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

Part of paid clinical trials in Los Angeles, California.

Sponsor
Satellos Bioscience, Inc.
Study ID
NCT06867107
Phase
PHASE2
Status
Enrolling By Invitation

Conditions

  • Duchenne Muscular Dystrophy (DMD)

Eligibility Criteria

Sex
MALE
Age
16 Years - 40 Years
Healthy Volunteers
Not accepted

Interventions

  • SAT-3247 — DRUG
    AAK1 inhibitor

Study Details

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 25 months- for a total of 24 months of treatment including the duration of the SAT-3247-CL-101 study. The study will enroll up to 30 participants including those that previously participated in the SAT-3247-CL-101 study.

Key Dates

First listed
Mar 10, 2025
Start date
Aug 20, 2025
Status verified
Sep 2026
Primary completion
Sep 30, 2027
Completion
Sep 30, 2027

Study Design

Enrollment
30 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

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

Primary Outcome Measure

Treatment emergent adverse events [ Time Frame: 24 months ]

Locations (3)

FacilityCityStateZIPSite coordinators
University of California, Los AngelesLos AngelesCalifornia90095-
Nationwide Children's HospitalColumbusOhio43205-
St. Jude Children's Research HospitalMemphisTennessee38105-

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