Clinical trials: Disabilities, Intellectual
Down Syndrome
A Double-blind, Placebo-controlled Comparative Study and Open-label Extension Study to Confirm the Efficacy and Safety of E2020 in Subjects With Down Syndrome Having Regression Symptoms and Disabled Activities of Daily Living.
The purpose of this double-blind, placebo-controlled, comparative study and open-label extension study is to confirm the efficacy and safety of E2020 in subjects with Down syndrome having regression symptoms and disabled…
- Trial name
- NCT02094053
- Testing
- E2020-Donepezil hydrochloride
- Where
- 10 sites in Japan
- Volunteers
- 36 people
- Sponsor
- Eisai Co., Ltd.
Myotonic Dystrophy 1, Huntington Disease
NIPD on cffDNA for Triplet Repeat Diseases
The purprose of this study is to develop and validate an analytical NIPD test for triplet repeat disesases by NGS analysis from maternal blood, searching for the familial mutation in families at risk of having one of the…
- Conditions
- Myotonic Dystrophy 1, Huntington Disease, Fragile X Syndrome
- Trial name
- NCT04698551
- Testing
- Non invasive prenatal diagnosis
- Where
- France
- Volunteers
- 36 people
- Sponsor
- University Hospital, Montpellier
Intellectual Disability, Attention Deficit Hyperactivity Disorder
Evaluation of a Home-based Parenting Support Program: Parenting Young Children
Background: Parents with intellectual and developmental disabilities (IDDs) have a tendency to provide insufficient caregiving and often need parenting support to prevent neglect and child removal.
- Conditions
- Intellectual Disability, Attention Deficit Hyperactivity Disorder, Autism Spectrum Disorder
- Trial name
- PYC (NCT05935722)
- Testing
- Parenting Young Children, Treatment as Usual
- Where
- 5 sites in Sweden
- Volunteers
- 60 people
- Sponsor
- Örebro University, Sweden
Gastreoesophageal Reflux Disease
Pharmacokinetics of Enteral Omeprazole Suspension in Patients With Cerebral Palsy and Mental Retardation
Gastroesophageal reflux disease and reflux-esophagitis are a major chronic problem in most children with cerebral palsy and mental retardation.
- Trial name
- NCT00426595
- Testing
- Administration of omeprazole suspension, omeprazole administered as a multi-unit-pellet system
- Where
- Belgium
- Volunteers
- 14 people
- Sponsor
- University Hospital, Ghent
Epilepsy, Intracranial Tumors
Magnetoencephalography in Children
This study is a single-center observational clinical study, which evaluates the diagnostic value of magnetoencephalography (MEG) in the diagnosis of neurodevelopmental diseases in children, such as the localization of epileptic…
- Conditions
- Epilepsy, Intracranial Tumors, Cerebrovascular Disease, Autism
- Trial name
- NCT06668519
- Testing
- Magnetoencephalography
- Where
- China
- Volunteers
- 1,500 people
- Sponsor
- Children's Hospital of Fudan University
X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD)
Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
Study ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age.
- Conditions
- X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
- Trial name
- NCT02204904
- Testing
- Allo-HSCT
- Where
- 13 sites in United States, Argentina and 5 more
- Volunteers
- 59 people
- Sponsor
- Genetix Biotherapeutics Inc.
Fragile X Syndrome
Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome
The purpose of this study is to determine whether the medication oxytocin is an effective and tolerable treatment in adolescent males with fragile X syndrome (FraX) for improving socially appropriate behaviors and reducing social…
- Trial name
- NCT01254045
- Testing
- oxytocin 24IU, oxytocin 48IU
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Stanford 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
Intellectual Disability
A Pilot Trial of Mindfulness Based Intervention Program for People With Intellectual Disabilities in a Low Income Setting
The proposed study is a pre-post intervention study to investigate the acceptability and feasibility of a mindfulness based program for people with intellectual disabilities and their caregivers in a low income setting in…
- Trial name
- NCT03501914
- Testing
- Mindfulness
- Where
- Pakistan
- Volunteers
- 12 people
- Sponsor
- Pakistan Institute of Living and Learning
Sanfilippo Syndrome
Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
Sanfilippo syndrome Type A, or Mucopolysaccharidosis (MPS) IIIA, is a rare lysosomal storage disease caused by deficiency of the enzyme heparan N-sulfatase (sulfamidase).
- Trial name
- NCT02060526
- Testing
- Recombinant human heparan N-sulfatase [rhHNS]
- Where
- 10 sites in United States, Argentina and 6 more
- Volunteers
- 21 people
- Sponsor
- Shire
Down Syndrome
Effects of Plyometric Exercises on Static and Dynamic Balance of Children With Down,s Syndrome.
Down syndrome is a condition in which a person has an extra chromosome. Typically, a baby is born with 46 chromosomes. Down syndrome is a genetic condition that causes mild to serious physical and developmental problems.
- Trial name
- NCT05951543
- Testing
- plyometric exercises, dynamic balance group control group
- Where
- Pakistan
- Volunteers
- 20 people
- Sponsor
- Riphah International University
Asthma in Children, Intellectual Disabilities (F70-F79)
Smart Inhaler and Remote Asthma Management in Adolescents With Intellectual and Developmental Disabilities
Correct use of daily medications containing inhaled corticosteroids is key for asthma control, yet children with intellectual and developmental disabilities (IDD) face additional barriers to proper inhaler use.
- Conditions
- Asthma in Children, Intellectual Disabilities (F70-F79), Developmental Disability, Autism Spectrum Disorder
- Trial name
- IDD (NCT07746921)
- Testing
- Use of active remote smart inhaler in the home setting, Use of passive remote smart inhaler in the home setting
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Children's Hospital Los Angeles
Lesch-Nyhan Syndrome
Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease
OBJECTIVES: I. Determine the effects of aminoimidazole carboxamide riboside (AICAR) on hematologic manifestations of Lesch-Nyhan disease. II.
- Trial name
- NCT00004314
- Testing
- aminomidazole carboxamide riboside
- Volunteers
- 2 people
- Sponsor
- National Center for Research Resources (NCRR)
Mucopolysaccharidosis II
Long-term Follow-Up for RGX-121
RGX-121-5101 is an observational long-term follow-up study to the RGX-121-101 or RGX-121-1102 studies where participants received RGX-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfatase gene…
- Trial name
- NCT04597385
- Testing
- Long-term Follow-Up
- Where
- 4 sites in United States, Brazil
- Volunteers
- 27 people
- Sponsor
- REGENXBIO Inc.
Down Syndrome, Trisomy 21
JASPER Intervention in Down Syndrome
The goal of this study is to determine whether JASPER (Joint Attention, Symbolic Play, Engagement, Regulation), which is an intensive, targeted early behavioral intervention focused on a developmentally based approach for…
- Trial name
- NCT03653143
- Testing
- JASPER
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Boston Children's Hospital
Mucopolysaccharidosis, Hurler Syndrome
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Rationale: Chemotherapy administration before a donor stem cell transplant is necessary to stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome
- Trial name
- NCT01043640
- Testing
- Campath-1H, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Intellectual Disabilities Nutritional, Intellectual Disability
Dietary Intervention and Adults With Intellectual Disabilities
Goal The overall aim of the study is to improve the health of persons with Intellectual disabilities
- Trial name
- NCT04436692
- Testing
- Nudging
- Where
- Norway
- Volunteers
- 32 people
- Sponsor
- University of Oslo
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
Rett Syndrome, Tourette Syndrome
The Role of Probiotics PS128 in Movement Disorders
Pediatric movement disorders comprise of a large number of different neurological diseases including Rett syndrome (RTT) and Tourette syndrome.
- Conditions
- Rett Syndrome, Tourette Syndrome, Tic Disorders
- Trial name
- NCT03259971
- Testing
- Probiotic-Lactobacillus plantarum PS128
- Where
- Taiwan
- Volunteers
- 500 people
- Sponsor
- National Taiwan University Hospital
Autism Spectrum Disorder (ASD), Intellectual Disability
Verbal Intent and Gaze Cues on Action Prediction in ASD
A 2×2×3 mixed design (intent word × object type × group: TD, ASD, ID) was used. Age-matched children watched videos and judged which object a model would pick; their choices and eye movements were recorded.
- Trial name
- NCT07597694
- Where
- China
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- University of Electronic Science and Technology of China