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A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy of Natalizumab on Reducing Disability Progression in Subjects With Secondary Progressive Multiple Sclerosis, With optional Open-label Extension

Part 1:The primary objective of the study is to investigate whether treatment with natalizumab slows the accumulation of disability not related to relapses in subjects with SPMS. The secondary objectives of this study are to determine in this study…

A Phase 3, Multi-National, Double-Blind, Randomized, Placebo-Controlled, Stratified, Parallel Group, Study to Evaluate the Safety, Tolerability and Efficacy of Tirasemtiv in Patients with Amyotrophic Lateral Sclerosis (ALS)

Protocol version Amendment 1, 31-Jul-2015, paragraph 2.1, page 28:Primary objective:The primary objective is to assess the effect of tirasemtiv versus placebo on respiratory function in patients with ALS.Secondary objectives:* Evaluation of…

Prevention of depression and poor physical function in older persons with vitamin D supplementation

Primary objectives: 1. Does vitamin D supplementation improve depressive symptoms in older persons? 2. Does vitamin D supplementation improve physical performance and functional limitations in older persons?Secondary objectives: 3. Does vitamin D…

A randomized, double-blind, placebo-controlled, multi-center study of the safety and efficacy of Dexpramipexole in subjects with amyotrophic lateral sclerosis.

Primary:The primary objective of the study is to evaluate the efficacy of oral administration of dexpramipexole 150 mg twice daily compared to placebo for 12 months in subjects with ALS.Secondary:The secondary objectives of the study are to evaluate…

An Open-Label, Multicenter, Extension Study to Evaluate the Long-Term Safety and Efficacy of Dexpramipexole (BIIB050) in Subjects With Amyotrophic Lateral Sclerosis

Primary:The primary objective of the study is to evaluate the long-term safety profile of dexpramipexole in subjects with ALS.Secondary:The secondary objective of this study is to evaluate the longterm efficacy of dexpramipexole in this study…

A Phase 3, multi-center, randomized, Double-Blind, double-dummy, active controlled, parallel group study to evaluate the efficacy and safety of RPC1063 administered orally to relapsing multiple sclerosis patients.

Primary Objective:To assess whether the clinical efficacy of RPC1063 is superior to interferon (IFN) *-1a (Avonex®) inreducing the rate of clinical relapses in patients with RMS.Secondary Objectives:- To assess the effect of RPC1063 on the…

Functional effects of botulinum toxin in the hip adductors and subsequent exercise in patients with hereditary spastic paraplegia

This study aims to provide evidence for the notion that reduction of spasticity by bilateral BTX injections in the hip adductor group followed by self-administered stretching exercises and agility training improves lateral stepping and gait…

Effects of oral levosimendan (ODM-109) on respiratory function in patients with ALS: open label extension for patients completing study 3119002

Is to evaluate long-term safety of oral levosimendan in amyotrophic lateral sclerosis (ALS) patients.

A Phase 3, Randomized, Open-Label, Parallel-Group Study to Compare the Pharmacodynamics, Pharmacokinetics, Efficacy, Safety, Tolerability, and Immunogenicity of Multiple Subcutaneous Injections of Efgartigimod PH20 SC with Multiple Intravenous Infusions of Efgartigimod in Patients with Generalized Myasthenia Gravis (ADAPT)

Rationale:The aim of this study is to investigate the pharmacodynamics (PD), pharmacokinetics (PK), safety, tolerability, immunogenicity, and clinical efficacy of efgartigimod coformulated with recombinant human hyaluronidase PH20 (rHuPH20)…

Effects of oral Levosimendan (ODM-109) on respiratory function in patients with ALS

The primary objective of this study is to confirm that levosimendan can significantly improve respiratory function measured by supine slow vital capacity (SVC) in amyotrophic lateral sclerosis (ALS) patients.The secondary objective is to confirm…

A Phase 3, Open-Label Extension Study of Tirasemtiv for Patients with Amyotrophic Lateral Sclerosis (ALS) who Completed VITALITY-ALS (CY 4031)

The primary objective is to assess the long-term safety and tolerability of tirasemtiv, inpatients with ALS

An Open-Label Uncontrolled Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Activity of Nipocalimab in Children Aged 2 to less than 18 years with Generalized Myasthenia Gravis

This study has been transitioned to CTIS with ID 2022-502539-21-00 check the CTIS register for the current data. The purpose of this study is to determine the effect of nipocalimab on total serum immunoglobulin G (IgG) in pediatric participants 2 to…

A Randomized, Controlled, Open-Label, Rater-Blinded, Phase 3b Study of the Efficacy, Safety, and Tolerability of 6-Week Extended Interval Dosing (EID) of Natalizumab (BG00002) in Subjects With Relapsing-Remitting Multiple Sclerosis witching From Treatment With 4-Week Natalizumab Standard Interval Dosing (SID) in Relation to Continued SID Treatment - Followed by an Open-Label Crossover Extension Study Comprising Subcutaneous and Intravenous Natalizumab Administration

Part 1: The primary objective of this study is to evaluate the efficacy of natalizumab extended interval dosing (EID) in subjects who have previously been treated with natalizumab standard interval dosing (SID) for at least 12 months, in relation to…

A multicenter, randomized, double-blind, placebo-controlled study to investigate the efficacy and safety of FAB122 in patients with Amyotrophic Lateral Sclerosis

To assess the effect of treatment with 100 mg of FAB122 (edaravone) on disease progression in patients with ALS.

Safety and efficacy of tauroursodeoxycholic acid (TUDCA) as add-on treatment in patients affected by amyotrophic lateral sclerosis (ALS)

The primary objective of the study is:To assess the efficacy of TUDCA in slowing disease progression in patients with ALS during the treatment period compared to the lead-in phase), as measured by ALSFRS-R.

Phase IIIb, open-label, single-arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101 administered intrathecally (1.2 x 1014 vector genomes) to participants 2 < 18 years of age with spinal muscular atrophy (SMA) who have discontinued treatment with nusinersen (Spinraza®) or risdiplam (Evrysdi®)

The purpose of this study is to characterize the safety and tolerability of OAV101 IT in participants who have discontinued treatment with nusinersen (Spinraza®) or risdiplam (Evrysdi®). The data from this study will expand on the data generated…

A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel, Multicenter Study to Evaluate the Safety and Efficacy of ALXN1720 in Adults with Generalized Myasthenia Gravis.

This study has been transitioned to CTIS with ID 2023-508284-77-00 check the CTIS register for the current data. The purpose of this study is to evaluate the safety and efficacy of ALXN1720 for the treatment of gMG in adults with autoantibodies…

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS)

Primary objective: To evaluate the effect of ravulizumab compared with placebo on amyotrophic lateral sclerosis functional rating scale-revised (ALSFRS-R) score in adult patients with amyotrophic lateral sclerosis (ALS).Secondary objectives:1. To…

A PHASE 3, MULTI-CENTER, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED TRIAL TO EVALUATE THE EFFICACY AND SAFETY OF RELDESEMTIV IN PATIENTS WITH AMYOTROPHIC LATERAL SCLEROSIS (ALS)

The purpose of this study is to evaluate whether the new drug reldesemtiv is effective and safe in patients with ALS. The primary objective is to assess the effect of reldesemtiv versus placebo on functional outcomes in ALS.The secondary objectives…

Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

Primary-Assess the efficacy of apitegromab compared with placebo using the HFMSE in patients 2 through 12 years oldKey secondary-Assess the efficacy of apitegromab compared with placebo based on the number of patients with clinical improvement in…