Clinical trials: Spinal Muscular Atrophy (SMA)
Spinal Muscular Atrophy
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S.
- Trial name
- WeSMA (NCT05232929)
- Testing
- Risdiplam
- Where
- 38 sites in United States, Puerto Rico
- Volunteers
- 403 people
- Sponsor
- Genentech, Inc.
Cerebral Palsy, Acquired Brain Injury
Safety and Usability of a Robotic Gait Device for Children and Adolescents With Neurological or Neuromuscular Disease in Their Natural Environment
The goal of this clinical trial is to analyze the usability and safety of the robotic gait device EXPLORER in children with cerebral palsy, acquired brain injury and spinal muscular atrophy.
- Conditions
- Cerebral Palsy, Acquired Brain Injury, Spinal Muscular Atrophy
- Trial name
- NCT06167954
- Testing
- EXPLORER
- Where
- 5 sites in Spain
- Volunteers
- 50 people
- Sponsor
- MarsiBionics
Spinal Muscular Atrophy, Spine Deformity
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRx™ System in delivering nusinersen in subjects with spinal muscular atrophy (SMA).
- Conditions
- Spinal Muscular Atrophy, Spine Deformity, Scoliosis
- Trial name
- PIERRE (NCT05866419)
- Testing
- ThecaFlex DRx System
- Where
- 21 sites in United States, Germany and 3 more
- Volunteers
- 90 people
- Sponsor
- Alcyone Therapeutics, Inc
Prostate Cancer
A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer Drugs
ANDROMEDA is a first-in-human, Phase I/II, open-label, multicenter study of AZD9750 in participants with metastatic prostate cancer.
- Trial name
- ANDROMEDA (NCT07336446)
- Testing
- AZD9750, AZD5305
- Where
- 5 sites in United States, Australia and 2 more
- Volunteers
- 6 people
- Sponsor
- AstraZeneca
Spinal Muscular Atrophy
MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA) is caused by the homozygous loss of the Survival Motor Neuron (SMN) 1 gene, which leads to degeneration of spinal alpha-motor neurons and muscle atrophy.
- Trial name
- MAP_THE_SMA-01 (NCT05769465)
- Testing
- disease modifying treatments
- Where
- Italy
- Volunteers
- 247 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Circulating Tumor Cell, Metastatic Prostate Cancer
Cabazitaxel in mCRPC Patients With AR-V7 Positive Circulating Tumor Cells (CTCs)
After failure on docetaxel, which has been the standard first line therapy for patients with metastatic castration-resistant prostate cancer (mCRPC), several treatment options are currently available.
- Trial name
- CABA-V7 (NCT03050866)
- Testing
- Cabazitaxel, Antihistamine, Corticosteroid
- Where
- 9 sites in Netherlands
- Volunteers
- 140 people
- Sponsor
- Erasmus Medical Center
Spinal Muscular Atrophy
Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA)
The primary objective of this research protocol is to study and follow the course of motor neuron loss in individuals with spinal muscular atrophy (SMA) using the electrophysiological technique of motor unit number estimation…
- Trial name
- NCT04139343
- Where
- United States
- Volunteers
- 67 people, healthy volunteers welcome
- Sponsor
- Ohio State University
Infantile Spinal Muscular Atrophy of Type 2 or 3
Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA)
This clinical trial aims to test a new physio-therapeutic approach tailored to type 2 and 3 Spinal Muscular Atrophy patients, based on physical training in swimming-pool.
- Trial name
- ExerASI (NCT02061189)
- Testing
- Physical exercise in a swimming pool during 6 months
- Where
- France
- Volunteers
- 19 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Muscular Atrophy, Spinal
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
- Trial name
- PUPFISH (NCT05808764)
- Testing
- Risdiplam
- Where
- 13 sites in United States, Belgium and 6 more
- Volunteers
- 11 people
- Sponsor
- Hoffmann-La Roche
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
TRIAL READY (Clinical Trial Readiness)
This study, being conducted under the auspice of the CReATe Consortium, will enroll patients with ALS and related disorders as well as healthy controls, with the goal of facilitating clinical validation of leading…
- Conditions
- Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, ALS-Frontotemporal Dementia, Primary Lateral Sclerosis
- Trial name
- NCT03912987
- Where
- 3 sites in United States
- Volunteers
- 610 people, healthy volunteers welcome
- Sponsor
- University of Miami
Spinal Muscular Atrophy
Equine-Assisted Physiotherapy for Spinal Muscular Atrophy
This prospective interventional study evaluates the immediate and sustained effects of intensive equine-assisted physiotherapy in children with spinal muscular atrophy (SMA).
- Trial name
- HIPOSMA (NCT05341453)
- Testing
- Individual physiotherapy (SMA-SOC-N), Equine-Assisted Physiotherapy Based on Neuroproprioceptive Facilitation and Inhibition, Therapeutic grooming
- Where
- Czechia
- Volunteers
- 21 people
- Sponsor
- Charles University, Czech Republic
Muscular Dystrophy, Duchenne, Orthotic Devices
The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder
The goal of this clinical trial is to investigate the effect of a muscle-mimicking, fabric-type shoulder orthosis on functional movements of the upper limb in patients with neuromuscular disorder.
- Conditions
- Muscular Dystrophy, Duchenne, Orthotic Devices, Upper Extremity, Neuromuscular Diseases (NMD)
- Trial name
- NCT06363357
- Testing
- Shoulder orthosis
- Where
- South Korea
- Volunteers
- 8 people
- Sponsor
- Seoul National University Hospital
Spinal Muscular Atrophy
Implementation of FEES in Spinal Muscle Atrophy
The major aim of this project is to assess comprehensively frequency and extent of dysphagia and bulbar dysfunction in SMA1, 2, and 3 patients by applying FEES and validated dysphagia scores.
- Trial name
- DYS-SMA (NCT04773470)
- Testing
- Flexible Endoscopic Evaluation of Swallowing
- Where
- Germany
- Volunteers
- 79 people
- Sponsor
- University of Giessen
Spinal Muscular Atrophy
Sun May Arise on SMA : Newborn Screening of Spinal Muscular Atrophy in Belgium
Medico-economic study of Newborn screening of Spinal Muscular Atrophy
- Trial name
- NCT03554343
- Testing
- test for SMN1 exon 7 deletion
- Where
- Belgium
- Volunteers
- 136,339 people
- Sponsor
- Centre Hospitalier Régional de la Citadelle
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
Spinal Muscular Atrophy
Respiratory Muscle Training in Patients With Spinal Muscular Atrophy (SMA).
The goal of this clinical trial is to study respiratory muscle training in patients with Spinal Muscular Atrophy (SMA). The main questions it aims to answer are: Is a home-based individualized training program for the inspiratory…
- Trial name
- RESISTANT (NCT05632666)
- Testing
- Inspiratory and expiratory muscle training at a therapeutic intensity, Inspiratory and expiratory muscle training at a non-therapeutic intensity
- Where
- Netherlands
- Volunteers
- 30 people
- Sponsor
- UMC Utrecht
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
Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 4
Spinal Cord Stimulation in Spinal Muscular Atrophy
Spinal cord stimulation (SCS) has shown remarkable efficacy in restoring motor function in people with spinal cord injury by recruiting afferent input to enhance the responsiveness of spared neural circuits to residual cortical…
- Trial name
- SCSinSMA (NCT05430113)
- Testing
- Spinal Cord Stimulator (octopolar Medtronic Vectris Leads)
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Marco Capogrosso
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
Spinal Muscular Atrophy (SMA)
Robot-assisted Training
The goal of this clinical trial is to evaluate the long-term effects of isokinetic rehabilitation training in patients with spinal muscular atrophy (SMA).
- Trial name
- NCT06648486
- Testing
- With the assistance of wearable isokinetic training robot （ 7 children with SMA type II）
- Where
- China
- Volunteers
- 13 people
- Sponsor
- Peking University Third Hospital