Clinical trials: SMA
Spinal Muscular Atrophy (SMA)
Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development
Spinal muscular atrophy (SMA) is the leading genetic cause of death of infants. Strong preclinical evidence suggests that effective therapy must be delivered as early as possible to prevent progression of the disease.
- Trial name
- NCT01736553
- Where
- 15 sites in United States
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Ohio State University
Spinal Muscular Atrophy
A Registered Cohort Study on SMA
Spinal muscular atrophy (SMA) is an autosomal recessive disease that causes progressive muscle wasting and weakness due to loss of motor neurons in the spinal cord.
- Trial name
- NCT04010604
- Where
- China
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Wan-Jin Chen
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy
Wearable Technology to Assess Gait Function in SMA and DMD
The purpose of this project is to devise instrumented insoles capable of accurately measuring gait at each footfall, over multiple hours in any environment.
- Trial name
- NCT04193085
- Testing
- Observational
- Where
- United States
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Columbia University
Spinal Muscular Atrophy
SMN Circular RNAs as Potential Biomarkers for the Therapeutic Response to Nusinersen in Spinal Muscular Atrophy Patients
The first cure for Spinal Muscular Atrophy (SMA; Nusinersen) has been approved by FDA in 2017. Although it improves the clinical picture of most SMA patients, not all exhibit the same response to treatment.
- Trial name
- NCT05761262
- Where
- Italy
- Volunteers
- 91 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Spinal Muscular Atrophy (SMA)
An International Federated Model for Wearable-derived Remote Longitudinal Motor Monitoring in Young Children With Spinal Muscular Atrophy Compared With Healthy Controls: Active-NBS Study (UK)
Active-NBS is a study to evaluate the muscle development of patients with spinal muscular atrophy (SMA) who are diagnosed at birth. Medicines have become available in the last decade, and many patients are treated very early.
- Trial name
- Active-NBS UK (NCT07543003)
- Where
- United Kingdom
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- University of Oxford
Spinal Muscular Atrophy Type 3, Mitochondrial Myopathy
Oxidative Capacity and Exercise Tolerance in Ambulatory SMA
This proposal will focus on (1) estimating oxidative capacity of specific muscle groups during exercise using near infrared spectroscopy and (2) describing body composition to better understand exercise capacity and mitochondrial…
- Trial name
- NCT02895789
- Where
- United States
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Columbia University
Muscular Atrophy, Spinal, Child, Only
Procedural Sedation for Pediatric Patients With Spinal Muscular Atrophy
Background and Aim: Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by progressive symmetrical weakness and atrophy of proximal muscles causing from degeneration of anterior horn cells of spinal cord.
- Conditions
- Muscular Atrophy, Spinal, Child, Only, Anesthesia
- Trial name
- NCT05291962
- Testing
- Retrospective observational study
- Where
- Turkey (Türkiye)
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Ankara City Hospital Bilkent
Pelvic Organ Prolapse
Evaluation of Neuromuscular Morphometry of the Vaginal Wall
In this prospective study, women aged between 40 and 75 years who had not undergone any vaginal surgery and had not undergone any abdominal prolapse surgery were included.
- Trial name
- NCT06363838
- Where
- Turkey (Türkiye)
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Gaziosmanpasa Research and Education Hospital
Spinal and Bulbar Muscular Atrophy, Kennedys Disease
Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)
Background: SBMA is an inherited chronic disease. It affects males in mid to late adulthood. It causes slowly progressive weakness of muscles and hand tremors. Researchers want to learn more about the effects of SBMA.
- Conditions
- Spinal and Bulbar Muscular Atrophy, Kennedys Disease, Motor Neuron Disease
- Trial name
- NCT04944940
- Where
- United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Spinal Muscular Atrophy
Prenatal Carrier Screening for Spinal Muscular Atrophy Among Thai Pregnant Women
Spinal muscular atrophy (SMA) prenatal carrier screening is recommended by American College of Medical Genetics (ACMG) and American College of Obstetrics and Gynecology (ACOG).
- Trial name
- NCT04859179
- Where
- Thailand
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Mahidol University
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
TRIAL READY (Clinical Trial Readiness)
This study, being conducted under the auspice of the CReATe Consortium, will enroll patients with ALS and related disorders as well as healthy controls, with the goal of facilitating clinical validation of leading…
- Conditions
- Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, ALS-Frontotemporal Dementia, Primary Lateral Sclerosis
- Trial name
- NCT03912987
- Where
- 3 sites in United States
- Volunteers
- 610 people, healthy volunteers welcome
- Sponsor
- University of Miami
Spinal Muscular Atrophy
Spinal Muscular Atrophy and Cardiac Autonomic Function
Respiratory dysfunction is the major cause of morbidity and mortality in patients with spinal muscular atrophy (SMA). Air stacking is a clearance airway technique frequently used but its effects on cardiac autonomic function in…
- Trial name
- NCT01522079
- Testing
- Air stacking manuever
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Centro Universitário Augusto Motta
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
Muscular Dystrophies, Myopathies
Observational Study to Observe Variations of Gait Parameters in Patients With Neuromuscular Diseases
This study has the general objective of observing walking parameters during a clinical test to objectively estimate fatigue in patients with neuromuscular diseases.
- Conditions
- Muscular Dystrophies, Myopathies, Spinal Muscular Atrophy (SMA), Charcot Marie Tooth Disease
- Trial name
- NCT06666816
- Testing
- inapplicable
- Where
- 2 sites in Italy
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- IRCCS Eugenio Medea
Congenital Mirror Movements
Role of the SMA During Unimanual and Bimanual Movements Preparation: the Mirror Movements Paradigm
The aim of the project is to study the role of secondary motor area (more precisely the supplementary motor area, or SMA) during unimanual and bimanual voluntary movements externally (cue) or internally (subject's choice)…
- Trial name
- MOMIC2 (NCT02073604)
- Testing
- Healthy volunteers, Congenital mirror movements
- Where
- France
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Spinal Muscular Atrophy
Aerobic Training in Patients With Spinal Muscular Atrophy Type III
Spinal muscular atrophy type III, (SMAIII) is a disease in the nerve cells in the spinal cord which leads to to progressive muscle weakness and atrophy. No effective treatment is available for SMA.
- Trial name
- NCT02003937
- Testing
- Aerobic conditioning
- Where
- Denmark
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
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
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
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, 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