CURRENT RECRUITING TRIALS
For more information, please contact nm.neurocru@mcgill.ca
1821-FSH-301 Losmapimod (FSHD)
This is a study to evaluate the safety and efficacy of losmapimod in treating patients with Facioscapulohumeral Muscular Dystrophy (FSHD) over 48 weeks.
CRU-SCIG (gMyasthenia Gravis)
Switch from intravenous to subcutaneous immunoglobulin in patients with myasthenia gravis (MG) and chronic inflammatory demyelinating polyneuropathy (CIDP): A single-arm, open-label, prospective pilot study to determine efficacy, treatment satisfaction, health care resource utilization and tolerability
The aim of this study will investigate efficacy, treatment satisfaction, health care resource utilization and tolerability of SCIg in MG and CIDP patients previously treated with IVIg. . The data gathered during the study may also be useful for medical professionals and researchers in the future.
MOM-M281-011 (Nipocalimab - gMyasthenia Gravis)
A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis
Myasthenia gravis (MG) is a rare, heterogeneous, neuromuscular disease characterized by fluctuating, fatigable muscle weakness. MG is caused by pathogenic autoantibodies that impair cholinergic transmission in the postsynaptic membrane at the neuromuscular junction and impair or prevent muscle contraction.
Nipocalimab (also referred to as JNJ-80202135 or M281) is a fully human, aglycosylated immunoglobulin (Ig)G1 monoclonal antibody (mAb) designed to selectively bind, saturate, and block the IgG binding site on the endogenous neonatal Fc receptor (FcRn).
This study will consist of a screening phase (up to 4 weeks), treatment phase (a 24-week double-blind placebo-controlled phase, and an open-label extension [OLE] phase [up to 2 years]) and a follow-up safety visit (up to 8 weeks after last infusion of study intervention). Efficacy evaluations will include assessments such as Myasthenia Gravis – Activities of Daily Living (MG-ADL) score.
The overall duration of study will be up to 4 years and 8 months.
LUMINESCE (gMyasthenia Gravis - Satralizumab)
The purpose of this study is to compare the effects, good or bad, of subcutaneous (under the skin) injections of satralizumab in combination with current treatment (background therapy) in patients with gMG.
This study will also collect more data on the long-term safety and efficacy of satralizumab and continue providing you with satralizumab treatment during an open label extension period.
For this study, 240 male and female participants, including 20 adolescents, will be recruited at 120 sites. The rest of the participants will be aged 18+ years. The Montreal Neurological Institute-Hospital will only recruit adult patients aged 18 years and older.
HBS-101-CL-005 (Myotonic Dystrophy)
The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
The secondary objectives of this study are to assess the impact of pitolisant on fatigue, cognitive function and the burden of disease along with assessing the long-term safety and effectiveness of pitolisant in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
GALILEO Vertex VX-670 (Myotonic Dystrophy)
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
For more information, please contac
The Team
Dr. Oliver Blanchard
Principal Investigator
Dr. Rami Massie
Principal Investigator
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For more information, please contac
Dr. Erin O’Ferrall
Principal Investigator
Angela Genge
Principal Investigator
UPCOMING TRIALS
ARROWHEAD ARO-DM1 (Myotonic Dystrophy)
A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy Who Are 18 to 65 Years Old
This is a Phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single and multiple ascending doses of ARO-DM1 compared to placebo in male and female subjects with Type 1 Myotonic Dystrophy (DM1).
Dr. Daria Trojan
Principal Investigator
Dr. Bernard Brais
Principal Investigator
ACTIVE (NOT RECRUITING) TRIALS
Fulcrum Gene Therapy (FSHD)
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD) with Open-Label Extension (OLE)
The purpose of the 48 week placebo-controlled treatment period of this study is is to determine whether losmapimod may slow down or stop the progression of the disease in individuals with FSHD by preventing or decreasing DUX4 expression in skeletal muscle and other affected organs. This study will also evaluate how you feel when you are taking the study drug and assess how you think the study drug may be affecting the progression of your disease.
HELIOS ALNYLAM (hATTR Amyloidosis)
The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will receive vutrisiran or the reference comparator patisiran during the Treatment Period. The Treatment Period is followed by a Treatment Extension Period during which all participants in the patisiran group will switch to vutrisiran. This study will use the placebo arm of the APOLLO study (NCT01960348) as an external comparator for the primary and most other efficacy endpoints.
RAVULIZUMAB (Generalized Myasthenia Gravis)
From clinicaltrials.gov:
Inclusion criteria
- Diagnosed with Myasthenia Gravis at least 6 months (180 days) prior to the date of the Screening Visit as confirmed by specific criteria.
- Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at screening.
- MG-ADL profile must be ≥ 6 at screening and randomization (Day 1).
- Vaccinated against meningococcal infections within 3 years prior to, or at the time of, initiating study drug to reduce the risk of meningococcal infection (N meningitidis).
Spinraza (SMA)
A prospective, multi-center, observational study of the safety, tolerability and effectiveness of SPINRAZA in adult patients with Spinal Muscular Atrophy
From clinicaltrials.gov:
This is a longitudinal, observational study of adult patients with genetically confirmed chromosome 5q SMA to examine the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) for up to 30 months.
This is a prospective, longitudinal, multi-center, observational study designed to evaluate the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) in ambulatory and non-ambulatory adult patients with SMA. Subjects with SMA II/III that are 18 years to 70 years of age who are planning to initiate treatment with SPINRAZA® (nusinersen) as part of their clinical care plan will be enrolled in this study. This study does not provide SPINRAZA® (nusinersen) or cover costs associated with standard clinical care.These patients will be treated by their respective physicians according to standard clinical practice. Study visits including standardized assessments of strength and function will occur at baseline, day 15 after treatment initiation, day 30, day 70, and then 4-month intervals through month 30.
RAISE (gMG)
From clinicaltrials.gov:
The RAISE study is a multicenter, randomized, double-blind, placebo controlled study to confirm the efficacy, safety, and tolerability of zilucoplan in subjects with generalized Myasthenia Gravis. Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or placebo for 12 weeks.
Vanessa Bertone
Team Lead