Clinical trials: Neuromuscular Diseases in Children
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
Constipation, Childhood Acute Lymphoblastic Leukemia
Comparison of Gastrointestinal Motility in Healthy Children and Children With Constipation
To gain a better understanding of the underlying dysmotility in constipation in children, we will use a novel minimal invasive method called 3D Transit.
- Conditions
- Constipation, Childhood Acute Lymphoblastic Leukemia, Neurofibromatosis 1, Chronic Constipation With Overflow
- Trial name
- NCT03981510
- Testing
- 3D-Transit
- Where
- Denmark
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- University of Aarhus
X-Linked Myotubular Myopathy
A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no therapies for this serious condition.
- Trial name
- ASPIRO (NCT03199469)
- Testing
- Resamirigene bilparvovec
- Where
- 6 sites in United States, Canada and 2 more
- Volunteers
- 27 people
- Sponsor
- Astellas Gene Therapies
Scoliosis; Idiopathic, Infantile, Scoliosis; Juvenile
Physical Activity, Motor Competence, Pulmonary Function, and Quality of Life in Children With Severe Spinal Disease
Observation study about physical activity, motor competence, pulmonary function, and health related quality of life in children, surgically treated for early onset scoliosis
- Conditions
- Scoliosis; Idiopathic, Infantile, Scoliosis; Juvenile, Scoliosis Neuromuscular, Scoliosis in Neurofibromatosis
- Trial name
- NCT03494829
- Where
- Norway
- Volunteers
- 50 people
- Sponsor
- Oslo University Hospital
Neurofibromatosis Type 1
Effects of Physical Training on Bone and Muscle Quality, Muscle Strength, and Motor Coordination in Children With NF1
A physical training program will improve quality of life, participation in physical activity, motor coordination, muscle strength, and bone and muscle strength in children with neurofibromatosis type 1.
- Trial name
- NCT01058330
- Testing
- Plyometric training program
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Shriners Hospitals for Children
Cancer
Study to Investigate Safety, Pharmacokinetic (PK), Pharmacodynamic (PD) and Clinical Activity of Trametinib in Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Subjects With Cancers Harboring V600 Mutations
This was a 4-part (Part A, Part B, Part C and Part D), Phase I/IIa, multi-center, open label, study in pediatric subjects with refractory or recurrent tumors.
- Trial name
- NCT02124772
- Testing
- Trametinib, Dabrafenib
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 139 people
- Sponsor
- Novartis Pharmaceuticals
Duchenne Dystrophy Muscular
Biomechanical and Morphological Changes in Dystrophic Muscle
The loss of ability to walk in many children with DMD (Duchenne muscular Dystrophy) is a pejorative event. Biomechanical and morphological unknowledge about the loss of the walk ability in children with DMD is an obstacle in…
- Trial name
- MARCHE-DMD (NCT02472990)
- Where
- 10 sites in France
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University Hospital, Brest
Acellular Pertussis, Diphtheria
Post-marketing Surveillance to Assess the Safety of Infanrix-IPV Vaccine Among Infants and Children in Korea
The purpose of this study is to collect safety information following routine vaccination with Infanrix-IPV among infants and children in Korea.
- Conditions
- Acellular Pertussis, Diphtheria, Tetanus, Poliomyelitis
- Trial name
- NCT01568060
- Testing
- Infanrix-IPV data collection
- Where
- South Korea
- Volunteers
- 645 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Duchenne or Becker Muscular Dystrophy
Effectiveness of Aquatic Therapy in Children With Duchenne and Becker Muscular Dystrophy
The purpose of this study is ; to evaluate the effects of aquatic therapy applied in addition to conventional physical therapy on balance, functionality and quality of life in children with Duchenne and Becker muscular dystrophy.
