Clinical trials: Disabilities, Intellectual
Adrenomyeloneuropathy, Adrenoleukodystrophy
A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy
Study of the use of Lorenzo's oil in adults with adrenomyeloneuropathy, the adult form of Lorenzo's oil.
- Trial name
- NCT00545597
- Testing
- Lorenzo's oil
- Where
- United States
- Volunteers
- 240 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Neonatal Seizure, Hypoxic-Ischemic Encephalopathy
Neonatal Seizure Registry - Developmental Functional EValuation
The NSR-DEV study is a longitudinal cohort study of around 280 Neonatal Seizure Registry participants that aims to evaluate childhood outcomes after acute symptomatic neonatal seizures, as well as examine risk factors for…
- Conditions
- Neonatal Seizure, Hypoxic-Ischemic Encephalopathy, Stroke, Intracranial Hemorrhages
- Trial name
- NSR-DEV (NCT04337697)
- Where
- 9 sites in United States
- Volunteers
- 188 people
- Sponsor
- University of California, San Francisco
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.
Prader Willi Syndrome, Obsessive-compulsive Disorder
Behavioral Treatment of Obsessive-Compulsive Symptoms in Youth With Prader-Willi Syndrome: A Pilot Project
Obsessive-compulsive (OC) symptoms are often present among youth with Prader-Willi Syndrome (PWS). They are also associated with considerable problems in the daily functioning of the child and his/her family.
- Trial name
- NCT00742664
- Testing
- Cognitive-behavioral therapy, Wait-list control
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- University of South Florida
Intellectual Disabilities (F70-F79)
Dental Treatment and Oral Rehabilitation for Adult Individuals With Intellectual Disability in General Anaesthesia
This study evaluates the types of dental procedures performed in general anaesthesia for adult persons with intellectual disability, as well as factors affecting the decision to perform the dental treatment in general…
- Trial name
- DTADGA (NCT03823976)
- Testing
- Types of the dental procedures in general anaesthesia
- Where
- Serbia
- Volunteers
- 274 people
- Sponsor
- University of Novi Sad
Prader-Willi Syndrome, Constipation
The Intestinal Function in People With Prader-Willi Syndrome
The projects aim is to investigate the intestinal function of patients suffering from Prader-Willi Syndrome. The methods used are ultrasonographic measurement of the rectal diameter and gastrointestinal transit time
- Conditions
- Prader-Willi Syndrome, Constipation, Incontinence
- Trial name
- NCT01523288
- Where
- Denmark
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Aarhus University Hospital
Obesity Prevention, Pediatric Obesity
Relationship Between Eating Patterns, Body Composition and the Detection of Fatty Liver in Children and Adolescents With Trisomy 21: LiverTy Project
Childhood obesity is a growing public health issue affecting millions of children worldwide, increasing the risk of metabolic and cardiovascular diseases in adulthood.
- Conditions
- Obesity Prevention, Pediatric Obesity, Trisomy 21, Down Syndrome (Trisomy 21)
- Trial name
- LiverTy (NCT06888570)
- Testing
- To evaluate fat liver content in the cases/controls population.
- Where
- Spain
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- IMDEA Food
Prader-Willi Syndrome
Effect of Intranasal Oxytocin on Dysphagia in Children and Adolescents With Prader-Willi Syndrome
This phase 2B is designed to test the effectiveness of intranasal Oxytocin on Prader Willi Syndrome (PWS). This is a prospective, multicentre, randomised, double-blind, Phase 2B clinical study planned to include around 24 PWS…
- Trial name
- DYSMOT (NCT05298085)
- Testing
- Oxytocin nasal spray
- Where
- 4 sites in France
- Volunteers
- 25 people
- Sponsor
- University Hospital, Toulouse
Fragile X Syndrome
Metformin in Children and Adults With Fragile X Syndrome
Fragile X Syndrome (FXS) is caused by loss of FMR1 expression on the X chromosome that leads to increased mRNA translation, which results in hyperactivation of ERK (extracellular signal-regulated kinase) and mTORC1 (mechanistic…
- Trial name
- NCT03722290
- Testing
- Metformin
- Where
- Canada
- Volunteers
- 15 people
- Sponsor
- Université de Sherbrooke
Neurodegenerative Diseases, Leukodystrophy
Home Exercise for Individuals With Neurodegenerative Disease
The primary goal of this study is to address the need for targeted therapeutic interventions for impairments that impact walking in related neurodegenerative diseases.
