Campus Alert: Find the latest UMMS campus news and resources at umassmed.edu/coronavirus

Search Close Search
Page Menu

Clinical Trials

Clinical trials DuchenneThe Duchenne Program at UMassMed is committed to providing the best possible care while helping to develop the treatments of tomorrow. Our ultimate goal is a world without Duchenne muscular dystrophy. It takes teamwork, dedication, and careful science to get there, and we are committed to being a part of the solution. To help advance new potential treatments, we’re participating in a variety of clinical trials. If you are interested in any of the clinical trials below, please email DMDresearchprogram@umassmed.edu.

The following clinical trials are active at our site:

0367-CL-0102: Astellas Pharma Inc.

A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD).

This study is comprised of a 4-week pre-treatment screening period, 24-week treatment period and 4-week post-treatment follow-up period. The 24-week treatment period consists of a 12-week double-blind (DB) part and 12-week open-label extension (OLE) part and each part includes a 2 week Low-dose Period and a 10-week High-dose Period.

To learn more details about the trial

To learn more about his investigational drug

Would you like to participate in this trial?


RACER 53: NS Pharma Inc.

A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD).

NS Pharma is conducting a Phase 3 randomized, double-blind, placebo-controlled, multi-center study to assess the efficacy and safety of Viltolarsen in ambulant boys with Duchenne muscular dystrophy. Eligible patients with out-of-frame deletion mutations amenable to exon 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 80 mg/kg Viltolarsen or placebo for up to 48 weeks.

To learn more details about the trial

To learn more about this investigational drug

Would you like to participate in this trial?


IGNITE DMD: Solid Biosciences

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Solid Biosciences is conducting a clinical trial in which the microdystrophin gene is delivered intravenously with a viral vector. Patients will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 2 years. This study is currently on FDA hold.

To learn more details about the trial

To learn more about this investigational medicine


SPITFIRE: Hoffman LaRoche

PHASE 3 STUDY OF RG6206 ANTI-MYOSTATIN ADNECTIN IN PEOPLE WITH DUCHENNE

Pharmaceutical company Hoffman-LaRoche (often known as “Roche”) is testing a myostatin Inhibitor in children with Duchenne to find out if RG6206 is effective, safe and well tolerated in boys with Duchenne. The study includes ambulatory boys with Duchenne ages 6-11 years who are on steroid treatment. The study is coming to a close and we are not currently recruiting patients.