Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

Conditions

• DMD
• Duchenne
• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Neuromuscular Diseases

Eligibility Criteria

Sex

MALE

Age

7 Years - 9 Years

Healthy Volunteers

Not accepted

Interventions

• SAT-3247 — DRUG
  SAT-3247 is a selective AAK1 inhibitor for oral tablet administration which promotes functional rescue of asymmetric satellite cell division, resulting in the robust production of muscle progenitor cells, subsequent improvement in muscle regeneration, and enhanced muscle function.
• Placebo — DRUG
  matching placebo oral tablets

Study Details

Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and \< 10 years. The trial has two parts. In Part 1, the trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy. In Part 2, the trial will study two doses of SAT-3247 for an additional 9 months.

Key Dates

First listed

Dec 17, 2025

Start date

Dec 8, 2025

Status verified

Aug 2026

Primary completion

Mar 31, 2027

Completion

Feb 28, 2028

Study Design

Enrollment

51 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Active Comparator: SAT-3247 60 mg
  Part 1: SAT-3247 60 mg oral tablets administered daily for 12 weeks. Part 2: SAT-3247 60 mg oral tablets administered daily for an additional 9 months after completing Part 1
• Active Comparator: SAT-3247 120 mg
  Part 1: SAT-3247 120 mg oral tablets administered daily for 12 weeks. Part 2: SAT-3247 120 mg oral tablets administered daily for an additional 9 months after completing Part 1; note the 120 mg dose will not be studied in the US and Canada
• Placebo Comparator: placebo
  Part 1: placebo oral tablets administered daily for 12 weeks

Primary Outcome Measure

Safety of SAT-3247 [ Time Frame: 12 weeks in Part 1 and up to 12 months in part 2 ]

Central Contacts

• Satellos Medical Information
  +1 647-660-1780
  [email protected]

Locations (7)

Find similar trials in Los Angeles, CA

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