Clinical trials: Spinal Muscle Atrophy
Tremor, Involuntary Movements
Hyperkinetic Movements in Patients With Disease of Motor Neurons and Their Response to Treatment With Nusinersen
Hyperkinetic movement disorders in patients with diseases of motor neurons will be studied. Patients with spinal muscular atrophy (SMA) and motor neuron disease patients will be studied.
- Conditions
- Tremor, Involuntary Movements, SMA, MND (Motor Neurone DIsease)
- Trial name
- NCT04825119
- Testing
- Nusinersen
- Where
- Slovenia
- Volunteers
- 110 people
- Sponsor
- University Medical Centre Ljubljana
Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy
Study of ALS Reversals 4: LifeTime Exposures
Hypothesis: There exists patients who have met ALS or PMA diagnostic criteria and subsequently experienced robust and sustained improvement, i.e.
- Conditions
- Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy, Motor Neuron Disease
- Trial name
- StARLiTE (NCT03706391)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Duke University
Spinal Muscular Atrophy (SMA)
Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy
There is a general physiological rule that any organ or system needs some minimal amount of activity to prevent its atrophy or degeneration.
- Trial name
- STIM-SMA (NCT06977269)
- Testing
- High-frequency repetitive transcranial magnetic stimulation, lumbar puncture
- Where
- Ukraine
- Volunteers
- 20 people
- Sponsor
- Charitable Foundation Children with Spinal Muscular Atrophy
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
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene…
- Trial name
- HINALEA 2 (NCT05861999)
- Testing
- Risdiplam
- Where
- 17 sites in United States, Germany and 4 more
- Volunteers
- 19 people
- Sponsor
- Hoffmann-La Roche
Infantile-onset Spinal Muscular Atrophy
Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
To provide access to nusinersen to eligible patients with Infantile-onset Spinal Muscular Atrophy (SMA) (consistent with Type 1) to address a high-unmet medical need.
- Trial name
- NCT02865109
- Testing
- Nusinersen
- Where
- 7 sites in Colombia, New Zealand and 1 more
- Sponsor
- Biogen
Spinal Muscular Atrophy
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
In this study, researchers will learn more about the use of a higher dose of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA).
- Trial name
- ASCEND (NCT05067790)
- Testing
- Nusinersen
- Where
- 22 sites in United States, Germany and 3 more
- Volunteers
- 45 people
- Sponsor
- Biogen
SMA
Study to Evaluate the Efficacy of Riluzole in Children and Young Adults With Spinal Muscular Atrophy (SMA)
This is a multicentric, randomized, double-blind study versus placebo, with two parallel groups treated to evaluate the efficacy and the tolerance of Riluzole in children and young adults (6 to 20 years of age) with SMA.
- Trial name
- ASIRI (NCT00774423)
- Testing
- Riluzole
- Where
- France
- Volunteers
- 141 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Spinal Muscular Atrophy
Development of a Multidisciplinary Network for Clinical and Laboratory Research for SMA
The goal of this observational study is to to establish profiles of clinical progression in patients affected by the different types of SMA (type I, II and III) treated with the currently approved drugs using a structured battery…
- Trial name
- NCT05866939
- Volunteers
- 400 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
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
Spinal Muscular Atrophy
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in individuals with SMA who are between the ages…
- Trial name
- SOLAR (NCT07444476)
- Testing
- Salanersen
- Where
- 3 sites in United States, China
- Volunteers
- 90 people
- Sponsor
- Biogen
Duchenne's Muscular Dystrophy, Scoliosis
Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne's Muscular Dystrophy
Nearly all patients with Duchenne's Muscular Dystrophy (DMD) have scoliosis. Posterior instrumented spinal fusion, which is a surgery to correct scoliosis, has been shown to improve quality of life and satisfaction of both…
- Trial name
- NCT00207857
- Where
- United States
- Sponsor
- Children's Healthcare of Atlanta
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
Muscular Atrophy, Spinal
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP).
- Trial name
- PIERRE-PK (NCT06555419)
- Testing
- Nusinersen, ThecaFlex DRx System
- Where
- 21 sites in United States, France and 5 more
- Volunteers
- 78 people
- Sponsor
- Biogen
Spinal Muscular Atrophy, Neonatal Hyperbilirubinemia
Establishing Novel Detection Techniques for Various Genetic-Related Diseases by Applying DHPLC Platform.
In this, here we want to present a new method for analysis variation in gene copy number for patients and carriers of SMA. This is a relative quantitation method and, therefore, relies on the inclusion of one or more internal…
- Conditions
- Spinal Muscular Atrophy, Neonatal Hyperbilirubinemia, Colon Cancer
- Trial name
- NCT00154960
- Where
- Taiwan
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
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
Spinal Muscular Atrophy
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA).
- Trial name
- CHERISH (NCT02292537)
- Testing
- Nusinersen
- Where
- 24 sites in United States, Canada and 8 more
- Volunteers
- 126 people
- Sponsor
- Biogen
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
Spinal Muscular Atrophy
European Registry of Patients With Infantile-onset Spinal Muscular Atrophy
IO-SMA-Registry is a prospective, longitudinal and observational study which objective is to collect prospectively information on longevity, psychomotor development and respiratory function of patients with infantile-onset spinal…
- Trial name
- NCT03339830
- Where
- 6 sites in France
- Volunteers
- 100 people
- Sponsor
- Institut de Myologie, France
Duchenne Muscular Dystrophy, Spinal Muscular Atrophy
Flu Vaccine Study in Neuromuscular Patients 2011
The purpose of the study is to compare the immune response of two different injection methods (Intramuscular V.S. Subcutaneous) of the 2011-2012 seasonal Influenza (Flu) vaccine among patients with neuromuscular conditions who…
- Conditions
- Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Congenital Muscular Dystrophy
- Trial name
- NCT01422200
- Testing
- 2011-2012 seasonal flu vaccine Subcutaneous, 2011-2012 seasonal flu vaccine Intramuscular
- Volunteers
- 22 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati