Lochmüller Lab

Our clinical research team is excited to share five new clinical trials activated across both pediatric and adult sites in Ottawa, CHEO and TOH. We are recruiting patients with Duchenne Muscular Dystrophy (DMD) (pediatric), Facioscapulohumeral muscular dystrophy (FSHD) (adult), and inflammatory myopathy (adult). We are expecting additional trials for myotonic dystrophy to open in early 2024. A complete list of our current clinical studies is available here. More details about the new opportunities follow below. 

New Trial Opportunities

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER) 

Investigator: Dr. Hugh McMillan 

Sponsor: Dyne 

The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

A Study Of PGN-EDO51 In Participants With Duchene Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51) 

Investigator: Dr. Hugh McMillan 

Sponsor: PepGen Inc 

The primary purpose of the study is to evaluate the safety and tolerability of multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne muscular dystrophy (DMD).

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

Investigator: Dr. Hanns Lochmüller 

Sponsor: Avidity 

This first-in-human, 3-part, multi-center, Phase 1/2, randomized, double-blind, placebo-controlled study isdesigned 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.

Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) – Natural History Study 

Investigator: Dr. Hanns Lochmüller 

Sponsor: University of Kansas Medical Center 

The primary goal of this proposal is to collect motor and functional outcomes specific to FSHD over time. By collecting measures specific to FSHD, this will help ensure the best level of clinical care is being provided. This study also hopes to speed up drug development by gaining a better understanding of how having FSHD impacts motor function and other health outcomes and how big a change in motor function would be clinically meaningful to those with FSHD.

A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. (ALKIVIA) 

Investigator: Dr. Hanns Lochmüller 

Sponsor: Argenx 

This study’s purpose is to measure the treatment response from efgartigimod PH20 SC compared with placebo in participants with Idiopathic Inflammatory Myopathy (IIM). Participants with the IIM subtypes of dermatomyositis, immune-mediated necrotizing myopathy, or certain other subtypes of polymyositis will be included in the study. Treatment response will be measured by Total improvement score.

See All of the Lochmüller Lab’s Trials here.