Condition: Myotonic Dystrophy · Sponsor: Sanofi
This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1). The purpose of this study is to evaluate the safety and efficacy of SAR446268 in knocking down dystrophia myotonica protein kinase (DMPK) messenger ribonucleic acid (mRNA) levels and improving neuromuscular function in DM1 participants receiving a single intravenous (IV) administration of SAR446268. The study consists of a dose escalation part (Part A) during which single ascending doses of SAR446268 will be evaluated in 3 distinct cohorts and an optional fourth dose cohort. Once a safe and effective dose is identified, additional participants will be treated in Part B, the dose expansion phase of the study. The study duration will be 112 weeks (approximately 2 years) for each participant in Parts A and B respectively and includes an optional pre-screening period, approximately 8-week screening phase and a 104-week follow-up period post-SAR446268 administration.
This description comes directly from the study's public registry record.
Trial Transparency email recommended (Toll free for US & Canada) · 800-633-1610 · contact-us@sanofi.com
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| University of Florida, 2004 Mowry Road - Site Number: 8400005 | Gainesville, Florida, United States | Recruiting |
| University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001 | Tampa, Florida, United States | Recruiting |
| Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003 | New York, New York, United States | Recruiting |
| Virginia Commonwealth University Medical Center- Site Number : 8400006 | Richmond, Virginia, United States | Recruiting |
| Hospital Italiano de Buenos Aires, Juan Domingo Peron 4190 - Site Number: 0320001 | Buenos Aires, Argentina | Recruiting |
| Investigational Site Number : 0360001 | Brisbane, Queensland, Australia | Recruiting |
| The Montreal Neurological Institute and Hospital, 3801 rue University - Site Number: 1240001 | Montreal, Quebec, Canada | Recruiting |
| Investigational Site Number : 3760002 | Ramat Gan, Israel | Recruiting |
| Investigational Site Number : 8260002 | Newcastle upon Tyne, United Kingdom | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06844214