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.

A Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients

AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA).

Part A is a single dose design with 1 cohort (dose level). In Part A, the patient duration is 6 months as the treatment period is 1 day followed by a 6 month follow-up period.

Part B is a multiple-ascending dose design with 2 cohorts (dose levels). In Part B, the patient duration is 6 months as the treatment period is 3 months followed by a 3 month follow-up period.

https://www.clinicaltrials.gov/study/NCT05027269?cond=Myotonic%20Dystrophy&term=avidity&rank=1

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.

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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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A Phase 1/​2A, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type 1  (DM1) 

 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.

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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.

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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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.

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A Phase 3, Multicenter, Randomized, Double- Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects with Generalized Myasthenia Gravis

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.

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