Clinical trials: Intellectual Disabilities
Cerebral Palsy, Caregiver Burden
Awareness Levels of Caregivers of Disabled Children
Disability-or apology; It is the inability or incompleteness of individuals to fulfill their roles in life, such as age, gender, culture, social and psychological factors, due to their inadequacies.
- Conditions
- Cerebral Palsy, Caregiver Burden, Disabilities Multiple, Muscular Dystrophy
- Trial name
- caregivers (NCT05228405)
- Testing
- All of the disabled children's caregivers will answer the Questionnaire of Disease, Physiotherapy and Sports Awareness
- Where
- Turkey (Türkiye)
- Volunteers
- 56 people
- Sponsor
- Kahramanmaras Sutcu Imam University
Asperger's Disorder, Mental Retardation
Cognitive Neuroscience of Autism Spectrum Disorders
Background: Autism spectrum disorders (ASDs) are a group of developmental disorders that affect communication, social interaction, and behavior.
- Conditions
- Asperger's Disorder, Mental Retardation, Children, Adult
- Trial name
- NCT01031407
- Where
- United States
- Volunteers
- 678 people
- Sponsor
- National Institute of Mental Health (NIMH)
Balance, Down Syndrome
Pilates Exercises and Down Syndrome
Down syndrome is one of the genetic disorders that affect postural control and balance in children. Balance involves controlling the position of the body in space to achieve stability and orientation.
- Conditions
- Balance, Down Syndrome, Pilates Exercises
- Trial name
- NCT05928949
- Testing
- pilates exercises
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Cairo University
Neurodevelopmental Disorders, Autism
Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders
There are currently no approved medications for the treatment of anxiety in children and youth with neurodevelopmental disorders (NDDs), both common and rare.
- Conditions
- Neurodevelopmental Disorders, Autism, Autism Spectrum Disorder, Fragile X Syndrome
- Trial name
- CALM (NCT06081348)
- Testing
- Sertraline
- Where
- 7 sites in Canada
- Volunteers
- 130 people
- Sponsor
- Holland Bloorview Kids Rehabilitation Hospital
Mucopolysaccharidosis I, Mucopolysaccharidosis II
Registry of Patients Diagnosed With Lysosomal Storage Diseases
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of…
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
- Trial name
- LSD Registry (NCT05619900)
- Testing
- There is no intervention
- Where
- United States
- Volunteers
- 250 people
- Sponsor
- University of California, San Francisco
Pyruvate Dehydrogenase Complex Deficiency
Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency
This is a medical research study to test a medication in patients with a disease called Pyruvate Dehydrogenase Complex (PDC) Deficiency.
- Trial name
- NCT06340685
- Testing
- Triheptanoin
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Jirair Krikor Bedoyan
Down Syndrome
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (HCl) (Aricept) In Treating Cognitive Dysfunction Exhibited By Children With Down Syndrome
The purpose of this study is to determine whether donepezil HCl is effective and safe in improving cognitive dysfunction exhibited by children and adolescents with Down syndrome (DS).
- Trial name
- NCT00570128
- Testing
- Donepezil HCl
- Where
- 31 sites in United States
- Volunteers
- 129 people
- Sponsor
- Eisai Inc.
Rett Syndrome
Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms
This study evaluates the safety, tolerability and efficacy of Sarizotan in reducing respiratory abnormalities in Rett Syndrome in an initial double blind 24 week period followed by an open label treatment phase of up to 168 weeks…
- Trial name
- STARS (NCT02790034)
- Testing
- Sarizotan low dose, Sarizotan high dose
- Where
- 14 sites in United States, Australia and 3 more
- Volunteers
- 129 people
- Sponsor
- Newron Pharmaceuticals SPA
Subglottic Stenosis (SGS), Pierre Robin Sequence (PRS)
Functional Modeling of the Pediatric Airway
The investigators hypothesize that a functional computational model that simulates the mechanical and aerodynamic behavior of the upper airway in children with Pierre Robin Sequence (PRS) and laryngeal lesions (e.g.
- Conditions
- Subglottic Stenosis (SGS), Pierre Robin Sequence (PRS), Micrognathia, Normal Controls From CT Scans of the Upper Airway
- Trial name
- NCT01690078
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- University of North Carolina, Chapel Hill
Hepato-splenomegaly, Dysostosis Multiplex
Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)
Development of a new MS-based biomarker for the ear-ly and sensitive diagnosis of GM1/GM2 from blood
- Conditions
- Hepato-splenomegaly, Dysostosis Multiplex, Seizures, Mental Retardation
- Trial name
- BioGM1/BioGM2 (NCT02298647)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Danon Disease
Natural History of Danon Disease
This is a multicenter, international, non-interventional, natural history study designed to collect longitudinal retrospective clinical information on patients with Danon disease (DD).
- Trial name
- NCT05548855
- Testing
- Heart Transplant, Cardiac Assistive Device
- Where
- 15 sites in Czechia, France and 3 more
- Volunteers
- 59 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Obstructive Sleep Apnea, Down Syndrome
Medications for Obstructive Sleep Apnea In Children With Down Syndrome
This is a randomized, double blind, cross-over study of the combination of atomoxetine and oxybutynin (ato-oxy) in children with DS and OSA documented by polysomnography (PSG).
- Trial name
- MOSAIC (NCT04115878)
- Testing
- Atomoxetine and oxybutynin (ato-oxy)
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- University of Arizona
Adrenoleukodystrophy, Metachromatic Leukodystrophy
HSCT for High Risk Inherited Inborn Errors
Hematopoietic stem cell transplantation has proven effective therapy for individuals with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease).
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease
- Trial name
- NCT00383448
- Testing
- Clofarabine, Total body Irradiation, Melphalan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Fragile X Syndrome
Biomarker and DNA Collection in Subjects Participating in Protocol 22003
The subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22003 will be offered participation in 22003A which will evaluate secreted protein before and after treatment with STX209 and…
- Trial name
- NCT00892580
- Testing
- STX209
- Where
- 8 sites in United States
- Volunteers
- 32 people
- Sponsor
- Seaside Therapeutics, Inc.
Down Syndrome
DS-Connect®: The Down Syndrome Registry
The goal of this study is to develop a registry for Down syndrome (DS) to facilitate research participation by individuals with Down syndrome.
- Trial name
- NCT01950624
- Where
- United States
- Volunteers
- 100,000 people
- Sponsor
- University of Colorado, Denver
Prader-Willi Syndrome
Targeting the Gut Microbiome for Prader-Willi Syndrome Treatment
The gut microbiome has recently emerged as a major contributor to obesity, systemic inflammation, and metabolic disease. Furthermore, intestinal bacteria are crucial players in the gut-brain axis, regulating a broad range of…
- Trial name
- BALPWS (NCT03548480)
- Testing
- Probiotic
- Where
- Spain
- Volunteers
- 39 people
- Sponsor
- Fundació Sant Joan de Déu
Phelan-McDermid Syndrome, Autism Spectrum Disorder
Mapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome
The purpose of this study is to comprehensively characterize PMS using standardized medical, cognitive, and behavioral measures and to track the natural history of the syndrome using repeated longitudinal assessments.
- Conditions
- Phelan-McDermid Syndrome, Autism Spectrum Disorder, Intellectual Disability
- Trial name
- NCT02461420
- Where
- 5 sites in United States
- Volunteers
- 207 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Down Syndrome
A Study of RG1662 in Adults and Adolescents With Down Syndrome (CLEMATIS)
This multi-center, randomized, double-blind, 3-arm, parallel-group, placebo-controlled study will evaluate the efficacy and safety of RG1662 in adults and adolescents with Down syndrome.
- Trial name
- NCT02024789
- Testing
- RG1662
- Where
- 37 sites in United States, Argentina and 8 more
- Volunteers
- 173 people
- Sponsor
- Hoffmann-La Roche
Neurodevelopmental Disorders, Autism Spectrum Disorder
Patterns of Neurodevelopmental Disorders
The purpose of this study is to systematically evaluate the results of medical investigations to identify symptom and biological patterns and common etiologies of neurodevelopmental disorders.
- Conditions
- Neurodevelopmental Disorders, Autism Spectrum Disorder, Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal Infection, Pediatric Acute-Onset Neuropsychiatric Syndrome
- Trial name
- NCT06213090
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Richard Frye
Mucopolysaccharidosis II
Study of Idursulfase-beta (GC1111) in Hunter Syndrome
This study evaluates the efficacy and safety of three doses of GC1111 in patients with Hunter Syndrome. Participants will be randomized to one of three doses of GC1111 or comparator.
- Trial name
- NCT02663024
- Testing
- idursulfase beta, idursulfase
- Volunteers
- 20 people
- Sponsor
- Green Cross Corporation