Clinical trials: Motor Neuron Disorder
ALS, Motor Neuron Disease
Deep Phenotyping in Patients With ALS
This study aims to establish a biorepository and phenotyping database to investigate longitudinal changes in ALS subjects. Blood, including DNA and RNA, cerebrospinal fluid (CSF) and electrophysiologic measures will be collected…
- Trial name
- NCT02819765
- Where
- 7 sites in United States
- Volunteers
- 32 people
- Sponsor
- Barrow Neurological Institute
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
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult participants with ALS, FTD, or mixed ALS/FTD phenotype with a documented…
- Trial name
- NCT05683860
- Testing
- WVE-004 10 mg Q12W
- Where
- 3 sites in Netherlands, United Kingdom
- Volunteers
- 8 people
- Sponsor
- Wave Life Sciences USA, Inc.
Spinal Muscular Atrophy
A Registry of Cases of Spinal Muscular Atrophy
Because no long-term record of prenatal Spinal Muscular Atrophy (SMA) treatment exists, this study will address an important gap in the understanding of the safety and benefit of treatment while in the womb.
- Trial name
- NCT07805278
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- St. Jude Children's Research Hospital
Amyotrophic Lateral Sclerosis
ALS Phase II Study of NX210c
This study will investigate the efficacy, safety, tolerability and pharmacokinetics (PK) of multiple intravenous infusions of NX210c, at two dose levels, in patients with Amyotrophic lateral sclerosis (ALS).
- Trial name
- SEALS (NCT06365216)
- Testing
- NX210c
- Where
- 16 sites in France
- Volunteers
- 80 people
- Sponsor
- Axoltis Pharma
Circulating Tumor Cell, Metastatic Prostate Cancer
Cabazitaxel in mCRPC Patients With AR-V7 Positive Circulating Tumor Cells (CTCs)
After failure on docetaxel, which has been the standard first line therapy for patients with metastatic castration-resistant prostate cancer (mCRPC), several treatment options are currently available.
- Trial name
- CABA-V7 (NCT03050866)
- Testing
- Cabazitaxel, Antihistamine, Corticosteroid
- Where
- 9 sites in Netherlands
- Volunteers
- 140 people
- Sponsor
- Erasmus Medical Center
Amyotrophic Lateral Sclerosis, Motor Neuron Disease
Safety of Intrathecal Riluzole in Patients With Amyotrophic Lateral Sclerosis
The purpose of this study is to investigate the safety and tolerability of intrathecal riluzole in adults with amyotrophic lateral sclerosis.
- Trial name
- NCT07093268
- Testing
- Intrathecal Riluzole
- Where
- 2 sites in Australia
- Volunteers
- 10 people
- Sponsor
- Brain Trust Bio
Motor Neuron Diseases, Spinocerebellar Ataxias
Rare Disease Registry Database for Movement Disorders in Southern Anhui Province
According to the World Health Organization (WHO) definition, a rare disease is a disease affecting fewer than 6.5 people per 10,000; in China, it generally refers to diseases with a prevalence of less than 1/10,000 (i.e., with a…
- Conditions
- Motor Neuron Diseases, Spinocerebellar Ataxias, Hereditary Muscular Dystrophy, Hereditary Spastic Paraplegia
- Trial name
- W-MoSCA (NCT07838883)
- Where
- China
- Volunteers
- 180 people
- Sponsor
- First Affiliated Hospital of Wannan Medical College
Spinal Muscular Atrophy
Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms)
Spinal Muscular Atrophy (SMA) is an autosomal recessive disease caused by a mutation of exon 7, in 95% of cases, encoding the gene for the motor neuron survival protein called SMN1 (Survival Motor Neuron) located on chromosome…
- Trial name
- NUSI-AD-5qSM (NCT04576494)
- Testing
- Monthly assessments of functional motor abilities by a trained therapist, Nusinersen
- Where
- France
- Volunteers
- 4 people
- Sponsor
- CHU de Reims
Amyotrophic Lateral Sclerosis
Nightime NIV Initiation in Amyotrophic Lateral Sclerosis in an Outpatient Setting
In patients with amyotrophic lateral sclerosis (ALS), non-invasive mechanical ventilation (NIV) is usually initiated in an in-hospital regime.
- Trial name
- NCT02759003
- Testing
- Nightime NIV initiation
- Volunteers
- 50 people
- Sponsor
- Fondazione Salvatore Maugeri
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
Spinal Muscular Atrophy (SMA), Cerebral Palsy (CP)
Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders
Both GIP and GLP-2 reduce bone resorption (measured as CTX) in healthy adult individuals. In this study, we will investigate whether GIP and GLP-2 reduce CTX in children with spinal muscular atrophy, duchenne muscular dystrophy…
- Conditions
- Spinal Muscular Atrophy (SMA), Cerebral Palsy (CP), Duchenne Muscular Dystrophy (DMD)
- Trial name
- NCT07254988
- Testing
- Meal test, GIP + GLP-2
- Where
- Denmark
- Volunteers
- 3 people
- Sponsor
- University of Copenhagen
Amyotrophic Lateral Sclerosis
Repeated Mesenchymal Stem Cell Injections in ALS
An open-label, single-center clinical trial to evaluate the safety and efficacy of repeated intrathecal administrations of autologous bone marrow derived mesenchyme stem cells in ALS patients.
- Trial name
- NCT04821479
- Testing
- Mesenchymal stem cells (MSC)
- Volunteers
- 20 people
- Sponsor
- Hadassah Medical Organization
ALS (Amyotrophic Lateral Sclerosis)
Extended Study of RAG-17 in the Treatment of Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation
This study primarily evaluates the safety, tolerability, and efficacy of RAG - 17 in adult ALS patients with SOD1 - mutated genes in the real - world setting.
- Trial name
- NCT07077668
- Testing
- A sterile aqueous solution of RAG - 17 preparation (sodium salt)
- Volunteers
- 10 people
- Sponsor
- Beijing Tiantan Hospital
Amyotrophic Lateral Sclerosis
Safety/Efficacy Study of 2nd Cycle Treatment After 6 Months of 1st Cycle HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell Treatment in ALS
The purpose of this study is to evaluate the safety and efficacy of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNRCS-Allo-ALS-02 inj"), through intrathecal delivery for the repeated treatment after 6 months of…
- Trial name
- NCT03214146
- Testing
- HYNRCS-Allo inj
- Where
- South Korea
- Volunteers
- 6 people
- Sponsor
- Hanyang University Seoul Hospital
Amyotrophic Lateral Sclerosis (ALS)
Pilot-Study of Thalidomide in Amyotrophic Lateral Sclerosis (ALS)
Neuroinflammation has recently emerged as a significant contributor to motor neuron damage. ALS tissue is characterized by inflammatory changes that are observed in both sporadic and familial ALS and in the ALS superoxide…
- Trial name
- NCT00231140
- Testing
- Thalidomide (drug)
- Where
- Germany
- Volunteers
- 40 people
- Sponsor
- Charite University, Berlin, Germany
Muscular Atrophy, Spinal
Progressive Strength Training in Spinal Muscular Atrophy
The proposed feasibility study is necessary to test if children and young adults will participate in and adhere to a 12-week, home-based, supervised progressive strength training exercise program and to obtain preliminary data…
- Trial name
- NCT01233817
- Testing
- Progressive strength training
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- University of Utah
Spinal Muscular Atrophy
Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)
The aim of this project is to establish a network of clinical teams including the major neuromuscular centers in Europe. We plan to work together to find the best common outcome measures for the following multicenter therapeutic…
- Trial name
- NCT01611610
- Testing
- Functional tests
- Volunteers
- 4 people
- Sponsor
- Institut de Myologie, France
Metastatic Castration Resistant Prostate Cancer
A Study of AC176 for the Treatment of Metastatic Castration Resistant Prostate Cancer
This clinical trial is evaluating a drug called AC176 in participants with metastatic castration resistant prostate cancer (mCRPC) who have progressed on at least two prior systemic therapies.
- Trial name
- NCT05241613
- Testing
- AC176
- Where
- 5 sites in United States
- Volunteers
- 28 people
- Sponsor
- Accutar Biotechnology Inc
Efficacy and Safety
Efficacy and Safety of Deep Cervical Lymph Node-vein Bypass Surgery in ALS Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease, affects motor neurons, causing progressive muscle atrophy and weakness.
- Trial name
- NCT06351735
- Testing
- Deep cervical lymph node-vein bypass surgery
- Volunteers
- 3 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine