Clinical trials: Neuron Disease, Motor
Muscular Atrophy, Spinal
A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function
This is a multi-center, open-label, non-randomized, parallel-group, 2-part study to evaluate the effect of hepatic impairment on the PK and safety and tolerability of a single oral dose of risdiplam compared to matched healthy…
- Trial name
- NCT03920865
- Testing
- Risdiplam
- Where
- 3 sites in United States
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Spinal Cord Injuries, Amyotrophic Lateral Sclerosis
Diaphragmatic Pacer Placement: Anesthetic Management (DP)
The diaphragmatic pacemaker (DP) has proven its utility in replacing mechanical ventilation (MV) in patients with chronic spinal cord injury (SCI) and Amyotrophic Lateral Sclerosis (ALS), by improving the patients quality of life…
- Conditions
- Spinal Cord Injuries, Amyotrophic Lateral Sclerosis, Respiratory Paralysis
- Trial name
- DP (NCT02942953)
- Testing
- Diaphragmatic Pacer
- Volunteers
- 16 people
- Sponsor
- Institut Guttmann
Amyotrophic Lateral Sclerosis
Impact of Nuedexta on Bulbar Physiology and Function in ALS
Nuedexta is FDA approved for the treatment of pseudobulbar affect in ALS patients and anecdotal reports of improvements in speech, salivation or swallowing have been reported.
- Trial name
- NCT03883581
- Testing
- dextromethorphan HBr and quinidine sulfate
- Where
- 2 sites in United States
- Volunteers
- 28 people
- Sponsor
- University of Florida
Amyotrophic Lateral Sclerosis (ALS)
A Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS
The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of SNUG01 in in adult subjects with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT07169175
- Testing
- SNUG01
- Where
- 4 sites in United States, China
- Volunteers
- 21 people
- Sponsor
- SineuGene Therapeutics Co., Ltd.
Amyotrophic Lateral Sclerosis, ALS
An Efficacy and Safety Study of Ravulizumab in ALS Participants
The purpose of the study is to assess the efficacy and safety of ravulizumab for the treatment of adult participants with ALS.
- Trial name
- NCT04248465
- Testing
- Ravulizumab
- Where
- 95 sites in United States, Australia and 15 more
- Volunteers
- 382 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Amyotrophic Lateral Sclerosis
The Efficacy and Safety of Vitamin E Mixed Tocotrienols In Patients With Amyotrophic Lateral Sclerosis (ALS)
There is currently no effective treatment in ALS. Oxidative stress, probably interacting with other neurodegenerative processes, is hypothesized to play a leading role in pathogenesis.
- Trial name
- NCT04140136
- Testing
- Tocotrienols
- Where
- Malaysia
- Volunteers
- 20 people
- Sponsor
- University of Malaya
Stroke
The Role of MNS in Improving Motor Performance
Many studies have demonstrated the usefulness of repetitive task practice by using robotic devices, including Lokomat, for the treatment of lower limb paresis.
- Trial name
- NCT02971371
- Testing
- Lokomat
- Volunteers
- 24 people
- Sponsor
- IRCCS Centro Neurolesi Bonino Pulejo
ALS, Amyotrophic Lateral Sclerosis
A Study to Explore the Role of Gut Flora in ALS
This study seeks to correlate microbiome sequencing data with information provided by patients and their medical records regarding ALS
- Conditions
- ALS, Amyotrophic Lateral Sclerosis, Motor Neuron Disease
- Trial name
- NCT04150809
- Testing
- No intervention
- Where
- United States
- Sponsor
- ProgenaBiome
Amyotrophic Lateral Sclerosis
Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment
The Phoenix Trial is a randomized double blind placebo controlled Phase III trial to evaluate the safety and efficacy of AMX0035 for treatment of ALS
- Trial name
- Phoenix (NCT05021536)
- Testing
- AMX0035
- Where
- 69 sites in United States, Belgium and 10 more
- Volunteers
- 664 people
- Sponsor
- Amylyx Pharmaceuticals Inc.
Amyotrophic Lateral Sclerosis
Low Dose IL-2 in the Treatment of Immune-associated ALS Syndrome
The purpose of this study was to evaluate the efficacy and safety of low-dose IL-2 in the treatment of immunorelated ALS syndrome.
