Clinical trials: Motor Disorders
- Condition Motor Disorders
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Emotion Management Skills, Lifestyle, Healthy
The Impact of Enhanced Music & Natural Visuals on Emotional Health
The investigators are conducting research on factors related to the self-regulation of mood and arousal states across a range of everyday activities as well as different levels of stress.
- Conditions
- Emotion Management Skills, Lifestyle, Healthy, Lifestyle Intervention, Recovery
- Trial name
- NCT07676110
- Testing
- soundBrilliance software application - Emotional Health
- Where
- 3 sites in United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- soundBrilliance LLC
Healthy Volunteer, Muscular Atrophy, Spinal
Ability of Muscle Imaging and Motor Function Measure (MFM) to Detect Changes in Disease Progression in Ambulant Spinal Muscular Atrophy Patients Compared to Healthy Volunteers.
This non-drug, single center, 24-week, longitudinal study in ambulant spinal muscular atrophy (SMA) patients and in age- and gender-matched healthy volunteers will assess the detection of disease progression by magnetic resonance…
- Trial name
- NCT02044029
- Where
- Switzerland
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Alzheimer Disease
Memesto Wearable Device for Persons With Dementia
An estimated 70% of the 7.2+ million people in the U.S. with Alzheimer's Disease and Alzheimer's Disease-Related Dementias experience agitation, characterized by poorly organized and purposeless psychomotor activity that…
- Trial name
- NCT05153161
- Testing
- Memesto
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Edgewater Safety Systems, Inc.
Spinal Muscular Atrophy
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
- Trial name
- SMNRx (NCT01494701)
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 28 people
- Sponsor
- Biogen
Muscular Atrophy, Spinal
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
This expanded access program (EAP) will provide access to risdiplam for eligible participants with Type 1 or Type 2 spinal muscular atrophy (SMA) before it is commercially available in the United States for the indication of SMA.
- Trial name
- NCT04256265
- Testing
- Risdiplam
- Where
- 29 sites in United States
- Sponsor
- Genentech, Inc.
Muscular Atrophy, Spinal
A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD)
In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA).
- Trial name
- ONWARD (NCT04729907)
- Testing
- Nusinersen
- Where
- 39 sites in United States, Brazil and 14 more
- Volunteers
- 115 people
- Sponsor
- Biogen
Spinal Muscular Atrophy Type I
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT05614531
- Testing
- EXG001-307 injection
- Where
- 2 sites in China
- Volunteers
- 12 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Spinal Muscular Atrophy Type I
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Trial name
- NCT07070999
- Testing
- GB221
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- Gemma Biotherapeutics
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved…
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- SAPPHIRE (NCT05156320)
- Testing
- Apitegromab
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 188 people
- Sponsor
- Scholar Rock, Inc.
Pulmonary Disease, Chronic Obstructive
The Study for Evaluating Factors Relating to Daily Step Counts and Physical Activity in Japanese Patients With COPD.
This study is conducted as a multicenter, non-interventional, cross-sectional study. Patients will be enrolled in the study by a continuous registration method after patients provide written informed consent.
- Trial name
- STEP-COPD (NCT03642613)
- Where
- Japan
- Volunteers
- 505 people
- Sponsor
- AstraZeneca
Amyotrophic Lateral Sclerosis
Individual Patient Expanded Access IND of Autologous HBadMSCs for the Treatment of Amyotrophic Lateral Sclerosis
The drug for this submission is Hope Biosciences' autologous, adipose-derived culture-expanded mesenchymal stem cells (HB-adMSCs) for the treatment of a single patient with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT04514952
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Mood, Depression Disorders
Technology Exposure and Child Health: Wellness Impact and Social Effects: An Observational Product Registry Study
The goal of this study is to understand how children's mobile device usage (smartphones or tablets), including social media use and online games, are related to their mental wellness and mental health, as well as some aspects of…
- Conditions
- Mood, Depression Disorders, Anxiety Disorders, Sleep
- Trial name
- TECHWISE (NCT06664944)
- Where
- United States
- Volunteers
- 912 people, healthy volunteers welcome
- Sponsor
- Aura
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult participants with ALS, FTD, or mixed ALS/FTD phenotype with a documented…
- Trial name
- NCT05683860
- Testing
- WVE-004 10 mg Q12W
- Where
- 3 sites in Netherlands, United Kingdom
- Volunteers
- 8 people
- Sponsor
- Wave Life Sciences USA, Inc.
Muscular Dystrophies, Spinal Muscular Atrophy
Schulze Muscular Dystrophy Ability Clinical Study
The primary objective of the Schulze study is to evaluate the function of the upper limbs of subjects diagnosed with neuromuscular disorders, with and without use of the Abilitech Assist device in the clinic and home…
- Conditions
- Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT05409079
- Testing
- Abilitech Assist
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- AbiliTech Medical Inc.
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
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
- 11 sites in United States, Germany and 2 more
- Volunteers
- 21 people
- Sponsor
- Hoffmann-La Roche
Tourette Syndrome
Safety, Tolerability, Pharmacokinetic, and Efficacy Study of AZD5213 in Adolescents With Tourette's Disorder
This is a two-part, randomized, multi-center, blinded study in adolescents with Tourette's Disorder. There will be an up to 21-day screening period in which subject eligibility will be determined.
- Trial name
- NCT01904773
- Where
- 7 sites in United States
- Volunteers
- 29 people
- Sponsor
- AstraZeneca
Spinal Muscular Atrophy Type 3
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.
- Trial name
- NCT06421831
- Testing
- GC101
- Where
- China
- Volunteers
- 21 people
- Sponsor
- GeneCradle Inc
Spinal Muscular Atrophy, SMA
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones…
- Conditions
- Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- OPAL (NCT07047144)
- Testing
- Apitegromab, Nusinersen, Risdiplam
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 52 people
- Sponsor
- Scholar Rock, Inc.
Type I Spinal Muscular Atrophy
Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy.
- Trial name
- NCT06576388
- Testing
- EXG001-307 injection
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd