Clinical trials: Motor Neurone Disease
Amyotrophic Lateral Sclerosis
A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)
The purpose of this study is to assess the long-term safety and tolerability of reldesemtiv in patients with ALS who have successfully completed dosing in the Phase 3 clinical trial, CY 5031 (also known as COURAGE-ALS)
- Trial name
- NCT05442775
- Testing
- Reldesemtiv
- Where
- 41 sites in United States, Australia and 7 more
- Volunteers
- 71 people
- Sponsor
- Cytokinetics
Amyotrophic Lateral Sclerosis
To Determine the Maximum Tolerated Dose (MTD) of HK-001 in Healthy Volunteers
Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts. After single dose administration, followed by an independent Data and Safety Monitoring Board (DSMB) meeting…
- Trial name
- NCT03651349
- Testing
- HK-001
- Where
- Taiwan
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Everfront Biotech Co., Ltd.
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
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
Safety and Efficacy of TRO19622 as add-on Therapy to Riluzole Versus Placebo in Treatment of Patients Suffering From ALS
The purpose of the assay is to assess the safety and the efficacy of TRO19622 330 mg QD as add-on therapy to riluzole 50 mg bid in the treatment of patients suffering from ALS, as compared to placebo, assessed by the 18-month…
- Trial name
- MITOTARGET (NCT00868166)
- Testing
- Olesoxime, Riluzole
- Where
- 15 sites in Belgium, France and 3 more
- Volunteers
- 512 people
- Sponsor
- Hoffmann-La Roche
Neuromuscular Disorders, Neurodevelopmental Disorders
Efficacy and Usability of the EXPLORER Exoskeleton in Children With Neurodevelopmental Disorders in Natural Environments
Neurodevelopmental disorders often lead to abnormal development of the Central Nervous System (CNS), frequently causing motor dysfunctions such as an inability to stand and walk.
- Trial name
- NCT06765070
- Testing
- EXPLORER
- Where
- 6 sites in Spain
- Volunteers
- 15 people
- Sponsor
- MarsiBionics
Spinal Muscular Atrophy
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA).
- Trial name
- NCT01839656
- Testing
- nusinersen
- Where
- 4 sites in United States, Canada
- Volunteers
- 21 people
- Sponsor
- Biogen
Amyotrophic Lateral Sclerosis
Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ZYIL1 in Patients With Amyotrophic Lateral Sclerosis
ZYIL1 is expected to show benefit in patients with Amyotrophic Lateral Sclerosis (ALS). The present study aims to determine the safety, tolerability, pharmacokinetics, and pharmacodynamics of ZYIL1 when administered to subjects…
- Trial name
- NCT05981040
- Testing
- ZYIL1 capsules 25 mg and ZYIL1 capsules 50 mg
- Where
- 7 sites in India
- Volunteers
- 24 people
- Sponsor
- Zydus Lifesciences Limited
Amyotrophic Lateral Sclerosis
A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis
This 8-week randomized, open-label evaluation will examine the acute safety and tolerability of 4 different dosing regimens of Acthar to inform dose selection for future studies of Acthar in patients with Amyotrophic Lateral…
- Trial name
- NCT01906658
- Testing
- Repository corticotropin injection
- Where
- 17 sites in United States
- Volunteers
- 43 people
- Sponsor
- Mallinckrodt
Spinal Muscular Atrophy Type I
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
This is a Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular atrophy (SMA) Type 1 and who are genetically defined by a biallelic…
- Trial name
- NCT03837184
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 4 sites in Japan, South Korea and 1 more
- Volunteers
- 2 people
- Sponsor
- Novartis Gene Therapies
Amyotrophic Lateral Sclerosis
RT001 in Amyotrophic Lateral Sclerosis
RT001-014 is a Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects with Amyotrophic Lateral Sclerosis
- Trial name
- NCT04762589
- Testing
- RT001
- Where
- 4 sites in Estonia, Latvia and 2 more
- Volunteers
- 43 people
- Sponsor
- Biojiva LLC
Spinal Muscular Atrophy (SMA), Carrier Screening
Multi-disease Carrier Screening Test Validation
The purpose of this study is to collect blood samples to enable validation of genetic testing for diseases within a multi-disease carrier screening panel.
- Conditions
- Spinal Muscular Atrophy (SMA), Carrier Screening, Genetic Testing
- Trial name
- NCT01663584
- Testing
- Blood draw
- Where
- United States
- Sponsor
- Natera, Inc.
Spinal Muscular Atrophy
ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
The primary purpose is to address critical evidence in the treatment landscape for Spinal Muscular Atrophy (SMA), specifically focusing on the intrathecal formulation of onasemnogene abeparvovec-brve (ITVISMA®). U.S.
- Trial name
- NCT07448610
- Testing
- Onasemnogene Abeparvovec-brve
- Where
- 6 sites in United States
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
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.
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
ALS, ALS (Amyotrophic Lateral Sclerosis)
Systems Biology of Amyotrophic Lateral Sclerosis (ALS)
The goal of this observational study is to identify molecular features in multiple clinical samples and/or the patient environment that are associated with ALS.
- Trial name
- NCT07007390
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Viome
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.
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.
Amyotrophic Lateral Sclerosis
A Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis
This study is conducted to evaluate the safety and tolerability of the drug product sNN0029, containing the growth factor VEGF165, when administered directly into one of the fluid filled cavities in the brain using an implanted…
- Trial name
- NCT00800501
- Testing
- sNN0029
- Where
- Belgium
- Volunteers
- 18 people
- Sponsor
- Newron Sweden AB
Spinal Muscular Atrophy
A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia
The aim of this retrospective medical chart review is to describe the clinical outcomes, clinical characteristics, and demographics of patients with spinal muscular atrophy (SMA) type 1 treated with onasemnogene abeparvovec (OA)…
- Trial name
- SMA-REAL (NCT07737756)
- Volunteers
- 6 people
- Sponsor
- Novartis Pharmaceuticals