Clinical trials: Muscular Atrophy, Spinal, open to volunteers
Spinal Muscular Atrophy (SMA)
Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy
There is a general physiological rule that any organ or system needs some minimal amount of activity to prevent its atrophy or degeneration.
- Trial name
- STIM-SMA (NCT06977269)
- Testing
- High-frequency repetitive transcranial magnetic stimulation, lumbar puncture
- Where
- Ukraine
- Volunteers
- 20 people
- Sponsor
- Charitable Foundation Children with Spinal Muscular Atrophy
Metastatic Hormone-Sensitive Prostate Cancer (mHSPC)
TulmiSTAR-02: A Phase I/II Open-label Study of Tulmimetostat in Combination With Darolutamide vs. Darolutamide, and Tulmimetostat With Abiraterone in Patients With Metastatic Hormone-sensitive Prostate Cancer (mHSPC)
The purpose of this study is to evaluate the safety, tolerability, and efficacy of two different treatment combinations of tulmimetostat in participants with de novo or recurrent metastatic hormone-sensitive prostate cancer…
- Trial name
- TulmiSTAR-02 (NCT07190300)
- Testing
- Tulmimetostat, Darolutamide, Abiraterone
- Where
- 31 sites in United States, Australia and 12 more
- Volunteers
- 181 people
- Sponsor
- Novartis Pharmaceuticals
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Spinal and Bulbar Muscular Atrophy, Kennedys Disease
Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)
Background: SBMA is an inherited chronic disease. It affects males in mid to late adulthood. It causes slowly progressive weakness of muscles and hand tremors. Researchers want to learn more about the effects of SBMA.
- Conditions
- Spinal and Bulbar Muscular Atrophy, Kennedys Disease, Motor Neuron Disease
- Trial name
- NCT04944940
- Where
- United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Spinal Muscular Atrophy (SMA)
Advanced Multi-omics and -System Approaches to Identify Novel Biomarkers for SMA
Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder caused by mutations in the SMN1 gene, leading to motor neuron degeneration and progressive muscle atrophy.
- Trial name
- MOSAiC-SMA (NCT07883655)
- Where
- 3 sites in Italy
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Istituto Giannina Gaslini
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
Spinal Muscular Atrophy
A Registered Cohort Study on SMA
Spinal muscular atrophy (SMA) is an autosomal recessive disease that causes progressive muscle wasting and weakness due to loss of motor neurons in the spinal cord.
- Trial name
- NCT04010604
- Where
- China
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Wan-Jin Chen
Spinal Muscular Atrophy (SMA)
Service Evaluation for Newborn Screening for Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA) is a disease that causes progressive muscle weakness. There are many types- the most severe and most common, type 1, affects very young babies, and is life-threatening if untreated.
- Trial name
- SENS (NCT07888868)
- Where
- 3 sites in United Kingdom
- Volunteers
- 750,000 people
- Sponsor
- University of Oxford
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
Psoriatic Arthritis, Osteoarthitis
CARTIZ Registry: Cartilage, Arthropathy and Imaging Under Tirzepatide in Zone-stratified Cohorts - A Four-Institute Mexican Observational Registry
CARTIZ is a prospective observational clinical registry of adults in Mexico receiving tirzepatide (a dual GLP-1/GIP receptor agonist) under an independent clinical indication - typically type 2 diabetes, insulin resistance…
- Conditions
- Psoriatic Arthritis, Osteoarthitis, Knee, Diabetes (DM)
- Trial name
- CARTIZ (NCT07567378)
- Testing
- Tirzepatide
- Where
- Mexico
- Volunteers
- 30 people
- Sponsor
- JULIO GRANADOS MONTIEL
Spinal Muscular Atrophy 1
Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Regarding Feeding and Communication
Spinal Muscular Atrophy Type 1 (SMA )Type 1 is a severe, early-onset neuromuscular condition that typically leads to profound weakness and impaired bulbar function-affecting swallowing, feeding, speech, and airway protection.
- Trial name
- NCT07596277
- Where
- United Kingdom
- Volunteers
- 15 people
- Sponsor
- Guy's and St Thomas' NHS Foundation Trust
Tetraplegia, Spinal Cord Injuries
BrainGate2: Feasibility Study of an Intracortical Neural Interface System for Persons With Tetraplegia
The purpose of this study is to obtain preliminary device safety information and demonstrate proof of principle (feasibility) of the ability of people with tetraplegia to control a computer cursor and other assistive devices with…
- Conditions
- Tetraplegia, Spinal Cord Injuries, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions
- Trial name
- BrainGate2 (NCT00912041)
- Testing
- Placement of the BrainGate2 sensor(s) into the motor-related cortex
- Where
- 6 sites in United States
- Volunteers
- 27 people
- Sponsor
- Leigh R. Hochberg, MD, PhD.
Muscular Atrophy, Spinal
Natural History of SMA
This is an investigator initiated observational study with the aim to record several aspects of function, care and adverse events in a large cohort of SMA patients followed longitudinally by using a structured academic disease…
- Trial name
- iSMAR (NCT05755451)
- Where
- 5 sites in Italy
- Volunteers
- 1,200 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth
Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers
In recent years, knowledge of neuromuscular diseases has advanced considerably, and new therapeutic avenues are beginning to emerge.
- Conditions
- Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth, Muscular Dystrophy, Myotonic Dystrophy
- Trial name
- Myokinesis (NCT07321977)
- Testing
- Gait analysis via video capture
- Where
- France
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS)
Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)
The goal of this clinical trial is to evaluate the safety and preliminary efficacy of an implantable device that records and stimulates different areas of the brain to allow adults affected by disabling paralysis (see Eligibility…
- Conditions
- Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS), Muscular Disorders, Atrophic, Brain Stem Stroke
- Trial name
- INTENT (NCT07521930)
- Testing
- INTENT Neural Interface System
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Johns Hopkins University
SMA - Spinal Muscular Atrophy
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously received nusinersen.
- Trial name
- NCT06971094
- Testing
- GC101 adeno-associated virus injection
- Where
- 7 sites in China
- Volunteers
- 50 people
- Sponsor
- GeneCradle Inc
Spinal Muscular Atrophy
CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in Spain
The goal of this observational study is to set up a longitudinal data collection study to perform a long-term follow-up of SMA patients in a real-world setting, and to standardise and homogenize the data collection process for…
- Trial name
- CUIDAME (NCT07231549)
- Where
- Spain
- Volunteers
- 1,000 people
- Sponsor
- Fundació Sant Joan de Déu
Spinal Muscular Atrophy (SMA)
HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness
This single-arm pilot study will assess the preliminary effectiveness of an intensive motor skill intervention (HABIT-ILE) combined with functional strength training (FST) in children with SMA who are receiving disease-modifying…
- Trial name
- NCT07488338
- Testing
- Combined Hand-Arm Bimanual Intensive Therapy Including Lower Extremities (HABIT-ILE) + functional strength training (FST)
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Teachers College, Columbia University
Spinal Stenosis Lumbar, Sarcopenia
Sarcopenia and Interlaminar Epidural Steroid Injection Outcomes in Lumbar Spinal Stenosis
This prospective observational study aims to investigate the association between sarcopenia and clinical outcomes following interlaminar epidural steroid injection in patients with lumbar spinal stenosis.
- Trial name
- NCT07855419
- Where
- Turkey (Türkiye)
- Volunteers
- 70 people
- Sponsor
- Marmara University
Cerebrovascular Accident, Post-polio Syndrome
Nomad P-KAFO Study
The goal of this clinical trial is to evaluate the impact of using the Nomad powered KAFO in people who have had a musculoskeletal or neurological injury that has affected their ability to walk.
- Conditions
- Cerebrovascular Accident, Post-polio Syndrome, Spinal Cord Injuries, Multiple Sclerosis
- Trial name
- NCT05644522
- Testing
- Indego Nomad® Powered Knee-Ankle-Foot Orthosis (P-KAFO) (Parker Hannifin Corp., Cleveland, OH)
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Shirley Ryan AbilityLab