Clinical trials: Muscular Atrophy, Spinal
Inclusion Body Myositis (IBM), Motor Neuron Disease, Amyotrophic Lateral Sclerosis
The Aim of the Study is to Characterize Patients Exhibiting Muscular Atrophy and Weakness Who Require a Muscle Biopsy for Diagnosis (IBM vs Atypical MND), Through an Innovative Technique Which Enables the Evaluation of the Neuromuscular Junction Involvement in Each Condition
The aim of the study is to characterize 30 patients exhibiting muscular atrophy and weakness who require a muscle biopsy for diagnosis (suspected inclusion body myopathy vs atypical motor neuron disease), through an innovative…
- Trial name
- TDP-signatures (NCT07796776)
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS San Raffaele
Spinal Muscular Atrophy
A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients
This study aims to assess the safety and effectiveness of onasemnogene abeparvovec intrathecal injection in spinal muscular atrophy (SMA) patients in clinical practice in Japan.
- Trial name
- NCT07767890
- Where
- 4 sites in Japan
- Volunteers
- 80 people
- Sponsor
- Novartis Pharmaceuticals
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
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
The purpose of this project is to investigate the utility of the SMA EFFORT, an SMA-specific patient-reported outcome measure, to assess perceived physical fatigability that is anchored to intensity and duration of activities.
- Trial name
- NCT06955897
- Testing
- Observational
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Columbia University
Spasticity Secondary to Either a Disorder or Trauma, Spinal Cord Injury (SCI)
Myobloc Atrophy Study
The present pilot study is designed to assess the extent to which BOTOX and MYOBLOC cause muscle atrophy in spastic patients. The primary objective is to assess whether there is statistically significant difference in muscle…
- Conditions
- Spasticity Secondary to Either a Disorder or Trauma, Spinal Cord Injury (SCI), Brain Injury, Tumor
- Trial name
- MAS (NCT02052024)
- Testing
- Botox, MYOBLOC
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Georgetown University
Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy
Study of ALS Reversals 2: Genetic Analyses
The purpose of this study is to try to understand why reversals of amyotrophic lateral sclerosis (ALS) and primary muscular atrophy (PMA) take place.
- Trial name
- StAR2 (NCT03464903)
- Where
- United States
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Duke University
Muscular Atrophy, Spinal
A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Types II and III Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Types II and III SMA; and…
- Trial name
- NCT00568802
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Stanford University
Spinal Muscular Atrophy
Personalized Medicine for SMA: a Translational Project
Major breakthroughs in the treatment for Spinal muscular atrophy (SMA) have been recently achieved with various therapeutic approaches that increase full-length SMN protein levels.
- Trial name
- NCT05779956
- Where
- 2 sites in Italy
- Volunteers
- 90 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Muscular Atrophy, Spinal, Child, Only
Procedural Sedation for Pediatric Patients With Spinal Muscular Atrophy
Background and Aim: Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by progressive symmetrical weakness and atrophy of proximal muscles causing from degeneration of anterior horn cells of spinal cord.
- Conditions
- Muscular Atrophy, Spinal, Child, Only, Anesthesia
- Trial name
- NCT05291962
- Testing
- Retrospective observational study
- Where
- Turkey (Türkiye)
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Ankara City Hospital Bilkent
Spinal Muscular Atrophy (SMA)
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which…
- Trial name
- MANATEE (NCT05115110)
- Testing
- RO7204239, Risdiplam
- Where
- 30 sites in United States, Australia and 10 more
- Volunteers
- 97 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy, Spinal Muscular Atrophy
Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor
Spinal cord injuries and people with Duchenne Muscular Dystrophy or Infant Spinal Muscular Atrophy (ISA) are prone to pain and pressure sores associated with prolonged sitting.
- Trial name
- Es-Alert (NCT04335942)
- Testing
- Alert "AFNOR 3.6"., Alertes " AFNOR 3.6 " et alertes " Guidelines ".
- Where
- France
- Volunteers
- 36 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Post-Polio Syndrome
Post-Poliosyndrome Treated With Intravenous Immunoglobulin (IvIg)
Inflammatory cytokines in the cerebrospinal fluid may contribute to the new muscle weakness, fatigue and pain experienced by patients with post-polio syndrome. Intravenousimmunoglobulin (IvIg) reduces this inflammation.
- Trial name
- NCT00231439
- Testing
- IvIg
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Haukeland University Hospital
Prostate Cancer
Study to Compare Capsule and Liquid Formulations of Enzalutamide After Single Dose Administration Under Fasting Conditions in Prostate Cancer
Background: Enzalutamide is given to treat metastatic prostate cancer. But it takes 4 pills to deliver the effective dose. This can be difficult for people already taking multiple drugs.
