Clinical trials: Neuron Disease, Motor
Amyotrophic Lateral Sclerosis
Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis
The purpose of this study was to evaluate the safety of intramuscular administration of Engensis in Participants with Amyotrophic Lateral Sclerosis as compared to Placebo.
- Trial name
- REViVALS-1A (NCT04632225)
- Testing
- Engensis
- Where
- 5 sites in United States, South Korea
- Volunteers
- 18 people
- Sponsor
- Helixmith Co., Ltd.
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi.
- Conditions
- Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
- Trial name
- NCT04042025
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 31 sites in United States, Australia and 7 more
- Volunteers
- 85 people
- Sponsor
- Novartis Gene Therapies
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients
This is a phase 1/2a randomized, double-blind study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of study drug AJ201 in subjects with Spinal and Bulbar Muscular Atrophy (SBMA).
- Trial name
- NCT05517603
- Testing
- AJ201
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- AnnJi Pharmaceutical Co., Ltd.
Spinal and Bulbar Muscular Atrophy
Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy
The purpose of this study was to determine if BVS857 is safe, tolerable and increases thigh muscle thickness in patients with spinal bulbar and muscular atrophy (SBMA).
- Trial name
- NCT02024932
- Testing
- BVS857
- Where
- 6 sites in United States, Denmark and 2 more
- Volunteers
- 37 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis
IC14 for ALS Patients Expanded Access
The primary objective is to provide the investigational product, IC14, at the dose of 8 mg/kg intravenously every 2 weeks for 12 weeks to 6 participants with amyotrophic lateral sclerosis (ALS).
- Trial name
- EAP (NCT04309604)
- Testing
- IC14
- Where
- United States
- Sponsor
- Implicit Bioscience
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.
Spinal and Bulbar Muscular Atrophy
Specified Drug-Use Survey of Leuprorelin Acetate Injection Kit 11.25 mg "All-Case Investigation: Spinal and Bulbar Muscular Atrophy (SBMA)"
The purpose of this survey is to evaluate the long-term safety and efficacy of leuprorelin acetate injection kit 11.25 mg in patients with spinal and bulbar muscular atrophy (SBMA) in the routine clinical setting.
- Trial name
- NCT03555578
- Testing
- Leuprorelin Acetate
- Where
- Japan
- Volunteers
- 1,905 people
- Sponsor
- Takeda
ALS (Amyotrophic Lateral Sclerosis), TBI Traumatic Brain Injury
Augmented Reality BCI Longitudinal Study for Persons With ALS, Stroke, TBI and SCI Utilizing Cognixion + Apple Vision Pro
The goal of this study is refine the usability of a BCI capable communication platform. The study will take place in the United States area and will enroll up to 10 participants with late stage ALS, traumatic brain injury (TBI)…
- Conditions
- ALS (Amyotrophic Lateral Sclerosis), TBI Traumatic Brain Injury, SCI - Spinal Cord Injury, Stroke
- Trial name
- NCT07209943
- Testing
- Cognixion + Apple Vision Pro
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Cognixion
Type 2 Spinal Muscular Atrophy
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
This was a Phase III multi-center, single dose (1.2 x 10^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never…
- Trial name
- STEER (NCT05089656)
- Testing
- OAV101
- Where
- 42 sites in United States, Brazil and 11 more
- Volunteers
- 126 people
- Sponsor
- Novartis Pharmaceuticals
Muscular Atrophy, Spinal
A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies
In this study, researchers will know more about the effects of nusinersen, also known as Spinraza®, in pregnant participants with spinal muscular atrophy, also known as SMA.
- Trial name
- NCT05789758
- Testing
- No Intervention
- Where
- 14 sites in United States, United Kingdom
- Volunteers
- 20 people
- Sponsor
- Biogen
Amyotrophic Lateral Sclerosis
A 12-week, Multicenter, Safety and Dose-ranging Study of 3 Oral Doses of TCH346 in Patients With Amyotrophic Lateral Sclerosis
This study is the first to be performed in Amyotrophic Lateral Sclerosis (ALS) patients with the novel compound TCH346. Its purpose is to evaluate the safety and clinical effects of 3 dose levels of TCH 346 compared to placebo in…
- Trial name
- NCT00036413
- Testing
- TCH346
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis
Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients With ALS
This study will evaluate whether prolonged oral levosimendan can preserve respiratory function more effectively than placebo, resulting in better patient functionality as measured by the ALSFRS-R scale.
- Trial name
- REFALS (NCT03505021)
- Testing
- Levosimendan
- Where
- 104 sites in United States, Australia and 12 more
- Volunteers
- 496 people
- Sponsor
- Orion Corporation, Orion Pharma
Healthy
A Multiple Dose Study to Investigate Safety, Tolerability and Pharmacokinetics of TBN
The purpose of this study is to assess the safety, tolerability and pharmacokinetics (i.e. how study drug is taken up by the body) of TBN in healthy participants.
- Trial name
- NCT04667013
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Guangzhou Magpie Pharmaceuticals Co., Ltd.
Amyotrophic Lateral Sclerosis (ALS)
Noninvasive Assessment of Neuromuscular Disease Using Electrical Impedance Myography
The purpose of this protocol is to test a new Electrical Impedance Myography (EIM) device and study its reliability and ability to differentiate ALS patients from healthy controls.
- Trial name
- NCT01803386
- Testing
- EIM Measurements
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Skulpt, Inc.
Amyotrophic Lateral Sclerosis
Extension Study Evaluating The Safety And Tolerability of AMX0035
The primary objective is to evaluate the safety and tolerability of AMX0035 over 108 weeks of open label treatment for participants previously enrolled in Study A35-004 (PHOENIX).
- Trial name
- NCT05619783
- Testing
- AMX0035
- Where
- 46 sites in Belgium, France and 9 more
- Volunteers
- 352 people
- Sponsor
- Amylyx Pharmaceuticals Inc.
Amyotrophic Lateral Sclerosis
Clinical Trial of Ultra-high Dose Methylcobalamin for ALS
To examine the clinical efficacy and safety of ultra-high dose (50mg, im, twice a week) methylcobalamin in retarding the progression of symptoms in amyotrophic lateral sclerosis (ALS) patients, we enroll ALS patients diagnosed by…
- Trial name
- JETALS (NCT03548311)
- Testing
- methylcobalamin, saline solution
- Where
- 16 sites in Japan
- Volunteers
- 130 people
- Sponsor
- Eisai Co., Ltd.
Amyotrophic Lateral Sclerosis
Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year
This study assessed the effect of tirasemtiv versus placebo on respiratory function in patients with ALS.
- Trial name
- VITALITY-ALS (NCT02496767)
- Testing
- Tirasemtiv
- Where
- 81 sites in United States, Belgium and 9 more
- Volunteers
- 744 people
- Sponsor
- Cytokinetics
Amyotrophic Lateral Sclerosis
A follow-on Study to Assess Long-term Safety and Tolerability of i.c.v Administration of sNN0029 in Patients With ALS
This is an open-label, follow-on phase 1 study to assess the long-term safety and tolerability of continuous i.c.v administration of 4 μg sNN0029/day in patients with ALS who previously participated in study sNN0029-003
- Trial name
- NCT02269436
- Testing
- sNN0029 infusion solution
- Where
- 2 sites in Belgium, Netherlands
- Volunteers
- 11 people
- Sponsor
- Newron Sweden AB
Spinal Muscular Atrophy
A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
The primary objective of the study is to explore the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments.
- Trial name
- DigiNOA (NCT05109637)
- Testing
- Konectom NMD Application
- Where
- 5 sites in Germany
- Volunteers
- 93 people
- Sponsor
- Biogen
Muscular Atrophy, Spinal
Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection
The primary objective of this study is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in China.The secondary objectives are to collect data on the efficacy and the pharmacokinetics of…
- Trial name
- PANDA (NCT04419233)
- Testing
- Nusinersen Sodium Injection
- Where
- 9 sites in China
- Volunteers
- 50 people
- Sponsor
- Biogen