Clinical trials: Intellectual Disabilities
Rett Syndrome
Validating Innovative Biosensors for Rett Autonomic Symptom Tracking
The VIBRANT study aims to validate biosensors to objectively and directly measure symptoms in Rett syndrome and create confidence in the use of these devices in clinical trials.
- Trial name
- VIBRANT (NCT06338267)
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Rett Syndrome Research Trust
Rett Syndrome, RTT
Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
To evaluate the efficacy of cannabidiol oral solution (GWP42003-P, CBD-OS) in reducing symptom severity when compared with placebo, in participants with Rett syndrome.
- Trial name
- ARCH (NCT03848832)
- Testing
- GWP42003-P
- Where
- 26 sites in United States, Italy and 2 more
- Volunteers
- 29 people
- Sponsor
- Jazz Pharmaceuticals
Down Syndrome, Trisomy 21
Down Syndrome Metabolic Health Study
The purpose of this research study is to determine which measures best capture cardiovascular disease (CVD) risk and type 2 diabetes (T2DM) risk in children and adolescents with Down syndrome (DS).
- Trial name
- NCT01821300
- Where
- 2 sites in United States
- Volunteers
- 257 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Weight Loss, Food Habits
School Intervention With Daily Physical Activity and Healthy Food for Students With an Intellectual Disability.
A whole of school intervention with daily physical activity and healthy food for students with intellectual disabilities. The aim is to provide plenty of concrete examples of healthy life style choices and using school personnel…
- Conditions
- Weight Loss, Food Habits, Mental Retardation
- Trial name
- NCT01291238
- Testing
- Healthier, Smarter, Happier
- Where
- Sweden
- Volunteers
- 145 people
- Sponsor
- Karolinska Institutet
Prader Willi Syndrome, Hypoglycemia
Hypoglycemia in Prader-Willi Syndrome
This project will study whether infants with Prader-Willi Syndrome experience low blood sugars after short periods of fasting. This study will also evaluate metabolic markers in the blood to determine if infants with Prader-Willi…
- Trial name
- NCT01897363
- Testing
- Infants with Prader-Willi Syndrome
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- University of Florida
Mucopolysaccharidosis II
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme…
- Trial name
- NCT04251026
- Testing
- tividenofusp alfa
- Where
- 7 sites in United States, Canada and 2 more
- Volunteers
- 47 people
- Sponsor
- Denali Therapeutics Inc.
Autism Spectrum Disorder, Intellectual Disability
TMS for the Investigation of Brain Plasticity in Autism Spectrum Disorders
The main purpose is to study brain plasticity (the changes that occur in the brain through experience) in individuals with autism spectrum disorder (ASD).
- Trial name
- NCT02836405
- Testing
- Transcranial Magnetic Stimulation
- Where
- 2 sites in United States
- Volunteers
- 164 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Developmental Disability, Developmental Dyslexia
Postural Control, Grip Strength and Body Posture in Children With Intellectual Disability and Dyslexia
This study investigates physical changes in children with Dyslexia and Intellectual Disability. Participants divided into three groups and Body Posture, Postural Control and Hand Grip Strengths was evaluated.
- Conditions
- Developmental Disability, Developmental Dyslexia, Intellectual Disability
- Trial name
- NCT04250701
- Testing
- Body Posture Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- Ahmet Emir
Fragile X Syndrome, Disruptive Behavior
Treatment of Disruptive Behaviors in Fragile X Syndrome
Disruptive behaviors such as self-injury, aggression, and property destruction pose significant health-related issues to children diagnosed with fragile X syndrome (FXS), impacting the child's quality of life and causing…
- Trial name
- NCT03510156
- Testing
- Behavior analytic treatment
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Stanford University
Williams Beuren Syndrome
Efficacy of Minoxidil in Children With Williams-Beuren Syndrome
The Williams-Beuren syndrome (WBS) is a sporadic congenital disorder characterized by a multisystem developmental impairment. This syndrome is caused by a microdeletion in chromosome 7q11.23 that encompasses loss of the elastin…
- Trial name
- Williams (NCT00876200)
- Testing
- Minoxidil
- Where
- 18 sites in France
- Volunteers
- 21 people
- Sponsor
- Hospices Civils de Lyon
Fragile X Syndrome
Validation of Saliva in the Esoterix Genetic Laboratories Fragile X Assay
The purpose of this research is to determine if saliva samples can be used as an alternate sample type to test for fragile X. By using saliva instead of blood, it would be easier for patients to have fragile X testing.
