Clinical trials: SMA
Spinal Muscular Atrophy (SMA)
A Study of the Safety and Preliminary Efficacy of NKG001 in Pediatric Patients With Type 1 or Type 2 Spinal Muscular Atrophy
This study is a single-center, open-label, single-arm, non-randomized, single-dose, dose-escalation investigator-initiated trial (IIT) designed to evaluate the safety, tolerability, and preliminary efficacy of NKG001 Injection…
- Trial name
- NCT07617779
- Testing
- NKG001
- Where
- China
- Volunteers
- 21 people
- Sponsor
- Nikegen Pharmaceutical (Hangzhou) Company Limited
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
Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth
Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers
In recent years, knowledge of neuromuscular diseases has advanced considerably, and new therapeutic avenues are beginning to emerge.
- Conditions
- Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth, Muscular Dystrophy, Myotonic Dystrophy
- Trial name
- Myokinesis (NCT07321977)
- Testing
- Gait analysis via video capture
- Where
- France
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
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
SMA
EAP of Apitegromab for Patients With Spinal Muscular Atrophy
The purpose of this expanded access program (EAP) is to provide access to apitegromab for eligible patients with spinal muscular atrophy (SMA) prior to approval by the local regulatory agency .
- Trial name
- NCT06877689
- Testing
- Apitegromab
- Sponsor
- Scholar Rock, Inc.
SMA - Spinal Muscular Atrophy
Adrenal& Bone Complications in Paediatric Patients Living With Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a genetic disorder that is known to cause damage to specialised nerve cells, called motor neurons.
- Trial name
- SMA-ABC (NCT07530926)
- Where
- United Kingdom
- Volunteers
- 550 people
- Sponsor
- Sheffield Children's NHS Foundation Trust
Neuromuscular Diseases (NMD), ALS (Amyotrophic Lateral Sclerosis)
Invasive Home Ventilation in Denmark
The aim of this study is to describe national trends over the past 10 years in patients receiving invasive home mechanical ventilation (HMV) in Denmark.
- Conditions
- Neuromuscular Diseases (NMD), ALS (Amyotrophic Lateral Sclerosis), Spinal Cord Injuries (SCI), Duchenne Muscular Dystrophy (DMD)
- Trial name
- HOMEVENT DK (NCT07467187)
- Where
- Denmark
- Volunteers
- 450 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Spinal Muscular Atrophy (SMA), Cerebral Palsy (CP)
Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders
Both GIP and GLP-2 reduce bone resorption (measured as CTX) in healthy adult individuals. In this study, we will investigate whether GIP and GLP-2 reduce CTX in children with spinal muscular atrophy, duchenne muscular dystrophy…
- Conditions
- Spinal Muscular Atrophy (SMA), Cerebral Palsy (CP), Duchenne Muscular Dystrophy (DMD)
- Trial name
- NCT07254988
- Testing
- Meal test, GIP + GLP-2
- Where
- Denmark
- Volunteers
- 3 people
- Sponsor
- University of Copenhagen
SMA - Spinal Muscular Atrophy
HABIT-ILE + FST in Children With SMA
This single-arm pilot study will assess the feasibility and preliminary effectiveness of an intensive motor skill intervention (HABIT-ILE) combined with functional strength training (FST) in children with SMA who are receiving…
- Trial name
- NCT07223320
- Testing
- Combined Hand-Arm Bimanual Intensive Therapy Including Lower Extremities (HABIT-ILE) + functional strength training (FST)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Teachers College, Columbia University
1 Hz Real rTMS to the Pre-SMA, 1 Hz Sham rTMS to the Pre-SMA
TMS for Exposure Therapy Resistant OCD
TETRO is a multi-center placebo-controlled double-blind randomized controlled trial with an intervention phase of 5-7 weeks and a follow-up phase of 12 months in 250 adult (18 years and older) OCD patients who show…
- Trial name
- TETRO (NCT05331937)
- Testing
- repetitive transcranial magnetic stimulation (rTMS)
- Where
- 8 sites in Netherlands
- Volunteers
- 250 people
- Sponsor
- Amsterdam UMC, location VUmc
SMA - Spinal Muscular Atrophy, Gene Therapy
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
- Trial name
- STR1VE (NCT03306277)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 16 sites in United States
- Volunteers
- 22 people
- Sponsor
- Novartis Gene Therapies
SMA
Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in patients with spinal muscular atrophy (SMA) Type 1 who meet enrollment criteria and are genetically defined by a…
- Trial name
- STRIVE-EU (NCT03461289)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 10 sites in Belgium, France and 2 more
- Volunteers
- 33 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved…
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- SAPPHIRE (NCT05156320)
- Testing
- Apitegromab
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 188 people
- Sponsor
- Scholar Rock, Inc.
