Clinical trials: Disabilities, Intellectual
Fragile X Syndrome
A Prospective Open-label Study of Aripiprazole in Fragile X Syndrome
The purpose of this study is to determine the effectiveness and tolerability of aripiprazole in the treatment of children and adolescents with Fragile X Syndrome.
- Trial name
- NCT00420459
- Testing
- Aripiprazole
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Indiana University
Intellectual Disabilities, Speech Delay
Pivotal Response Treatment for Individuals With Intellectual Disabilities
The investigators will assess the efficacy of Pivotal Response Treatment (PRT) in the treatment of communication deficits in children with intellectual disabilities.
- Conditions
- Intellectual Disabilities, Speech Delay, Language Disorder
- Trial name
- NCT01988623
- Testing
- Pivotal Response Treatment (PRT)
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Stanford University
Infantile Refsum's Disease, Zellweger Syndrome
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
OBJECTIVES: I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism II.
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders
- Trial name
- NCT00007020
- Testing
- Cholic Acids
- Where
- United States
- Volunteers
- 85 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Visual Rehabilitation, Visual Impairment, Autism Spectrum Disorder, Developmental Disabilities, Intellectual Disability, Behavioral Symptoms
Evaluation of a Program to Enhance Ophthalmic Compliance in Children and Adolescents With Special Needs
The goal of this clinical trial is to learn about the effects of the structured program incorporating contextual, behavioral, and cognitive strategies to improve compliance of children and adolescents with special needs during…
- Trial name
- NCT06717282
- Testing
- visual rehabilitation for ophthalmic compliance, Using contextual, behavioral, and cognitive-based stragegies to develop training programs
- Where
- Taiwan
- Volunteers
- 20 people
- Sponsor
- National Taiwan University Hospital
Fragile X Syndrome
An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome
This is a 4-year, open-label extension (OLE) study for subjects completing one of two double-blind clinical trials with BPN14770, Study BPN14770-CNS-301 (in adult males) and Study BPN14770-CNS-204 (in adolescent males).
- Trial name
- NCT05367960
- Testing
- Zatolmilast/ BPN14770
- Where
- 17 sites in United States
- Volunteers
- 314 people
- Sponsor
- Tetra Discovery Partners
Williams-Beuren Syndrome, Micro-duplication 7q11.23 Syndrome
Quantification of Elastin Markers Synthesis in Williams-Beuren Syndrome and 7q11.23 Micro-duplication Syndrome
Introduction: Williams-Beuren syndrome is a rare genetic disorder caused by a 7q11.23 microdeletion. The phenotype associates vasculopathy (arterial stenosis, hypertension), dimorphism and intellectual disability.
- Conditions
- Williams-Beuren Syndrome, Micro-duplication 7q11.23 Syndrome, Vasculopathy
- Trial name
- ELAST7 (NCT04051086)
- Testing
- Physical examination and Urine and blood samples, Urine and blood samples
- Where
- France
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
Prader Willi Syndrome, Obesity
Characterization of Transcriptional Regulators of Ghrelin Hormone Which Causes Genetic Obesity
The problem point of the Prader-Willi Syndrome (PWS) patient is the obesity which is intense and the plasma ghrelin level which increases unusual from the recently PWS patients was discovered.
- Trial name
- NCT01404624
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- Samsung Medical Center
Prader-Willi Syndrome
Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome
The primary objective of this study is to assess the efficacy of cannabidiol oral solution on hyperphagia-related behavior in patients with Prader-Willi Syndrome (PWS).
- Trial name
- NCT02844933
- Testing
- Cannabidiol
- Where
- 7 sites in United States
- Volunteers
- 7 people
- Sponsor
- Radius Pharmaceuticals, Inc.
X-linked Adrenoleukodystrophy
IT and IV Lentiviral Gene Therapy for X-ALD
This is a Phase I/II clinical trial of gene therapy for treating X-linked adrenoleukodystrophy using a high-safety, high-efficiency, self-inactivating lentiviral vector (LV) TYF-ABCD1 to functionally correct the defective gene.