- Trial name
- NCT06186310
- Testing
- Aquatic Therapy Group, Conventional Physical Therapy Group
- Where
- Turkey (Türkiye)
- Volunteers
- 16 people
- Sponsor
- Gaziosmanpasa Research and Education Hospital
Cerebral Palsy (CP)
Constraint-induced Movement Therapy Combined With Functional Electrical Stimulation in Children With Cerebral Palsy
This randomized controlled trial aims to compare the effectiveness of Constraint-Induced Movement Therapy (CIMT) combined with Functional Electrical Stimulation (FES) versus CIMT alone in improving upper limb function, reducing…
- Trial name
- CIMT-FES-CP (NCT07587476)
- Testing
- Constraint-Induced Movement Therapy Combined with Functional Electrical Stimulation, Constraint-Induced Movement Therapy
- Where
- Pakistan
- Volunteers
- 46 people
- Sponsor
- Montiha Azeem
Scoliosis Correction, Iron Deficiency
IntraVenous Iron in Kids With Iron Deficiency and Scoliosis Study
Adolescents and young adults undergoing spinal fusion surgery for the correction of scoliosis and other spinal deformity are at high risk of perioperative iron deficiency and anemia, yet the means and evidence for optimizing iron…
- Conditions
- Scoliosis Correction, Iron Deficiency, Transfusion Blood, Surgery Complications
- Trial name
- IV-KIDS (NCT07217873)
- Testing
- Ferric carboxymaltose IV
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- Columbia University
Orofacial Myofunctional Disorders
The Effect of Orofacial Myofunctional Therapy on Neuromuscular Outcomes and Orofacial Function in Children and Adolescents in Estonia
The aim of the present study is to evaluate the effects of semi-standardized myofunctional therapy on the functional and neuromuscular characteristics of the orofacial system in children and adolescents aged 7-14 years using a…
- Trial name
- NCT07828769
- Testing
- Semi-standardized Orofacial Myofunctional Therapy
- Where
- Estonia
- Volunteers
- 80 people
- Sponsor
- Tartu University Hospital
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
Becker Muscular Dystrophy, Muscular Dystrophies
Defining Endpoints in Becker Muscular Dystrophy
This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)
- Conditions
- Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
- Trial name
- GRASP-01-002 (NCT05257473)
- Where
- 11 sites in United States, New Zealand and 1 more
- Volunteers
- 80 people
- Sponsor
- Virginia Commonwealth University
Cerebral Palsy, Spasticity
Dutch National ITB Study in Children With Cerebral Palsy
The objective of this study is to determine the efficacy and safety of treatment with intrathecal baclofen for severe spasticity in children with cerebral palsy.
- Trial name
- NCT00367068
- Testing
- baclofen, intrathecal, infusion pump for intrathecal baclofen, Synchromed, Medtronic
- Where
- Netherlands
- Volunteers
- 18 people
- Sponsor
- Maastricht University
Spinal Muscular Atrophy
Spinal Muscular Atrophy and School Transitions
A qualitative questionnaire-based study to explore the experiences of children and young people with Spinal Muscular Atrophy (SMA) and their families regarding school transition and educational participation.
- Trial name
- NCT07720518
- Where
- United Kingdom
- Volunteers
- 20 people
- Sponsor
- Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust
Metabolic Disease, Mitochondrial Diseases
Genetic Diagnosis in Inborn Errors of Metabolism
Inborn Errors of metabolism comprise a large number of rare conditions with a collective incidence of around 1/2000 newborns. Many disorders are treatable provided that a correct diagnosis can be established in time, and for many…
- Conditions
- Metabolic Disease, Mitochondrial Diseases, Epilepsy in Children, Epilepsy
- Trial name
- NCT06376279
- Testing
- IEM-EP
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Region Stockholm
Duchenne Muscular Dystrophy (DMD)
A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD
The primary objective of Parts 1 and 2 of the study were to establish the histologic effects of givinostat administered chronically at the selected daily dose.
- Trial name
- NCT01761292
- Testing
- Givinostat
- Where
- 4 sites in Italy
- Volunteers
- 20 people
- Sponsor
- Italfarmaco
Duchenne Muscular Dystrophy
Aerobic Exercise in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is the most common neuromuscular disease in childhood with an estimate incidence of 1 in 3500 to 5000 male births.
- Trial name
- NCT04173234
- Testing
- Aerobic Training, Home exercise program
- Where
- Turkey (Türkiye)
- Volunteers
- 19 people
- Sponsor
- Hacettepe University
Poliomyelitis
Study to Evaluate the Safety of Co-administration of GSK Biologicals' Poliorix and Infanrix in Healthy Children in Russia
The purpose of this study is to assess reactogenicity and safety of simultaneous administration of GSK Biologicals' inactivated poliomyelitis vaccine Poliorix and GSK Biologicals' DTPa-vaccine Infanrix vaccines in healthy…
- Trial name
- NCT01094171
- Testing
- Poliorix TM, Infanrix TM
- Where
- 2 sites in Russia
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline