Trial Review

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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06131983




Registration number
Help: Registration number
NCT06131983
Ethics application status
Help: Ethics application status
Date submitted
Help: Date submitted
8/11/2023
Date registered
Help: Date registered
15/11/2023
Date last updated
Help: Date last updated
5/10/2026

Titles & IDs
Public title
Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1
Scientific title
A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 (SRP-1001) in Adult Patients and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1
Additional trial identifiers [1] 0 0
2023-509748-89-00
Additional trial identifiers [2] 0 0
SRP-1001-101
Universal Trial Number (UTN)
Trial acronym
Related trial records

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

Intervention/exposure
Study type
Interventional
Description of the intervention or exposure
Treatment: Drugs - SRP-1001 for Injection
Treatment: Drugs - Placebo

Experimental: SRP-1001 - SRP-1001 for injection

Placebo comparator: Placebo - Sodium chloride (0.9%)


Treatment: Drugs: SRP-1001 for Injection
Single or multiple doses of SRP-1001 by intravenous (IV) infusion

Treatment: Drugs: Placebo
Calculated volume to match active treatment by IV infusion
Intervention code [1] 0 0
Treatment: Drugs
Description of the comparator or control
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment-Emergent Adverse Events Over Time Through End of Study
Timepoint [1] 0 0
Part 1: Up to Day 90; Part 2: Up to Day 360
Secondary outcome [1] 0 0
PK of SRP-1001: Maximum Observed Plasma Concentration (Cmax)
Timepoint [1] 0 0
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
Secondary outcome [2] 0 0
PK of SRP-1001: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)
Timepoint [2] 0 0
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
Secondary outcome [3] 0 0
PK of SRP-1001: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)
Timepoint [3] 0 0
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
Secondary outcome [4] 0 0
PK of SRP-1001: Area Under the Plasma Concentration Versus Time from Zero to Infinity (AUCinf)
Timepoint [4] 0 0
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
Secondary outcome [5] 0 0
PK of SRP-1001: Terminal Elimination Half-Life (t1/2)
Timepoint [5] 0 0
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
Secondary outcome [6] 0 0
PK of SRP-1001: Systemic Clearance (CL)
Timepoint [6] 0 0
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
Secondary outcome [7] 0 0
PK of SRP-1001: Volume of Distribution (Vss)
Timepoint [7] 0 0
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
Secondary outcome [8] 0 0
PK of SRP-1001: Recovery of Unchanged Drug in Urine Over 0-24 Hours (Amount Excreted: Ae)
Timepoint [8] 0 0
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
Secondary outcome [9] 0 0
PK of SRP-1001: Fraction of Drug Excreted in Urine as Percent of IV Dose (Fe)
Timepoint [9] 0 0
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
Secondary outcome [10] 0 0
PK of SRP-1001: Renal Clearance (CLr)
Timepoint [10] 0 0
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose

Eligibility
Key inclusion criteria
Key

* Genetically confirmed FSHD1 based on screening evaluation or source verifiable medical record
* Clinical severity score between 3 and 8 (scale, 0 to 10)
* Must have an eligible lower extremity muscle for biopsy as determined from MRI by a central reader, with muscle fat fraction =10% and less than approximately 40%
* Males or nonpregnant, nonlactating females =18 years of age who do not plan to become pregnant during the study, with an upper age limit of =70 years
* Able and willing to provide written informed consent prior to the performance of any study specific procedures
* Participants with a body mass index (BMI) between 18.0 and 35.0 kilograms/square meter, inclusive. A participant with FSHD1 and a BMI outside this range may be allowed into the study at the discretion of the principal investigator.
* Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader
* A 12-lead electrocardiogram at screening with no abnormalities that may compromise participant's safety in the study
* Participants of childbearing potential and their partners must use highly effective contraception during the study and for at least 9 months following the end of study or last dose of study medication, whichever is later. Males must not donate sperm during the study from Day 1 until at least 9 months following the end of study or last dose of study medication, whichever is later.

Key
Minimum age
16 Years
Maximum age
70 Years
Sex
Both males and females
Healthy volunteers
No
Key exclusion criteria
* Human immunodeficiency virus (HIV) infection as shown by presence of anti-HIV antibody (seropositive) at screening
* Seropositive for hepatitis B or hepatitis C at screening
* Uncontrolled hypertension
* Severe cardiovascular disease
* History of thrombolic events
* Platelet count less that the lower limit of normal at screening
* History or presence of: a hypercoagulable state, nephrotic range proteinuria, antiphospholipid antibody syndrome, myeloproliferative disease, inability to ambulate, use of hormone-based contraceptives.
* Any contraindication to muscle biopsy or MRI

Note: additional inclusion/exclusion criteria may apply per protocol

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
Parallel
Other design features
Phase
Phase 1
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
19/06/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/12/2028
Actual
Sample size
Target
60
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Liverpool Hospital - Liverpool
Recruitment hospital [2] 0 0
Wesley Research Institute - Auchenflower
Recruitment hospital [3] 0 0
Sunshine Coast University Hospital - Birtinya
Recruitment hospital [4] 0 0
Alfred Health - Melbourne
Recruitment postcode(s) [1] 0 0
2170 - Liverpool
Recruitment postcode(s) [2] 0 0
4066 - Auchenflower
Recruitment postcode(s) [3] 0 0
4575 - Birtinya
Recruitment postcode(s) [4] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
Alberta
Country [2] 0 0
Canada
State/province [2] 0 0
Quebec
Country [3] 0 0
Germany
State/province [3] 0 0
München
Country [4] 0 0
Germany
State/province [4] 0 0
Ulm
Country [5] 0 0
Italy
State/province [5] 0 0
Milan
Country [6] 0 0
Italy
State/province [6] 0 0
Roma
Country [7] 0 0
Netherlands
State/province [7] 0 0
Leiden
Country [8] 0 0
New Zealand
State/province [8] 0 0
Auckland
Country [9] 0 0
Spain
State/province [9] 0 0
Barcelona
Country [10] 0 0
Spain
State/province [10] 0 0
Madrid
Country [11] 0 0
Spain
State/province [11] 0 0
Valencia

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Sarepta Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Sarepta Therapeutics, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4
Address 0 0
Country 0 0
Phone 0 0
1-888-SAREPTA (1-888-727-3782)
Fax 0 0
Email 0 0
[email protected]
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/NCT06131983