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Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT)
NeuroNEXT was created to conduct studies of treatments for neurological diseases through partnerships with academia, private foundations, and industry. collaborator. https://neuronext.orgCReATe Consortium
The Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) Consortium will enroll patients with sporadic and familial forms of amyotrophic lateral sclerosis, frontotemporal dementia (FTD), primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), and progressive muscular atrophy (PMA).
https://www.rarediseasesnetwork.org/cms/create/The Pre-symptomatic Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study (Pre-fALS)
Pre-fALS is a prospective natural history and biomarker study of people not yet affected with ALS, but who are at genetic risk for developing ALS.
https://clinicaltrials.gov/ct2/show/NCT00317616The Myasthenia Gravis Rare Disease Network (MGNet)
The Myasthenia Gravis Rare Disease Network (MGNet) is a consortium of academic medical centers partnering with the Myasthenia Gravis Foundation of America and Conquer MG as well as collaborators in other research groups and industry. https://www.rarediseasesnetwork.org/mgnet
Arimoclomol in Amyotropic Lateral Sclerosis
This is a multicenter, randomized, double-blind, placebo-controlled, parallel group trial to evaluate the efficacy and safety of arimoclomol in amyotropic lateral sclerosis (ALS).
https://clinicaltrials.gov/ct2/show/NCT03491462Disease-Modifying Treatments for Myasthenia Gravis (PROMISE-MG)
This study is designed to address the evidence gaps in a real-world setting and help patients with MG choose treatments that are best suited to them.
https://clinicaltrials.gov/ct2/show/NCT03490539Brief Description: The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial.
https://clinicaltrials.gov/ct2/show/NCT04297683An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS) (VALOR (Part C))
The primary objective of Parts A and B of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB067 (Tofersen) in adults with ALS.
https://www.clinicaltrials.gov/ct2/show/NCT02623699A Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 in Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis
The primary objective of this study is to evaluate the safety and tolerability of BIIB078 in adults with C9ORF72-Amyotrophic Lateral Sclerosis (ALS).
https://clinicaltrials.gov/ct2/show/NCT03626012Phenotype, Genotype & Biomarkers in ALS and Related Disorders
Brief Summary: The goals of this study are: (1) to better understand the relationship between the phenotype and genotype of amyotrophic lateral sclerosis (ALS) and related diseases, including primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA), and frontotemporal dementia (FTD); and (2) to develop biomarkers that might be useful in aiding therapy development for this group of disorders.
https://clinicaltrials.gov/ct2/show/NCT02327845 -
Carbunar
Topiramate for Cryptogenic Sensory Peripheral Neuropathy in Metabolic Syndrome (CSPN) (TopCSPN)
The TopCSPN trial is a double blinded randomized placebo controlled study of oral topiramate as a potential disease modifying therapy for cryptogenic sensory peripheral neuropathy (CSPN).
https://clinicaltrials.gov/ct2/show/NCT02878798A Phase 2 Study of KZR-616 to Evaluate Safety and Efficacy in Patients With Active Polymyositis or Dermatomyositis (PRESIDIO)
This is a Phase 2 randomized, double-blind, placebo-controlled, crossover, multicenter study to evaluate the safety, tolerability, efficacy, Pharmacokinetics (PK) and Pharmacodynamics (PD) of treatment with KZR-616 in patients with active Polymyositis (PM) or Dermatomyositis (DM).
https://clinicaltrials.gov/ct2/show/NCT04033926 -
Saporta
Topiramate for Cryptogenic Sensory Peripheral Neuropathy in Metabolic Syndrome (CSPN) (TopCSPN)
The TopCSPN trial is a double blinded randomized placebo controlled study of oral topiramate as a potential disease modifying therapy for cryptogenic sensory peripheral neuropathy (CSPN).
https://clinicaltrials.gov/ct2/show/NCT02878798Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others (INC-6601)
This is an observational longitudinal study to determine the natural history and genotype-phenotype correlations of disease causing mutations in Charcot Marie Tooth disease (CMT) type 1B (CMT1B), 2A (CMT2A), 4A (CMT4A), and 4C (CMT4C).https://clinicaltrials.gov/ct2/show/NCT01193075
Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2 (INC-6602)
Detailed Description: This project is to understand modifier genes and how they influence the severity of disease expression, along with identifying new forms of CMT which have not been genetically determined
https://clinicaltrials.gov/ct2/show/NCT01193088 -
Granit
Descartes-08 CAR-T Cells in Generalized Myasthenia Gravis (MG)
This is a Phase Ib/IIa study to evaluate the safety and preliminary efficacy of Descartes-08 CAR T-cells in patients with Generalized Myasthenia Gravis
https://clinicaltrials.gov/ct2/show/NCT04146051Disease-Modifying Treatments for Myasthenia Gravis (PROMISE-MG)
This study is designed to address the evidence gaps in a real-world setting and help patients with MG choose treatments that are best suited to them.
https://clinicaltrials.gov/ct2/show/NCT03490539HEALEY ALS Platform Trial
Brief Description: The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.
https://clinicaltrials.gov/ct2/show/NCT04297683An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS) (VALOR (Part C))
The primary objective of Parts A and B of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB067 (Tofersen) in adult with ALS.
https://www.clinicaltrials.gov/ct2/show/NCT02623699A Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 in Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis
The primary objective of this study is to evaluate the safety and tolerability of BIIB078 in adults with C9ORF72-Amyotrophic Lateral Sclerosis (ALS).
https://clinicaltrials.gov/ct2/show/NCT03626012Arimoclomol in Amyotropic Lateral Sclerosis
This is a multicenter, randomized, double-blind, placebo-controlled, parallel group trial to evaluate the efficacy and safety of arimoclomol in amyotropic lateral sclerosis (ALS).
https://clinicaltrials.gov/ct2/show/NCT03491462The Pre-symptomatic Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study (Pre-fALS)
Pre-fALS is a prospective natural history and biomarker study of people not yet affected with ALS, but who are at genetic risk for developing ALS.
https://clinicaltrials.gov/ct2/show/NCT00317616Phenotype, Genotype & Biomarkers in ALS and Related Disorders
The goals of this study are: (1) to better understand the relationship between the phenotype and genotype of amyotrophic lateral sclerosis (ALS) and related diseases, including primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA), and frontotemporal dementia (FTD); and (2) to develop biomarkers that might be useful in aiding therapy development for this group of disorders.
https://clinicaltrials.gov/ct2/show/NCT02327845 -
Sharma
A Study of GDC-0134 to Determine Initial Safety, Tolerability, and Pharmacokinetic Parameters in Participants With Amyotrophic Lateral Sclerosis
This first-in-human, double-blind, placebo-controlled Phase I study will be conducted in participants with amyotrophic lateral sclerosis (ALS) to explore safety, tolerability, and pharmacokinetic (PK) properties of GDC-0134.
https://clinicaltrials.gov/ct2/show/NCT02655614Study of the Efficacy and Safety of Immune Globulin Intravenous (Human) Flebogamma® 5% DIF in Patients With Post-polio Syndrome (FORCE)
This is a multicenter, prospective, randomized, placebo-controlled, double-blind, parallel group clinical trial with adaptive dose selection in subjects with post polio syndrome (PPS).
https://www.clinicaltrials.gov/ct2/show/NCT02176863Safety Study of Oral Edaravone Administered in Subjects With ALS
The objective of this study is to evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral Sclerosis (ALS) over 24 and 48 weeks.
https://clinicaltrials.gov/ct2/show/NCT04165824A Study to Test Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
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).
https://clinicaltrials.gov/ct2/show/NCT03971422A Study to Assess the Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves) (ADHERE)
This is a Phase 2 study to evaluate the safety and efficacy of the subcutaneous formulation of efgartigimod in adults with CIDP.
https://clinicaltrials.gov/ct2/show/NCT04281472