Clinical trials: Muscle Wasting
Spinal Muscular Atrophy
Spinal Muscular Atrophy Neonatal Screening Program
Spinal muscular atrophy (SMA) is a group of disorders caused by the degeneration of the motor neuron cells of the anterior horn of the spinal cord and, in some subtypes, of the bulbar motor neurons.
- Trial name
- NCT06310421
- Testing
- Screening SMA test
- Where
- 2 sites in Italy
- Volunteers
- 11,500 people
- Sponsor
- IRCCS Burlo Garofolo
Sarcopenia
Sarcopenia Physical Activity and Metabolomic
Muscle failure (sarcopenia or dynapenia) is a factor of frailty and therefore, ultimately, of loss of autonomy in the elderly. Currently, no biomarker of muscle failure has a high sensitivity, specificity and positive predictive…
- Trial name
- SPAM (NCT05199207)
- Testing
- Physical activity programm
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Nice
Prader-Willi Syndrome, Sarcopenia
Ultrasound to Assess Sarcopenia in Prader Willi Syndrome
The aim of the study is to examine the clinical applicability of ultrasound as a diagnostic tool for sarcopenia in individuals with PWS by investigating the association between ultrasound-derived measurements, DXA-derived…
- Conditions
- Prader-Willi Syndrome, Sarcopenia, Metabolic Syndrome
- Trial name
- PWS (NCT06448871)
- Testing
- Ultrasound assessment of the right lower extremity muscles, Dual-energy X-ray absorptiometry (DXA) scan
- Where
- Taiwan
- Volunteers
- 49 people
- Sponsor
- Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation
Spinal Muscular Atrophy (SMA)
Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy
The primary objective of the study is to evaluate anxiety level during intrathecal administration (IT) under standard of care (SOC) and virtual reality (VR) conditions using a reliable self-rating scale.
- Trial name
- REALITY (NCT05354414)
- Testing
- Virtual reality, Standard of Care
- Where
- 12 sites in France
- Volunteers
- 63 people
- Sponsor
- Biogen
Non Invasive Ventilation, Spinal Muscular Atrophy
Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIV
SMA (Spinal Muscular Atrophy) is a rare neuromuscular disease characterized by motoneuron damage. Symptoms consist of respiratory involvement with numerous respiratory infections and eventually respiratory failure, for which NIV…
- Conditions
- Non Invasive Ventilation, Spinal Muscular Atrophy, Respiratory Bacterial Infection
- Trial name
- AVNIR (NCT05712330)
- Testing
- non invasive ventilation samples, patient samples, parent questionnaire
- Where
- France
- Volunteers
- 8 people
- Sponsor
- Centre Hospitalier Universitaire, Amiens
Stroke, Hemiplegia Following Ischemic Stroke
Improving STEP in Stroke Patients
Stroke is the leading cause of acquired disability in adults. Stroke causes death in 10% of patients, disability and functional handicap in 60% of cases.
- Conditions
- Stroke, Hemiplegia Following Ischemic Stroke, Disability Physical, Spasticity as Sequela of Stroke
- Trial name
- I-STEP (NCT07403149)
- Testing
- REMARCHE® shoes
- Volunteers
- 58 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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 Muscular Atrophy
Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
In this observational study, researchers are looking at the effects of spinal muscular atrophy (SMA) drugs on the muscles and nerve cells in patients with SMA.
- Trial name
- NCT06532474
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- St. Jude Children's Research Hospital
Hip Fractures, Sarcopenia
Feasibility of High Density Sit-to-stand Functional Resistance Training in Patients With Hip Fracture
The objective of this study is to examine if functional high-density, high-volume chair rise training is feasible for increasing lower extremity strength without causing excessive hip pain during execution in patients with hip…
- Conditions
- Hip Fractures, Sarcopenia, Fall Injury
- Trial name
- NCT05840315
- Testing
- Basic intervention and nutritional intervention, Sit-to-stand, Leg press
- Where
- Denmark
- Volunteers
- 30 people
- Sponsor
- Horsholm Municipality
Muscular Atrophy, Spinal
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA).
- Trial name
- FIREFISH (NCT02913482)
- Testing
- Risdiplam
- Where
- 32 sites in United States, Belgium and 14 more
- Volunteers
- 62 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy, Neuromuscular Disorders
Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting
Clinical trials organization in several neuromuscular disorders (NMD) has some specific issues. Nonambulant status and difficulties with transportation are among them.
- Trial name
- NCT02235090
- Testing
- Direct current stimulation of cervical spinal cord
- Where
- Ukraine
- Volunteers
- healthy volunteers welcome
- Sponsor
- Charitable Foundation Children with Spinal Muscular Atrophy
Cerebral Palsy, Spinal Muscular Atrophy
Pediatric Powered Wheelchair Standing Devices: An Exploratory Study
For children who use a power wheelchair, a powered wheelchair standing device (PWSD) may be considered for daily use. A PWSD allows a child to electronically move between sitting and standing and can be driven in either position.
- Conditions
- Cerebral Palsy, Spinal Muscular Atrophy, Spinal Cord Injuries, Spina Bifida
- Trial name
- NCT05117827
- Testing
- Power Wheelchair Standing Device
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Grand Valley State University
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III
The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy types II or III; and to determine if the drug has an effect on SMN mRNA and protein levels.
- Trial name
- NPTUNE01 (NCT00439569)
- Testing
- sodium phenylbutyrate
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Westat
Prostate Cancer, Radiotherapy Side Effect
PROstate CAncer Radiotherapy - Bowel Quality of Life (PROCAR-BQ)
This is a prospective pilot study to evaluate the mean increase of bowel symptoms after pelvic radiotherapy (RT) in prostate cancer (PCa) patient using the validated \& newly translated EORTC-QLQ PRT20 module.
- Conditions
- Prostate Cancer, Radiotherapy Side Effect, Bowel Dysfunction, Quality of Life
- Trial name
- PROCAR-BQ (NCT05880446)
- Testing
- Bowel symptoms & QoL assessment
- Where
- Belgium
- Volunteers
- 41 people
- Sponsor
- Jules Bordet Institute
Spinal Muscular Atrophy
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA).
- Trial name
- NCT01839656
- Testing
- nusinersen
- Where
- 4 sites in United States, Canada
- Volunteers
- 21 people
- Sponsor
- Biogen
Rehabilitation
Early Rehabilitation in Critical Illness Survivors
Background: Few studies showed that early rehabilitation on the Intensive Care Unit (ICU) results in a better functional outcome at hospital discharge and reduced hospital stay.
- Trial name
- ER (NCT02754505)
- Testing
- Early rehabilitation, Usual care
- Where
- Austria
- Volunteers
- 53 people
- Sponsor
- Danube Hospital
Healthy Volunteer, Muscular Atrophy, Spinal
Ability of Muscle Imaging and Motor Function Measure (MFM) to Detect Changes in Disease Progression in Ambulant Spinal Muscular Atrophy Patients Compared to Healthy Volunteers.
This non-drug, single center, 24-week, longitudinal study in ambulant spinal muscular atrophy (SMA) patients and in age- and gender-matched healthy volunteers will assess the detection of disease progression by magnetic resonance…
- Trial name
- NCT02044029
- Where
- Switzerland
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy
Holter of Movement in Patients with SMA Undergoing Treatment.
Acti-SMA is a multi-centric academic study. It aims to monitor the progress of patients with spinal muscular atrophy under treatment with Spinraza° or risdiplam.
- Trial name
- NCT04888702
- Testing
- Actimyo
- Where
- 3 sites in Belgium, Romania
- Volunteers
- 30 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Muscular Atrophy, Spinal
A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Type I Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Type I SMA; and to detect the…
- Trial name
- NCT00568698
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Stanford University
Spinal Muscular Atrophy
A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With Spinal Muscular Atrophy
This is a multisite randomized controlled registry-based trial to evaluate the efficacy of an 8-week home-based exergaming intervention as compared to usual care on occupational satisfaction of children and youth (8-18 years old…
- Trial name
- INFORM SMA (NCT06396325)
- Testing
- Tales from the Magic Keep™, Usual Care
- Where
- 5 sites in Canada
- Volunteers
- 22 people
- Sponsor
- Maryam Oskoui, MD, MSc, FRCPC, FAAN