Clinical trials: Motor Neuron Disorder
Muscular Atrophy, Spinal
A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis
In this single center study blood samples for biomarker analysis will be collected from patients with spinal muscular atrophy. Up to 21 mL blood will be drawn from eligible patients at a single visit.
- Trial name
- NCT01910168
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Hoffmann-La Roche
SMA - Spinal Muscular Atrophy
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously received nusinersen.
- Trial name
- NCT06971094
- Testing
- GC101 adeno-associated virus injection
- Where
- 7 sites in China
- Volunteers
- 50 people
- Sponsor
- GeneCradle Inc
Motor Neuron Disease, Amyotrophic Lateral Sclerosis, ALS (Amyotrophic Lateral Sclerosis)
Music Therapy Protocol to Support Bulbar and Respiratory Functions in ALS
This study evaluates potential of music therapy treatment to support breathing, speech, swallow and cough of persons with amyotrophic lateral sclerosis (ALS).
- Conditions
- Motor Neuron Disease, Amyotrophic Lateral Sclerosis, ALS (Amyotrophic Lateral Sclerosis), Dysarthria, Flaccid, Dysarthria, Spastic
- Trial name
- NCT03604822
- Testing
- Music therapy
- Where
- 2 sites in Russia, United Kingdom
- Volunteers
- 8 people
- Sponsor
- Alisa Apreleva
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.
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
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 (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
Muscular Atrophy, Spinal
A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies
The aim of this study was to assess real-world dosing patterns, long-term healthcare costs, and characteristics of SMA patients who received treatment with DMTs. DMTs included onasemnogene abeparvovec, nusinersen, and risdiplam.
- Trial name
- NCT07378943
- Where
- United States
- Volunteers
- 4,114 people
- Sponsor
- Novartis Pharmaceuticals
Spinal Muscular Atrophy (SMA)
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor…
- Trial name
- NCT04174157
- Testing
- Prospective observational registry, Zolgensma
- Where
- 99 sites in United States, Greece and 9 more
- Volunteers
- 700 people
- Sponsor
- Novartis Pharmaceuticals
Muscular Atrophy, Spinal
A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy
The aim of this study was to evaluate real-world treatment patterns and clinical outcomes in pediatric SMA patients in the Czech and Slovak Republics, using data from the Registry of muscular Dystrophy (REaDY).
- Trial name
- NCT07474311
- Where
- Czechia
- Volunteers
- 213 people
- Sponsor
- Novartis Pharmaceuticals
Child With Cancer, Muscular Atrophy, Spinal
A BHI to Increase Hope Level and Stress Level of Parents With a CMC
Being a parent of a child with medical complexity (CMC) poses an enormous stress because these CMC have a multisystem disease, a severe neurologic condition or cancer which may result in premature death.
- Conditions
- Child With Cancer, Muscular Atrophy, Spinal, Child With Cerebral Palsy, Endocrine System Diseases
- Trial name
- NCT05269693
- Testing
- Brief Hope Intervention
- Where
- China
- Volunteers
- 40 people
- Sponsor
- The Hong Kong Polytechnic University
Motor Neurone Disease
Assessment of the Effects of Sheffield Support Snood in MND Patients
The aim of this study is to evaluate the effects on MND patients of a new collar specifically designed for people affected by neck weakness: the Sheffield Support Snood.
- Trial name
- NCT02464852
- Testing
- Head movements, Activities of daily living (drinking, washing hand and eating), Sheffield Support Snood
- Where
- United Kingdom
- Volunteers
- 15 people
- Sponsor
- Sheffield Teaching Hospitals NHS Foundation Trust
Spinal Muscular Atrophy
Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America
The natural history of SMA patients has changed, due to the improvements in treatment and technological advances. The systematic collection of data from routine clinical practice in multiple Latin American countries, harmonized…
- Trial name
- RegistrAME (NCT05475691)
- Where
- 18 sites in Argentina, Brazil and 4 more
- Volunteers
- 361 people
- Sponsor
- Hospital Israelita Albert Einstein
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
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.
- Trial name
- NCT06288230
- Testing
- vesemnogene lantuparvovec
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Lantu Biopharma
Muscular Atrophy, Spinal
A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy
The aim of this study was to evaluate treatment patterns, SMA-related complications and services, and all-cause medical encounters of patients with SMA receiving multiple DMTs in the real world.
- Trial name
- NCT07403214
- Where
- United States
- Volunteers
- 4,805 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis
Early Access Program for Ulefnersen in Children, Adolescents and Adults With FUS-associated Amyotrophic Lateral Sclerosis (FUS-ALS)
This expanded access program provides access to ulefnersen for eligible patients with FUS-associated amyotrophic lateral sclerosis (FUS-ALS) who meet protocol-defined eligibility criteria.
- Trial name
- NCT07862959
- Testing
- Ulefnersen
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Spinal Muscular Atrophy (SMA), Cerebral Palsy (CP)
Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders
Both GIP and GLP-2 reduce bone resorption (measured as CTX) in healthy adult individuals. In this study, we will investigate whether GIP and GLP-2 reduce CTX in children with spinal muscular atrophy, duchenne muscular dystrophy…
- Conditions
- Spinal Muscular Atrophy (SMA), Cerebral Palsy (CP), Duchenne Muscular Dystrophy (DMD)
- Trial name
- NCT07254988
- Testing
- Meal test, GIP + GLP-2
- Where
- Denmark
- Volunteers
- 3 people
- Sponsor
- University of Copenhagen
Muscle Tone Poor, Spinal Muscular Atrophy
Myotonometer Intra- and Inter Examiner Reliability on Spinal Muscular Atrophy
The primary aim of the study was to measure the intra-rater and inter-rater reliability of MyotonPRO in measuring postural muscle tone and mechanical properties in individuals with spinal muscular atrophy (SMA).
- Trial name
- NCT05521217
- Testing
- MyotonPRO
- Where
- 2 sites in Turkey (Türkiye)
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Istanbul Medipol University Hospital
Amyotrophic Lateral Sclerosis (ALS)
Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.
- Trial name
- NCT07743268
- Testing
- nL-TARDB-006
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- n-Lorem Foundation