Clinical trials: Muscle Loss
Spinal Muscular Atrophy
SMN Circular RNAs as Potential New Targets and Biomarkers for SMA
Spinal Muscular Atrophy (SMA) is a life-threatening disease in infancy that is caused by inactivating mutations in the Survival Motor Neuron 1 (SMN1) gene1,2.
- Trial name
- CircSMA (NCT05760209)
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Spinal Muscular Atrophy
SMN Circular RNAs as Potential Biomarkers for the Therapeutic Response to Nusinersen in Spinal Muscular Atrophy Patients
The first cure for Spinal Muscular Atrophy (SMA; Nusinersen) has been approved by FDA in 2017. Although it improves the clinical picture of most SMA patients, not all exhibit the same response to treatment.
- Trial name
- NCT05761262
- Where
- Italy
- Volunteers
- 91 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Epilepsy; Seizure, Neuromuscular Diseases
North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2
The "North Carolina Clinical Genomic Evaluation by Next-gen Exome Sequencing, 2 (NCGENES 2)" study is part of a larger consortium project investigating the clinical utility, or net benefit of an intervention on patient and family…
- Conditions
- Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability
- Trial name
- NCGENES2 (NCT03548779)
- Testing
- Pre-visit prep, usual care + exome seq
- Where
- 3 sites in United States
- Volunteers
- 548 people
- Sponsor
- University of North Carolina, Chapel Hill
Spinal Muscular Atrophy (SMA)
An International Federated Model for Wearable-derived Remote Longitudinal Motor Monitoring in Young Children With Spinal Muscular Atrophy Compared With Healthy Controls: Active-NBS Study (UK)
Active-NBS is a study to evaluate the muscle development of patients with spinal muscular atrophy (SMA) who are diagnosed at birth. Medicines have become available in the last decade, and many patients are treated very early.
- Trial name
- Active-NBS UK (NCT07543003)
- Where
- United Kingdom
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- University of Oxford
Sarcopenia, Child Development
Determination of Reference Values for Handgrip Strength and Bioimpedance Values in Healthy Turkish Children
In this study, our aim is to determine the reference values for the handgrip force and bioimpedance values in healthy Turkish children.
- Trial name
- NCT06001931
- Testing
- handgrip force and bioimpedance values in healthy Turkish children.
- Where
- Turkey (Türkiye)
- Volunteers
- 800 people, healthy volunteers welcome
- Sponsor
- Istanbul Physical Medicine Rehabilitation Training and Research Hospital
Spinal Muscular Atrophy Type 3, Mitochondrial Myopathy
Oxidative Capacity and Exercise Tolerance in Ambulatory SMA
This proposal will focus on (1) estimating oxidative capacity of specific muscle groups during exercise using near infrared spectroscopy and (2) describing body composition to better understand exercise capacity and mitochondrial…
- Trial name
- NCT02895789
- Where
- United States
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Columbia University
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
- 29 sites in United States, Australia and 10 more
- Volunteers
- 97 people
- Sponsor
- Hoffmann-La Roche
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 Amyotrophy, Infantile Spinal Muscular Atrophy
Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused by the degeneration of motor neurons in the anterior horn of the spinal cord, due to the absence of the SMN1 gene and the resulting lack of SMN…
- Conditions
- Spinal Amyotrophy, Infantile Spinal Muscular Atrophy, Juvenile Spinal Muscular Atrophy
- Trial name
- FANTASI-SMART (NCT06562283)
- Testing
- Thumb test, Grip test, Quadriceps Intermittent Fatigue test (QIF test))
- Where
- 4 sites in France
- Volunteers
- 80 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Spinal Muscular Atrophy (SMA)
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
The goal of this clinical trial is to learn about the safety, and tolerability of giving an experimental gene therapy called SPX-01 to an infant with spinal muscular atrophy (SMA) type 1.
- Trial name
- NCT07903376
- Testing
- SPX-01
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Exir Gene Pharmed
Mechanical Ventilation
Study of Muscle Effects of BYM338 in Mechanically Ventilated Patients
The purpose of this study is to evaluate the effect of BYM338 on muscle mass and function in Group III failure to wean patients, as compared to placebo controls.