- Trial name
- NCT01616589
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Esoterix Genetic Laboratories, LLC
Down Syndrome
Liq-NOL Efficacy in Pediatric Patients With Down Syndrome
The purpose of this study is to measure the effects of LiQ-NOL supplementation on language production using the Clinical Evaluation of Language Fundamentals test, language sampling using the mean length of utterance test, and…
- Trial name
- NCT00891917
- Testing
- Ubiquinol-10 Syrup
- Where
- United States
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Intellectual Developmental Disorder, Malformations
The Belgian Genome Resource to Resolve Rare Diseases
Whole-exome (WES) or whole-genome sequencing (WGS) are recommended as first- or second-tier molecular tests for patients with developmental disorders (DD), but the clinical utility of WGS continues to be debated.
- Conditions
- Intellectual Developmental Disorder, Malformations, Dysmorphia, Developmental Delay (Disorder)
- Trial name
- BeSolveRD (NCT07051213)
- Testing
- Whole exome sequencing, Whole genome Sequencing
- Where
- Belgium
- Volunteers
- 567 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Rubinstein-Taybi Syndrome
Diagnosis of RSTS: Identification of the Acetylation Profiles as Epigenetic Markers for Assessing Causality of CREBBP and EP300 Variants.
Rubinstein-Taybi syndrome (RSTS) is a rare and severe congenital developmental disorder characterized by congenital anomalies and intellectual disability with a long term memory deficit.
- Trial name
- GENEPI (NCT04122742)
- Testing
- skin biopsy for the primary fibroblast culture and a 15 ml blood sample (3 unnamed samples of 5ml) in each of the 4 SRT patients included., Generation of Induced Pluripotent Stem Cells (iPSC) from fibroblasts obtained by skin biopsy, Histone acetylation profiles of cells of SRT patients with CREBBP mutations
- Where
- France
- Volunteers
- 154 people
- Sponsor
- University Hospital, Bordeaux
Hunter Syndrome
Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
This long-term study will provide Elaprase treatment to children enrolled in this study and will utilize data from both enrolled patients and Hunter Outcome Survey (HOS) patient registry data to conduct the primary growth…
- Trial name
- NCT02455622
- Testing
- Elaprase for intravenous (IV) infusion
- Where
- 8 sites in United States, Dominican Republic and 6 more
- Volunteers
- 21 people
- Sponsor
- Shire
Rett Syndrome
Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome
Using a tele-research approach, we will recruit, enroll, guide and support carers and participants to engage in computer based activities (modified virtual reality) with the primary outcome of reducing stereotypies and increasing…
- Trial name
- NCT05012475
- Testing
- Modified Virtual Reality Gaming
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Georgetown University
Intellectual Disability
Improving the Health of Parents and Their Adolescent and Transition-age Youth With Intellectual and Developmental Disabilities
This study will determine the comparative effectiveness of Go Act, a tailored advocacy curriculum versus Peer parent-directed peer learning for increasing parent activation for parents of youth with intellectual and developmental…
- Trial name
- NCT05986305
- Testing
- Go Act, Peer
- Where
- 2 sites in United States
- Volunteers
- 406 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
Prader-Willi Syndrome, Obesity
Effects of Transcranial Direct Current Stimulation (tDCS) on Individuals With Prader-Willi Syndrome
Prader-Willi Syndrome (PWS) is a multisystemic genetic disease characterized by hypotonia, mental retardation, hyperphagia, and uncontrollable hunger due to hypothalamic dysfunction, caused by dysregulation of genes located in…
- Conditions
- Prader-Willi Syndrome, Obesity, Depressive Disorder, Obsessive-Compulsive Disorder
- Trial name
- NCT03324906
- Testing
- tDCS
- Where
- Brazil
- Volunteers
- 40 people
- Sponsor
- Federal University of São Paulo
Quality of Life, Down Syndrome
OHRQoL and Parental Perception in a Group of Children and Adolescents With Down Syndrome: A Cross
The aim of the study is to evaluate Oral Health related quality of life of children and adolescents with down syndrome and Parental Perception, knowledge and Attitude on oral health conditions of children and adolescents with…
- Conditions
- Quality of Life, Down Syndrome, Oral Health
- Trial name
- NCT04394221
- Where
- Egypt
- Volunteers
- 194 people
- Sponsor
- Cairo University
Prader-Willi Syndrome
The Intervention of Obesity in Children With Prader-Willi Syndrome Using Prebiotics and Probiotics
Prader-Willi syndrome (PWS) is a rare genetic disease, with hyperappetite and severe obesity. At present, there is no effective drugs and interventions to help control the appetite of PWS patients.
- Trial name
- NCT05791604
- Testing
- probiotics including B. lactis B420, B. lactis HN019, B. animalis Bb-12, L. rhamnosus GG, Prebiotics with galactomannan and oligofructose
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Children's Hospital of Fudan University