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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05291091




Registration number
NCT05291091
Ethics application status
Date submitted
14/03/2022
Date registered
22/03/2022

Titles & IDs
Public title
Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
Scientific title
A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy
Secondary ID [1] 0 0
EDG-5506-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Becker Muscular Dystrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Sevasemten 10 mg
Treatment: Drugs - Sevasemten 5 mg
Treatment: Drugs - Sevasemten 12.5 mg
Treatment: Drugs - Placebo

Experimental: Adult Cohort 1 - Drug: Sevasemten Drug: Placebo

Experimental: Adult Cohort 2 - Drug: Sevasemten Drug: Placebo

Experimental: Adult Cohort 6 - Drug: Sevasemten Drug: Placebo

Experimental: Adolescent Cohort 4 - Drug: Sevasemten Drug: Placebo

Experimental: Adolescent Cohort 5 - Drug: Sevasemten Drug: Placebo


Treatment: Drugs: Sevasemten 10 mg
Sevasemten is administered orally once per day

Treatment: Drugs: Sevasemten 5 mg
Sevasemten is administered orally once per day

Treatment: Drugs: Sevasemten 12.5 mg
Sevasemten is administered orally once per day

Treatment: Drugs: Placebo
Placebo is administered orally once per day
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of adverse events in those treated with sevasemten or placebo
Timepoint [1] 0 0
12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
Primary outcome [2] 0 0
Severity of adverse events in those treated with sevasemten or placebo
Timepoint [2] 0 0
12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
Primary outcome [3] 0 0
Change from Baseline in serum Creatine Kinase
Timepoint [3] 0 0
12 Months (CANYON Cohorts 1, 2)
Primary outcome [4] 0 0
Change from Baseline in the North Star Ambulatory Assessment scale
Timepoint [4] 0 0
18 months (GRAND CANYON Cohort 6)
Secondary outcome [1] 0 0
Change from Baseline in the protein fast skeletal muscle Troponin I
Timepoint [1] 0 0
12 months (CANYON Cohorts 1, 2), 18 months (GRAND CANYON Cohort 6)
Secondary outcome [2] 0 0
Change from Baseline in the North Star Ambulatory Assessment scale
Timepoint [2] 0 0
12 Months (CANYON Cohorts 1, 2)
Secondary outcome [3] 0 0
Change from Baseline in the North Star Assessment for Limb-Girdle Type Muscular Dystrophies scale
Timepoint [3] 0 0
12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Secondary outcome [4] 0 0
Change from Baseline in the 10-meter walk/run test
Timepoint [4] 0 0
12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Secondary outcome [5] 0 0
Change from Baseline in 100-meter timed test
Timepoint [5] 0 0
12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Secondary outcome [6] 0 0
Change from Baseline in stride velocity (95th percentile)
Timepoint [6] 0 0
18 Months (GRAND CANYON Cohort 6)
Secondary outcome [7] 0 0
Pharmacokinetics as measured by steady state plasma concentration
Timepoint [7] 0 0
12 Months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
Secondary outcome [8] 0 0
Change from Baseline in growth as assessed by height centile on World Health Organization growth charts
Timepoint [8] 0 0
12 months (CANYON Cohorts 4, 5)
Secondary outcome [9] 0 0
Month 18 change from Baseline in fat fraction of upper leg muscles as assessed by Magnetic Resonance Imaging
Timepoint [9] 0 0
18 months (GRAND CANYON Cohort 6)

Eligibility
Key inclusion criteria
The CANYON Study including the adolescent cohorts are fully enrolled.

GRAND CANYON eligibility is listed below.

Key

1. Adults (aged 18 to 50 years, inclusive) with a documented dystrophin mutation and phenotype consistent with Becker muscular dystrophy, and history of being ambulatory beyond 16 years of age without steroids; history of being ambulatory beyond 18 years of age with steroids.
2. Able to complete the 100-meter timed test in < 200 seconds with or without use of mobility aid devices.
3. Able to perform the North Star Ambulatory Assessment scale and achieve a score of 5 to 32, inclusive.

Key
Minimum age
12 Years
Maximum age
50 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
1. Medical history or clinically significant physical examination/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study. This includes contraindications to magnetic resonance imaging such as non-compatible implanted medical devices or severe claustrophobia.
2. Cardiac echocardiogram ejection fraction < 40%
3. Forced vital capacity predicted <60% or using daytime ventilatory support
4. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
5. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
6/07/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2026
Actual
Sample size
Target
170
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
St Vincent's Hospital Melbourne - Fitzroy
Recruitment postcode(s) [1] 0 0
VIC, 3065 - Fitzroy
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Indiana
Country [8] 0 0
United States of America
State/province [8] 0 0
Iowa
Country [9] 0 0
United States of America
State/province [9] 0 0
Kansas
Country [10] 0 0
United States of America
State/province [10] 0 0
Maryland
Country [11] 0 0
United States of America
State/province [11] 0 0
Massachusetts
Country [12] 0 0
United States of America
State/province [12] 0 0
Minnesota
Country [13] 0 0
United States of America
State/province [13] 0 0
Missouri
Country [14] 0 0
United States of America
State/province [14] 0 0
New York
Country [15] 0 0
United States of America
State/province [15] 0 0
North Carolina
Country [16] 0 0
United States of America
State/province [16] 0 0
Ohio
Country [17] 0 0
United States of America
State/province [17] 0 0
Pennsylvania
Country [18] 0 0
United States of America
State/province [18] 0 0
Texas
Country [19] 0 0
United States of America
State/province [19] 0 0
Virginia
Country [20] 0 0
Belgium
State/province [20] 0 0
Gent
Country [21] 0 0
Belgium
State/province [21] 0 0
Leuven
Country [22] 0 0
Belgium
State/province [22] 0 0
Liège
Country [23] 0 0
Denmark
State/province [23] 0 0
Copenhagen
Country [24] 0 0
France
State/province [24] 0 0
Marseille
Country [25] 0 0
France
State/province [25] 0 0
Nantes Cedex
Country [26] 0 0
France
State/province [26] 0 0
Nice Cedex 1
Country [27] 0 0
France
State/province [27] 0 0
Paris
Country [28] 0 0
Germany
State/province [28] 0 0
Munich
Country [29] 0 0
Israel
State/province [29] 0 0
Jerusalem
Country [30] 0 0
Israel
State/province [30] 0 0
Petah Tiqwa
Country [31] 0 0
Italy
State/province [31] 0 0
Milan
Country [32] 0 0
Italy
State/province [32] 0 0
Padova
Country [33] 0 0
Italy
State/province [33] 0 0
Rome
Country [34] 0 0
Netherlands
State/province [34] 0 0
Leiden
Country [35] 0 0
New Zealand
State/province [35] 0 0
Auckland
Country [36] 0 0
Spain
State/province [36] 0 0
Barcelona
Country [37] 0 0
Spain
State/province [37] 0 0
San Sebastian
Country [38] 0 0
Spain
State/province [38] 0 0
Valencia
Country [39] 0 0
United Kingdom
State/province [39] 0 0
London
Country [40] 0 0
United Kingdom
State/province [40] 0 0
Newcastle
Country [41] 0 0
United Kingdom
State/province [41] 0 0
Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Edgewise Therapeutics, Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Medpace, Inc.
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
ImagingNMD
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Commercial sector/industry
Name [3] 0 0
SYSNAV
Address [3] 0 0
Country [3] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Joanne Donovan, MD, PhD
Address 0 0
Edgewise Therapeutics, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Edgewise Therapeutics
Address 0 0
Country 0 0
Phone 0 0
720-262-7002
Fax 0 0
Email 0 0
studies@edgewisetx.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05291091