Clinical trials: Neuron Disease, Motor
Muscular Atrophy, Spinal
A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)
The purpose of this open-label, single arm study is to further evaluate long-term tolerability, safety and efficacy outcomes of olesoxime in participants with Spinal Muscular Atrophy (SMA) who previously participated in one of…
- Trial name
- NCT02628743
- Testing
- Olesoxime
- Where
- 24 sites in Belgium, France and 5 more
- Volunteers
- 131 people
- Sponsor
- Hoffmann-La Roche
Amyotrophic Lateral Sclerosis (ALS)
Safety Study of XT-150 in Participants With ALS
This is a Phase 1, open-label, multi-center safety study of XT-150 in adult participants with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT06704347
- Testing
- XT-150
- Where
- 4 sites in United States
- Volunteers
- 8 people
- Sponsor
- Xalud Therapeutics, Inc.
Amyotrophic Lateral Sclerosis (ALS), Angina, Stable
Quest to Analyze One Thousand Humans Meditating
This study will be focused on assessing the molecular, physiological, and emotional correlates of an intensive meditation experience in the context of a retreat setting in a large 2000 plus-person cohort comprised of healthy and…
- Conditions
- Amyotrophic Lateral Sclerosis (ALS), Angina, Stable, Anxiety, Asthma
- Trial name
- QUANTUM (NCT06583395)
- Testing
- Advanced multi-component meditation practice
- Where
- United States
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Tobias Moeller-Bertram
Healthy Volunteers
A Study of VRG50635 in Healthy Volunteers
The primary objectives of this study are to investigate the safety and tolerability of VRG50635 and to determine how VRG50635 is absorbed by the body.
- Trial name
- NCT06286475
- Testing
- VRG50635
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Verge Genomics
Spinal Muscular Atrophy
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.
- Trial name
- NCT06288230
- Testing
- vesemnogene lantuparvovec
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Lantu Biopharma
ALS (Amyotrophic Lateral Sclerosis)
Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis
This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000.
- Trial name
- KOANEWA (NCT07401121)
- Testing
- AAV9 Gene therapy
- Where
- Australia
- Volunteers
- 15 people
- Sponsor
- Celosia Therapeutics Pty Ltd
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
A Study of NIDO-361 in Patients With SBMA
The purpose of this study is to evaluate the safety, tolerability, and efficacy of NIDO-361 in adult patients with Spinal and Bulbar Muscular Atrophy (SBMA).
- Trial name
- PIONEER KD (NCT06411912)
- Testing
- NIDO-361
- Where
- 5 sites in Denmark, Italy and 2 more
- Volunteers
- 54 people
- Sponsor
- Nido Biosciences, Inc.
Amyotrophic Lateral Sclerosis (ALS)
A Multicenter, Dose Ranging Safety and Pharmacokinetics Study of Arimoclomol in ALS
The primary purpose of this study is to evaluate the safety and tolerability of arimoclomol in ALS patients following 90 days of dosing. In addition, the amount of arimoclomol in blood and cerebrospinal fluid will be measured.
- Trial name
- NCT00244244
- Testing
- arimoclomol
- Where
- 10 sites in United States
- Volunteers
- 80 people
- Sponsor
- CytRx
Amyotrophic Lateral Sclerosis
Exploratory Study of Biotelemetry in Amyotrophic Lateral Sclerosis
Study 201283 is an exploratory, non-controlled, non-drug study in Amyotrophic Lateral Sclerosis (ALS) subjects. This study is being conducted as the first step for developing new meaningful measure(s) which might prove to be more…
- Trial name
- NCT02447952
- Testing
- Faros Sensor (FS) and LifeInsight Hub, Fast Fix electrode patch, Quantitative Measure of Speech (Core Phase Only)
- Where
- 2 sites in United Kingdom
- Volunteers
- 25 people
- Sponsor
- GlaxoSmithKline
Spinal Muscular Atrophy
A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients
This study aims to assess the safety and effectiveness of onasemnogene abeparvovec intrathecal injection in spinal muscular atrophy (SMA) patients in clinical practice in Japan.
- Trial name
- NCT07767890
- Where
- 4 sites in Japan
- Volunteers
- 80 people
- Sponsor
- Novartis Pharmaceuticals
Primary Lateral Sclerosis, Adult, 1
"Individual Patient Expanded Access IND to Treat Primary Lateral Sclerosis"
This Individual Patient Expanded Access IND has been created as requested by an 83-year-old man who suffers Primary Lateral Sclerosis and for which the drugs currently approved are not providing an improvement over the…
- Trial name
- NCT04825613
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Amyotrophic Lateral Sclerosis, Motor Neuron Disease
Phase 1 Dose Escalation and PK Study of Cu(II)ATSM in ALS/MND
Multicenter, open-label , single and multiple dose-escalation and pharmacokinetic study
- Trial name
- NCT02870634
- Testing
- Cu(II)ATSM
- Where
- 2 sites in Australia
- Volunteers
- 50 people
- Sponsor
- Collaborative Medicinal Development Pty Limited
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 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
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, Spinal Muscular Atrophy Type 3
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- TOPAZ (NCT03921528)
- Testing
- SRK-015
- Where
- 16 sites in United States, Italy and 2 more
- Volunteers
- 58 people
- Sponsor
- Scholar Rock, Inc.
Amyotrophic Lateral Sclerosis
Regulatory T Cells for Amyotrophic Lateral Sclerosis
Phase 1 Safety Run-in Study of 6 patients followed by Phase 1b Randomized, Double Blind, Placebo Control Trial of CK0803, neurotropic, allogeneic, umbilical cord blood derived T regulatory (Treg) cells in additional 60 patients…
- Trial name
- REGALS (NCT05695521)
- Testing
- CK0803, Excipient
- Where
- 3 sites in United States
- Volunteers
- 66 people
- Sponsor
- Cellenkos, Inc.
Amyotrophic Lateral Sclerosis, ALS
Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis
The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow-derived stem cells("HYNR-CS inj"), through intrathecal delivery for the treatment in patients with ALS. This study consists of 2 steps.
- Trial name
- NCT01363401
- Testing
- HYNR-CS inj, Control group
- Where
- South Korea
- Volunteers
- 72 people
- Sponsor
- Corestemchemon, Inc.
Amyotrophic Lateral Sclerosis, ALS
Open Label Study to Evaluate Ciprofloxacin/Celecoxib Combination in Patients With ALS
This is an open label, off label study, to provide interested ALS patients with Ciprofloxacin/Celecoxib fixed dose combination, while assessing safety and tolerability and routine disease progression measures (ALSFRS-R and Vital…
- Trial name
- NCT04165850
- Testing
- Fixed dose combination Ciprofloxacin/Celecoxib
- Where
- Israel
- Volunteers
- 16 people
- Sponsor
- NeuroSense Therapeutics Ltd.
Type I Spinal Muscular Atrophy
Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy.
- Trial name
- NCT06576388
- Testing
- EXG001-307 injection
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd