Clinical trials: SMA1
Infantile Spinal Muscular Atrophy
Test of Aquatic Mobility of SMA Infants
Spinal muscular atrophy (SMA) is a genetic neurodegenerative disease impacting spinal cord motor neurons, leading to motor and respiratory issues and, ultimately, death.
- Trial name
- BAINSMA (NCT06152302)
- Testing
- Mobility study in dry conditions, Mobility study in water conditions
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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 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 Type I, Spinal Muscular Atrophy Type II
Body Weight Support Harness System in Spinal Muscular Atrophy
The goal of this pilot interventional study is to learn about the use of an in-home harness system in children who have been treated for spinal muscular atrophy. The main questions it aims to answer are: 1.
- Trial name
- NCT05715749
- Testing
- In-home body weight support harness system
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Nationwide Children's Hospital
Spinal Muscular Atrophy Type I
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I
The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy type I; and to determine if the drug has an effect on SMN mRNA and protein levels.
- Trial name
- NPTUNE 02 (NCT00439218)
- Testing
- sodium phenylbutyrate
- Where
- 5 sites in United States
- Volunteers
- 5 people
- Sponsor
- Westat
Type I Spinal Muscular Atrophy
Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy.
- Trial name
- NCT06576388
- Testing
- EXG001-307 injection
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Spinal Muscular Atrophy
Prospective Evaluation of Infants With Spinal Muscular Atrophy:
SPOT SMA is a prospective NIH-supported clinical study targeting pre-symptomatic or recently diagnosed infants and children with Spinal Muscular Atrophy (SMA) types 1, 2, or 3 and their healthy control siblings less than 36…
- Trial name
- SPOTSMA (NCT02831296)
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Spinal Muscular Atrophy 1
A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I
This is a phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I.
- Trial name
- NCT07554924
- Testing
- SKG0201 Injection
- Where
- 3 sites in China
- Volunteers
- 11 people
- Sponsor
- Lanyue Biotech (Hangzhou) Co., Ltd.
SMA1
Exploring Bulbar Function, Speech And Communication Development in SMA Type 1
5q-spinal muscular atrophy (5q-SMA) is a rare, autosomal recessive neuromuscular disease characterized by degeneration of motor neurons in the spinal cord and lower brainstem with progressive muscle atrophy, weakness, and…
- Trial name
- ExSpAnD SMA (NCT05954455)
- Where
- United Kingdom
- Volunteers
- 30 people
- Sponsor
- Institute of Child Health
Spinal Muscular Atrophy
Infants With Spinal Muscular Atrophy Type I
Background: Spinal muscular atrophy type 1 (SMA 1) causes severe muscle weakness and problems with eating and breathing. The symptoms begin in infancy, and children affected with SMA 1 often die in early childhood.
- Trial name
- NCT01547871
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
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
Spinal Muscular Atrophy Type I
Impact of Motor and Oral Motor Function on Quality of Life in Children With SMA
The aim of this study is to investigate the impact of motor and oral motor functions on the quality of life in children aged 2-4 years diagnosed with Spinal Muscular Atrophy (SMA) Type I.
- Trial name
- NCT06864767
- Testing
- Neuro-sensory Motor Developmental Assessment, Functional Oral Intake Scale, Behavioral Pediatrics Feeding Assessment Scale
- Where
- Turkey (Türkiye)
- Volunteers
- 23 people
- Sponsor
- Zeynep HOŞBAY
Infantile Spinal Muscular Atrophy
Trial Evaluating the Interest of Noninvasive Ventilation in NAVA Mode in Respiratory Decompensations Children With Infantile Spinal Muscular Atrophy Type II
The new NAVA® ventilatory mode with the SERVO-i ventilator (Maquet®) uses the electrical activity of the diaphragm (EADi) as a marker for triggering the respiratory cycle.
- Trial name
- NAVASI (NCT03395795)
- Testing
- NAVA
- Where
- France
- Volunteers
- 1 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Spinal Muscular Atrophy 1
Palliative Care in Spinal Muscular Atrophy (SMA) 1
The purpose of this study is to evaluate the quality of supportive and palliative care for SMA type 1 patients.
- Trial name
- ASI1 (NCT01862042)
- Testing
- Follow-up diary and questionnaire
- Where
- France
- Volunteers
- 39 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Spinal Muscular Atrophy Type I
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
This is a Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular atrophy (SMA) Type 1 and who are genetically defined by a biallelic…
- Trial name
- NCT03837184
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 4 sites in Japan, South Korea and 1 more
- Volunteers
- 2 people
- Sponsor
- Novartis Gene Therapies
Muscular Atrophy, Spinal
International SMA Patient Registry
This is a registry of individuals affected by Spinal Muscular Atrophy (SMA) and/or family members of individuals affected by SMA.
- Trial name
- NCT00466349
- Where
- United States
- Volunteers
- 3,000 people
- Sponsor
- Indiana University
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 1
Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Regarding Feeding and Communication
Spinal Muscular Atrophy Type 1 (SMA )Type 1 is a severe, early-onset neuromuscular condition that typically leads to profound weakness and impaired bulbar function-affecting swallowing, feeding, speech, and airway protection.
- Trial name
- NCT07596277
- Where
- United Kingdom
- Volunteers
- 15 people
- Sponsor
- Guy's and St Thomas' NHS Foundation Trust
Spinal Muscular Atrophy (SMA)
Advanced Multi-omics and -System Approaches to Identify Novel Biomarkers for SMA
Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder caused by mutations in the SMN1 gene, leading to motor neuron degeneration and progressive muscle atrophy.
- Trial name
- MOSAiC-SMA (NCT07883655)
- Where
- 3 sites in Italy
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Istituto Giannina Gaslini
Spinal Muscular Atrophy
A Pilot Study of Biomarkers for Spinal Muscular Atrophy
The goal of this pilot study is to identify a marker or panel of markers in the blood or urine from a wide range of Spinal Muscular Atrophy (SMA) patients that segregates with measures of clinical severity.
- Trial name
- BforSMA (NCT00756821)
- Where
- 18 sites in United States, Canada
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- Carelon Research