Clinical trials: Disabilities, Intellectual
Mucopolysaccharidosis Type II
Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis II
The Mucopolysaccharidoses (MPS) are a family of genetic metabolic disorders, caused by specific enzyme deficiencies which result in accumulation of partially degraded glycosaminoglycans (GAGs) within various tissues.
- Trial name
- COHERE (NCT06022380)
- Where
- United Kingdom
- Volunteers
- 46 people
- Sponsor
- Manchester University NHS Foundation Trust
Down Syndrome
Effects of Rhythmic Aerobic Exercises on Functional Balance in Children With Down Syndrome
Down syndrome (DS) is the most commonly occurring chromosomal condition and it affects more than 400,000 people in the United States. The chances of having a child with DS increases as the mother ages.
- Trial name
- NCT06196983
- Testing
- Rhythmic aerobic exercises, Control group, strength and balance exercises
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- Riphah International University
Prader-Willi Syndrome, Obesity
Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Beloranib) in Obese Subjects With Prader-Willi Syndrome
The purpose of this study is to evaluate efficacy and safety of ZGN-440 (beloranib) in obese adolescent and adult subjects with Prader-Willi Syndrome.
- Trial name
- bestPWS (NCT02179151)
- Testing
- ZGN-440 for Injectable Suspension
- Where
- 15 sites in United States
- Volunteers
- 108 people
- Sponsor
- Zafgen, Inc.
Intellectual Disability, Fragile X Syndrome
Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual Disabilities
This study is a randomized, double-blind, placebo-controlled, crossover trial of extended-release liquid methylphenidate (XRMPH) to evaluate the sensitivity of the NIH Toolbox Cognition Battery (NIHTB-CB) to changes in cognition…
- Conditions
- Intellectual Disability, Fragile X Syndrome, Down Syndrome, Attention Deficit Hyperactivity Disorder
- Trial name
- NCT05301361
- Testing
- Methylphenidate Oral Solution
- Where
- 3 sites in United States
- Volunteers
- 68 people
- Sponsor
- University of California, Davis
Rare Diseases, Albinism
Implementation of Long-read Sequencing for the Diagnosis of Rare Diseases.
Following on from the third national plan for rare diseases (PNMR3), the main objectives of the PNMR4 are to reduce diagnostic uncertainty and dead ends and to strengthen translational research to promote diagnosis and the…
- Conditions
- Rare Diseases, Albinism, Intellectual Disability
- Trial name
- LongEpi (NCT07400913)
- Testing
- Long-read sequencing
- Where
- France
- Volunteers
- 150 people
- Sponsor
- University Hospital, Bordeaux
Rett Syndrome
An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome
Rett Syndrome (RTT) is a complex disorder resulting from mutations in a gene encoding the MeCP2 protein. Currently, there are no methods to fix the abnormal gene, however, animal studies suggest that the symptoms of RTT can be…
- Trial name
- NCT02562820
- Testing
- Intravenous infusion
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- The Cleveland Clinic
Intellectual Disability
Compassionate Imagery for People With An Intellectual Disability
The current study aims to explore the adaptation of compassionate imagery for people with an intellectual disability who are experiencing mental health difficulties.
- Trial name
- NCT05339191
- Testing
- Compassionate image workshop
- Where
- United Kingdom
- Volunteers
- 4 people
- Sponsor
- NHS Lanarkshire
Prader-Willi Syndrome
Growth Hormone in Children Under 2 Years With Prader-Willi in Hospital of Sabadell
The PWS is a genetic disease with intellectual disabilities associated with multiple manifestations in other body systems. It is characterized by hypothalamic-pituitary abnormalities with severe hypotonia during the early years…
- Trial name
- NCT02205450
- Testing
- Recombinant Somatropin
- Where
- Spain
- Volunteers
- 16 people
- Sponsor
- Corporacion Parc Tauli
Down Syndrome
MOTOR PROFICIENCY AND HEALTH-RELATED FITNESS IN DOWN SYNDROME
People with Down syndrome (DS) seem to have a lower physical fitness than their peers without disabilities. Purpose: To examine the correlation of gross motor proficiency to health-related physical fitness ( HRPF) in children…
- Trial name
- NCT07702201
- Where
- Egypt
- Volunteers
- 20 people
- Sponsor
- Cairo University
Disabilities, Intellectual, Disability, Developmental
Appropriate Medication Use for Individuals With Intellectual Disabilities - A Matter of Life
The innovation idea is to develop valuable new knowledge about safe drug use in people with intellectual and/or developmental disabilities (IDDs).
- Trial name
- NCT06160102
- Testing
- Develop and implement the Framework for safe drug use in people with intellectual and/or developmental disabilities
- Where
- Norway
- Volunteers
- 110 people
- Sponsor
- Vestvagoy Municipality
Fragile X Syndrome
Probiotic Intervention for Microbiome Modifications and Clinical Improvements in Fragile X Syndrome
The primary objective of this clinical trial is to evaluate efficacy of probiotic mixture which contains Lactobacillus casei, Lactobacillus salivarius and Bifidobacterium breve, in children with Fragile X aged 3-18 years.
