Open-Label Extension of EDG-5506 in Participants with Becker Muscular Dystrophy

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Edgewise Therapeutics Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05], Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

22 Feb 2024 → ongoing

Decision date (initial)

2025-09-23

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Edgewise Therapeutics

External identifiers

EU CT number

2023-506231-15-00

WHO UTN

U1111-1294-7667

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety, Efficacy

To assess the safety and tolerability of long-term treatment of sevasemten

Secondary objectives 1

1. To assess the change in individual safety parameters

Conditions and MedDRA coding

Becker Muscular Dystrophy

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-003394-PIP01-23

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. 1. Males with a diagnosis of BMD and participation in EDG-5506-002, EDG-5506-201, or EDG-5506-202 (Note: Adolescents will be enrolled in countries with regulatory approval to enroll individuals in that age range). Participants are eligible if they complete the respective prior study visits as follows: − EDG-5506-002: Complete the final study Visit 27 [Month 24]; or, completion of the ET visit prior to Visit 27 [Month 24] − EDG-5506-201: Complete the final study visit (Cohorts 1, 2, 4, and 5: Visit 12 [Month 12]; Cohort 6: Visit 11 [Month 18]) − EDG-5506-202: Complete at least 36 weeks of open-label treatment (Visit 14 [Week 52])
2. 2.Willing to comply with contraception requirements described in the protocol (Section 5.3.1.)
3. 3. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

Exclusion criteria 7

1. 1. Medical Conditions: Any clinically significant changes during or following participation in EDG-5506-002, EDG-5506-201, or EDG-5506-202 that would affect the potential safety of the participant to receive sevasemten.
2. 2. Prior/Concomitant Therapy: Receiving strong cytochrome P450 (CYP) 3A4 inhibitors or moderate/strong CYP3A4 inducers.
3. 3. Prior/Concomitant Therapy: Receipt of an investigational drug other than sevasemten within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.
4. 4. Prior/Concomitant Therapy: Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months. − Receipt of systemic or topical low dose (equivalent to ≤5 mg prednisone daily total dose)corticosteroids for indications other than BMD or receipt for a short duration (≤10 days in previous 6 months), along with receipt of chronic inhaled/intranasal steroids, is permitted.
5. 5. Other Exclusions: Participants who are unlikely to comply with the study protocol or, in the opinion of the Investigator, would not be a suitable candidate for participation in the study.
6. 6. Other Exclusions: Participants with a known allergy to sevasemten or its excipients.
7. 7. Other Exclusions: Participants who are submitted to an institution by virtue of an order of a court or government authority to comply with Section 40a No. 2 of the German Medical Products Act.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 1. Incidence, frequency, and severity of TEAEs and SAEs

Secondary endpoints 2

1. 1. Incidence laboratory test-related TEAEs
2. 2. Change from Baseline in: − Safety labs − Vital Signs − ECG parameters − Cardiac function as assessed by an echocardiogram − Pulmonary function as assessed by FEV1, FVC

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Edgewise Therapeutics Inc.

Sponsor organisation

Edgewise Therapeutics Inc.

Address

1715 38th Street

City

Boulder

Postcode

80301-2603

Country

United States

Scientific contact point

Organisation

Edgewise Therapeutics Inc.

Contact name

Edgewise Clinical Studies

Public contact point

Organisation

Edgewise Therapeutics Inc.

Contact name

Edgewise Clinical Studies

Third parties 1

Locations

7 EU/EEA countries · 17 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 70 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

15 submissions — initial application plus substantial / non-substantial modifications