG-5506
Daily oral dose


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of AEs in those treated with EDG-5506 [ Time Frame: 25 Months ]
  2. Frequency of AEs in those treated with EDG-5506 [ Time Frame: 25 Months ]
  3. Severity of AEs in those treated with EDG-5506 [ Time Frame: 25 Months ]

Secondary Outcome Measures :
  1. Incidence of treatment-emergent abnormal clinical chemistry test results [ Time Frame: 24 Months ]
  2. Incidence of treatment-emergent abnormal hematology test results [ Time Frame: 24 Months ]
  3. Incidence of treatment-emergent abnormal coagulation test results [ Time Frame: 24 Months ]
  4. Incidence of treatment-emergent abnormal urinalysis test results [ Time Frame: 24 Months ]
  5. Number of participants with changes in clinical chemistry [ Time Frame: 24 Months ]
  6. Number of participants with changes in hematology [ Time Frame: 24 Months ]
  7. Number of participants with changes in coagulation [ Time Frame: 24 Months ]
  8. Number of participants with changes in urinalysis [ Time Frame: 24 Months ]
  9. Number of participants with changes in vital signs [ Time Frame: 24 Months ]
  10. Number of participants with changes in physical examination [ Time Frame: 24 Months ]
  11. Number of participants with changes in ECG PR Interval [ Time Frame: 24 Months ]
  12. Number of participants with changes in ECG QRS Interval [ Time Frame: 24 Months ]
  13. Number of participants with changes in ECG QT Interval [ Time Frame: 24 Months ]
  14. Number of participants with changes in ECG QTc Interval [ Time Frame: 24 Months ]
  15. Number of participants with changes in FVC [ Time Frame: 24 Months ]
    Assessed by spirometry

  16. Number of participants with changes in FEV1 [ Time Frame: 24 Months ]
    As assessed by spirometry


Other Outcome Measures:
  1. Number of participants with changes in biomarkers of muscle fiber damage [ Time Frame: 24 Months ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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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): NCT05160415


Locations
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United States, Georgia
Rare Disease Research
Atlanta, Georgia, United States, 30329
Sponsors and Collaborators
Edgewise Therapeutics, Inc.
Medpace, Inc.
Investigators
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Study Chair: Sam Collins, MBBS, PhD Edgewise Therapeutics, Inc.
More Information
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Additional Information:

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Responsible Party: Edgewise Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05160415    
Other Study ID Numbers: EDG-5506-002
First Posted: December 16, 2021    Key Record Dates
Last Update Posted: November 3, 2022
Last Verified: October 2022
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.:
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