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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06867107




Registration number
NCT06867107
Ethics application status
Date submitted
4/03/2025
Date registered
10/03/2025

Titles & IDs
Public title
A Long-term Follow-up Study for Participants That Completed the SAT-3247-CL-101 Study
Scientific title
An Open-Label Long-Term Follow-up Study of SAT-3247 in Patients with Duchenne Muscular Dystrophy (DMD) That Participated in SAT-3247-CL-101
Secondary ID [1] 0 0
SAT-3247-LT-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy (DMD) 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 - SAT-3247

Experimental: Treatment Arm - SAT-3247 60 mg administered orally in a 5-days on/2-days off (weekday) dosing regimen


Treatment: Drugs: SAT-3247
AAK1 inhibitor
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and tolerability
Timepoint [1] 0 0
11 months
Secondary outcome [1] 0 0
SAT-3247 effect on fat fraction in biceps brachii
Timepoint [1] 0 0
12 months
Secondary outcome [2] 0 0
SAT-3247 effects on muscle force
Timepoint [2] 0 0
12 months
Secondary outcome [3] 0 0
Potential for improvement in muscle function with treatment of SAT-3247
Timepoint [3] 0 0
12 months

Eligibility
Key inclusion criteria
* Previously participated in the SAT-3247-CL-101 parent clinical trials.
* Continued status of stable glucocorticosteroid dose or no glucocorticosteroid dose from parent clinical trial.
* Continued stable doses of prescription medicines (excluding glucocorticosteroids) and over-the-counter medicines and/or herbal supplements for supportive care from parent clinical trial.
* Ability to understand the nature of the trial and any hazards of participating.
* Ability to communicate satisfactorily with the investigator and physiotherapist and to participate in and comply with the requirements of the entire trial including scheduled visits, procedures, laboratory tests, questionnaires, wearable devices, and study restrictions.
* Willingness to give written consent or assent (if not of cognitive capacity of consent in the jurisdiction where the study is being conducted) and parent/legal guardian willing to give written consent to participate (if participant is not of cognitive capacity to consent) after reading the information and consent form, and after having the opportunity to discuss the trial with the investigator or their delegate.
* All participants, if sexually active, agree to follow the contraception requirements and sperm donation limitations of the trial as described in the protocol.
Minimum age
18 Years
Maximum age
40 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* Presence of acute medical condition, chronic illness or history of chronic illness (other than DMD) sufficient to invalidate the participant's participation in the trial or make it unnecessarily hazardous in the judgment of the investigator.
* Participants expected to require spine surgeries or hospitalizations for non-acute health needs within 12 months.
* Participants with acute gastrointestinal symptoms (e.g., nausea, vomiting, diarrhea, heartburn) or acute infection (such as influenza) or a significant infection or known inflammatory process at Screening.
* Severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator.
* Development of symptomatic cardiomyopathy since completion of the parent trial.
* Inability to swallow tablets.

a. Tablets can be split or crushed and stirred into flavored beverages or food (e.g., apple sauce, yogurt) followed by immediate administration.
* Receipt of an investigational product (including prescription medicines and investigational devices) as part of another clinical trial since completion of the parent trial or in the follow-up period of another clinical trial at the time of Screening for this study.

a. Use of deflazacort or vamorolone in jurisdictions where these are considered investigational as they have not received health authority marketing authorization will not be exclusionary.
* Possibility that the participant will not cooperate with the requirements of the protocol or is unable or unwilling to comply with the study requirements according to investigator's decision.
* Employee, contractors, or consultants of the Sponsor, the CRO, and/or study site or their relatives.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/05/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/06/2026
Actual
Sample size
Target
10
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
St. Vincent Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
- Melbourne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Satellos Bioscience, 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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Satellos Medical Affairs/Clinical Development
Address 0 0
Country 0 0
Phone 0 0
+61 3 8736 1750
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


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/NCT06867107