- Trial name
- NCT03478904
- Testing
- Enzalutamide Liquid, Enzalutamide Capsule
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- National Cancer Institute (NCI)
Castration-resistant Prostate Cancer, Metastatic Cancer
pTVG-HP DNA Vaccine With or Without pTVG-AR DNA Vaccine and Pembrolizumab in Patients With Castration-Resistant, Metastatic Prostate Cancer
This trial will evaluate the use of one versus two DNA vaccines, delivered concurrently with PD-1 blockade using pembrolizumab followed by treatment with pembrolizumab alone, and delivered over a prolonged period of time (for a…
- Conditions
- Castration-resistant Prostate Cancer, Metastatic Cancer, Prostate Cancer
- Trial name
- NCT04090528
- Testing
- pTVG-HP, pTVG-AR, Pembrolizumab
- Where
- 2 sites in United States
- Volunteers
- 60 people
- Sponsor
- University of Wisconsin, Madison
Spinal Muscular Atrophy (SMA)
Effects of Different Stiffness Settings During Robotic Isokinetic Training in Children With SMA
This randomized, within-participant, contralateral-limb-controlled clinical trial will compare individually optimized compliant and rigid stiffness settings of a portable isokinetic knee training robot in children with spinal…
- Trial name
- NCT07822711
- Testing
- Portable Isokinetic Knee Training Robot With an Individually Optimized Compliant Setting, Portable Isokinetic Knee Training Robot With a Rigid Setting
- Where
- China
- Volunteers
- 13 people
- Sponsor
- Peking University Third Hospital
Prostate Cancer
An Open Label Prostate Cancer Study in Japanese Patients
The primary aim of study is to gain an initial assessment of safety and tolerability of AZD3514 in Japanese patients together with assessing Pharmacokinetics (PK) and gaining a preliminary assessment of anti-tumour action.
- Trial name
- NCT01351688
- Testing
- AZD3514
- Where
- 2 sites in Japan
- Volunteers
- 13 people
- Sponsor
- AstraZeneca
Spinal Muscular Atrophy
Motor Development and Orthoses in Spinal Muscular Atrophy (SMA)
Spinal Muscular Atrophy (SMA) is neurodegenerative disease of anterior horn cells of spinal cord and represents the second more frequent pathology in childhood.
- Trial name
- NCT00961103
- Where
- Italy
- Volunteers
- 80 people
- Sponsor
- Azienda USL Reggio Emilia - IRCCS
Spinal Muscular Atrophy (SMA)
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor…
- Trial name
- NCT04174157
- Testing
- Prospective observational registry, Zolgensma
- Where
- 99 sites in United States, Greece and 9 more
- Volunteers
- 700 people
- Sponsor
- Novartis Pharmaceuticals
Motor Neuron Disease, Amyotrophic Lateral Sclerosis, Motor Neuron Disease Progressive Spinal Muscle Atrophy
Non-invasive Spinal, Cortical, and Sensorimotor Biomarkers in Motor Neurone Disease
Substantial variability exists in the onset, and rate of degeneration across individuals with Motor Neurone Disease (MND) or Amyotrophic Lateral Sclerosis (ALS).
- Conditions
- Motor Neuron Disease, Amyotrophic Lateral Sclerosis, Motor Neuron Disease Progressive Spinal Muscle Atrophy, Primary Lateral Sclerosis, Multiple Sclerosis
- Trial name
- NCT06320444
- Testing
- 232 Electrode Electrophysiology (EEG-ECG-EMG-EXG)
- Where
- Ireland
- Volunteers
- 240 people, healthy volunteers welcome
- Sponsor
- University of Dublin, Trinity College
Amyotrophic Lateral Sclerosis, Brainstem Stroke
Optimizing BCI-FIT: Brain Computer Interface - Functional Implementation Toolkit
This project adds to non-invasive BCIs for communication for adults with severe speech and physical impairments due to neurodegenerative diseases.
- Conditions
- Amyotrophic Lateral Sclerosis, Brainstem Stroke, Muscular Dystrophies, Parkinson's Disease and Parkinsonism
- Trial name
- BCI-FIT (NCT04468919)
- Testing
- BCI-FIT multi-modal access, BCI-FIT adaptive signal modeling, BCI-FIT active querying
- Where
- United States
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- Oregon Health and Science University