Clinical trials: Disabilities, Intellectual
Hypofibrinogenemia, Afibrinogenemia
Human Fibrinogen Concentrate in Pediatric Cardiac Surgery
The goal of the study is to determine whether the use of Human Fibrinogen Concentrate (RiaSTAP) will decrease blood loss and the need for component blood therapy in neonates and infants undergoing cardiopulmonary bypass.
- Conditions
- Hypofibrinogenemia, Afibrinogenemia, Bleeding Disorders
- Trial name
- RiaSTAP (NCT02822599)
- Testing
- RiaStAP, Saline
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Nicklaus Children's Hospital f/k/a Miami Children's Hospital
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
- Trial name
- NCT06430385
- Testing
- ION440
- Where
- 12 sites in United States, Austria and 3 more
- Volunteers
- 48 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Down Syndrome
Effect of Sensory Integration on Gross and Balance on Children With Down Syndrome
To investigate the effect of sensory integration on gross motor function in children with Down syndrome. To investigate the effect of sensory integration on balance in children with Down syndrome.
- Trial name
- NCT07785011
- Testing
- BALANCE AND SENSORY INTEGRATION
- Where
- Egypt
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Raheel Sanad
Autism Disorder, Multiple Disabilities
Impact and Determinants of Success of a Mobile Multidisciplinary Intervention System Specialized in Pain Management for Patients With Moderate to Severe Intellectual Development Disorder, Isolated or Associated With an ASD or Multiple Disability
Pain is described by patients with a Neurodevelopmental Disorder (NDD), including Autism Spectrum Disorder (ASD), Intellectual Development Disorder (IDD) or multiple disabilities, and the family as one of the main associated…
- Conditions
- Autism Disorder, Multiple Disabilities, Pain, Intellectual Developmental Disorder
- Trial name
- MoDIDol (NCT07302048)
- Testing
- MoDIDol intervention
- Where
- France
- Volunteers
- 80 people
- Sponsor
- University Hospital, Brest
Intellectual Disability
Augmentative and Alternative Communication in Games With Virtual Reality in Intellectual Disabilities
Background: Games using motion capture from webcam have become increasingly popular. A population that lacks much stimulus in the teaching-learning process has under-explored the benefits of this kind of games: The Intellectually…
- Trial name
- NCT03033797
- Testing
- MoviLetrando, MoveHero
- Where
- Brazil
- Volunteers
- 165 people
- Sponsor
- Faculdade de Medicina do ABC
Health-Related Behavior, Quality of Life
PODER Familiar: Health Promotion for Latino Families of Children With IDD
The goal of this clinical trial is to test a culturally tailored health promotion intervention for children with IDD and their families.
- Conditions
- Health-Related Behavior, Quality of Life, Psychological Distress, Diet, Healthy
- Trial name
- PODER (NCT05713617)
- Testing
- PODER Familiar
- Where
- 2 sites in United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- University of Texas at Austin
Fragile X Syndrome
Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40
This study is to investigate the safety, tolerability and efficacy of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in males with Fragile X Syndrome (FXS) with confirmed full FMR1 mutation treated over a 10 week period in an…
- Trial name
- IMPACT-FXS (NCT04823052)
- Testing
- Sulindac (HLX-0201), dose strength 1, Sulindac (HLX-0201), dose strength 2, Gaboxadol (HLX-0206)
- Where
- 10 sites in United States, Australia
- Sponsor
- Healx Limited
Genetic Disorder, Asperger Disorder
Pediatric Patients With Metabolic or Other Genetic Disorders
Background: Some patients with unusual genetic conditions are referred to the National Institutes of Health (NIH). They may not be eligible to join current research studies.
- Conditions
- Genetic Disorder, Asperger Disorder, Autism Spectrum Disorder, Fragile X Syndrome
- Trial name
- NCT02769949
- Where
- United States
- Volunteers
- 96 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Autism Spectrum Disorder
Interest of Oxytocin as an Adjuvant Treatment of Psycho-educational Measures in Challenging Behaviors in Children With Autism Spectrum Disorders and Moderate to Severe Intellectual Disability: Feasibility and Safety Study.
