An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101

NCT06867107 | Enrolling By Invitation Phase 2

• 1 other identifier: SAT-3247-LT-001
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 2

Brief Summary

This is an open-label long-term safety and efficacy study of orally administered SAT-3247 in patients with DMD that previously participated in SAT-3247-CL-101. The study will assess the long-term safety, tolerability and potential efficacy of long-term dosing of 60 mg of orally administered SAT-3247 in a 5-days on/2-days off (i.e. weekday dosing) regimen in an open-label design through 11 months- for a total of 12 months of treatment including the duration of the SAT-3247-CL-101 study. The study will enroll up to 10 participants that previously participated in the SAT-3247-CL-101 study.

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

muscle regeneration; SAT-3247

Outcome Measures

Primary Outcomes (1)

• Treatment emergent adverse events

  Incidence, temporal profile, and severity of treatment emergent adverse events (TEAEs)

  11 months

Secondary Outcomes (3)

• SAT-3247 effect on fat fraction in biceps brachii

  12 months

• SAT-3247 effects on muscle force

  12 months

• Potential for improvement in muscle function with treatment of SAT-3247

  12 months

Study Arms (1)

Treatment Arm

EXPERIMENTAL

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

Drug: SAT-3247

Interventions

SAT-3247 DRUG

AAK1 inhibitor

Treatment Arm

Eligibility Criteria

• Age: 18 Years - 40 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Satellos Bioscience, Inc.: lead

Study Sites (2)

The Royal Children's Hospital

Melbourne, Victoria, 3052, Australia

Location

St. Vincent Hospital

Melbourne, Victoria, Australia

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: open-label study of 60 mg SAT-3247 in 5-days on/2-days off (i.e., weekday dosing) regimen

Study Record Dates

• First Submitted: March 4, 2025
• First Posted: March 10, 2025
• Study Start: August 20, 2025
• Primary Completion (Estimated): August 30, 2026
• Study Completion (Estimated): August 30, 2026
• Last Updated: April 23, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

AU (2)