Clinical trials: Muscular Atrophy, Spinal
Spinal Muscular Atrophy
Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular Atrophy with 3 copies of SMN2 and deletion of SMN1.
- Trial name
- STRONG (NCT03381729)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 11 sites in United States
- Volunteers
- 32 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy
Motor Function and Activity of Daily Life in Spinal Muscular Atrophy
This study will incorporate patients/ caregivers' perspectives to investigate the performance in daily activities of individuals with SMA and how it relates to their motor function abilities.
- Trial name
- NCT05931211
- Where
- Taiwan
- Volunteers
- 100 people
- Sponsor
- National Taiwan University Hospital
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)
Post-Polio Syndrome
Post-Poliosyndrome Treated With Intravenous Immunoglobulin (IvIg)
Inflammatory cytokines in the cerebrospinal fluid may contribute to the new muscle weakness, fatigue and pain experienced by patients with post-polio syndrome. Intravenousimmunoglobulin (IvIg) reduces this inflammation.
- Trial name
- NCT00231439
- Testing
- IvIg
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Haukeland University Hospital
Myelomeningocele, Spinal Cord Injury
Reciprocating Gait Orthoses for Paraplegia Patients
Ambulation would bring many physiological and psychological benefits and getting up and walking has been a dream for paraplegia patients.The reciprocating gait orthoses (RGOs) for paraplegics particularly draws research…
- Conditions
- Myelomeningocele, Spinal Cord Injury, Spinal Muscular Atrophy
- Trial name
- UPGO (NCT02227407)
- Testing
- RGOs(reciprocating gait orthoses)
- Where
- Taiwan
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
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
Spina Bifida, Neuromuscular Diseases
Metformin in Children With Motor Deficit
Obesity with insulin resistance in the paediatric population provides an increasing challenge. Children with neurological or neuromuscular diseases are even more prone to obesity: their locomotor impairment leads to an…
- Trial name
- NCT00720161
- Testing
- Metformin
- Where
- Belgium
- Volunteers
- 42 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Androgenetic Alopecia
Association of Polymorphisms in the Androgen Receptor Gene and Finasteride Response in Women With Androgenetic Alopecia
Previous studies of finasteride treatment in women with hair loss have failed to show positive results, yet, some women have responded anecdotally.
- Trial name
- NCT01052870
- Testing
- Finasteride
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- HairDx, LLC
Spinal Muscular Atrophy
Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
Risdiplam Exchange (RISE) is a study of spinal muscular atrophy (SMA) patients who crossover to 36 months of open-label risdiplam monotherapy following a comparable period of nusinersen treatment.
- Trial name
- RISE (NCT05522361)
- Testing
- Risdiplam
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Clinic for Special Children
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2…
- Trial name
- HINALEA 1 (NCT05861986)
- Testing
- Risdiplam
- Where
- 16 sites in United States, Germany and 2 more
- Volunteers
- 28 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy
A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022
Spinal Muscular Atrophy (SMA) is a rare neuromuscular condition, characterised by loss of motor neurons as a result of a mutation in the survival motor neuron gene.
- Trial name
- NCT05994950
- Where
- 2 sites in United Kingdom
- Volunteers
- 30 people
- Sponsor
- Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust
Spinal Muscular Atrophy
Newborn Screening for Spinal Muscular Atrophy (SMA) - a Proof of Principle Study Using Anonymised Blood Spots.
Spinal muscular atrophy (SMA) is a motor neuron disorder caused by the absence of a functional survival of motor neuron 1, telomeric (SMN1) gene. Type I SMA, a lethal disease of infancy, accounts for the majority of cases.
- Trial name
- NCT05237544
- Where
- United Kingdom
- Volunteers
- 5,065 people, healthy volunteers welcome
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Spinal Muscular Atrophy (SMA)
VRehab-SMA Phase 1.2
Spinal muscular atrophy is a genetic disorder characterized by progressive muscle weakness, severely impacting patients' motor abilities.
- Trial name
- NCT07578454
- Testing
- VRehab SMA device
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- University of Oxford
Spinal Muscular Atrophy
Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
With the advent of new treatments for ASI, new phenotypes are emerging. The investigators propose to describe these new phenotypes by prospectively following children with ASI of all types treated with TRS and aged under 16 for 2…
- Trial name
- PHENO SMART (NCT06321965)
- Testing
- evaluation of muscle function, First-line cognitive assessment, second-line cognitive assessment
- Where
- 8 sites in France
- Volunteers
- 60 people
- Sponsor
- Hospices Civils de Lyon
SMARD1, CMT2S
Gene Therapy for IGHMBP2-Related Diseases
Open-label, single intrathecal injection study of a AAV9 vector carrying the IGHMBP2 gene for IGHMBP2-related diseases.
- Trial name
- NCT05152823
- Testing
- Gene Therapy
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Megan Waldrop
Spinal Muscular Atrophy
Clinical Study of Spinal Muscular Atrophy
The investigators propose to prepare for clinical trials where SMA patients are asked to join the research effort. The visits will include questions, physical exam, blood drawing, and sometimes X-rays and a skin biopsy.
- Trial name
- NCT00443066
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- Columbia University
Spinal Muscular Atrophy
Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies
The aim of the study is to propose a method for quantifying motor function in infants with spinal muscular atrophy treated with innovative therapies using inertial sensors.
- Trial name
- IMUSMA (NCT04833348)
- Testing
- Motor function measurement using inertial sensors
- Where
- France
- Volunteers
- 35 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Spinal Muscular Atrophy, Muscular Dystrophy
National Registry for Egyptian Pediatric Neuromuscular Diseases
Our aim is to establish multi-center national Egyptian database of information for inherited and acquired neuromuscular diseases in infants and children from 0 to 18 years of age.
- Conditions
- Spinal Muscular Atrophy, Muscular Dystrophy, Muscle Diseases, Myasthenic Syndromes
- Trial name
- NCT02124616
- Where
- Egypt
- Volunteers
- 200 people
- Sponsor
- Ain Shams University
Spinal Muscular Atrophy, Merosin Deficient Congenital Muscular Dystrophy
Fat and Glucose Metabolism in Fed and Fasted State in Patients With Low Skeletal Muscle Mass
In a study from 2003 the investigators showed that adult patients with very low skeletal muscle mass (spinal muscular atrophy (SMA) type II, Duchenne muscular dystrophy, congenital muscular dystrophy) are prone to develop…
- Trial name
- NCT03970135
- Testing
- Fasting
- Where
- Denmark
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
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