Clinical trials: Neurodegenerative Disease, Hereditary
Spinocerebellar Ataxia Type 3
Optimizing Parameters of Transcranial Temporal Interference Stimulation for Spinocerebellar Ataxia Type 3
The purpose of this research study is to identify the optimal biological dose for tTIS that targets DN in SCA3 while achieving the best possible balance between therapeutic efficacy and safety.
- Trial name
- NCT07743866
- Testing
- Transcranial Temporal Interference Stimulation (tTIS)
- Where
- China
- Volunteers
- 24 people
- Sponsor
- First Affiliated Hospital of Fujian Medical University
Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A
Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
P2-SAF-301 is an open-label interventional study without administration of investigational product, evaluating the long-term safety and tolerability of intracerebral SAF-301 previously administered to 4 patients with Sanfilippo…
- Trial name
- NCT02053064
- Testing
- SAF-301
- Where
- France
- Volunteers
- 4 people
- Sponsor
- LYSOGENE
Tourette Syndrome, Tic Disorders
Study of Tics in Patients With Tourette's Syndrome and Chronic Motor Tic Disorder
This study will investigate which areas of the brain are primarily involved in and responsible for tics in patients with Tourette's syndrome and chronic motor disorder.
- Trial name
- NCT00033995
- Where
- United States
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Huntington Disease
Move to Improve: Telehealth Exercise to Music for HD
The purpose of this study is to determine if a movement to music exercise program delivered via telehealth is feasible and safe for individuals with neurodegenerative disease and their caregivers (Aim 1).
- Trial name
- MtI (NCT05270681)
- Testing
- Movement to Music
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Ohio State University
Metastatic Cancer
A Study of Reduced-dose Radiation in People With Metastatic Tumors With a Genetic Change
This study will test whether reduced-dose radiotherapy is an effective treatment for metastatic tumors with an ATM mutation. The researchers want to find the lowest dose of radiation that would still be effective to treat these…
- Trial name
- NCT05010031
- Testing
- Palliative radiotherapy
- Where
- 7 sites in United States
- Volunteers
- 45 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Infantile Spinal Muscular Atrophy
Test of Aquatic Mobility of SMA Infants
Spinal muscular atrophy (SMA) is a genetic neurodegenerative disease impacting spinal cord motor neurons, leading to motor and respiratory issues and, ultimately, death.
- Trial name
- BAINSMA (NCT06152302)
- Testing
- Mobility study in dry conditions, Mobility study in water conditions
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Alzheimers Disease, Dementia
Dominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia. A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset Alzheimer's Disease Caused by a Genetic Mutation.
The purpose of this study is to assess the safety, tolerability, biomarker and cognitive efficacy of investigational products in subjects who are known to have an Alzheimer's disease-causing mutation by determining if treatment…
- Conditions
- Alzheimers Disease, Dementia, Alzheimers Disease, Familial
- Trial name
- DIAN-TU (NCT04623242)
- Testing
- Gantenerumab, Solanezumab
- Where
- 25 sites in United States, Australia and 6 more
- Volunteers
- 194 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Machado-Joseph Disease / Spinocerebellar Ataxia 3
Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease
This is an exploratory, randomized, parallel-group, dose escalation and dose-controlled study without a placebo arm. Eligible patients will be randomized in a 1:1 ratio (double-blind) to receive Cabaletta in 2 doses, once weekly…
- Trial name
- NCT02147886
- Testing
- Cabaletta for IV infusion once weekly during 24 weeks
- Where
- Israel
- Volunteers
- 15 people
- Sponsor
- Bioblast Pharma Ltd.
Neurofibromatosis Type 1
Molecular Aspects of Preimplantation Genetic Diagnosis for NF1
Neurofibromatosis 1 (NF1) is a multisystem disorders characterized by skin abnormalities, such as café-au-lait spots and neurofibromas, learning disabilities, skeletal anomalies and vascular complications.
- Trial name
- NF1 (NCT05149469)
- Where
- France
- Volunteers
- 60 people
- Sponsor
- University Hospital, Strasbourg, France
Huntington's Disease
A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease
This is a multicenter, multinational, randomized, parallel-group, double-blind, placebo-controlled, dose range finding study to compare the efficacy and safety of different doses of pridopidine versus placebo in the treatment of…
- Trial name
- NCT02006472
- Testing
- Pridopidine
- Where
- 58 sites in United States, Australia and 10 more
- Volunteers
- 408 people
- Sponsor
- Prilenia
Microsatellite Stable (MSS) Colon Cancer, Locally Advanced Colon Cancer
Node-Sparing Short-Course Radiation With CAPOX and Sintilimab for MSS Locally Advanced Colon Cancer: A Randomized, Prospective, Multicenter Study
This study aims to evaluate the use of node-sparing short-course radiotherapy combined with chemotherapy and Sintilimab, or chemotherapy alone, as neoadjuvant therapy for MSS-type locally advanced colon cancer.
- Trial name
- mRCAT-C (NCT06594692)
- Testing
- Node-Sparing Short-Course Radiotherapy Combined with CAPOX and Sintilimab, CAPOX Chemotherapy
- Where
- 2 sites in China
- Volunteers
- 140 people
- Sponsor
- Sir Run Run Shaw Hospital
Spinocerebellar Ataxia Type 2
Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2
Adult participants with spinocerebellar ataxia type 2 (SCA2) who carry ≥33 cytosine, adenine, guanine (CAG) repeats in the ATXN2 gene, and who have met all protocol eligibility criteria will be randomized to receive a single dose…
- Trial name
- NCT06672445
- Testing
- ARO-ATXN2 Injection
- Where
- 16 sites in Australia, Canada and 6 more
- Volunteers
- 39 people
- Sponsor
- Arrowhead Pharmaceuticals
Amyloidosis Cardiac
Frontline Lenalidomide for AL Amyloidosis Involving Myocardium
This phase II clinical trial aimed at influencing the improvement of major organ functions, especially the objective response rate, in Amyloid light-chain amyloidosis involving myocardium.
- Trial name
- NCT04298372
- Testing
- Lenalidomide 25mg
- Where
- South Korea
- Volunteers
- 2 people
- Sponsor
- Seoul National University Hospital
Cardiomyopathy
Observational Epidemiological Study of Patients Suffering From Systemic Amyloidosis
observational epidemiological study of patients affected by systemic amyloidosis
- Trial name
- AMI-ER (NCT06866912)
- Where
- Italy
- Volunteers
- 750 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Friedreich Ataxia
Measurement of Frataxin mRNA in Biofluids
The purpose of this research study is to determine a way to measure frataxin messenger RNA (mRNA) in fluids such as blood and cerebrospinal fluid (CSF) from patients with Friedreich's ataxia (FRDA).
- Trial name
- NCT06496451
- Testing
- Spinal tap for CSF collection, Venipuncture for blood sample
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of Florida
Inclusion Body Myositis, Myotonic Dystrophy
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Currently, there are no standardised fall risk scores or guidelines on when to use appropriate assistive gait devices (AGDs) for people with neuromuscular disorders (NMDs).
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy, Limb-girdle and Facioscapulohumeral Muscular Dystrophies, Pompe Disease
- Trial name
- FBIndex (NCT06605612)
- Testing
- FBIndex
- Where
- Germany
- Volunteers
- 108 people
- Sponsor
- LMU Klinikum
Schwannomatosis, Pain
Phase 2 Study of Tanezumab in Subjects With Moderate to Severe Pain Due to Schwannomatosis
The primary objective of this study is to determine whether the administration of tanezumab, an anti-nerve growth factor (NGF) antibody, improves pain relief in schwannomatosis patients receiving background non-NSAID therapy.
- Trial name
- NCT04163419
- Testing
- Tanezumab
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Massachusetts General Hospital
Friedreich's Ataxia
A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World
In this study, researchers will learn more about a disease called Friedrich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their…
- Trial name
- NCT07887828
- Volunteers
- 40 people
- Sponsor
- Biogen
Huntington Disease
Clinical Extension Study for Safety and Efficacy Evaluation of Cellavita-HD Administration in Huntington's Patients.
Cellavita-HD is a stem-cell therapy for Huntington's Disease. Open label, single treatment, extension study for long-term safety and efficacy evaluation of Cellavita-HD intravenous administration in Huntington's disease patients…
- Trial name
- ADORE-EXT (NCT04219241)
- Testing
- Cellavita-HD
- Where
- Brazil
- Volunteers
- 35 people
- Sponsor
- Azidus Brasil
Myotonic Dystrophy
DM1 Heart Registry - DM1 Respiratory Registry
Myotonic dystrophy type 1 (DM1) is the most frequent neuromuscular disease in adults. DM1 patients have an impaired prognosis (mean age of death <60 years) due to cardiac and respiratory complications.
- Trial name
- DM1-Heart-R (NCT01136330)
- Where
- France
- Volunteers
- 914 people
- Sponsor
- Institut de Myologie, France
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