- Trial name
- NCT01868685
- Testing
- BYM338
- Sponsor
- Novartis Pharmaceuticals
End-stage Kidney Disease, End-stage Renal Disease
The Prevalence of Sarcopenia in a Cohort of Haemodialysis Patients and Its Correlation With Mortality
The study aims to explore the correlation between the prevalence of sarcopenia and one year mortality amongst patients on maintenance hemodialysis
- Conditions
- End-stage Kidney Disease, End-stage Renal Disease, Dialysis Dependent Chronic Kidney Disease, Sarcopenia
- Trial name
- NCT06477978
- Testing
- Hand grip function testing
- Where
- Egypt
- Volunteers
- 274 people
- Sponsor
- Kasr El Aini Hospital
Sarcopenia, Osteoporosis
Efficacy of Structured Exercise-Nutritional Intervention on Sarcopenia in Patients With Osteoporosis
This is a prospective parallel group, double-blinded randomized controlled trial. Subjects are randomized into two groups - the intervention group and waitlist control group.
- Trial name
- NCT06287502
- Testing
- β-hydroxy β-methylbutyrate (HMB), Resistance, aerobic, flexibility and balance exercise training
- Where
- Hong Kong
- Volunteers
- 13 people
- Sponsor
- Kowloon Hospital, Hong Kong
Spinal Muscular Atrophy
Valproate and Levocarnitine in Children With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA), an autosomal recessive disorder, is characterized by muscle weakness due to degeneration of anterior horn cells in the spinal cord and brain stem nuclei.
- Trial name
- NCT01671384
- Testing
- Valproate, Levocarnitine
- Where
- India
- Volunteers
- 60 people
- Sponsor
- All India Institute of Medical Sciences
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi.
- Conditions
- Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
- Trial name
- NCT04042025
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 31 sites in United States, Australia and 7 more
- Volunteers
- 85 people
- Sponsor
- Novartis Gene Therapies
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
SMA
A Head-to-head Study Comparing the Functional Value of Two Models of Robotically Assisted Rehabilitation in SMA (Spinal Muscular Atrophy) Patients
A head-to-head study comparing the functional value of two models of robotically assisted rehabilitation in patients with SMA. A single-center, randomized, single-blinded, comparative study of Robotically Assisted Verticalization…
- Trial name
- SMArt (NCT06322654)
- Testing
- Robotically Assisted Locomotion, Robotically Assisted Verticalization
- Where
- Poland
- Volunteers
- 200 people
- Sponsor
- Wiktor Dega University Orthopedic and Rehabilitation Hospital
Spinal Muscular Atrophy, Developmental Delay
Effects of Developmental Support for Mothers of Infants With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder causing progressive muscle weakness and atrophy.
- Trial name
- NCT07547189
- Testing
- Online Early Intervention Program for Infants with SMA Type 1
- Where
- Turkey (Türkiye)
- Volunteers
- 8 people
- Sponsor
- Medipol University
Spinal and Bulbar Muscular Atrophy
Specified Drug-Use Survey of Leuprorelin Acetate Injection Kit 11.25 mg "All-Case Investigation: Spinal and Bulbar Muscular Atrophy (SBMA)"
The purpose of this survey is to evaluate the long-term safety and efficacy of leuprorelin acetate injection kit 11.25 mg in patients with spinal and bulbar muscular atrophy (SBMA) in the routine clinical setting.
- Trial name
- NCT03555578
- Testing
- Leuprorelin Acetate
- Where
- Japan
- Volunteers
- 1,905 people
- Sponsor
- Takeda
ACL Reconstruction
Protein Supplementation After ACL Surgery
The purpose of the study is to assess the longitudinal post-operative care effects of protein supplementation on muscle atrophy in the post-operative period following ACL reconstruction.
- Trial name
- NCT06311058
- Testing
- Whey protein isolate, Protein + BAA
- Where
- United States
- Volunteers
- 102 people
- Sponsor
- The Methodist Hospital Research Institute