- Trial name
- NCT03727555
- Testing
- Intrathecal and intravenous LV gene therapy
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Self-Injurious Behavior, Mental Retardation
Self-Injury: Diagnosis and Treatment
Self-injurious behavior is behavior in which a person hurts or harms himself. This behavior sometimes occurs in people with mental retardation or autism.
- Trial name
- NCT00065936
- Testing
- Naltrexone hydrochloride, Transcutaneous sensory nerve stimulation
- Where
- 2 sites in United States
- Volunteers
- 37 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Prader-Willi Syndrome
Study to Assess the Efficacy and Safety of Eutropin in Prader-Willi Syndrome
Evaluate the efficacy and safety after treatment of Eutropin® inj. compared to Genotropin® in infants/toddlers with Prader-Willi syndrome
- Trial name
- NCT02204163
- Testing
- Eutropin, Genotropin
- Where
- 3 sites in South Korea
- Volunteers
- 34 people
- Sponsor
- LG Life Sciences
Menkes Disease
Copper Histidinate Treatment for Menkes Disease
This expanded access protocol provides subcutaneous copper histidinate for Menkes disease patients under 6 years of age.
- Trial name
- NCT04074512
- Testing
- copper histidinate
- Where
- 20 sites in United States
- Sponsor
- Sentynl Therapeutics, Inc.
Prader-Willi Syndrome
Cortisol Activity in Patients With Prader-Willi Syndrome and Healthy Controls
The purpose of the study is to find out if people with Prader-Willi syndrome have a difference in the protein which changes inactive cortisone to the active stress hormone cortisol.
- Trial name
- NCT00932932
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Children's Mercy Hospital Kansas City
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
Intellectual Disability
Mental Health Crises in Youth With IDDs
This study is an unmatched, case-control study of 150 youth (Ages 7-17) with a parent reported Intellectual Developmental Disability (IDD) who present to Rady Children's Hospital Emergency Department with a Mental Health Crisis…
- Trial name
- NCT05471232
- Testing
- Genomic sequencing and molecular diagnostic results, if any., EHR-Based Network Model
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Rady Pediatric Genomics & Systems Medicine Institute
Intellectual Disability, Mild, Educational Problems
Therapy Program for Academic Skills in Children With Intellectual Disability
The aim of this study is to investigate the effect of a physical and occupational therapy program on academic skills in a sample of Egyptian children with intellectual disability.
- Trial name
- NCT05900830
- Testing
- play therapy, common educational methods
- Where
- Egypt
- Volunteers
- 20 people
- Sponsor
- Beni-Suef University
Williams Syndrome
Response Inhibition Training for Children With Williams Syndrome
The investigators will conduct a pilot study investigating the effectiveness of a computerized response inhibition training program at reducing the response inhibition difficulties often seen in children with Williams syndrome…
- Trial name
- WSRIT (NCT02212314)
- Testing
- Response Inhibition Training
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of Wisconsin, Milwaukee
Obesity, Prader-Willi Syndrome
Increased Expression of Adiponectin Receptor 2 in the Mononuclear Cells in Children With Prader-Willi Syndrome
The inflammatory process is involved in the pathogenesis of obesity. Prader-Willi syndrome (PWS) is a genetic model of syndromic obesity.
- Trial name
- NCT00800852
- Volunteers
- healthy volunteers welcome
- Sponsor
- Samsung Medical Center
Attention Deficit Disorder With Hyperactivity, Bipolar Disorder
Genetic Aspects of Neurologic and Psychiatric Disorders
The purpose of this study is to improve the understanding of the genetic causes of specific neurologic and psychiatric disorders.
- Conditions
- Attention Deficit Disorder With Hyperactivity, Bipolar Disorder, Mental Disorder Diagnosed in Childhood, Mental Retardation
- Trial name
- NCT00001544
- Where
- United States
- Volunteers
- 1,227 people
- Sponsor
- National Institute of Mental Health (NIMH)
Fragile X Syndrome, Autism Spectrum Disorders
Acamprosate in Youth With Fragile X Syndrome
Fragile X syndrome (FXS) is the most common inherited form of developmental disability. FXS is inherited from the carrier parent, most often the mothers.
- Trial name
- NCT01300923
- Testing
- Acamprosate
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Indiana University