Clinical trials: SMA1
Spinal Muscular Atrophy 1
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT02122952
- Testing
- AVXS-101
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Novartis Gene Therapies
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 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 1
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi.
- Trial name
- START (NCT03421977)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Novartis Gene Therapies
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
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
A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1
This is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).
- Trial name
- NCT06191354
- Testing
- SKG0201 Injection
- Where
- 3 sites in China
- Volunteers
- 12 people
- Sponsor
- Kun Sun
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
EEG Sleep Spindles, Motor Outcome
Sleep Spindles Organization as an Early Neural Marker of Neuromotor Outcome
The goal of this observational study is to test the effectiveness of quantitative early biomarkers in the sleep electroencephalogram (EEG), namely sleep spindles, as predictors of early sensorimotor maturation and long-term motor…
- Conditions
- EEG Sleep Spindles, Motor Outcome, Cerebral Palsy, SMA1
- Trial name
- SONNO (NCT06256887)
- Where
- 3 sites in Italy
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- IRCCS Fondazione Stella Maris
Spinal Muscular Atrophy Type I, Scoliosis
The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a serious neuromuscular disease characterized by the degeneration of alpha motor neurons in the spinal cord, resulting in progressive proximal muscle atrophy and denervation.
- Conditions
- Spinal Muscular Atrophy Type I, Scoliosis, Chest Deformities, Spinal Orthosis
- Trial name
- NCT05878418
- Testing
- Thoracolumbosacral Spinal Orthosis (TLSO), Trunk Exercise (TE), Pulmonary Rehabilitation(PR) and Pulmonary Care (PC)
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Medipol University
Spinal Muscular Atrophy
Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America
The natural history of SMA patients has changed, due to the improvements in treatment and technological advances. The systematic collection of data from routine clinical practice in multiple Latin American countries, harmonized…
- Trial name
- RegistrAME (NCT05475691)
- Where
- 18 sites in Argentina, Brazil and 4 more
- Volunteers
- 361 people
- Sponsor
- Hospital Israelita Albert Einstein
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
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 (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 Type I, Spinal Muscular Atrophy Type II
Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).
- Trial name
- NCT06632730
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Novartis
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
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)
Spinal Muscular Atrophy Type I
CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
This is a multi-center trial to test safety and evaluate early treatment intervention with valproic acid and carnitine in moderating SMA symptoms of Type I infants.
- Trial name
- NCT00661453
- Testing
- Valproic Acid and Levocarnitine
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 40 people
- Sponsor
- University of Utah
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
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.