The investigation team propose in this study to specifically evaluate the feasibility of using oxytocin in the form of an intranasal spray in a specific population of children with autism spectrum disorder and intellectual…
- Trial name
- OT-DEFI (NCT05864508)
- Testing
- Oxytocin nasal spray
- Where
- France
- Volunteers
- 30 people
- Sponsor
- University Hospital, Toulouse
Down Syndrome
The Impact of an 8-week Supported Exercise Program on Fitness and Symptoms in Adolescents With Down Syndrome
This study explores the effects of an eight-week exercise intervention for adolescents with Down syndrome on home exercise compliance.
- Trial name
- DSFIT (NCT03192540)
- Testing
- Exercise
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Boston Children's Hospital
Down Syndrome, Subclinical Hypothyroidism
Treatment Trial of Subclinical Hypothyroidism in Down Syndrome
The purpose of this research study is to learn about the effects of treating subclinical hypothyroidism (SCH) with thyroid hormone replacement in children and adolescents with Down syndrome (DS).
- Trial name
- NCT01832753
- Testing
- Levothyroxine
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
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
Randomized Controlled Study of Donepezil in Fragile X Syndrome
Fragile X syndrome (FraX) is the most common known heritable cause of human intellectual disability. Though recent research has revealed much about the genetic and neurobiological bases of FraX, knowledge about specific and…
- Trial name
- NCT01120626
- Testing
- donepezil, sugar pill
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Stanford University
Down Syndrome,Auxological Indexes, Auxological Parametars, Intrauterine Growth Restriction
Indicators of Growth, Nutritional Status and Comorbide Disorders of Newborns With Down Syndrome
Objective To access predictive values of the auxological parameters and indexes for risk of comorbid malformations in newborns with Down syndrome (DS) Study design In this cohort retrospective study, 141 newborns with proven…
- Trial name
- DownSy (NCT03553706)
- Where
- Croatia
- Volunteers
- 141 people
- Sponsor
- University Hospital of Split
Rett Syndrome
Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)
This 18-week open-label study examines the effectiveness of Full-Spectrum Medicinal Cannabis Plant Extract containing 0.08% THC (NTI164) in treating Rett syndrome (RTT) in children and young people.
- Trial name
- NCT06621043
- Testing
- NTI164
- Where
- Australia
- Volunteers
- 14 people
- Sponsor
- Fenix Innovation Group
Prader-Willi Syndrome
An Open-Label Study of DCCR Tablet in Patients With PWS
The primary objective of this study is to monitor the long-term safety of DCCR in PWS patients.
- Trial name
- NCT04086810
- Testing
- DCCR
- Sponsor
- Soleno Therapeutics, Inc.
Fragile X Syndrome (FXS), Autism Spectrum Disorder
Group CBT in Adolescents With Fragile X Syndrome and in Adolescents With Autism Spectrum Disorder
Fragile X Syndrome (FXS) is a rare genetic syndrome, caused by a mutation in the FMR1 gene located on the X chromosome. It is considered the leading hereditary cause of intellectual disability (ID) and the primary cause of Autism…
- Trial name
- NCT06677866
- Testing
- Cognitive behavioral therapy, Waiting List
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Bambino Gesù Hospital and Research Institute
Neurodevelopmental Disorders, Intellectual Developmental Disorder
High Depth Exome Sequencing on DNA From a Salivary Sample by Mouth Smear.
Despite technological advances, a genetic etiology has been identified in only about 50% to 60% of patients with Neurodevelopmental disorders (NDDs), with a higher diagnostic yield in the syndromic NDD and IDD subgroups.
- Trial name
- SEPASA (NCT07492199)
- Testing
- genetic
- Volunteers
- 50 people
- Sponsor
- Centre Hospitalier Universitaire de Besancon
Prader-Willi Syndrome
Brain Olfactory Pathways in Prader-Willi Syndrome
Studying the cerebral activity of children with Prader-Willi Syndrom (PWS) when the study propose to them nasal activations.
- Trial name
- PRADOL (NCT07006207)
- Testing
- olfactory stimulation, functional MRI
- Where
- France
- Volunteers
- 30 people
- Sponsor
- University Hospital, Toulouse
Prader-Willi Syndrome
Home-based SSP on Individuals With PWS
The Polyvagal Theory focuses on how function and structure changed in the vertebrate autonomic nervous system during evolution. The theory is named for the vagus, a major cranial nerve that regulates bodily state.
- Trial name
- NCT03871751
- Testing
- Safe and Sound Protocol
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Indiana University