Clinical trials: Neuromuscular Physiology
Neuromuscular Diseases
Effectiveness of Respiratory Physiotherapy in Children With Neuromuscular Disease
The main objective of this study is to determine whether the Respiratory Physiotherapy allows improvement or maintenance of respiratory function in children with Neuromuscular Diseases, against respiratory deterioration that…
- Trial name
- NCT02743702
- Testing
- RESPIRATORY PHYSIOTHERAPY, USUAL THERAPIES
- Where
- Spain
- Volunteers
- 21 people
- Sponsor
- University of Seville
Duchenne Muscular Dystrophy (DMD)
Twice Weekly Steroids and Exercise as Therapy for DMD
The study team will determine the potential of low dose twice weekly prednisone and whether exercise training can synergize to delay disease progression and improve muscle strength/physical function in boys with Duchenne muscular…
- Trial name
- NCT04322357
- Testing
- Prednisone, Exercise Training on standard steroid regimen, Exercise training on twice-weekly steroids
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- University of Florida
Muscular Dystrophy, Duchenne, Ambulatory Care
Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)
The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD.
- Trial name
- DELOS (NCT01027884)
- Testing
- Idebenone
- Where
- 23 sites in United States, Austria and 8 more
- Volunteers
- 65 people
- Sponsor
- Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
Use of Dynamic Arm Supports to Promote Activities of Daily Living in Individuals With DMD
This study is a longitudinal, randomized control trial evaluating the use of two commercially available dynamic arm support devices (1) Armon Ayura-Kinova and 2) JAECO WREX) to promote participation in activities of daily living…
- Trial name
- NCT03531788
- Testing
- Armon Ayura (Kinova), JAECO Wrex
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Roxanna Marie Bendixen
Spinal Muscular Atrophy
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥…
- Trial name
- SMART (NCT04851873)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 24 people
- Sponsor
- Novartis Pharmaceuticals
Muscular Dystrophies, Muscular Dystrophy, Duchenne
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne Muscular Dystrophy (DMD).
- Conditions
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- HOPE-2 (NCT03406780)
- Testing
- CAP-1002
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Capricor Inc.
Type 2 Spinal Muscular Atrophy
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
This was a Phase III multi-center, single dose (1.2 x 10^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never…
- Trial name
- STEER (NCT05089656)
- Testing
- OAV101
- Where
- 42 sites in United States, Brazil and 11 more
- Volunteers
- 126 people
- Sponsor
- Novartis Pharmaceuticals
Muscular Atrophy, Spinal
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)
This was a phase IV Open-label, single-arm, single-dose, multicenter study, to evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in…
- Trial name
- OFELIA (NCT05073133)
- Testing
- OAV101
- Where
- 5 sites in Argentina, Brazil
- Volunteers
- 16 people
- Sponsor
- Novartis Pharmaceuticals
Spinal Muscular Atrophy Type 3
Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) are one of the common physical disabilities in childhood. For SMA, progressive muscle weakness and early fatigue hamper the mobility of the sufferers.
- Trial name
- NCT03056144
- Testing
- whole body vibration therapy
- Where
- Hong Kong
- Volunteers
- 1 people
- Sponsor
- The Hong Kong Polytechnic University
Duchenne Muscular Dystrophy
Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT01207908
- Testing
- IGF-1
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Familial Dysautonomia
The Effects Of Bronchodilator Therapy On Respiratory And Autonomic Function In Patients With Familial Dysautonomia
Evaluate the effects of bronchodilator therapy on respiratory function. Our overall goal is to determine whether, in patients with familial dysautonomia (FD), there is a component of airway obstruction that is reversible.
- Trial name
- NCT01987219
- Testing
- Albuterol-sulphate, Ipratropium-bromide
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- NYU Langone Health
Neuromuscular Disease
Respiratory Muscle Strength in Patients With NMD
The purpose of this study is to determine if inspiratory muscle strength training (IMST) will impact maximal inspiratory pressure and pulmonary function in patients with neuromuscular disease.
- Trial name
- RMST (NCT01555905)
- Testing
- Threshold PEP or IMT device Phillips-Respironics
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- University of Florida
Spinal Muscular Atrophy
Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam
This was a Phase IIIb open-label, single arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101B in participants with SMA aged 2 to <18 years after the discontinuation of treatment with nusinersen or…
- Trial name
- STRENGTH (NCT05386680)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 27 people
- Sponsor
- Novartis Pharmaceuticals