Clinical trials: Neuron Disease, Motor
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
Muscular Atrophy, Spinal
A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy
The aim of this study was to evaluate treatment patterns, SMA-related complications and services, and all-cause medical encounters of patients with SMA receiving multiple DMTs in the real world.
- Trial name
- NCT07403214
- Where
- United States
- Volunteers
- 4,805 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis
Early Access Program for Ulefnersen in Children, Adolescents and Adults With FUS-associated Amyotrophic Lateral Sclerosis (FUS-ALS)
This expanded access program provides access to ulefnersen for eligible patients with FUS-associated amyotrophic lateral sclerosis (FUS-ALS) who meet protocol-defined eligibility criteria.
- Trial name
- NCT07862959
- Testing
- Ulefnersen
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Amyotrophic Lateral Sclerosis
A Safety and Efficacy Study of ARGX-119 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS)
This study aims to evaluate the safety of ARGX-119 in adults with ALS. The study will also assess the impact of ARGX-119 on ALS disease outcomes, including muscle function.
- Trial name
- ReALiSe (NCT06441682)
- Testing
- ARGX-119
- Where
- 9 sites in Belgium, Canada and 4 more
- Volunteers
- 60 people
- Sponsor
- argenx
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.
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
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
PIONEER-ALS is a Phase 1/2, multicenter, open-label, ascending dose, uncontrolled, first-in-human study that will evaluate the safety, tolerability and effects on clinical and biomarker endpoints of intracisternal administration…
- Trial name
- ALS (NCT07287397)
- Testing
- VTx-002, Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone
- Where
- 11 sites in United States, Belgium and 2 more
- Volunteers
- 36 people
- Sponsor
- Vector Y Therapeutics
ALS (Amyotrophic Lateral Sclerosis)
This Study Evaluates the Safety, Target Engagement, and Preliminary Efficacy of Galunisertib (TGF-βR1/ALK5 Inhibitor)Combined With Nerandomilast (PDE4 Inhibitor) in GREM2-positive ALS, a Biomarker-defined Subgroup Hypothesized to Reflect Heightened TGF-β/SMAD-driven Astrocytic and Fibrotic Signaling
Amyotrophic lateral sclerosis (ALS) is a relentlessly progressive neurodegenerative disorder characterized by loss of upper and lower motor neurons, leading to muscle weakness, respiratory decline, and eventual mortality.
- Trial name
- NCT07321860
- Testing
- Galunisertib + Nerandomilast Combination
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Gipfel Life Sciences GmbH
Metastatic Castration-resistant Prostate Cancer
A Study to Compare the Efficacy and Safety of BMS-986365 Versus the Investigator's Choice of Therapy in Participants With Metastatic Castration-resistant Prostate Cancer
The purpose of this study is to compare the efficacy and safety of BMS-986365 versus the investigator's choice of therapy in participants with Metastatic Castration-resistant Prostate Cancer.
- Trial name
- rechARge (NCT06764485)
- Testing
- BMS-986365, Enzalutamide, Abiraterone
- Where
- 227 sites in United States, Argentina and 24 more
- Volunteers
- 960 people
- Sponsor
- Celgene
Amyotrophic Lateral Sclerosis
Blood-Brain Barrier Opening Using MR-Guided Focused Ultrasound in Patients With Amyotrophic Lateral Sclerosis
The purpose of this study is to evaluate the safety, tolerability, and feasibility of Blood-Brain Barrier (BBB) opening using transcranial MRI-guided focused ultrasound in conjunction with an intravenous ultrasound contrast agent…
- Trial name
- NCT03321487
- Testing
- Blood-Brain Barrier opening with MRgFUS
- Where
- Canada
- Volunteers
- 4 people
- Sponsor
- InSightec
Spinal Muscular Atrophy
Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States
This global, retrospective, non-interventional, medical chart review (MCR), descriptive study collected patient-level data in regions outside the US.
- Trial name
- NCT06178393
- Where
- United States
- Volunteers
- 162 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis (ALS)
Placebo Controlled Study of ONO2506PO in the Presence of Riluzole in Patients With Amyotrophic Lateral Sclerosis (ALS)
The purpose of this study is to determine if oral treatment with ONO-2506PO in patients diagnosed with ALS, who have had onset of muscle weakness within 14 months of randomization, could lead to the slowing of decline in…
- Trial name
- NCT00403104
- Testing
- ONO-2506PO
- Where
- 26 sites in Austria, Belgium and 6 more
- Volunteers
- 420 people
- Sponsor
- Ono Pharmaceutical Co., Ltd.
Spasticity
Long-term Surveillance of the MedStream Programmable Infusion System
This post-approval study will primarily evaluate the long-term safety of the MedStream Programmable Infusion System when used in combination with Baclofen for the treatment of severe spasticity.
- Trial name
- NCT01712087
- Testing
- MedStream Programmable Infusion System
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Codman & Shurtleff
Amyotrophic Lateral Sclerosis (ALS)
Safety Extension Study of Oral Edaravone Administered in Subjects With Amyotrophic Lateral Sclerosis (ALS)
This is a Phase 3, international, multicenter, open-label, long-term extension study. The primary objective of this study is to evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral…
- Trial name
- NCT04577404
- Testing
- MT-1186
- Where
- 44 sites in United States, Canada and 4 more
- Volunteers
- 124 people
- Sponsor
- Shionogi
Amyotrophic Lateral Sclerosis, Motor Neuron Disease
Study of Electrical Impedance Myography (EIM) in ALS
This trial is studying Electrical Impedance Myography (EIM) for measuring muscle health. The trial is studying people with Amyotrophic Lateral Sclerosis (ALS), other neuromuscular diseases, and healthy volunteers to see if the…
- Conditions
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Charcot-Marie-Tooth Disease, Multiple Sclerosis
- Trial name
- NCT02011204
- Testing
- Electrical impedance myography (EIM)
- Where
- 6 sites in United States
- Volunteers
- 106 people, healthy volunteers welcome
- Sponsor
- Skulpt, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- ONYX (NCT05626855)
- Testing
- Apitegromab
- Where
- 49 sites in United States, Belgium and 7 more
- Volunteers
- 238 people
- Sponsor
- Scholar Rock, Inc.
Amyotrophic Lateral Sclerosis
A Study Evaluating the Safety and Tolerability of QRL-201 in ALS
The primary objective of this study is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS
- Trial name
- NCT05633459
- Testing
- Multiple ascending doses of QRL-201, QRL-201
- Where
- 15 sites in Belgium, Canada and 4 more
- Volunteers
- 69 people
- Sponsor
- QurAlis Corporation
Amyotrophic Lateral Sclerosis
Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis
The purpose of this study is to determine whether nebulized RNS60 is effective in the treatment of amyotrophic lateral sclerosis (ALS).
- Trial name
- NCT02988297
- Testing
- RNS60
- Sponsor
- Revalesio Corporation
Neurologic Disorder, Neurologic Diseases
Functional Outcomes and Control Using Synchron BCI - Australia
Functional Outcomes and Control Using Synchron BCI - Australia (FOCUS-AUS)
- Conditions
- Neurologic Disorder, Neurologic Diseases, MND (Motor Neurone DIsease), Motor Neuron Disease
- Trial name
- FOCUS-AUS (NCT07533903)
- Testing
- Stentrode
- Where
- Australia
- Volunteers
- 10 people
- Sponsor
- Synchron, Inc.
Amyotrophic Lateral Sclerosis
RAPA-501 Therapy for ALS
RAPA-501-ALS is a phase 2/3 expansion cohort study of RAPA-501 autologous hybrid TREG/Th2 cells in patients living with amyotrophic lateral sclerosis (pwALS).
- Trial name
- NCT04220190
- Testing
- RAPA-501 Autologous T stem cells
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Rapa Therapeutics LLC