RESOURCES & POLICIES
Clinical Trials
INSPIRE DUCHENNE
Duchenne muscular dystrophy (Duchenne)
SGT-003: A Phase 1/2 clinical trial of an investigational gene therapy treatment for Duchenne
A multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne.
Active and recruiting
Location: U.S., Canada, Italy, and the U.K.
IMPACT DUCHENNE
Duchenne muscular dystrophy (Duchenne)
SGT-003: A Phase 3 clinical trial of an investigational gene therapy treatment for Duchenne
A multicenter, randomized, double-blind, placebo-controlled study to investigate the efficacy of a single intravenous (IV) infusion of SGT-003 in pediatric ambulant male participants with Duchenne.
Active and recruiting
Location: Outside the U.S. (Australia, Canada, with planned expansion into additional countries, subject to receipt of regulatory approvals)
Falcon Study
Friedreich's ataxia (FA)
SGT-212: A Phase 1b clinical trial of an investigational gene therapy treatment for FA
A first in-human, open-label, dose-finding study to investigate the safety and tolerability of SGT-212, administered via intradentate nuclei (IDN) and intravenous (IV) infusion routes, in participants with FA.
Active and recruiting
Location: U.S.
Artemis Study
Catecholaminergic polymorphic ventricular tachycardia (CPVT)
SGT-501: A Phase 1b clinical trial of an investigational gene therapy treatment for CPVT
A multicenter, open-label, dose-finding study to investigate the safety and tolerability of a single intravenous (IV) infusion of SGT-501 in participants with CPVT.
Active and recruiting
Location: U.S. and Canada
The investigational products described on this website are investigational therapies that have not been approved by the U.S. Food and Drug Administration (FDA) or any other regulatory authority. The information provided is for general informational purposes only and is not intended to diagnose, treat, cure, or prevent any disease, nor does it constitute medical advice. Participation in a clinical trial involves risks and may not provide any direct medical benefit. Patients should consult with their healthcare provider to determine whether participation in a clinical trial is appropriate. Any descriptions of clinical development plans or expectations are subject to risks and uncertainties, and actual results may differ materially.