Clinical trials: Intellectual Disabilities
Down Syndrome
Parent Supported Weight Reduction in Down Syndrome
The purpose of this study is to determine whether a nutrition and physical activity education program for families of overweight or obese adolescents with Down syndrome is more effective when behavioral lifestyle change…
- Trial name
- NCT01256112
- Testing
- Parent Supported Behavioral Intervention, Nutrition/Activity Education (NAE)
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
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
Drumming Lessons' Influence on Children With Down Syndrome
The goal of this clinical trial is to learn if drumming lessons can increase self-control in children with Down syndrome. The main question it aims to answer is whether 2 months of drumming lessons can improve the behavioral…
- Trial name
- NCT07156318
- Testing
- 2 months of drumming lessons given by a professional drummer with extensive experience working with children with Down syndrome, Lessons after measurement
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Vanderbilt University
Rett Syndrome
Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome
Rett syndrome (RTT) is a disorder in which the nervous system does not develop properly. RTT generally affects girls, but there are some boys who have been diagnosed with RTT.
- Trial name
- NCT00069550
- Testing
- dextromethorphan, donepezil hydrochloride
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Hunter Syndrome
Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome
The purpose of the study is to collect data on CSF biomarkers in patients with Hunter Syndrome that would serve as reference data for comparison with cognitively impaired patients with Hunter syndrome, patients with other…
- Trial name
- NCT01449240
- Testing
- No treatment
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 10 people
- Sponsor
- Shire
Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical study (Study ALD-102 or Study…
- Conditions
- Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
- Trial name
- NCT02698579
- Testing
- No interventional drug product utilized in this follow-up study
- Where
- 13 sites in United States, Argentina and 7 more
- Volunteers
- 64 people
- Sponsor
- Genetix Biotherapeutics Inc.
Down Syndrome
Motor Intervention Whit the Intorus Tool on Children With Down Syndrome
The goal of this clinical trial is to test in people with down syndrome how does the INTORUS tool influence their psychomotor development.
- Trial name
- NCT05740046
- Testing
- INTORUS
- Where
- Spain
- Volunteers
- 30 people
- Sponsor
- University of Extremadura
Rett Syndrome
To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
Primary Objective • To investigate the efficacy of treatment with oral Bionetide versus placebo in girls and women with Rett syndrome Key Secondary Objective To investigate the efficacy of treatment with oral Bionetide versus…
- Trial name
- NCT06840496
- Testing
- Bionetide
- Where
- 18 sites in United States, Australia
- Volunteers
- 210 people
- Sponsor
- Biomed Industries, Inc.
Intellectual Disability, Developmental Delay
Establishing the Clinical Utility of First StepDx PLUS and NextStepDx PLUS Study
The study uses a randomized controlled study design of pediatric neurologists and developmental pediatricians and front-line (primary care) pediatricians to determine if use of FirstStepDx PLUS and Next StepDx PLUS are associated…
- Trial name
- NCT02414438
- Testing
- FirstStepDX PLUS and NextStepDX PLUS Webinar, FirstStepDX PLUS and NextStepDX PLUS Test Results
- Where
- United States
- Volunteers
- 217 people, healthy volunteers welcome
- Sponsor
- Qure Healthcare, LLC
Menkes Disease
NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease
This clinical trial will evaluate the safety, tolerability, dosing, and efficacy of Northera (Droxidopa) in children with Menkes disease aged 7 to 17 years who survived the major neurodegenerative and neurocognitive effects of…
- Trial name
- NCT07398508
- Testing
- Droxidopa Oral Product
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Stephen G. Kaler
Intellectual Disability
Targeted Next Generation Sequencing and Intellectual Disability
The purpose is to determine the benefit of next generation sequencing (NGS) targeted on genes involved in intellectual disability for etiologic diagnosis of intellectual disabilities.
- Trial name
- NGS-DI (NCT02889068)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Central Hospital, Nancy, France
Congenital Hypothyroidism
Clinical and Genetic Analysis in Congenital Hypothyroidism Due to Thyroid Dysgenesis.
Congenital hypothyroidism (CH) is a rare disease that affects 1 in 3500 newborn. This condition is detected consistently since the late 1970s in France, which has led to early care and a significant improvement in prognosis and…
- Trial name
- HYPOTYGEN (NCT01916018)
- Testing
- Clinical and radiologic exams and blood samples
- Where
- France
- Volunteers
- 558 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Leukemia, Myeloid, Acute, Leukemia, B-cell
Precision Exercise in Children With Malignant Hemopathies
In the early years of life and during adolescence, physical activity is crucial for good development of motor skills. It is even more so for those children and young people who are forced to undergo anti-cancer therapies and…
- Conditions
- Leukemia, Myeloid, Acute, Leukemia, B-cell, Leukemia, T Cell, Lymphoma, Hodgkin
- Trial name
- SportTherapy (NCT04090268)
- Testing
- Sport Therapy
- Where
- Italy
- Volunteers
- 380 people, healthy volunteers welcome
- Sponsor
- University of Milano Bicocca
Alcohol Use, Drug Use
Effectiveness of Take it Personal!
A controlled pre-post design study on Take it Personal! has demonstrated effectiveness in reducing the frequency and severity of youth use of alcohol, cannabis or other illicit drugs.
- Conditions
- Alcohol Use, Drug Use, Mild Intellectual Disability, IQ 50-70, Borderline Intellectual Disabilities (Intelligence Quotient 70-85)
- Trial name
- NCT05843474
- Testing
- Take it Personal!
- Where
- Netherlands
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Behavioural Science Institute
Intellectual Disability, Autism
Sensors for Communication for Persons Who Cannot Communicate Unequivocally
Some persons with intellectual disability or comprehensive cerebral palsy cannot communicate unequivocally how they are, how they react to situations and people, whether they are in pain or experience discomfort, anger or fear.
- Conditions
- Intellectual Disability, Autism, Cerebral Palsy
- Trial name
- NCT04199299
- Testing
- Heart rate and respiration rate sensors
- Where
- Norway
- Volunteers
- 100 people
- Sponsor
- University of Oslo
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
Healthy
Effectiveness of the Serious Game 'Broodles' for Siblings of Children With Visual Impairment and/or Intellectual Disability
The support for siblings of children with disabilities is scarce and fragmented, even though studies have shown that these siblings can benefit from support.
- Trial name
- NCT05376007
- Testing
- Serious game 'Broodles'
- Where
- Netherlands
- Volunteers
- 107 people, healthy volunteers welcome
- Sponsor
- VU University of Amsterdam
PTEN Gene Mutation, PTEN Hamartoma Tumor Syndrome
RAD001 and Neurocognition in PTEN Hamartoma Tumor Syndrome
Phosphatase and TENsin homolog (PTEN) gene germline mutations are associated with a spectrum of clinical manifestations characterized by neurocognitive deficits, intellectual disability, autism symptomatology, skin lesions…
- Trial name
- NCT02991807
- Testing
- RAD001
- Where
- 3 sites in United States
- Volunteers
- 46 people
- Sponsor
- Boston Children's Hospital
X-linked Adrenoleukodystrophy
Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy
X-linked adrenoleukodystrophy (ALD) is a genetic disorder affecting the brain and adrenal glands. Approximately one third of boys who are at risk will develop cerebral disease.
- Trial name
- NCT02233257
- Testing
- Lorenzo's Oil
- Where
- United States
- Sponsor
- University of Minnesota
Non Malignant Disorders, Immunodeficiencies
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients < 21…
- Conditions
- Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies
- Trial name
- NCT00744692
- Testing
- Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Duke University