A Randomized, Double-Blind, Multicenter, Placebo-Controlled Phase 3 Study with Open-Label Period to Evaluate the Efficacy and Safety of Inebilizumab in Adults with 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. 

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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. 

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Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

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.

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Une étude de phase 3, randomisée, à double insu et contrôlée par placebo évaluant l’efficacité et l’innocuité du rozanolixizumab chez des patients adultes atteints de myasthénie grave généralisée

L’objectif de cette étude est d’évaluer l’innocuité, la tolérabilité et l’efficacité du rozanolixizumab. L’essai examinera également comment le rozanolixizumab agit dans le corps humain et comment le corps réagit au rozanolixizumab.

A Phase III trial of pamrevlumab or placebo in combination with systemic corticosteroids in subjects with non-ambulatory Duchenne Muscular Dystrophy

From clinicaltrials.gov:

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))

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Étude de phase 3, multicentrique, à répartition aléatoire, à double insu et contrôlée par placebo visant à confirmer l’innocuité, la tolérabilité et l’efficacité du zilucoplan chez des sujets atteints de myasthénie grave généralisée

Le but de cette étude est de tester l’efficacité du zilucoplan et son innocuité chez les patients atteints de MGg.

Étude observationnelle, multicentrique, prospective évaluant l’innocuité (sûreté), la tolérabilité et l’efficacité potentielle du SPINRAZA chez les patients adultes atteints d’amyotrophie spinale

De clinicaltrials.gov. Malheureusement, les informations ne sont disponibles qu’en anglais. Pour plus d’informations en français, veuillez contacter Xin Di Dong.

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.

Étude pour évaluer l’efficacité et l’innocuité de l’immunoglobuline intraveineuse (humaine) Flebogamma® 5% DIF par rapport au placebo chez les patients atteints du syndrome de post-poliomyélite

Le but de cette recherche consiste à évaluer l’efficacité d’un concentré d’immunoglobuline humaine connu comme Flebogamma® 5% DIF (appelé ci-après Flebogamma) pour améliorer les symptômes du SPP, tels que la capacité de marche, la douleur, la qualité de vie en général, la fatigue et la force musculaire. Flebogamma est le médicament expérimental. Par expérimental, on entend qu’il n’a été approuvé par aucune autorité de santé (autorités gouvernementales) comme la Food and Drug Administration (FDA) américaine ou Santé Canada pour atténuer vos symptômes du SPP efficacement et de manière sécuritaire. Toutefois, Santé Canada ne s‘est pas objectée à l‘utilisation de Flebogamma dans le cadre de cette étude.

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A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (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.

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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

From clinicaltrials.gov:

Inclusion criteria

1. Diagnosed with Myasthenia Gravis at least 6 months (180 days) prior to the date of the Screening Visit as confirmed by specific criteria.
2. Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at screening.
3. MG-ADL profile must be ≥ 6 at screening and randomization (Day 1).
4. 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).

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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. 

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