Clinical trials: Neuron Disease, Motor
Amyotrophic Lateral Sclerosis
Study of AROSOD-1 in Adult Participants With Amyotrophic Lateral Sclerosis (ALS)
In this Phase 1 adult symptomatic patients with amyotrophic lateral sclerosis (ALS) carrying a superoxide dismutase 1 (SOD1) gene mutation thought to be causative of ALS, will receive single ascending doses of ARO-SOD1…
- Trial name
- NCT05949294
- Testing
- ARO-SOD1 Injection
- Sponsor
- Arrowhead Pharmaceuticals
Amyotrophic Lateral Sclerosis
Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
This study will assess the efficacy and safety of PTC857 treatment in participants diagnosed with ALS.
- Trial name
- CARDINALS (NCT05349721)
- Testing
- PTC857
- Where
- 55 sites in United States, Argentina and 11 more
- Volunteers
- 336 people
- Sponsor
- PTC Therapeutics
Upper Extremity Impairments, Cerebral Palsy (CP)
Obi Medical Robot: Evaluating Effectiveness Related to Usability
This mixed-methods usability study evaluates the effectiveness of the Obi Gen 3 robotic feeding device in meeting user needs among providers, caregivers, and patients.
- Conditions
- Upper Extremity Impairments, Cerebral Palsy (CP), ALS - Amyotrophic Lateral Sclerosis, Rett Syndrome
- Trial name
- NCT07151950
- Testing
- One-week trial of Obi Medical device, which provides robotic assistance with feeding to persons with upper extremity impairments who are unable to self-feed in the traditional manner using utensils
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Desin LLC
Healthy
A Multiple Dose Study to Investigate Safety, Tolerability and Pharmacokinetics of TBN
The purpose of this study is to assess the safety, tolerability and pharmacokinetics (i.e. how study drug is taken up by the body) of TBN in healthy participants.
- Trial name
- NCT04667013
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Guangzhou Magpie Pharmaceuticals Co., Ltd.
Amyotrophic Lateral Sclerosis
A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS
A phase 2 double-blind, placebo-controlled study of AL001 in participants with C9orf72-associated ALS.
- Trial name
- NCT05053035
- Testing
- AL001
- Where
- 3 sites in United States
- Volunteers
- 5 people
- Sponsor
- Alector Inc.
Amyotrophic Lateral Sclerosis
Study of ANX005 in Adults With Amyotrophic Lateral Sclerosis (ALS)
This study is a multi-center, open-label study of intravenous (IV) ANX005 in participants with ALS.
- Trial name
- NCT04569435
- Testing
- ANX005
- Where
- 8 sites in United States, Canada
- Volunteers
- 17 people
- Sponsor
- Annexon, Inc.
Amyotrophic Lateral Sclerosis
Safety/Efficacy of AVP-923 in the Treatment of Emotional Lability (Uncontrolled Crying & Laughing) in Patients With ALS
The purpose of this study is to compare and evaluate the safety of AVP-923 (dextromethorphan/quinidine) for the treatment of emotional lability in ALS patients.
- Trial name
- NCT00021697
- Testing
- AVP-923
- Where
- 17 sites in United States
- Volunteers
- 100 people
- Sponsor
- Avanir Pharmaceuticals
Amyotrophic Lateral Sclerosis
A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients With Amyotrophic Lateral Sclerosis (ALS)
The purpose of this study was to assess the effect of CK-2127107 (hereafter referred to as reldesemtiv) versus placebo on respiratory function and other measures of skeletal muscle function in patients with ALS.
- Trial name
- FORTITUDE-ALS (NCT03160898)
- Testing
- Reldesemtiv
- Where
- 64 sites in United States, Australia and 4 more
- Volunteers
- 458 people
- Sponsor
- Cytokinetics
Amyotrophic Lateral Sclerosis
Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year
This study assessed the effect of tirasemtiv versus placebo on respiratory function in patients with ALS.
- Trial name
- VITALITY-ALS (NCT02496767)
- Testing
- Tirasemtiv
- Where
- 81 sites in United States, Belgium and 9 more
- Volunteers
- 744 people
- Sponsor
- Cytokinetics
Muscular Atrophy, Spinal
Pediatric Spinal Muscular Atrophy (SMA) China Registry
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) treatments among pediatric Chinese participants with spinal muscular atrophy linked to chromosome 5q…
- Trial name
- NCT05042921
- Where
- 25 sites in China
- Volunteers
- 600 people
- Sponsor
- Biogen
Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis
European Home Mechanical Ventilation Registry
The European Home Mechanical Ventilation Registry (EHMVR) will enable a thorough evaluation of HMV by documenting the characteristics of HMV patients and their treatment.
- Conditions
- Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis, Spinal Cord Injury, Muscular Dystrophies
- Trial name
- EHMVR (NCT02315339)
- Where
- Germany
- Volunteers
- 37 people
- Sponsor
- ResMed
Muscular Atrophy, Spinal
Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants
The study is a randomized, single oral dose, crossover study in up to three parts to investigate the relative bioavailability and bioequivalence of two different formulations of risdiplam 5 mg (dispersible tablets) versus the…
- Trial name
- NCT04718181
- Testing
- risdiplam, omeprazole
- Where
- 4 sites in United States
- Volunteers
- 131 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Amyotrophic Lateral Sclerosis
A Study to Evaluate the Safety, Tolerability and Efficacy of ILB in Patients With Amyotrophic Lateral Sclerosis
This is a Phase 2a single-centre, open single-arm study in patients with Amyotrophic Lateral Sclerosis (ALS) of intermediate progression rate. Eligible subjects will be administered weekly doses of ILB.
- Trial name
- NCT03613571
- Testing
- ILB
- Where
- Sweden
- Volunteers
- 13 people
- Sponsor
- TikoMed AB
Amyotrophic Lateral Sclerosis
A Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL343 in Participants With Amyotrophic Lateral Sclerosis
This is a Phase 1b, multicenter, randomized, placebo-controlled, double-blind study of 28 days, followed by an 18-month open-label extension, designed to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of…
- Trial name
- NCT05006352
- Testing
- DNL343
- Where
- 7 sites in United States, Netherlands
- Volunteers
- 29 people
- Sponsor
- Denali Therapeutics Inc.
Amyotrophic Lateral Sclerosis (ALS), Amyotrophic Lateral Sclerosis &Amp; Other Neuromuscular Disorders
Amyotrophic Lateral Sclerosis: A Multicenter Retrospective Observational Registry
This retrospective observational study will analyze de-identified clinical data from patients with amyotrophic lateral sclerosis (ALS) collected at multiple centers over 7 years.
- Trial name
- NCT07143656
- Testing
- Imaging and Clinical Reports
- Where
- Poland
- Volunteers
- 36 people
- Sponsor
- Biocells Medical
Neurologic Disorder, Paralysis
EMPOWER Early Feasibility Study: Non-invasive BCI to Control a Wheelchair for People With Paralysis
Neuralis is an innovative assistive technology designed for individuals with severe neuromuscular conditions, enabling wheelchair control through EEG signals.
- Conditions
- Neurologic Disorder, Paralysis, Paralysis; Stroke, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06169696
- Testing
- Intervention/Treatment
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Synaptrix Labs Inc.
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, Motor Neuron Disease
MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)
This is a 24-month, Phase 2, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of pegcetacoplan in subjects with amyotrophic lateral sclerosis (ALS)
- Trial name
- NCT04579666
- Testing
- Pegcetacoplan (APL-2)
- Where
- 60 sites in United States, Australia and 12 more
- Volunteers
- 249 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Spinal Muscular Atrophy Type 3
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.
- Trial name
- NCT06421831
- Testing
- GC101
- Where
- China
- Volunteers
- 21 people
- Sponsor
- GeneCradle Inc
Frontotemporal Dementia, FTD
A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes
PBFT02 is a gene therapy for frontotemporal dementia intended to deliver a functional copy of the GRN gene to the brain. This study will assess the safety, tolerability and efficacy of this treatment in patients with…
- Conditions
- Frontotemporal Dementia, FTD, FTD-GRN, Dementia Frontotemporal
- Trial name
- upliFT-D (NCT04747431)
- Testing
- PBFT02
- Where
- 10 sites in United States, Australia and 3 more
- Volunteers
- 30 people
- Sponsor
- Passage Bio, Inc.