- Trial name
- NCT04952155
- Testing
- IL-2
- Where
- China
- Volunteers
- 13 people
- Sponsor
- Peking University Third Hospital
ALS (Amyotrophic Lateral Sclerosis)
Intermediate-size Patient Population Expanded Access Protocol
The purpose of this Expanded Access Program is to provide tazbentetol to ALS patients who are not eligible to enroll in an ALS clinical trial.
- Trial name
- NCT07088159
- Testing
- Tazbentetol
- Where
- 16 sites in United States
- Sponsor
- Spinogenix
Amyotrophic Lateral Sclerosis
Effects of Oral Levosimendan on Respiratory Function in Patients With Amyotrophic Lateral Sclerosis (ALS): Open-Label Extension
This study provides an opportunity for subjects in the REFALS (3119002; NCT03505021) study to continue treatment with oral levosimendan.
- Trial name
- REFALS-ES (NCT03948178)
- Testing
- Levosimendan
- Where
- 79 sites in United States, Australia and 12 more
- Volunteers
- 227 people
- Sponsor
- Orion Corporation, Orion Pharma
Amyotrophic Lateral Sclerosis
EIM Via the Myolex mScan as an ALS Biomarker
Amyotrophic lateral sclerosis (ALS) has been traditionally considered incurable and untreatable. But starting in the 1990s with the introduction of Riluzole, therapies are being discovered and ultimately approved for slowing…
- Trial name
- ElectricALS (NCT06491732)
- Testing
- Electrical Impedance Myography
- Where
- 6 sites in United States
- Volunteers
- 80 people
- Sponsor
- Beth Israel Deaconess Medical Center
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved…
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- SAPPHIRE (NCT05156320)
- Testing
- Apitegromab
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 188 people
- Sponsor
- Scholar Rock, Inc.
Amyotrophic Lateral Sclerosis ALS
Intestinal Microbiome Transplant in ALS
This is a 24-week study of intestinal microbiome transplant in people with ALS. All participants will be evaluated clinically in person for 4 visits. Blood and mailed/ fresh stool samples will also be collected.
- Trial name
- NCT07017946
- Testing
- MTP-101C
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Duke University
Hereditary Spastic Paraplegia, Neurodegenerative Diseases
Hereditary Spastic Paraplegia Genomic Sequencing Initiative (HSPseq)
The purpose of the HSP Sequencing Initiative is to better understand the role of genetics in hereditary spastic paraplegia (HSP) and related disorders.
- Conditions
- Hereditary Spastic Paraplegia, Neurodegenerative Diseases, Pediatric Disorder, Spasticity, Muscle
- Trial name
- NCT05354622
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Boston Children's Hospital
Amyotrophic Lateral Sclerosis (ALS)
Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
This is a single-center, open-label, dose-escalation, investigator-initiated pilot study to evaluate the safety, tolerability, dose-limiting toxicity (DLT), and pharmacodynamic signals of orelabrutinib in patients with…
- Trial name
- NCT07875972
- Testing
- Orelabrutinib
- Volunteers
- 12 people
- Sponsor
- Liu Qing
Spinal Muscular Atrophy 1
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi.
- Trial name
- START (NCT03421977)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy (SMA), Spinal Muscular Atrophy Type I
Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine Regions
The systematic inclusion of spinal muscular atrophy (SMA) in France's neonatal genetic screening (NGS) program, scheduled for September 2025, represents a major milestone in public health.
- Trial name
- PSYSMA (NCT07208903)
- Testing
- focus group discussions, Interview with the psychologist
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
Amyotrophic Lateral Sclerosis (ALS)
Transcranial Static Magnetic Stimulation (tSMS) and Potential Theranostic Biomarkers in Amyotrophic Lateral Sclerosis.
The objective of the present study is to assess the efficacy of tSMS in ALS patients. This will be achieved by monitoring: levels of NF-L and other potential innovative biomarkers, clinical progression, trough ALSFRS-R.
- Trial name
- NCT06834269
- Testing
- Transcranial magnetic stimulation (tSMS)
- Where
- Italy
- Volunteers
- 60 people
- Sponsor
- Campus Bio-Medico University
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