Clinical trials: Intellectual Disabilities
Hunter Syndrome
Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®
This study is being conducted to identify pediatric patients with Hunter syndrome who have neurodevelopmental disease characteristics, who are currently receiving treatment with Elaprase, and who may be suitable to participate in…
- Trial name
- NCT00937794
- Testing
- Neurobehavioral testing, Visual and auditory assessments
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Shire
Respiratory Syncytial Virus Infection
Synagis® Liquid 50mg, 100mg for Intramuscular Injection Special Investigation in Immunocompromised Children With Synagis®
This post marketing observational study (PMOS) was conducted in Japan during the 2013-2014 and 2014-2015 Respiratory Syncytial Virus (RSV) seasons to assess the safety and effectiveness of palivizumab for the prevention of…
- Trial name
- NCT02016690
- Volunteers
- 312 people
- Sponsor
- AbbVie
Cerebral Adrenoleukodystrophy (CALD)
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in the post-marketing setting (tradename Skysona) and to describe major…
- Trial name
- Stargazer (NCT06224413)
- Testing
- No Intervention
- Where
- 3 sites in United States
- Volunteers
- 120 people
- Sponsor
- Genetix Biotherapeutics Inc.
Autism Spectrum Disorder, Intellectual Disabilities
An Evaluation of Care (Education) and Treatment Reviews for People With Learning Disabilities and Autistic People
Aims In this research project, we will determine whether Care (Education) and Treatment Reviews are helpful for people with learning disabilities and autistic people and their families.
- Trial name
- CECiLiA (NCT06918483)
- Volunteers
- 695 people
- Sponsor
- Coventry and Warwickshire Partnership NHS Trust
Down Syndrome
Assessing Malleability of Spatial Abilities in Down Syndrome
Based on available literature and our own preliminary research, the researchers have concluded that persons with Down syndrome (DS) exhibit difficulties in utilizing the specific spatial abilities of mental rotation and…
- Trial name
- NCT05332912
- Testing
- Spatial Ability Experience
- Where
- United States
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- University of Alabama, Tuscaloosa
Down Syndrome, Postural Balance
Balance Bike Training in Down Syndrome
Background: Children with Down syndrome (DS) present with hypotonia, impaired balance and poor gross motor proficiency which lead to difficulty learning new motor skills, decreased engagement in physical activity, and limited…
- Trial name
- NCT03677245
- Testing
- Strider Balance Bike
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- University of Mississippi Medical Center
Williams Syndrome, Supravalvular Aortic Stenosis
Williams Syndrome (WS) and Supravalvar Aortic Stenosis (SVAS) DNA and Tissue Bank
Williams syndrome is a rare genetic disorder occurring in 1:8000-12,000 individuals. It is caused by the deletion of 25-27 coding genes, including elastin (ELN) on the 7th human chromosome.
- Conditions
- Williams Syndrome, Supravalvular Aortic Stenosis, Cardiovascular Disease
- Trial name
- NCT02706639
- Where
- 3 sites in United States
- Volunteers
- 305 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Prader-Willi Syndrome
A Pharmacokinetic and Safety Study of CSTI-500 in Subjects With Prader-Willi Syndrome
The purpose of this Phase 1 study is to evaluate the pharmacokinetics (PK) and safety of a single dose of CSTI-500 10 mg in subjects with Prader-Willi syndrome (PWS) between 13 and 50 years of age with a genetically confirmed…
- Trial name
- NCT05504395
- Testing
- CSTI-500
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- ConSynance Therapeutics
Down Syndrome, Sleep Disordered Breathing
Healthy Sleep for Children With Down Syndrome
The purpose of this multi-center observational study (utilizing the sites enrolling patients for the Pediatric Adenotonsillectomy Trial for Snoring (PATS) [1U011HL125307-O1A1]) is to gather data regarding children with Down…
- Trial name
- HELP-DS (NCT03922165)
- Testing
- Adenotonsillectomy
- Where
- 6 sites in United States
- Volunteers
- 26 people
- Sponsor
- Brigham and Women's Hospital
Neurodevelopmental Disorders With Severe Self-injuries
Efficacy of Cannabidiol in the Management of Self-injuries in Children and Adolescents With Severe Neurodevelopmental Disorders.
Self-injuries includes repetitive actions directed at oneself, such as biting, hitting, hitting one's head or limbs, slapping oneself, pulling one's hair, or hitting oneself in the eyes, which can lead to severe injury.
- Trial name
- CBD-SIB (NCT07564245)
- Testing
- Cannabidiol
- Where
- 2 sites in France
- Volunteers
- 21 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Mucopolysaccharidosis II
A Study of JR-141 in Patients With Mucopolysaccharidosis II
A Phase II/ III multicenter, open-label, single-group, designed to evaluate the efficacy and safety of study drug for the treatment of the MPS II.
- Trial name
- NCT03568175
- Testing
- JR-141
- Where
- 19 sites in Japan
- Volunteers
- 28 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Intellectual Disability, Attention Deficit Hyperactivity Disorder
Evaluation of a Home-based Parenting Support Program: Parenting Young Children
Background: Parents with intellectual and developmental disabilities (IDDs) have a tendency to provide insufficient caregiving and often need parenting support to prevent neglect and child removal.
- Conditions
- Intellectual Disability, Attention Deficit Hyperactivity Disorder, Autism Spectrum Disorder
- Trial name
- PYC (NCT05935722)
- Testing
- Parenting Young Children, Treatment as Usual
- Where
- 5 sites in Sweden
- Volunteers
- 60 people
- Sponsor
- Örebro University, Sweden
Dysarthria, Down Syndrome
Intensive Dysarthria Sessions in Adults and Children With Down Syndrome
This research study is designed to investigate the feasibility and treatment effects of a behavioral speech treatment in adults and children with Down Syndrome (DS) and dysarthria.
- Trial name
- NCT01684670
- Testing
- Speech sessions
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Duke University
Down Syndrome With and Without Auto Immune Abnormalities
Pilot Study of Auto-immune Abnormalities Associated With Down Syndrome
The purpose of the study is to identify biological data linked to auto immune abnormalities associated with Down Syndrome.
- Trial name
- IMMUTRI (NCT01041469)
- Where
- 2 sites in France
- Volunteers
- 72 people
- Sponsor
- Institut Jerome Lejeune
Angelman Syndrome, Fragile X Syndrome
Single Dose Pharmacokinetic (PK) Study
The trial is a Phase 1 Single Dose PK Study in Adolescent Subjects with Fragile X syndrome (FXS) or Angelman syndrome (AS). The primary objective of the study is to evaluate the pharmacokinetics (PK) of OV101 following a single 5…
- Trial name
- NCT03109756
- Testing
- OV101
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Ovid Therapeutics Inc.
Down Syndrome
Effect of Aerobic Exercises on Static & Dynamic Balance
To determine the effect of aerobic exercises on static \& dynamic balance of children in Down syndrome. When a person has Down syndrome, they have an extra chromosome. In the body, chromosomes are discrete collections of genes.
- Trial name
- NCT05915780
- Testing
- Cycling, Balance Board
- Where
- Pakistan
- Volunteers
- 20 people
- Sponsor
- Riphah International University
Mucopolysaccharidosis II
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
The purpose of this study is to determine whether the administration of iduronate-2-sulfatase enzyme in a weekly or every other week therapy frequency is safe and efficacious in patients with MPS II.
- Trial name
- NCT00069641
- Testing
- Iduronate-2-sulfatase enzyme replacement therapy
- Where
- 9 sites in United States, Brazil and 2 more
- Volunteers
- 96 people
- Sponsor
- Shire
Pyruvate Dehydrogenase Complex Deficiency Disease
Natural History and Advanced Genetic Study of Pyruvate Dehydrogenase Complex Deficiencies
Children and adults with pyruvate dehydrogenase complex deficiency (PDCD) are participating in a research study seeking to better understand the genetic causes, symptoms, usefulness of current treatments, and outcomes for these…
- Trial name
- NCT03056794
- Testing
- No intervention
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- University of Pittsburgh
Down Syndrome, Activity
Activities and Participation of Children With Down Syndrome
The aim of this study is to examine the activity participation levels of Down syndrome(DS) children and the restrictive or supportive factors affecting their participation levels and to investigate their effects on quality of…
- Conditions
- Down Syndrome, Activity, Participation, Quality of Life
- Trial name
- NCT05152251
- Testing
- Evaluation
- Where
- Turkey (Türkiye)
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Education, Intellectual Disabilities With Other Behavioral Symptoms
The Effect of Self-Care Education on Self-Care Skills and School Sociality
The aim of this study is to examine the effect of self-care education given to children with educable intellectual disabilities on children\'s self-care skills and school social behaviors.
- Trial name
- NCT06527118
- Testing
- ACTIVITIES TO LEARN SELF CARE SKILLS
- Where
- Turkey (Türkiye)
- Volunteers
- 34 people
- Sponsor
- Dokuz Eylul University