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

Primary Purpose

Becker Muscular Dystrophy

Status

Active

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

EDG-5506

About this trial

This is an interventional treatment trial for Becker Muscular Dystrophy focused on measuring Becker Muscular Dystrophy

Eligibility Criteria

18 Years - 55 Years (Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Participants who have completed Study EDG-5506-001.
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:

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.
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.

Sites / Locations

Rare Disease Research

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment

Arm Description

Drug: EDG-5506

Outcomes

Primary Outcome Measures

Incidence of AEs in those treated with EDG-5506

Frequency of AEs in those treated with EDG-5506

Severity of AEs in those treated with EDG-5506

Secondary Outcome Measures

Incidence of treatment-emergent abnormal clinical chemistry test results

Incidence of treatment-emergent abnormal hematology test results

Incidence of treatment-emergent abnormal coagulation test results

Incidence of treatment-emergent abnormal urinalysis test results

Number of participants with changes in clinical chemistry

Number of participants with changes in hematology

Number of participants with changes in coagulation

Number of participants with changes in urinalysis

Number of participants with changes in vital signs

Number of participants with changes in physical examination

Number of participants with changes in ECG PR Interval

Number of participants with changes in ECG QRS Interval

Number of participants with changes in ECG QT Interval

Number of participants with changes in ECG QTc Interval

Number of participants with changes in FVC

Assessed by spirometry

Number of participants with changes in FEV1

As assessed by spirometry

Full Information

NCT ID

NCT05160415

First Posted

December 2, 2021

Last Updated

October 28, 2022

Sponsor

Edgewise Therapeutics, Inc.

Collaborators

Medpace, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT05160415

Brief Title

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

Acronym

ARCH

Official Title

A Phase 1b, Open-label Study of the Safety and Pharmacokinetics of EDG-5506 in Adults With Becker Muscular Dystrophy

Study Type

Interventional

2. Study Status

Record Verification Date

October 2022

Overall Recruitment Status

Active, not recruiting

Study Start Date

December 28, 2021 (Actual)

Primary Completion Date

April 2024 (Anticipated)

Study Completion Date

April 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Edgewise Therapeutics, Inc.

Collaborators

Medpace, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Becker Muscular Dystrophy

Keywords

Becker Muscular Dystrophy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

12 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Treatment

Arm Type

Experimental

Arm Description

Drug: EDG-5506

Intervention Type

Drug

Intervention Name(s)

EDG-5506

Intervention Description

Daily oral dose

Primary Outcome Measure Information:

Title

Incidence of AEs in those treated with EDG-5506

Time Frame

25 Months

Title

Frequency of AEs in those treated with EDG-5506

Time Frame

25 Months

Title

Severity of AEs in those treated with EDG-5506

Time Frame

25 Months

Secondary Outcome Measure Information:

Title

Incidence of treatment-emergent abnormal clinical chemistry test results

Time Frame

24 Months

Title

Incidence of treatment-emergent abnormal hematology test results

Time Frame

24 Months

Title

Incidence of treatment-emergent abnormal coagulation test results

Time Frame

24 Months

Title

Incidence of treatment-emergent abnormal urinalysis test results

Time Frame

24 Months

Title

Number of participants with changes in clinical chemistry

Time Frame

24 Months

Title

Number of participants with changes in hematology

Time Frame

24 Months

Title

Number of participants with changes in coagulation

Time Frame

24 Months

Title

Number of participants with changes in urinalysis

Time Frame

24 Months

Title

Number of participants with changes in vital signs

Time Frame

24 Months

Title

Number of participants with changes in physical examination

Time Frame

24 Months

Title

Number of participants with changes in ECG PR Interval

Time Frame

24 Months

Title

Number of participants with changes in ECG QRS Interval

Time Frame

24 Months

Title

Number of participants with changes in ECG QT Interval

Time Frame

24 Months

Title

Number of participants with changes in ECG QTc Interval

Time Frame

24 Months

Title

Number of participants with changes in FVC

Description

Assessed by spirometry

Time Frame

24 Months

Title

Number of participants with changes in FEV1

Description

As assessed by spirometry

Time Frame

24 Months

Other Pre-specified Outcome Measures:

Title

Number of participants with changes in biomarkers of muscle fiber damage

Time Frame

24 Months

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

55 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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. 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Sam Collins, MBBS, PhD

Organizational Affiliation

Edgewise Therapeutics, Inc.

Official's Role

Study Chair

Facility Information:

Facility Name

Rare Disease Research

City

Atlanta

State/Province

Georgia

ZIP/Postal Code

30329

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Links:

URL

http://www.edgewisetx.com

Description

Sponsor Website

Learn more about this trial

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

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