Clinical trials: Motor Neurone Disease
Amyotrophic Lateral Sclerosis
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
PIONEER-ALS is a Phase 1/2, multicenter, open-label, ascending dose, uncontrolled, first-in-human study that will evaluate the safety, tolerability and effects on clinical and biomarker endpoints of intracisternal administration…
- Trial name
- ALS (NCT07287397)
- Testing
- VTx-002, Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone
- Where
- 11 sites in United States, Belgium and 2 more
- Volunteers
- 36 people
- Sponsor
- Vector Y Therapeutics
Foot Drop, Thigh Muscles Weakness (Hamstrings or Quadriceps)
Evaluation of the Safety and Performance of the NESS L300 Plus System
The study is designed to evaluate the safety and efficacy of using the L300 Plus as a neuroprosthesis device for walking in patients following an UMN injury or disease.
- Conditions
- Foot Drop, Thigh Muscles Weakness (Hamstrings or Quadriceps), Upper Motor Neuron Injury or Disease
- Trial name
- L300Plus (NCT01237860)
- Testing
- NESS L300 Plus System
- Where
- Israel
- Volunteers
- 45 people
- Sponsor
- Bioness Inc
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene…
- Trial name
- HINALEA 2 (NCT05861999)
- Testing
- Risdiplam
- Where
- 17 sites in United States, Germany and 4 more
- Volunteers
- 19 people
- Sponsor
- Hoffmann-La Roche
Tetraplegia/Tetraparesis, Quadriplegia
GB-PRIME: An Early Feasibility Study of a Precise Robotically Implanted Brain-Computer Interface for the Control of External Devices
The GB-PRIME Study is an early feasibility study designed to assess the clinical safety and functionality of the Neuralink N1 Implant and R1 Robot.
- Conditions
- Tetraplegia/Tetraparesis, Quadriplegia, Quadriplegia/Tetraplegia, Cervical Spinal Cord Injury
- Trial name
- GB-PRIME (NCT07127172)
- Testing
- N1 Implant, R1 Robot
- Where
- 2 sites in United Kingdom
- Volunteers
- 7 people
- Sponsor
- Neuralink Corp
Amyotrophic Lateral Sclerosis
A Study to Assess the Safety, Tolerability, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation
The ALSpire Study is a clinical trial evaluating the investigational drug BIIB105 in adults living with amyotrophic lateral sclerosis (ALS). The ALSpire Study consists of two parts: Part 1: 6-month placebo-controlled study.
- Trial name
- ALSpire (NCT04494256)
- Testing
- BIIB105
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 99 people
- Sponsor
- Biogen
ALS
Riluzole Oral Soluble Film Safety and Tolerability in Amyotrophic Lateral Sclerosis
The primary objective of this study is to assess the safety and tolerability, with emphasis on the oral cavity, of ROSF (containing riluzole 50mg) in subjects with amyotrophic lateral sclerosis (ALS) administered twice daily for…
- Trial name
- NCT03457753
- Testing
- Riluzole Oral Soluble Film
- Where
- United States
- Sponsor
- Aquestive Therapeutics
Amyotrophic Lateral Sclerosis, ALS
A Study to Evaluate the Safety, Efficacy, and Pharmacodynamics of PLL001 in ALS Patients
FIH, Phase 1/2, multi-centre, randomised, double-blind, placebo controlled study with an optional open-label dosing extension to assess the safety, tolerability, efficacy, and Pharmacodynamics (PD) of single or multiple (up to 48…
- Trial name
- NCT06513546
- Volunteers
- 153 people
- Sponsor
- PLL TX AUSTRALIA PTY LTD
Spinal Muscular Atrophy
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥…
- Trial name
- SMART (NCT04851873)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 24 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis
A Biomarker Study to Evaluate MN-166 in Subjects With Amyotrophic Literal Sclerosis (ALS)
This is a multi-center, open-label study of MN-166 (ibudilast) in subjects with ALS. To be eligible subjects must meet the El Escorial criteria of possible, laboratory-supported probable, probable, or definite criteria for a…
- Trial name
- NCT02714036
- Testing
- ibudilast
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- MediciNova
Amyotrophic Lateral Sclerosis
Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS
The goal of this clinical trial is to learn if the drug pridopidine works to treat amyotrophic lateral sclerosis in adults. It will also help to learn about the safety of pridopidine.
- Trial name
- PREVAiLS (NCT07322003)
- Testing
- Pridopidine
- Where
- 56 sites in United States, Belgium and 11 more
- Volunteers
- 500 people
- Sponsor
- Prilenia
ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy
Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System Disorders
This observational registry collects existing medical and routine care data from people with amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and other motor system disorders.
- Conditions
- ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy, Spinal Cord Diseases, Central Nervous System Diseases
- Trial name
- NCT07900672
- Testing
- Standard of Care (Investigator Choice)
- Where
- Germany
- Volunteers
- 2,000 people
- Sponsor
- Ambulanzpartner Soziotechnologie APST GmbH
Amyotrophic Lateral Sclerosis, Neuromuscular Disease
Connect-One: Early Feasibility Study of Connexus® Brain-Computer Interface (BCI)
The Connect-One Study is an early feasibility study to obtain preliminary device safety information for the Connexus Brain-Computer Interface (BCI).
- Conditions
- Amyotrophic Lateral Sclerosis, Neuromuscular Disease, Stroke, Tetraplegia/Tetraparesis
- Trial name
- CONNECT-ONE (NCT07357428)
- Testing
- Connexus Brain-Computer Interface
- Where
- 3 sites in United States
- Volunteers
- 2 people
- Sponsor
- Paradromics
Spinal Muscular Atrophy (SMA)
Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)
The goal of this multicenter, open-label, non-comparative, cohort study is to investigate the safety, immunogenicity, and efficacy of ANB-004 in children with spinal muscular atrophy.
- Trial name
- BLUEBELL (NCT05747261)
- Testing
- ANB-004, dose 1, ANB-004, dose 2, ANB-004, dose 3
- Where
- 7 sites in Belarus, Russia
- Volunteers
- 40 people
- Sponsor
- Biocad