Clinical trials: Spinal Muscular Atrophy Type 2
Muscular Atrophy, Spinal
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
This expanded access program (EAP) will provide access to risdiplam for eligible participants with Type 1 or Type 2 spinal muscular atrophy (SMA) before it is commercially available in the United States for the indication of SMA.
- Trial name
- NCT04256265
- Testing
- Risdiplam
- Where
- 29 sites in United States
- Sponsor
- Genentech, Inc.
Spinal Muscular Atrophy Type I
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Trial name
- NCT07070999
- Testing
- GB221
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- Gemma Biotherapeutics
Spinal Muscle Atrophy
A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
This multicenter, non-interventional, prospective study with a retrospective component aims to evaluate the real-world clinical outcomes, safety profile, and motor function improvements of Risdiplam treatment in participants with…
- Trial name
- NCT07531719
- Where
- 2 sites in Egypt
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
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.
Spinal Muscular Atrophy
Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy(SMA) Treated With Valproic Acid
The purpose of this study is to determine if the treatment with valproic acid can increase the muscle strength and motor ability of children with spinal muscular atrophy.
- Trial name
- NCT01033331
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- University of Sao Paulo General Hospital
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 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
SMA II, Cerebral Palsy
Paracetamol Study in Patients With Low Muscle Mass
To investigate the safety and toxicity related to paracetamol treatment in children and adults with respectively SMA and CP.
- Trial name
- NCT03648658
- Testing
- Paracetamol 120Mg/5mL Oral Suspension
- Where
- Denmark
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Mette Cathrine Oerngreen
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
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
Neuromuscular Diseases, Cerebral Palsy
Rehabilitative Effect of the Use of a Gait Exoskeleton in Patients With Neuromuscular Disease or Cerebral Palsy
The purpose of this multicentric study is to assess the rehabilitative effect derived from the use of the ATLAS exoskeleton in children with neuromuscular diseases or cerebral palsy and with a level of less than 3 or less in the…
- Conditions
- Neuromuscular Diseases, Cerebral Palsy, SMA II
- Trial name
- NCT04813601
- Testing
- ATLAS 2030
- Where
- Spain
- Volunteers
- 10 people
- Sponsor
- MarsiBionics
Muscular Atrophy, Spinal
A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Types II and III Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Types II and III SMA; and…
- Trial name
- NCT00568802
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Stanford University
Spinal Muscular Atrophy Type 2
GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy
This study explored dose escalation of single-arm, open, single intrathecal injection in patients with delayed onset type 2 SMA. The investigator plans to conduct 2 cohorts.
- Trial name
- GITOPWTSMA (NCT06772402)
- Testing
- GCB-001
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Genecombio Ltd.
Spinal Muscular Atrophy
Natural History of Types 2 and 3 SMA in Taiwan
The primary objective of this study is to investigate the natural history of spinal muscular atrophy (SMA) types 2 and 3 patients in Taiwan. This study will provide further insights into the clinical course SMA.
- Trial name
- NCT03300869
- Where
- Taiwan
- Volunteers
- 300 people
- Sponsor
- Kaohsiung Medical University Chung-Ho Memorial Hospital
Muscular Atrophy, Spinal
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
Multi-center, randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Risdiplam in adult and pediatric participants with Type 2 and Type 3 SMA.
- Trial name
- SUNFISH (NCT02908685)
- Testing
- Risdiplam
- Where
- 45 sites in United States, Belgium and 14 more
- Volunteers
- 231 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy
Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)
The aim of this project is to establish a network of clinical teams including the major neuromuscular centers in Europe. We plan to work together to find the best common outcome measures for the following multicenter therapeutic…
- Trial name
- NCT01611610
- Testing
- Functional tests
- Volunteers
- 4 people
- Sponsor
- Institut de Myologie, France
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- ONYX (NCT05626855)
- Testing
- Apitegromab
- Where
- 49 sites in United States, Belgium and 7 more
- Volunteers
- 238 people
- Sponsor
- Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- TOPAZ (NCT03921528)
- Testing
- SRK-015
- Where
- 16 sites in United States, Italy and 2 more
- Volunteers
- 58 people
- Sponsor
- Scholar Rock, Inc.
Spinal Muscular Atrophy Type III, Spinal Muscular Atrophy Type II
Spinal Cord Gray Matter Imaging in Spinal Muscular Atrophy
This study aims to measure the spinal cord gray matter in patients with spinal muscular atrophy (SMA) type II and III in comparison with age- and sex-matched healthy controls (HC) using rAMIRA (radially sampled averaged…
- Trial name
- NCT06137612
- Testing
- MRI
- Where
- Switzerland
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- University Hospital, Basel, Switzerland
Type 2 Spinal Muscular Atrophy, Type 3 Spinal Muscular Atrophy
Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy
NatHis-SMA is a prospective, longitudinal and interventional study of the natural history of patients with type 2 and 3 Spinal Muscular Atrophy (SMA).
- Trial name
- NatHis-SMA (NCT02391831)
- Testing
- Strength, function and activity measurements, Muscle MRI, Electrophysiology measurements
- Where
- 9 sites in Belgium, France and 1 more
- Volunteers
- 81 people
- Sponsor
- Institut de Myologie, France