Muscular Dystrophies, Myopathies
Observational Study to Observe Variations of Gait Parameters in Patients With Neuromuscular Diseases
This study has the general objective of observing walking parameters during a clinical test to objectively estimate fatigue in patients with neuromuscular diseases.
- Conditions
- Muscular Dystrophies, Myopathies, Spinal Muscular Atrophy (SMA), Charcot Marie Tooth Disease
- Trial name
- NCT06666816
- Testing
- inapplicable
- Where
- 2 sites in Italy
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- IRCCS Eugenio Medea
Spinal Muscular Atrophy (SMA)
Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System
Evaluate the diagnostic journey, patient experience, and disparities in the treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System.
- Trial name
- SMA (NCT07223489)
- Testing
- Telephone Interview
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Medstar Health Research Institute
SMA, DMD
Swiss Registry for Neuromuscular Disorders
The Swiss Patient Registry for DMD/BMD and SMA was launched in 2008 in order to give Swiss patients access to new therapies. It was founded with the financial support of several patient organizations and research foundations.
- Conditions
- SMA, DMD, BMD, IMD
- Trial name
- Swiss-Reg-NMD (NCT05102916)
- Where
- 19 sites in Switzerland
- Volunteers
- 2,000 people
- Sponsor
- University of Bern
Spinal Muscular Atrophy (SMA)
Neuroproprioceptive Equine-Assisted Physiotherapy for Spinal Muscular Atrophy
This study investigates whether Equine-Assisted Physiotherapy based on Neuro-proprioceptive "Facilitation and Inhibition" (NEUROEQUIP-SMA) can improve movement, posture, breathing, and quality of life in children with spinal…
- Trial name
- NEUROEQUIP (NCT07336602)
- Testing
- Equine-Assisted Physiotherapy based on Neuro-proprioceptive "Facilitation and Inhibition", Standard Individual Outpatient Physiotherapy Based on Neuro-proprioceptive Facilitation and Inhibition, Therapeutic grooming
- Where
- 4 sites in Czechia
- Volunteers
- 20 people
- Sponsor
- Charles University, Czech Republic
Spinal Muscular Atrophy (SMA)
Long Read Analysis in Spinal Muscular Atrophy - LOREASI
Spinal Muscular Atrophy (SMA) is a severe neuromuscular disease caused by deletion of the SMN1 gene, with the most severe form leading to death in children without treatment.
- Trial name
- LOREASI (NCT07332702)
- Testing
- blood sample
- Where
- France
- Volunteers
- 27 people
- Sponsor
- University Hospital, Rouen
Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD)
Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening
The Active NBS Liege study is a monocentric, academic, fully remote, observational study designed to validate digital measures of motor development in children with spinal muscular atrophy (SMA) or Duchenne muscular dystrophy…
- Trial name
- Active-NBS BE (NCT07286565)
- Testing
- MAIJU, Syde, Questionnaires
- Where
- Belgium
- Volunteers
- 100 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Spinal Muscular Atrophy (SMA), Spinal Muscular Atrophy Type I
Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine Regions
The systematic inclusion of spinal muscular atrophy (SMA) in France's neonatal genetic screening (NGS) program, scheduled for September 2025, represents a major milestone in public health.
- Trial name
- PSYSMA (NCT07208903)
- Testing
- focus group discussions, Interview with the psychologist
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France