Clinical research
In clinical research, individuals volunteer to participate in studies that aim to uncover better ways to treat, prevent, diagnose and understand human disease. Our clinical research includes clinical trials, where a new treatment or intervention is tested; patient registries, which collect information about disease epidemiology and specific features; and natural history studies, which monitor patients in detail to discover more about how diseases progress.
Hanns is a highly experienced investigator who has led multiple academic and commercially sponsored clinical research studies in Europe.
We are involved in clinical research at both CHEO (for pediatric studies) and TOH (for adult studies). In 2018 a new neuromuscular research centre opened at the Ottawa Hospital’s Civic Campus, and this provides a state-of-the-art trial facility which will increase the number of trials open to neuromuscular patients from Ottawa and the surrounding area.
NCT04003974 – A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 24-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD)
Role of Dr. Lochmuller: Principal Investigator (Ottawa)
Sponsor: Fulcrum Therapeutics
NCT03692312 – A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy (REACH CDM)
Role of Dr. Lochmuller: Principal Investigator (Ottawa)
Sponsor: AMO Pharma Limited
NCT04281485 – A Phase 3, multicentre, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne Muscular Dystrophy.
Role of Dr. Lochmuller: Principal Investigator (Ottawa)
Sponsor: Pfizer
NCT02500381 - A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
Role of Dr. Lochmuller: Principal Investigator (Ottawa)
Sponsor: Sarepta Therapeutics, Inc.
NCT03179631- A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension
Role of Dr. Lochmuller: Principal Investigator (Ottawa)
Sponsor: PTC Therapeutics
NCT04990388 - A Phase 1/2 First-in-human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses (Part 1: Open-label) and Repeat Doses (Part 2: Randomized, Double-blind, Placebo-controlled) of UX053 in Patients with GSD III
Role of Dr. Lochmuller: Principal Investigator (Ottawa)
Sponsor: Ultragenyx Pharmaceutical Inc
Sponsor: ReveraGen BioPharma, Inc.
NCT02736188: A Phase 3b Open-label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
Role Dr Lochmüller: Chief Investigator
Sponsor: Ultragenyx Pharmaceutical Inc
NCT02377921: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
Role Dr Lochmüller: Chief Investigator (Global)
Sponsor: Ultragenyx Pharmaceutical Inc
NCT01784679: GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
Role Dr Lochmüller: Chief Investigator (UK)
Sponsor: Ultragenyx Pharmaceutical Inc
NCT01302600: Phase II, Multicenter, Randomized, Adaptive, Double-blind, Placebo Controlled Study to Assess Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Year Old Spinal Muscular Atrophy (SMA) Patients.
Role Dr Lochmüller: Principal Investigator (Newcastle)
Sponsor: Trophos
NCT02628743 – Multicenter, Open-Label, Single Arm Study to Evaluate Long-term Safety, Tolerability, and Effectiveness of 10 mg/kg Olesoxime in Patients With SMA
Role Dr Lochmüller: Chief Investigator (UK)
Sponsor: Hoffmann-La Roche
NCT02240355 – A Multicenter, Randomized, Double Blind, Placebo Controlled, Multiple Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO6885247 Following 12 Weeks of Treatment in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)
Role Dr Lochmüller: Chief Investigator (UK)
Sponsor: Hoffmann-La Roche
NCT02118779 – Observational Prolonged Trial in Myotonic Dystrophy Type 1 to Improve Quality of Life Standards, a Target Identification Collaboration (OPTIMISTIC)
Role Dr Lochmüller: Principal Investigator (Newcastle)
Sponsor: Radboud University
NCT00527228 – Deflazacort in Dysferlinopathies (LGMD2B/MM) – a Double Blind, Placebo-controlled Clinical Study
Role Dr Lochmüller: Principal Investigator
Sponsor: Ludwig-Maximilians – University of Munich
NCT02831504– Myotonic Dystrophy type 1 (DM1) deep phenotyping to improve delivery of personalized medicine and assist in the planning, design and recruitment of clinical trials
Role Dr Lochmüller: Principal Investigator (Newcastle)
Sponsor: Newcastle upon Tyne Hospitals NHS Foundation Trust
NCT02858908 – Tideglusib (GSK3b inhibitor) in early-onset myotonic dystrophy
A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400mg Or 1000mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy
Role Dr Lochmüller: Chief Investigator (Newcastle)
Sponsor: AMO Pharma Ltd.
If you are a patient interested in participating in an Ottawa-based clinical trial, please contact coordinator Emilie Hill-Smith.
If you are a pharmaceutical company interested in Ottawa as a participating centre for your study, please contact Dr. Lochmüller directly.
For general inquiries about the Canadian neuromuscular clinical trial landscape from a patient perspective and advice about what it means to participate in clinical research, please contact Muscular Dystrophy Canada.
Investigators, pharmaceutical company representatives and other stakeholders interested in clinical trials in Canada are also encouraged to visit the NMD4C clinical trial concierge site to see how they can support you.
