Clinical trials: Muscle Wasting
Neuromuscular Disorders, Neurodevelopmental Disorders
Efficacy and Usability of the EXPLORER Exoskeleton in Children With Neurodevelopmental Disorders in Natural Environments
Neurodevelopmental disorders often lead to abnormal development of the Central Nervous System (CNS), frequently causing motor dysfunctions such as an inability to stand and walk.
- Trial name
- NCT06765070
- Testing
- EXPLORER
- Where
- 6 sites in Spain
- Volunteers
- 15 people
- Sponsor
- MarsiBionics
Muscle Loss
A Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Study of ACE-031 (ActRIIB-IgG1)in Healthy Postmenopausal Volunteers
Single center, randomized, single dose study to evaluate the safety, tolerability, pharmacokinetic and pharmacodynamic effects of ACE-031 in healthy postmenopausal volunteers
- Trial name
- NCT00755638
- Testing
- ACE-031
- Where
- Canada
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
SMA II
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 2 (SMA 2) patients.
- Trial name
- NCT05901987
- Testing
- GC101
- Where
- 5 sites in China
- Volunteers
- 33 people
- Sponsor
- GeneCradle Inc
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients
This is a phase 1/2a randomized, double-blind study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of study drug AJ201 in subjects with Spinal and Bulbar Muscular Atrophy (SBMA).
- Trial name
- NCT05517603
- Testing
- AJ201
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- AnnJi Pharmaceutical Co., Ltd.
Cerebral Palsy, SMA II
Evaluation of the Exoskeleton ATLAS 2030 as Robot-assisted Physical Therapy to Children With Neuromuscular Diseases
This study aims to evaluate the safety, usability and acceptability of a motorized mobility assistance exoskeleton. The protocol explores the use of the motorized device during static and dynamic rehabilitation sessions focused…
- Conditions
- Cerebral Palsy, SMA II, Neuromuscular Diseases
- Trial name
- NCT04837157
- Testing
- Exoskeleton rehabilitation
- Where
- Spain
- Volunteers
- 6 people
- Sponsor
- MarsiBionics
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III Non Ambulant
Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.
Assess the efficacy and the safety of olesoxime in SMA type 2 or type 3 non ambulant patients aged 3-25 years
- Trial name
- NCT01302600
- Testing
- Olesoxime
- Where
- 22 sites in Belgium, France and 5 more
- Volunteers
- 165 people
- Sponsor
- Hoffmann-La Roche
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Spinal Muscular Atrophy (SMA)
A Study of the Safety and Preliminary Efficacy of NKG001 in Pediatric Patients With Type 1 or Type 2 Spinal Muscular Atrophy
This study is a single-center, open-label, single-arm, non-randomized, single-dose, dose-escalation investigator-initiated trial (IIT) designed to evaluate the safety, tolerability, and preliminary efficacy of NKG001 Injection…
- Trial name
- NCT07617779
- Testing
- NKG001
- Where
- China
- Volunteers
- 21 people
- Sponsor
- Nikegen Pharmaceutical (Hangzhou) Company Limited
Spinal Muscular Atrophy (SMA)
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
The goal of this clinical trial is to learn about the safety, and tolerability of giving an experimental gene therapy called SPX-01 to an infant with spinal muscular atrophy (SMA) type 1.
- Trial name
- NCT07903376
- Testing
- SPX-01
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Exir Gene Pharmed
Spinal Muscular Atrophy
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2
- Trial name
- SPR1NT (NCT03505099)
- Testing
- onasemnogene abeparvovec-xioi
- Where
- 16 sites in United States, Australia and 4 more
- Volunteers
- 30 people
- Sponsor
- Novartis Gene Therapies
Muscular Atrophy, Spinal
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have been diagnosed with SMA through…
- Trial name
- STELLAR-1 (NCT07221669)
- Testing
- Salanersen
- Where
- 14 sites in United States, Australia and 5 more
- Volunteers
- 30 people
- Sponsor
- Biogen
Spinal Muscular Atrophy Type I
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
This is a Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular atrophy (SMA) Type 1 and who are genetically defined by a biallelic…
- Trial name
- NCT03837184
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 4 sites in Japan, South Korea and 1 more
- Volunteers
- 2 people
- Sponsor
- Novartis Gene Therapies
Muscular Atrophy, Spinal
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).
- Trial name
- Rainbowfish (NCT03779334)
- Testing
- Risdiplam
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 26 people
- Sponsor
- Hoffmann-La Roche
Muscular Atrophy, Spinal
A Study of the Safety of Nusinersen (Spinraza®) and Its Effects on Motor Function in Koreans Living With SMA Treated During Their Usual Medical Care
In this study, researchers will learn more about the effects of nusinersen, also known as Spinraza®, in Korean participants with spinal muscular atrophy, also known as SMA.
- Trial name
- STANDARD (NCT04317794)
- Testing
- Nusinersen Sodium Injection
- Where
- 16 sites in South Korea
- Volunteers
- 74 people
- Sponsor
- Biogen
Malnutrition, Calorie, Sarcopenia
Malnutrition Screening Study
This is a non-interventional, cross-sectional, non-randomized, observational study to screen for the risk of malnutrition and sarcopenia in outpatients aged 18 and over who attend hospital clinics for any reason in Turkey.
- Trial name
- NCT07256925
- Where
- 69 sites in Turkey (Türkiye)
- Volunteers
- 7,500 people
- Sponsor
- Abbott Nutrition
Muscular Atrophy, Spinal
Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients
A two-period, two-treatment, crossover study to evaluate the safety, tolerability and efficacy of amifampridine phosphate in ambulatory patients diagnosed with spinal muscular atrophy (SMA) Type 3.
- Trial name
- NCT03781479
- Testing
- Amifampridine Phosphate
- Where
- Italy
- Volunteers
- 13 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Sarcopenia, Body Composition
Validation of a Body-Composition Segmentation Software on a Diverse Public CT Scan Cohort
This study evaluates the standalone performance of Soma, a deep-learning software developed by Nucleo Research, Inc. for the automated segmentation of body-composition tissues (skeletal muscle, subcutaneous adipose tissue…
- Conditions
- Sarcopenia, Body Composition, Obesity
- Trial name
- SOMA (NCT07600866)
- Testing
- Soma Body-Composition Segmentation Software
- Where
- United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Nucleo Research, Inc.
Spinal Muscular Atrophy
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)
The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS…
- Trial name
- NCT01780246
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- Biogen
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
Motor Neuron Disease
A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.
The COMMEND Study will assess the safety and effectiveness of FLX-787 in men and women with Motor Neuron Disease [including Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS) or Progressive Muscular Atrophy…
- Trial name
- NCT03196375
- Testing
- FLX-787-ODT (orally disintegrating tablet)
- Where
- 35 sites in United States
- Volunteers
- 54 people
- Sponsor
- Flex Pharma, Inc.