Clinical trials: Neuron Disease, Motor
Amyotrophic Lateral Sclerosis
Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial
A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS) who have completed the ORARIALS-01 trial.
- Trial name
- NCT03836716
- Testing
- Arimoclomol
- Where
- 23 sites in United States, Belgium and 9 more
- Volunteers
- 120 people
- Sponsor
- ZevraDenmark
Spinal Muscular Atrophy
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.
- Trial name
- NCT05824169
- Testing
- GC101
- Where
- 4 sites in China
- Volunteers
- 18 people
- Sponsor
- GeneCradle Inc
ALS
Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS
The purpose of this study is to evaluate the pharmacokinetic and pharmacodynamic effect after a single dose or at steady state after multiple doses of AMX0035 in adults with sporadic ALS.
- Trial name
- NCT04987671
- Testing
- AMX0035
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Amylyx Pharmaceuticals Inc.
Spinal Muscular Atrophy, SMA
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones…
- Conditions
- Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- OPAL (NCT07047144)
- Testing
- Apitegromab, Nusinersen, Risdiplam
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 52 people
- Sponsor
- Scholar Rock, Inc.
Amyotrophic Lateral Sclerosis
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)
In this study, researchers will learn more about the safety of tofersen, also known as Qalsody®. This is a drug available for doctors to prescribe for people with a certain type of amyotrophic lateral sclerosis, also known as…
- Trial name
- NCT07223723
- Testing
- Tofersen
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Biogen
Alzheimer Disease, Mild Cognitive Impairment
Clinical Feasibility of Speech Phenotyping for Remote Assessment of Neurodegenerative and Psychiatric Disorders
The primary objective of the study is to evaluate the feasibility of eliciting continuous narrative speech in different neurodegenerative and psychiatric indications, using remote, self-administered speech tasks, as measured by…
- Conditions
- Alzheimer Disease, Mild Cognitive Impairment, Dementia With Lewy Bodies, Dementia, Vascular
- Trial name
- RHAPSODY (NCT04939818)
- Where
- 2 sites in United Kingdom
- Volunteers
- 172 people, healthy volunteers welcome
- Sponsor
- Novoic Limited
Prostate Cancer
A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer Drugs
ANDROMEDA is a first-in-human, Phase I/II, open-label, multicenter study of AZD9750 in participants with metastatic prostate cancer.
- Trial name
- ANDROMEDA (NCT07336446)
- Testing
- AZD9750, AZD5305
- Where
- 5 sites in United States, Australia and 2 more
- Volunteers
- 6 people
- Sponsor
- AstraZeneca
Cystic Fibrosis, Motor Neuron Disease
MN4000 for Treatment of CF and MND Patients in the Home Setting
The study was a non-randomized open label pilot study. It was an observational design conducted at one (1) site in the US. All enrolled subjects received treatment with the MN4000.
- Conditions
- Cystic Fibrosis, Motor Neuron Disease, Airway Clearance Impairment
- Trial name
- NCT03797677
- Testing
- MN 4000
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Hill-Rom
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
Amyotrophic Lateral Sclerosis
31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Amyotrophic Lateral Sclerosis (REPAIR-ALS)
REPAIR-ALS is a single-center open label pilot, sequential group, investigator and patient blinded study to assess the CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients who have been…
- Trial name
- REPAIR-ALS (NCT03843710)
- Testing
- Gold Nanocrystals
- Where
- United States
- Sponsor
- Clene Nanomedicine
Tetraplegia/Tetraparesis, Quadriplegia
Precise Robotically IMplanted Brain-Computer InterfacE
The PRIME Study is a first-in-human early feasibility study to evaluate the initial clinical safety and device functionality of the Neuralink N1 Implant and R1 Robot device designs in participants with tetraparesis or…
- Conditions
- Tetraplegia/Tetraparesis, Quadriplegia, Cervical Spinal Cord Injury, Amyotrophic Lateral Sclerosis
- Trial name
- PRIME (NCT06429735)
- Testing
- N1 Implant, R1 Robot
- Where
- 2 sites in United States
- Volunteers
- 15 people
- Sponsor
- Neuralink Corp
Amyotrophic Lateral Sclerosis
A Study of GDC-0134 to Determine Initial Safety, Tolerability, and Pharmacokinetic Parameters in Participants With Amyotrophic Lateral Sclerosis
This first-in-human, double-blind, placebo-controlled Phase I study will be conducted in participants with amyotrophic lateral sclerosis (ALS) to explore safety, tolerability, and pharmacokinetic (PK) properties of GDC-0134.
- Trial name
- NCT02655614
- Testing
- GDC-0134, Rabeprazole, Midazolam
- Where
- 10 sites in United States, Canada and 1 more
- Volunteers
- 54 people
- Sponsor
- Genentech, Inc.
Amyotrophic Lateral Sclerosis
A Study to Evaluate, Safety, Tolerability, and PK of AP-101 in Participants With ALS
The purpose of this study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.
- Trial name
- NCT05039099
- Testing
- AP-101
- Where
- 13 sites in United States, Belgium and 4 more
- Volunteers
- 73 people
- Sponsor
- AL-S Pharma
Muscular Atrophy, Spinal
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA).
- Trial name
- FIREFISH (NCT02913482)
- Testing
- Risdiplam
- Where
- 32 sites in United States, Belgium and 14 more
- Volunteers
- 62 people
- Sponsor
- Hoffmann-La Roche
Amyotrophic Lateral Sclerosis (ALS)
Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis
Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis
- Trial name
- UNITE-ALS (NCT07023835)
- Testing
- 50 mg Usnoflast, 75 mg Usnoflast
- Where
- 49 sites in United States, Australia and 10 more
- Volunteers
- 240 people
- Sponsor
- Zydus Therapeutics Inc.
Amyotrophic Lateral Sclerosis
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis
The primary objective of this study is to evaluate the safety, tolerability of single-ascending doses of BIIB100 in adults with amyotrophic lateral sclerosis (ALS).
- Trial name
- NCT03945279
- Testing
- BIIB100
- Where
- 9 sites in United States
- Volunteers
- 49 people
- Sponsor
- Biogen
Tetraplegia/Tetraparesis, Quadriplegia
UAE-PRIME: A Feasibility Study of a Precise Robotically Implanted Brain-Computer Interface for the Control of External Devices
The UAE-PRIME Study is a feasibility study designed to assess the initial clinical safety and functionality of the Neuralink N1 Implant and R1 Robot.
- Conditions
- Tetraplegia/Tetraparesis, Quadriplegia, Quadriplegia/Tetraplegia, Cervical Spinal Cord Injury
- Trial name
- UAE-PRIME (NCT06992596)
- Testing
- N1 Implant, R1 Robot
- Where
- United Arab Emirates
- Volunteers
- 10 people
- Sponsor
- Neuralink Corp
Muscular Atrophy, Spinal
A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)
In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA).
- Trial name
- RESPOND (NCT04488133)
- Testing
- Nusinersen
- Where
- 15 sites in United States, Germany and 3 more
- Volunteers
- 46 people
- Sponsor
- Biogen
Muscular Atrophy, Spinal
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)
This was a phase IV Open-label, single-arm, single-dose, multicenter study, to evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in…
- Trial name
- OFELIA (NCT05073133)
- Testing
- OAV101
- Where
- 5 sites in Argentina, Brazil
- Volunteers
- 16 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis
A Prospective Study to Evaluate the Correlation Between Oculometric Measurements and Clinical Endpoints in ALS Patients
This is a prospective study in a cohort of about 45 patients with ALS participating in the Neurosense PrimeC drug study (NCT05357950).
- Trial name
- NCT05407428
- Testing
- NeuraLight software-based platform
- Where
- Israel
- Volunteers
- 45 people
- Sponsor
- NeuraLight