- Trial name
- NCT06279858
- Testing
- Probiotics
- Where
- Serbia
- Volunteers
- 15 people
- Sponsor
- Specila hospital for cerebral palsy and developmental neurology
X Fragile Syndrome
Clinical Trial for Asses the Safety and Efficacy of Vitamin C and Vitamin E in Combination Versus Placebo for Treating Cognitive and Behavior Disorder in Children With Fragile X Syndrome
The purpose of this study is to determine vitamin C and vitamin E in combination are effective in the treatment of cognitive and behavior disorder in children with fragile X syndrome.
- Trial name
- SXF2-8 (NCT02942498)
- Testing
- Vitamin C 10mg/Kg Vitamin E 10 mg/Kg
- Where
- Spain
- Volunteers
- 34 people
- Sponsor
- Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud
Child Development Disorders, Pervasive
Assessing Suicide Risk in Adolescents With Developmental Delays
Background: Currently, there is no standard method to study suicide risk in youth with developmental delays or intellectual disabilities (DD/ID).
- Trial name
- NCT01517126
- Where
- Canada
- Volunteers
- 17 people
- Sponsor
- National Institute of Mental Health (NIMH)
Autism Spectrum Disorder, Intellectual Disability
Heart Rate Informed Changes in Care for Non-Communicating Patients
The overarching aim is to generate knowledge to reduce incidence of pain in non-verbal patients' everyday life. The trial will 1) evaluate how HR can be used to identify potentially painful care procedures that should be…
- Conditions
- Autism Spectrum Disorder, Intellectual Disability, Communication, Nonverbal
- Trial name
- NCT05738278
- Testing
- HR-informed change in routine, Delayed HR-informed specific change in routine
- Where
- 2 sites in Norway
- Volunteers
- 38 people
- Sponsor
- University of Oslo
Intellectual Disability
Physical Activity With Tailored E-health Support for Individuals With Intellectual Disability
The main objective of this project is to enhance physical activity in youths and adults with intellectual disabilities (IDs) by developing and testing a tailored e-heath support for motivation and participation in physical…
- Trial name
- PA-IDe (NCT04079439)
- Testing
- Physical activity with tailored e-health support
- Where
- Norway
- Volunteers
- 60 people
- Sponsor
- University Hospital of North Norway
Prader-Willi Syndrome
A Phase 3 Extension Study of RAD011 (Cannabidiol Oral Solution) in Patients With Prader-Willi Syndrome
This is a Phase 3 open-label extension (OLE) study in patients diagnosed with Prader-Willi Syndrome (PWS) who completed the Maintenance Period of the randomized, placebo-controlled Phase 2/3 study SCOUT-015.
- Trial name
- NCT05387798
- Testing
- RAD011
- Sponsor
- Radius Pharmaceuticals, Inc.
Down Syndrome
Effect of Neuro Developmental Therapy in Infants With Down Syndrome
Neurodevelopmental treatments are a progressive therapeutic method experienced thru qualified therapists for the improvement in affected ones.
- Trial name
- NCT04816409
- Testing
- conventional therapy, Neurodevelopmental therapy
- Where
- Pakistan
- Volunteers
- 20 people
- Sponsor
- Riphah International University
Down Syndrome (Trisomy 21)
AEF0217 in Participants With Down Syndrome
The goal of this clinical trial is to identify if AEF0217 show an improvement in adaptive behaviors (daily life activities) in adults and older adolescents with Down Syndrome. It will also learn about the safety of AEF0217.
- Trial name
- NCT07334912
- Testing
- AEF0217 100 µg, AEF0217 300 µg
- Where
- 10 sites in France, Italy and 1 more
- Volunteers
- 188 people
- Sponsor
- Aelis Farma
Hypothyroidism, Trisomy 21
Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients
Children with levothyroxine (T21) have developmental delay and other functional gastrointestinal (GI) issues that may negatively affect L-T4 tolerability and absorption.
- Trial name
- NCT04747275
- Testing
- Liquid stable levothyroxine (L-T4) Tirosint-SOL, Oral tablet levothyroxine (L-T4)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Children's Mercy Hospital Kansas City
Obstructive Sleep Apnea, Down Syndrome
Clinical Outcomes From Treatment and Evaluation of Obstructive Sleep Apnoea in Children With Down Syndrome
The goal of this observational study is to learn about the use of non-invasive ventilation for treatment of obstructive sleep apnoea syndrome in children with Down Syndrome.
- Conditions
- Obstructive Sleep Apnea, Down Syndrome, Quality of Life, Behavior
- Trial name
- REFRESHED (NCT06030349)
- Testing
- Non-invasive ventilation
- Where
- 6 sites in Ireland, United Kingdom
- Volunteers
- 200 people
- Sponsor
- Children's Health Ireland