Clinical trials: Spinal Muscular Atrophy
Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis
European Home Mechanical Ventilation Registry
The European Home Mechanical Ventilation Registry (EHMVR) will enable a thorough evaluation of HMV by documenting the characteristics of HMV patients and their treatment.
- Conditions
- Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis, Spinal Cord Injury, Muscular Dystrophies
- Trial name
- EHMVR (NCT02315339)
- Where
- Germany
- Volunteers
- 37 people
- Sponsor
- ResMed
Spinal Muscular Atrophy
Metabolomics of Children With SMA
The aim of the proposed project is to evaluate whether the metabolome of patients with spinal muscular atrophy (SMA) before the initiation of treatment with nusinersen differs from the metabolome of healthy individuals and…
- Trial name
- NCT04587492
- Testing
- Nusinersen
- Where
- 2 sites in Slovenia
- Volunteers
- 35 people
- Sponsor
- University Medical Centre Ljubljana
Prostate Cancer
An Open Label Prostate Cancer Study in Japanese Patients
The primary aim of study is to gain an initial assessment of safety and tolerability of AZD3514 in Japanese patients together with assessing Pharmacokinetics (PK) and gaining a preliminary assessment of anti-tumour action.
- Trial name
- NCT01351688
- Testing
- AZD3514
- Where
- 2 sites in Japan
- Volunteers
- 13 people
- Sponsor
- AstraZeneca
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
Postpolio Syndrome
Whole Body Vibration on Muscle Strength in Patients With Postpolio Syndrome
Post-polio syndrome (PPS) is a neurological disease that affects polio survivors at least 15 years after the initial polio infection.
- Trial name
- NCT04387864
- Testing
- Whole-body Vibration Device
- Where
- Turkey (Türkiye)
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Koç University
Neuromuscular Disorders, Neurodevelopmental Disorders
Safety, Usability, and Effectiveness of a Gait Exoskeleton for Children and Adolescents With Neurodevelopmental Disorders.
Neurodevelopmental disorders often result in abnormal development of the Central Nervous System (CNS), frequently causing motor dysfunctions such as the inability to stand and walk.
- Trial name
- NCT07159360
- Testing
- CLINICAL EXPLORER
- Where
- 2 sites in Mexico, Spain
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- MarsiBionics
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
Quality of Life and Participation of the Adult with Spinal Muscular Atrophy in France
Spinal muscular atrophy is a hereditary motorneuron disease caused by a mutation of the SMN1 gene, which is at the origin of a progressive limb and axial motor deficiency.
- Trial name
- QOLSMA (NCT05366465)
- Testing
- SMA adult patients
- Where
- 2 sites in France
- Volunteers
- 149 people
- Sponsor
- Hospices Civils de Lyon
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
French Register of Patients With Spinal Muscular Atrophy
The primary objectives of the study are to obtain clinically meaningful data on survival and outcomes of all the patients with spinal muscular atrophy (SMA) 5q types 1 through 4 (according to international classification), being…
- Trial name
- SMA (NCT04177134)
- Where
- France
- Volunteers
- 1,000 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Spinal Muscular Atrophy
A Registry of Cases of Spinal Muscular Atrophy
Because no long-term record of prenatal Spinal Muscular Atrophy (SMA) treatment exists, this study will address an important gap in the understanding of the safety and benefit of treatment while in the womb.
- Trial name
- NCT07805278
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- St. Jude Children's Research Hospital
Spinal Muscular Atrophy
Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States
This global, retrospective, non-interventional, medical chart review (MCR), descriptive study collected patient-level data in regions outside the US.
- Trial name
- NCT06178393
- Where
- United States
- Volunteers
- 162 people
- Sponsor
- Novartis Pharmaceuticals
Non Invasive Prenatal Diagnosis
NIPD on CFTC for Triplet Repeat Diseases
The purpose of this study is to develop and validate an analytical and clinical NIPD test for triplet repeat diseases by isolated circulating fetal trophoblastic cells (CFTC) analysis from maternal blood, searching for the…
- Trial name
- DIACCIMEX (NCT03087526)
- Testing
- Non invasive prenatal diagnosis
- Where
- 8 sites in France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
Spinal Muscular Atrophy (SMA)
Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)
The goal of this multicenter, open-label, non-comparative, cohort study is to investigate the safety, immunogenicity, and efficacy of ANB-004 in children with spinal muscular atrophy.
- Trial name
- BLUEBELL (NCT05747261)
- Testing
- ANB-004, dose 1, ANB-004, dose 2, ANB-004, dose 3
- Where
- 7 sites in Belarus, Russia
- Volunteers
- 40 people
- Sponsor
- Biocad
Libido, Receptors, Androgen
Genetic Polymorphism of the Androgen Receptor-Gene and Sexual Function in Middle Aged Women
It is known that with increasing age sexual desire is declining in women. Decreasing levels of androgens are believed to have an influence, but cannot explain the loss of libido completely.
- Conditions
- Libido, Receptors, Androgen, Polymorphism, Genetic, Androgen Effect
- Trial name
- CAG-Libido (NCT01977313)
- Where
- Switzerland
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Insel Gruppe AG, University Hospital Bern
Anarthria, Dysarthria
Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia
The purpose of this study is to obtain preliminary device safety information and demonstrate proof of principle (feasibility) of the ability of people with tetraplegia to control a computer cursor and other assistive devices with…
- Conditions
- Anarthria, Dysarthria, Tetraplegia, Spinal Cord Injuries
- Trial name
- BG-Speech-01 (NCT05724173)
- Testing
- BrainGate Neural Interface System
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Leigh R. Hochberg, MD, PhD.
Tetraplegia, Tetraplegia/Tetraparesis
[BrainConnexion] - Neurodevice Phase I Trial
This study aims to evaluate the safety of a wireless implantable neurodevice microsystem in tetraplegic patients, as well as the efficacy of the electrodes for long-term recording of neural activities and the successful control…
- Conditions
- Tetraplegia, Tetraplegia/Tetraparesis, Spinal Cord Injuries, Amyotrophic Lateral Sclerosis
- Trial name
- NCT03811301
- Testing
- BrainConnexion
- Where
- Singapore
- Volunteers
- 5 people
- Sponsor
- National Neuroscience Institute
Muscular Atrophy, Spinal
Two Different Treatment Modalities in Patients With Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA) is a severe neuromuscular disorder characterized by the degeneration of alpha motor neurons in the spinal cord, resulting in progressive muscle atrophy and weakness, particularly in proximal and…
- Trial name
- NCT06178653
- Testing
- Pulmonary Rehabilitation, Trunk Control Training
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Istanbul Medipol University Hospital
Neurologic Diseases, Hemiparesis
NuroSleeve Powered Brace & Stimulation System to Restore Arm Function
The purpose of this study is to investigate if a person with weakness or paralysis in one or both arms, can use the NuroSleeve combined powered arm brace (orthosis) and muscle stimulation system to help restore movement in one…
- Conditions
- Neurologic Diseases, Hemiparesis, Quadriplegia, Muscular Dystrophies
- Trial name
- NCT04798378
- Testing
- Neurosleeve
- Where
- 2 sites in United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Thomas Jefferson University
Spinal Muscular Atrophy
Spinal Muscular Atrophy on Wheels, Using Power Mobility
The group of children diagnosed with Spinal Muscular Atrophy (SMA) has serious restrictions on participation. SMA is a neuromuscular disease that leads to neuromusculoskeletal disorders that limit functional activities, sometimes…
- Trial name
- SMAPmob (NCT05589987)
- Testing
- power mobility
- Where
- 2 sites in Spain
- Volunteers
- 20 people
- Sponsor
- University of Castilla-La Mancha