Clinical trials: Neuromuscular Diseases in Children
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
Attention Deficit/Hyperactivity Disorder (ADHD)
The Efficacy and Safety of Phosphatidylserine-Omega3 in Children With Attention-Deficit/ Hyperactivity Disorder
The primary objective of this trial is to determine whether an oral administration of Phosphatidylserine-Omega3 would significantly improve the clinical symptoms of children suffering from ADHD.
- Trial name
- NCT00418184
- Testing
- Phosphatidylserine-Omega3, Colored cellulose tainted with fishy odor
- Where
- Israel
- Volunteers
- 200 people
- Sponsor
- Enzymotec
Diphtheria, Tetanus
Study of DAPTACEL® Administered as a 5th Dose in 4 to 6-Year-Old Children Previously Immunized With PENTACEL™
Objectives: To present the rates of adverse reactions after a dose of DAPTACEL® vaccine administered to children 4 to 6 years of age who have previously received four doses of PENTACEL™ vaccine.
- Conditions
- Diphtheria, Tetanus, Pertussis, Polio
- Trial name
- NCT00802867
- Testing
- Diphtheria and Tetanus Toxoids and Acellular Pertussis (DAPTACEL®)
- Where
- 19 sites in United States
- Volunteers
- 989 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
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
Cerebral Palsy
A Safety, Efficacy and Tolerability Study of Sativex for the Treatment of Spasticity in Children Aged 8 to 18 Years
A study to assess the effects of Sativex treatment on spasticity in a population of children and adolescents aged from 8 to 18 years with cerebral palsy or traumatic central nervous system injury.
- Trial name
- NCT01898520
- Testing
- Sativex
- Where
- 14 sites in Czechia, Israel and 1 more
- Volunteers
- 72 people
- Sponsor
- Jazz Pharmaceuticals
Congenital Myotonic Dystrophy
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).
- Trial name
- NCT03692312
- Testing
- Tideglusib
- Where
- 14 sites in United States, Australia and 3 more
- Volunteers
- 56 people
- Sponsor
- AMO Pharma Limited
Tetanus, Diphtheria
Study of DTap-IPV Compared to DAPTACEL® and IPOL® as the 5th Dose in Children 4 to 6 Years of Age
The study was designed to compare the safety and immunogenicity of DTap-IPV with DAPTACEL® + IPOL® as the 5th dose booster in children ≥ 4 to < 7 years of age in the US and Puerto Rico who were previously vaccinated with…
- Conditions
- Tetanus, Diphtheria, Pertussis, Measles
- Trial name
- NCT01346293
- Testing
- Diphtheria and Tetanus Toxoids and Acellular Pertussis + Measles, Mumps, Rubella + Varicella Virus, Diphtheria and Tetanus Toxoids and Acellular Pertussis + Poliovirus + MMR + Varicella Virus
- Where
- 72 sites in United States, Puerto Rico
- Volunteers
- 3,372 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Poliomyelitis
A Post-marketing Safety Study of GSK Bio IPV Vaccine (PoliorixTM) in Korean Children
Following licensing of PoliorixTM in Korea, this study will collect safety data about the routine use of this vaccine in 600 children according to the regulations of Korean Food and Drugs Administration (KFDA).
- Trial name
- NCT00514033
- Testing
- Poliomyelitis vaccine (inactivated) -PoliorixTM
- Where
- South Korea
- Volunteers
- 349 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Attention Deficit Hyperactivity Disorder
Concordance of the Quotient Attention Deficit Hyperactivity Disorder (ADHD) System Report With Teacher and Parent Assessments for Symptoms of ADHD
To demonstrate concordance between the Quotient ADHD System Report and standard DSM-IV based parent and teacher reported rating/assessment scales, currently the accepted method of symptom measurement for subjects diagnosed with…
- Trial name
- NCT01118650
- Sponsor
- BioBehavioral Diagnostics Company
Cerebral Palsy, Spasticity
Dose-response Study of Efficacy and Safety of Botulinum Toxin Type A to Treat Spasticity of the Arm(s) or of Arm(s) and Leg(s) in Cerebral Palsy
The purpose of this study is to determine whether injections of Botulinum toxin type A into muscles of one or both arms alone or in combination with injections into one or both legs are effective and safe in treating…
- Trial name
- XARA (NCT02002884)
- Testing
- IncobotulinumtoxinA (8 Units per kg body weight), IncobotulinumtoxinA (6 Units per kg body weight), IncobotulinumtoxinA (2 Units per kg body weight)
- Where
- 32 sites in United States, Argentina and 4 more
- Volunteers
- 351 people
- Sponsor
- Merz Pharmaceuticals GmbH
Meningococcal Meningitis, Tetanus
Study of Menactra® in Children Aged 4 to 6 Years When Administered Concomitantly With a Fifth Dose of DAPTACEL®
The purpose of this study is to evaluate the immunogenicity and safety of the concomitant administration of Menactra® vaccine and DAPTACEL® vaccine.
- Conditions
- Meningococcal Meningitis, Tetanus, Diphtheria, Pertussis
- Trial name
- NCT00355121
- Testing
- Polysaccharide Diphtheria Toxoid Conjugate Vaccine
- Where
- 26 sites in United States
- Volunteers
- 882 people, healthy volunteers welcome
- Sponsor
- Sanofi
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
Neurofibromatosis Type 1
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints.
- Trial name
- SPRINKLE (NCT05309668)
- Testing
- Selumetinib granule formulation, Selumetinib capsule formulation
- Where
- 16 sites in United States, Germany and 5 more
- Volunteers
- 36 people
- Sponsor
- AstraZeneca
Diphtheria, Tetanus
REVAXIS® Versus DT Polio® as a Second Booster in 6 Year-old Children
Primary objective: To demonstrate the non inferiority between REVAXIS® and DT Polio® when given as a second booster to healthy 6 year-old children . Secondary objectives: Additional immunogenicity assessments.
- Conditions
- Diphtheria, Tetanus, Poliomyelitis
- Trial name
- NCT00447525
- Testing
- REVAXIS®, DT-Polio®
- Where
- 64 sites in France
- Volunteers
- 760 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Poliomyelitis
Safety of Imovax Polio in Chinese Infants and Children
To describe the tolerance in terms of occurrence of serious adverse reactions and severe adverse reactions (injection site and systemic) within eight days after one dose of IMOVAX Polio™ administered in children and infants.
- Trial name
- NCT00258843
- Testing
- Inactivated Poliomyelitis vaccine
- Where
- China
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Traumatic Brain Injury
Pilot, Proof-of-Concept Study of Sublingual Tizanidine in Children With Chronic Traumatic Brain Injury (TBI)
Nightly administration of a unique, sublingual (under the tongue) formulation of tizanidine, a known anti-spasticity medication, has been shown in a previous study to improve sleep and next-day functioning in CP (cerebral palsy)…
- Trial name
- NCT00287157
- Testing
- Sublingual Tizanidine HCl
- Where
- Israel
- Volunteers
- 5 people
- Sponsor
- Teva GTC
Duchenne Muscular Dystrophy
A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
Study to characterize the single-state and steady-state dosing of oral deflazacort in pediatric and adolescents subjects.
- Trial name
- NCT02251600
- Testing
- Deflazacort
- Where
- 4 sites in United States
- Volunteers
- 24 people
- Sponsor
- PTC Therapeutics
Hepatitis B, Acellular Pertussis
Pneumococcal Vaccine Booster Study in Healthy Children 11-18 Months Old Previously Primed With the Same Vaccines
The purpose of this study is to assess the safety in terms of fever (rectal temperature) higher than 39 degree Celcius (°C) and the immunogenicity in terms of antibody response following a booster vaccination with pneumococcal…
- Conditions
- Hepatitis B, Acellular Pertussis, Tetanus, Poliomyelitis
- Trial name
- NCT00463437
- Testing
- Pneumococcal conjugate vaccine GSK1024850A, Prevenar, Infanrix hexa
- Where
- 63 sites in Germany, Poland and 1 more
- Volunteers
- 1,437 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Varicella, Measles
Immunogenicity and Safety Study of Proquad® and Infanrix® Hexa When Administered Concomitantly (V221-035)
Primary Objective: To demonstrate that ProQuad® can be administered concomitantly with a booster dose of Infanrix® hexa to healthy children 12 to 23 months of age without impairing either the antibody response rates to measles…
- Conditions
- Varicella, Measles, Mumps, Rubella
- Trial name
- NCT00432042
- Testing
- ProQuad®, Infanrix® hexa
- Where
- 50 sites in Germany, Italy
- Volunteers
- 955 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Duchenne Muscular Dystrophy
A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an…
- Trial name
- NCT05540860
- Testing
- Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3
- Where
- 14 sites in United States
- Volunteers
- 76 people
- Sponsor
- Edgewise Therapeutics, Inc.