Lochmüller Lab

Two clinical trial opportunities in FSHD at The Ottawa NeuroMuscular Centre now recruiting, FSHD community receive negative topline results from phase 3 losmapimod (REACH) trial.

Last week the Facioscapulohumeral muscular dystrophy (FSHD) community received disappointing news with the negative topline results from Fulcrum Therapeutics’ phase 3 clinical trial of losmapimod (REACH).

The REACH trial was the largest ever in FSHD, and included sites across Canada in Montreal, Calgary, and Ottawa. While the results from this study did not display the outcome that many were hoping for, the organization and delivery of the study from Fulcrum was commendable and the full study results will be shared to ensure that data from this trial benefits others developing treatments for FSHD.

The Canadian neuromuscular community will continue to provide opportunities for Canadians with FSHD to participate in clinical trials, and The NeuroMuscular Centre at The Ottawa Hospital is pleased to share two clinical trial opportunities for patients with FSHD under Dr Lochmuller as principal investigator. We will soon be recruiting additional participants for the Phase 1/​2 Study of AOC 1020 in Adults With FSHD (FORTITUDE), sponsored by Avidity Biosciences. The Study to Evaluate the Efficacy and Safety of Satralizumab in FSHD1 (REINFORCE), sponsored by Centre Hospitalier Universitaire de Nice, is currently open for recruitment.

Phase 1/ 2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FORTITUDE)

This first-in-human, 3-part, multi-center, Phase 1/2, randomized, double-blind, placebo-controlled study is designed to evaluate safety, tolerability, pharmacokinetics and to explore pharmacodynamics and efficacy of single and multiple-doses of AOC 1020 in adult participants with FSHD Type 1 and FSHD Type 2. The treatment tested is an antisense oligonucleotide against DUX4 that is given by intravenous infusion.

We are currently the sole Canadian site for the FORTITUDE study and are excited to open recruitment for a third cohort of patients after completing registration of two cohorts earlier this year.

For referrals and enquiries, please contact Jessica MacGregor, research coordinator at .

Study to Evaluate the Efficacy and Safety of Satralizumab in FSHD1 (REINFORCE)

The is a phase 2, interventional study to explore the satralizumab, an IL6-receptor (IL6-R) antagonist, for its efficacy in specifically reducing muscle and systemic inflammation. By antagonizing IL-6R downstream signaling, satralizumab holds promise in mitigating inflammation and potentially curtailing fibrofatty degeneration in FSHD. Satralizumab is a repurposed drug manufactured by Roche that is licensed in Canada for a different indication.

We are the sole Canadian site for the REINFORCE study, which is currently open for recruitment at the Ottawa Neuromuscular Centre, located at the Civic campus of The Ottawa Hospital. For referrals and enquiries, please contact Jessica MacGregor, research coordinator at .

Lochmüller Lab Clinical Research

You can read more about the Lochmüller lab’s clinical research activities, and see a full listing of our ongoing trials!