Clinical trials: Motor Neuron Diseases
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Amyotrophic Lateral Sclerosis
A Safety Study of sNN0029 Administration Via Intracerebroventricular Route to Patients With ALS
This is a phase I, multicentre randomised, double-blind, placebo-controlled trial to assess the safety and tolerability of continuous i.c.v.
- Trial name
- NCT01999803
- Testing
- sNN0029
- Where
- 2 sites in Belgium, Netherlands
- Volunteers
- 15 people
- Sponsor
- Newron Sweden AB
Spinal Muscular Atrophy
A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically…
- Trial name
- NURTURE (NCT02386553)
- Testing
- Nusinersen
- Where
- 21 sites in United States, Australia and 5 more
- Volunteers
- 25 people
- Sponsor
- Biogen
Amyotrophic Lateral Sclerosis (ALS)
Masitinib in Combination With Riluzole for the Treatment of Patients Suffering From Amyotrophic Lateral Sclerosis (ALS)
The objective is to compare the efficacy and safety of masitinib in combination with riluzole in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT02588677
- Testing
- Masitinib (4.5), Riluzole, Masitinib (3.0)
- Where
- Spain
- Volunteers
- 394 people
- Sponsor
- AB Science
Upper Motor Neuron Disease
Tracking Self-Reported Functional Needs and Quality of Life
The study aims to characterize the population of Cionic Neural Sleeve users and assess their health-related quality of life.
- Trial name
- NCT07284810
- Testing
- Cionic Neural Sleeve System
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Cionic, Inc.
Amyotrophic Lateral Sclerosis
Riluzole Oral Soluble Film (ROSF) Swallowing Safety in Amyotrophic Lateral Sclerosis (ALS)
The primary objective is to evaluate the effect, if any, of a single 50 mg dose of Riluzole Oral Soluble Film (ROSF) on swallowing safety in individuals with amyotrophic lateral sclerosis.
- Trial name
- ROSF (NCT03679975)
- Testing
- Riluzole Oral Soluble film (ROSF) 50 mg
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Aquestive Therapeutics
ALS, Amyotrophic Lateral Sclerosis
A Phase 1 Study to Investigate the Safety and Pharmacokinetics of Fosigotifator in Patients With Amyotrophic Lateral Sclerosis
Fosigotifator is an investigational drug being researched for the treatment of Amyotrophic Lateral Sclerosis. This is an up to 156-week, 2-part study.
- Trial name
- NCT04948645
- Testing
- Fosigotifator
- Where
- 14 sites in United States, Canada
- Volunteers
- 31 people
- Sponsor
- AbbVie
Spinal Muscular Atrophy
An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)
An open-label, multi-part, first-in-human study of oral branaplam in infants with Type 1 spinal muscular atrophy. The purpose of this study was to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD)…
- Trial name
- NCT02268552
- Testing
- branaplam
- Where
- 14 sites in Belgium, Bulgaria and 5 more
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis
Dose Escalation and Safety Study of Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis
The study is to determine the feasibility, safety, toxicity, and maximum tolerated (safe) dose of human spinal derived neural stem cell transplantation for the treatment of Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT01730716
- Testing
- Human spinal cord stem cell implantation
- Where
- 3 sites in United States
- Volunteers
- 18 people
- Sponsor
- Neuralstem Inc.
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
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
Alzheimer's Disease, Multiple Sclerosis
A Biospecimen and Clinical Data Study on Patients for Drug & Biomarker Discovery
The purpose of this research project is to collect and store blood samples and clinical data. Researchers can then use the stored samples in future studies.
- Conditions
- Alzheimer's Disease, Multiple Sclerosis, Parkinson's Disease, Huntington's Disease
- Trial name
- NCT01592552
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Neurologic Disorder, ALS
Functional Outcomes and Control Using Synchron BCI - Canada
Functional Outcomes and Control Using Synchron BCI - Canada (FOCUS-CAN)
- Conditions
- Neurologic Disorder, ALS, Motor Neuron Disease
- Trial name
- FOCUS-CAN (NCT07446114)
- Testing
- Stentrode
- Where
- 2 sites in Canada
- Volunteers
- 10 people
- Sponsor
- Synchron, Inc.
Amyotrophic Lateral Sclerosis
A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS)
The purpose of this study is to assess the effect of reldesemtiv versus placebo on functional outcomes in ALS.
- Trial name
- COURAGE-ALS (NCT04944784)
- Testing
- Reldesemtiv
- Where
- 84 sites in United States, Australia and 14 more
- Volunteers
- 489 people
- Sponsor
- Cytokinetics
Opioid Withdrawal
Dexmedetomidine in the Treatment of Symptoms Of Acute Opioid Withdrawal
This Phase 1b/2 inpatient study assessed the safety, pharmacokinetics, and early signs of efficacy of escalating doses of BXCL501 versus placebo following discontinuation of morphine maintenance.
- Trial name
- NCT04470050
- Testing
- Dexmedetomidine
- Where
- 3 sites in United States
- Volunteers
- 225 people
- Sponsor
- BioXcel Therapeutics 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.
Complete or Incomplete Paraplegia/quadriplegia, Spinal Cord Injury
Research on Wireless Brain Implant System for General Control of External Devices
The clinical trial aims to evaluate the safety and efficacy of the minimally invasive, wireless brain-machine interface system (WRS) in enabling general brain control of external devices, such as a cursor and other assistive…
- Conditions
- Complete or Incomplete Paraplegia/quadriplegia, Spinal Cord Injury, Brainstem Stroke, Amyotrophic Lateral Sclerosis
- Trial name
- RISE (NCT06829212)
- Testing
- WRS
- Where
- China
- Volunteers
- 4 people
- Sponsor
- Shanghai StairMed Technology Co., Ltd.
Amyotrophic Lateral Sclerosis
A Study of LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis
This study will examine what happens when patients with amyotrophic lateral sclerosis (ALS) are given an investigational medication (study drug) known as LTX-002.
- Trial name
- NeurALS (NCT07660614)
- Testing
- LTX-002
- Where
- 5 sites in Germany, Italy and 2 more
- Volunteers
- 56 people
- Sponsor
- Leal Therapeutics, Inc
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
Motor Neuron Disease
LIME Study (LFB IVIg MMN Efficacy Study)
The aim of this study is to evaluate the efficacy and safety of I10E (LFB 10% ready-to-use liquid human intravenous immunoglobulin) compared to Kiovig® for the maintenance treatment of MMN in a randomized, double-blind, active…
- Trial name
- LIME (NCT01951924)
- Testing
- Biological : I10E (Human normal Immunoglobulin for intravenous administration 100mg/mL), Biological: Kiovig® (Human normal Immunoglobulin for intravenous administration 100mg/mL)
- Where
- 18 sites in France, Italy and 2 more
- Volunteers
- 23 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies