CURRENT RECRUITING TRIALS
CRU-SCIG (Myasthenia 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.
MINT Inebilizumab (Myasthenia Gravis)
The purpose of this study is to determine whether the investigational drug, inebilizumab, is safe and effective in reducing the symptoms of myasthenia gravis.
In this study, the effect that inebilizumab has on your myasthenia gravis will be assessed by recording the state of your disease (worse, better, stable), whether you need more treatment for myasthenia gravis, how you feel about your health, and other measures of whether the drug might be working to prevent flares (activity) of myasthenia gravis.
The study will also assess side effects—both those that you report and those that might be measured in your lab tests-and whether your body makes antibodies against inebilizumab. Antibodies against inebilizumab could reduce the amount of inebilizumab in the blood and the effects of inebilizumab on your myasthenia gravis.
For this study, 252 male and female participants will be recruited at approximately 100 sites. The participants will be over the age of 18.
SATRALIZUMAB (Myasthenia Gravis)
A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study To Evaluate Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Patients With Generalized Myasthenia Gravis
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.
NIPOCALIMAB (gMyasthenia Gravis)
RESOLUTE (Pompe Disease)
The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.
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.
The purpose of the MycarinGstudy is to demonstrate the clinical efficacy and to assess safety and tolerability of rozanolixizumab in patients with generalized myasthenia gravis (MG).More information
This is a global, randomized, double-blind trial of pamrevlumab or placebo in combination with systemic corticosteroids in subjects with non-ambulatory Duchenne muscular dystrophy, aged 12 years and older. Approximately 90 male subjects will be randomized at a 1:1 ratio to Arm A (pamrevlumab + systemic corticosteroid) or Arm B (placebo+ systemic corticosteroid), respectively.
Subjects will be randomized in a 1:1 ratio to one of the two study treatment arms; pamrevlumab or placebo in combination with systemic steroids.
This trial has three study periods:
- Screening period: Up to 4 weeks
- Treatment period: 52 weeks
- Safety Follow-up period/final assessment: 4 weeks (Week 56 (+/-3 days))
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.
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.
FORCE (Post-Polio Syndrome)
A multicenter, prospective, randomized, placebo-controlled, double-blind, parallel-group clinical trial to assess the efficacy and safety of Immune Globulin Intravenous (Human) Flebogamma® 5% DIF in patients with Post-Polio Syndrome)
The aim of this research is to test how well a human immunoglobulin concentrate named Flebogamma® 5% DIF (hereafter referred to as Flebogamma) improves your PPS symptoms such as walking capacity, pain, overall quality of life, fatigue and muscle strength. Flebogamma is the investigational drug. Investigational means that is has not been approved by any health authority (government authorities) such as the US Food and Drug Administration (FDA) or Health Canada (HC) for improving your PPS symptoms effectively and safely. However, HC does not object to the use of Flebogamma in this study.
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)
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis
- 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).
PXT3003 (Charcot Marie Tooth)
The purpose of this study is to investigate how effective PXT3003 is when compared to a placebo on the disease progression and symptoms of CMT1A. PXT3003 consists of a combination of very low doses of 3 currently approved pharmaceutical ingredients: (RS)-baclofen, naltrexone HCl, and D-sorbitol, which are thought to act in different ways to treat CMT1A.
The placebo used in this study looks exactly like PXT3003 but it does not contain any active medication. Placebos are sometimes called sugar pills, dummy treatments, or shams. We are using a placebo to compare with PXT3003, and to ensure that the changes you report in your health, good or bad, are not only due to chance. In this information and consent form, the use of “study drug” refers either to PXT3003 or to the placebo.
The study also aims to assess the safety and tolerability (whether side effects of a medication can be handled by patients) of PXT3003.
For this study, 350 male and female participants will be recruited at 50 sites globally. The participants will be aged between 16 to 65 years. Only adults 18 years and older will be recruited at the Montreal Neurological Institute-Hospital.