Clinical trials: Intellectual Developmental Disorder
Adrenoleukodystrophy
Newborn Screening for Adrenoleukodystrophy
To test if the routine newborn screening dried blood spots can be used to test if elevation of C26:0 lysophosphatidylcholine (C26:0-lyso-PC), a status indicating adrenoleukodystrophy (ALD)
- Trial name
- NCT02952482
- Testing
- newborns testing for ALD
- Where
- Taiwan
- Volunteers
- 45,796 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Intellectual Disability, Quality of Life
The Effect of Gardening Activities on the Quality of Life of Students With Mild Intellectual Disability
This study will be conducted to determine the effect of gardening activities applied to mildly intellectually disabled secondary school students on their quality of life and to examine their views on these activities in depth.
- Trial name
- NCT07504874
- Testing
- Horticultural activity
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Ordu University
Atopic Dermatitis, Alopecia Areata
Treatment of Atopic Dermatitis and Alopecia Areata With Abrocitinib in Individuals With Down Syndrome
This is a single-center, open-label, basket phase 2b trial that will enroll Down Syndrome (DS) participants with at least one inflammatory skin condition (Atopic Dermatitis (AD) and/or Alopecia Areata (AA)).
- Trial name
- NCT07242638
- Testing
- Abrocitinib
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Prader-Willi Syndrome
PWS European Blood Bank for Infants and Controls From 0 to 48 Months
The present project aims to determine the underlying mechanisms for the switch from failure to thrive to excessive weight gain and hyperphagia with impaired satiety in PWS.
- Trial name
- NCT02529085
- Testing
- blood samples
- Where
- 6 sites in Belgium, France and 4 more
- Volunteers
- 215 people, healthy volunteers welcome
- Sponsor
- University Hospital, Toulouse
Autism Spectrum Disorder, Intellectual Disability
Transcranial Magnetic Stimulation Studies in Autism Spectrum Disorders
Autism Spectrum Disorder (ASD) is the most prevalent of the developmental disorders and their incidence is rising. However, the variability in the behavioral symptoms is large.
- Trial name
- NCT02591446
- Where
- United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Rhode Island Hospital
Intellectual Disability, Epilepsy
MEHMO Natural History and Biomarkers
This observational natural history study will follow individuals with MEHMO (Mental disability, Epileptic seizure, Hypopituitarism/Hypogenitalism, Microcephaly, Obesity) syndrome or an eIF2-pathway related disorder, who have…
- Conditions
- Intellectual Disability, Epilepsy, Hypogonadisms, Microcephaly
- Trial name
- NCT06019182
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Rett Syndrome
The Role of Dietary Calcium for the Treatment of Osteopenia in Girls With Rett Syndrome
Randomized, placebo-controlled trial of oral calcium supplementation for osteopenia in girls and women with Rett syndrome
- Trial name
- NCT05352373
- Testing
- calcium
- Volunteers
- 32 people
- Sponsor
- Baylor College of Medicine
Rett Syndrome, Dysbiosis
Protective Role of Pre-/ Post-biotics on Gut Inflammation, Dysbiosis, and Life Quality in Rett Syndrome (Biotics_RTT)
The study will examine the potential efficacy and safety of two pre- and post-biotics on markers for gut inflammation and intestinal microbiota ecology in patients with Rett syndrome.
- Conditions
- Rett Syndrome, Dysbiosis, Epilepsy, Quality of Life
- Trial name
- Biotics_RTT (NCT05420805)
- Testing
- ALAC, inulin, FOS, and sodium butyrate, Sodium butyrate and zinc oxide
- Where
- Italy
- Volunteers
- 28 people
- Sponsor
- Azienda Ospedaliera Universitaria Senese
Down Syndrome
Role of Early Motor Experience in Infants With Down Syndrome
Infants with Down syndrome show significant delays and weaknesses in motor, cognitive, and language development compared to typically developing infants.
- Trial name
- NCT05144373
- Testing
- Gross motor intervention, gross and fine motor intervention
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- Georgia State 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
Rett Syndrome, MECP2 Duplication dIsorder
Natural History of Rett Syndrome & Related Disorders
The purpose of this study is to advance understanding of the natural history of Rett syndrome (RTT), MECP2-duplication disorder (MECP2 Dup), CDKL5, FOXG1, and individuals with MECP2 mutations who do not have RTT including the…
- Conditions
- Rett Syndrome, MECP2 Duplication dIsorder, CDKL5 Disorder, FOXG1 Syndrome
- Trial name
- NCT02738281
- Where
- 14 sites in United States
- Volunteers
- 1,044 people
- Sponsor
- University of Alabama at Birmingham
X-Linked Adrenoleukodystrophy
Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.
- Trial name
- NCT03196765
- Testing
- Sobetirome (NV1205)
- Where
- 13 sites in Argentina, Australia and 6 more
- Sponsor
- NeuroVia, Inc.
Prader-Willi Syndrome, Hyperphagia
Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome
This Phase 2b/3 double-blind, placebo-controlled study will evaluate the safety, tolerability, and effects of livoletide on food-related behaviors in patients with Prader-Willi Syndrome (PWS).
- Trial name
- ZEPHYR (NCT03790865)
- Testing
- Livoletide
- Where
- 37 sites in United States, Australia and 6 more
- Volunteers
- 158 people
- Sponsor
- Millendo Therapeutics SAS
Rett Syndrome
Effects of Creatine Supplementation in Rett Syndrome
Creatine supplementation in RTT: a randomized controlled trial Rett Syndrome (RTT) is a neurodevelopmental disorder characterised by apparently normal early development (stage 1 of RTT) followed by loss of purposeful hand use…
- Trial name
- NCT01147575
- Testing
- Creatine monohydrate
- Where
- Austria
- Volunteers
- 21 people
- Sponsor
- Medical University of Vienna
Down Syndrome
Effects of Play Based Therapy on Attention Level of Children With Down Syndrome
The aim of the study is to examine the attention level of children with down syndrome receiving play based therapy as compared to children receiving child centered play therapy.
- Trial name
- NCT06893666
- Testing
- Play based therapy, Child centered play therapy
- Where
- Pakistan
- Volunteers
- 20 people
- Sponsor
- Riphah International University
Lesch-Nyhan Syndrome
Physiotherapy Assessment Based on the ICF Model in The Lesch-Nyhan Syndrome: Case Report
Lesch-Nyhan Syndrome (LNS) is a genetic disorder that results in a deficit in the hypoxanthine-guanine phosphoribosyltransferase (HPRT) enzyme, which affects purine metabolism.
- Trial name
- NCT05548751
- Where
- Turkey (Türkiye)
- Volunteers
- 1 people
- Sponsor
- Uskudar University
Adrenoleukodystrophy
Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
OBJECTIVES: I. Evaluate the efficacy of interferon beta and thalidomide in male patients with adrenoleukodystrophy who show evidence of brain inflammatory response and are receiving concurrent glyceryl trierucate and glyceryl…
- Trial name
- NCT00004450
- Testing
- glyceryl trierucate, glyceryl trioleate, interferon beta
- Volunteers
- 60 people
- Sponsor
- FDA Office of Orphan Products Development
Down Syndrome
Correlations Phenotype / Genotype in Down Syndrome
The purpose of this study is to correlate phenotype and genotype of Down syndrome patients in order to identify the biochemical reactions involved in their mental retardation and their other phenotypic characteristics.
- Trial name
- INTREPID (NCT01034280)
- Where
- France
- Volunteers
- 524 people
- Sponsor
- Institut Jerome Lejeune
Hypotonia, Muscle, Obstructive Sleep Apnea
Long-term Evaluation of Nasopharyngeal Airway in Hypotonia
This research is studying the long term use of a nasal airway device (self-supporting nasopharyngeal airway; "ssNPA") in children with hypotonic upper airway obstruction to learn about its effectiveness and tolerability as a…
- Conditions
- Hypotonia, Muscle, Obstructive Sleep Apnea, Nasal Airway Obstruction
- Trial name
- NCT06614582
- Testing
- Self-Supporting Nasopharyngeal Airway (ssNPA)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- University of Michigan
Sleep Apnea, Obstructive, Down Syndrome
A Pilot Study to Evaluate the Hypoglossal Nerve Stimulator in Adolescents With Down Syndrome and Obstructive Sleep Apnea
Obstructive sleep apnea (OSA) affects up to 1% of the general pediatric population and is associated with adverse behavior and quality of life, as well as long term cardiopulmonary system complications.
- Trial name
- NCT02344108
- Testing
- Inspire® Upper Airway Simulation System (Model 3028 IPG )
- Where
- 5 sites in United States
- Volunteers
- 42 people
- Sponsor
- Christopher Hartnick, M.D.