Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis

Rehabilitation in SOD1 ALS Treated With Tofersen

NCT05725759 ·Status: ENROLLING_BY_INVITATION

Intermediate-size Patient Population Expanded Access Protocol

NCT07088159 ·Status: AVAILABLE

First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)

NCT06351592 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

An Intermediate Size Expanded Access Protocol of AMX0035 for ALS

NCT05286372 ·Status: APPROVED_FOR_MARKETING

A Trial of Tocilizumab in ALS Subjects

NCT02469896 ·Status: COMPLETED ·Phase: PHASE2

Expanded Access Protocol of BHV-0223 for Patients With Amyotrophic Lateral Sclerosis (ALS)

NCT03537807 ·Status: NO_LONGER_AVAILABLE

An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)

NCT01494701 ·Status: COMPLETED ·Phase: PHASE1

A Phase 1 Study to Investigate the Safety and Pharmacokinetics of Fosigotifator in Patients With Amyotrophic Lateral Sclerosis

NCT04948645 ·Status: TERMINATED ·Phase: PHASE1

Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene Mutations

NCT01041222 ·Status: COMPLETED ·Phase: PHASE1

Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

NCT06100276 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)

NCT02865109 ·Status: NO_LONGER_AVAILABLE

Frequency of SOD1 and C9orf72 Gene Mutations in French ALS

NCT04819555 ·Status: COMPLETED

Phase1 First in Human Ascending Dose Study to Evaluate the Safety and Tolerability of FC-12738 in Health Adults

NCT05978908 ·Status: COMPLETED ·Phase: PHASE1

Study of AROSOD-1 in Adult Participants With Amyotrophic Lateral Sclerosis (ALS)

NCT05949294 ·Status: WITHDRAWN ·Phase: PHASE1

Patients With ALS and Other Motor Disorders Will be Treated With Mesenchymal Cell Exosome Solution

NCT07105371 ·Status: COMPLETED ·Phase: PHASE1

Two Intermediate Expanded Access Protocols (EAP) CNMAu8.EAP01 and CNMAu8.EAP02 for ALS

NCT05281484 ·Status: NO_LONGER_AVAILABLE

An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)

NCT02052791 ·Status: COMPLETED ·Phase: PHASE1

Multiple Doses of AT-1501-A201 in Adults With ALS

NCT04322149 ·Status: COMPLETED ·Phase: PHASE2

Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis (ALS)

NCT00748501 ·Status: COMPLETED ·Phase: PHASE2

Safety and Dosimetry of a New Radiotracer to Detect Misfolded SOD1 Associated With Amyotrophic Lateral Sclerosis

NCT05974579 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1

A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy

NCT05548556 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2

Study on the Safety and Efficacy of RAG-21 in the Treatment of Amyotrophic Lateral Sclerosis Patients With FUS Gene Mutations

NCT07080801 ·Status: NOT_YET_RECRUITING ·Phase: EARLY_PHASE1

FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)

NCT04768972 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

Intermediate Expanded Access Protocol CNMAu8.EAP04

NCT06408727 ·Status: TEMPORARILY_NOT_AVAILABLE

Randomized, Double-blind, Safety and Efficacy of Recombinant Human Erythropoietin in Amyotrophic Lateral Sclerosis

NCT03835507 ·Status: RECRUITING ·Phase: PHASE1/PHASE2