- Conditions
- Neurodegenerative Diseases, Leukodystrophy, Ataxia, LBSL
- Trial name
- NCT05443906
- Testing
- Home exercise
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Down Syndrome, Obstructive Sleep Apnea
Positive Airway Pressure (PAP) for Children With Down Syndrome (DS) and Obstructive Sleep Apnea Syndrome (OSAS)
Determine the efficacy of family-informed intervention (INT) vs standard clinical care over a period of twelve months in children with obstructive sleep apnea and Down Syndrome.
- Trial name
- NCT04132999
- Testing
- Family-informed intervention, Standard Clinical Care
- Where
- 3 sites in United States
- Volunteers
- 27 people
- Sponsor
- University of Miami
Down Syndrome
Effects of Infant Massage on Development of Down Syndrome Babies
The aim of ths study is to know the effects of infant massage in the maturation of the baby with Down syndrome. Scale Psychomotor Development Early Childhood Brunet-Lézine-Revised will be used to measure the maturational…
- Trial name
- NCT03084497
- Testing
- Infant Massage
- Where
- Spain
- Volunteers
- 32 people
- Sponsor
- University of Seville
Down Syndrome, Fetal Aneuploidy
Specimen Collection From Pregnant Women at Increased Risk for Fetal Aneuploidy
The specimen collection is designed for the purpose of the development of a noninvasive prenatal test for T21.
- Trial name
- NCT01429389
- Where
- 14 sites in United States, Canada
- Volunteers
- 2,000 people
- Sponsor
- Sequenom, Inc.
Behavior Disturbance, Muscle Hypotonia
Effect of Massage Therapy in a Multisensory Environment for Intellectual Disabilities
The aim of this study is to evaluate the effectiveness of multisensory environment (MSE) and/or massage therapy (MT) in residents with severe and profound intellectual disabilities (ID) who received massage therapy in MSE…
- Trial name
- MT-MSE (NCT02120820)
- Testing
- Multisensory environment (MSE), Massage therapy (MT), Control group
- Where
- Hong Kong
- Volunteers
- 240 people
- Sponsor
- The Hong Kong Polytechnic University
Fragile X Syndrome
A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome
The purpose of this study is to determine whether NNZ-2566 is safe and well tolerated in the treatment of Fragile X Syndrome in adolescent and adult males.
- Trial name
- NCT01894958
- Testing
- NNZ-2566
- Where
- 17 sites in United States
- Volunteers
- 72 people
- Sponsor
- Neuren Pharmaceuticals Limited
Velocardiofacial Syndrome, Williams Syndrome
The Psychiatric and Cognitive Phenotypes in Velocardiofacial Syndrome
The purpose of this study is to investigate the Psychiatric and Cognitive Phenotypes in Velocardiofacial Syndrome (VCFS), Williams Syndrome (WS)and Fragile X Syndrome Characterization, Treatment and Examining the Connection to…
- Conditions
- Velocardiofacial Syndrome, Williams Syndrome, Fragile X Syndrome
- Trial name
- VCFS (NCT00768820)
- Testing
- methylphenidate, fluoxetin, risperidone
- Where
- Israel
- Volunteers
- 400 people
- Sponsor
- The Chaim Sheba Medical Center
Down Syndrome
Shared Book Reading to Promote Mental Well-being Among Young People with and Without Down's Syndrome
Abstract Background: With the progress of society and the deepening of people's understanding of Down syndrome (DS), research has shifted focus on how to improve the quality of life and education for young people with DS.
- Trial name
- NCT06813625
- Testing
- Shared book reading intervention
- Where
- China
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Macau
Rett Syndrome
Pharmacological Treatment of Rett Syndrome With Statins
This is a phase 2 , open label, dose escalating study of Lovastatin in Rett syndrome.
- Trial name
- Statins (NCT02563860)
- Testing
- Lovastatin
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Montefiore Medical Center
Down Syndrome
A 4-Week Safety Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia
This is a prospective, randomized, double-blind, placebo-controlled, parallel-group, three-arm, multicenter study of the safety and PK of ELND005 administered orally for 4 weeks.
- Trial name
- DS201 (NCT01791725)
- Testing
- ELND005
- Where
- 3 sites in United States
- Volunteers
- 23 people
- Sponsor
- OPKO Health, Inc.
Down Syndrome, Alzheimer Disease
Brain Outcomes With Lifestyle Change in Down Syndrome
The goal of this study is to determine if weight loss or changes in dietary intake can help prevent of delay adults with Down syndrome from developing Alzheimer's Disease Adults with Down syndrome without dementia will be…
- Conditions
- Down Syndrome, Alzheimer Disease, Obesity
- Trial name
- BOLD (NCT05985486)
- Testing
- Diet, Health Education
- Where
- United States
- Volunteers
- 81 people
- Sponsor
- University of Kansas Medical Center
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