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First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort

NCT06565208 · Status: COMPLETED · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 77

Last updated 2025-05-15

No results posted yet for this study

Summary

This is a first-in-human (FIH), Phase 1 study of orally administered SAT-3247 in healthy adult volunteers (HVs) and adult participants with DMD to determine safety, tolerability, pharmacokinetics and pharmacodynamics.

Conditions

Interventions

DRUG

SAT-3247

SAT-3247 is an oral tablet that is a potent, muscle penetrant, small molecule inhibitor of AAK1; inhibition of AAK1 rescues perturbed asymmetric division of satellite stem cells, resulting in increased muscle regeneration in animal models of DMD. In vitro and in vivo animal pharmacology studies have demonstrated the efficacy of SAT-3247 in improving muscle strength and the necessary target coverage to maximize functional muscle improvement.

DRUG

matched placebo

matched placebo

Sponsors & Collaborators

  • Satellos Bioscience, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
40 Years
Sex
MALE
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2024-08-21
Primary Completion
2025-04-28
Completion
2025-04-28

Countries

  • Australia

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06565208 on ClinicalTrials.gov