Clinical trials: Disabilities, Intellectual
Prader-Willi Syndrome, Aggression
Guanfacine Extended Release for the Reduction of Aggression and Self-injurious Behavior Associated With Prader-Willi Syndrome
This is a placebo-controlled clinical trial to assess whether Guanfacine Extended Release (GXR) reduces aggression and self injurious behavior in individuals with Prader Willi Syndrome (PWS).
- Conditions
- Prader-Willi Syndrome, Aggression, Self-Injurious Behavior, Pathologic Processes
- Trial name
- PWS-GXR (NCT05657860)
- Testing
- Guanfacine Extended Release
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Maimonides Medical Center
Prader-Willi Syndrome, Self-Injurious Behavior
Treatment of Self-Injurious Behavior in Individuals With Prader-Willi Syndrome
Prader-Willi syndrome (PWS) is a genetic disorder usually caused by the deletion of a specific gene. One of the symptoms of PWS is self-injurious behavior (SIB); a common form of SIB in PWS patients is skin picking.
- Trial name
- NCT00065923
- Testing
- Topiramate
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Inherited Metabolic Diseases, Lysosomal Storage Disorders
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Eligible research subjects will receive an unrelated umbilical cord blood transfusion as a possible cure for their inherited metabolic disease.
- Conditions
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism
- Trial name
- UCBT-002 (NCT00654433)
- Testing
- ALD-101
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- Aldagen
Gaucher's Disease
Effects of Enzyme Replacement in Gaucher's Disease
Gaucher disease is a lysosomal storage disease resulting from glycocerebroside accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase.
- Trial name
- NCT00001289
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
MECP2 Duplication Syndrome
A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)
Methyl-CpG binding protein 2 (MECP2) is a dosage-sensitive, X-linked gene critical for central nervous system development and functional maintenance, which gain-of-function causes MECP2 duplication syndrome (MDS).
- Trial name
- HERO (NCT06615206)
- Testing
- HG204
- Where
- China
- Volunteers
- 6 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
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
Intellectual Disability, Mild to Moderate
Adaptive Mixed Reality for Students With Intellectual Disabilities
This study design investigates the empirical efficacy of an Adaptive Mixed Reality Learning Environment (AMRLE) tailored for educable adolescents and young adults (aged 10-20) diagnosed with mild-to-moderate intellectual…
- Trial name
- AMRLE (NCT07647926)
- Testing
- Adaptive Mixed Reality Learning Environment (AMRLE)
- Where
- Egypt
- Volunteers
- 44 people
- Sponsor
- King Salman Center for Disability Research
Intellectual Disability
Compassionate Imagery for People With An Intellectual Disability
The current study aims to explore the adaptation of compassionate imagery for people with an intellectual disability who are experiencing mental health difficulties.
- Trial name
- NCT05339191
- Testing
- Compassionate image workshop
- Where
- United Kingdom
- Volunteers
- 4 people
- Sponsor
- NHS Lanarkshire
Alcohol Use, Drug Use
Effectiveness of Take it Personal!
A controlled pre-post design study on Take it Personal! has demonstrated effectiveness in reducing the frequency and severity of youth use of alcohol, cannabis or other illicit drugs.
- Conditions
- Alcohol Use, Drug Use, Mild Intellectual Disability, IQ 50-70, Borderline Intellectual Disabilities (Intelligence Quotient 70-85)
- Trial name
- NCT05843474
- Testing
- Take it Personal!
- Where
- Netherlands
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Behavioural Science Institute
Learning Disabilities, Cerebral Palsy
Efficacy of AAC for Functional Communication
There is a considerable number children who are unable to speak, and our currently not being provided with any AAC options in their therapeutic facilities.
- Conditions
- Learning Disabilities, Cerebral Palsy, Down Syndrome
- Trial name
- NCT04583332
- Testing
- Rhymes, Individual Items in Rhymes, Objects
- Where
- Pakistan
- Volunteers
- 12 people
- Sponsor
- Riphah International University
Intellectual Disability, Neurodevelopmental Disorder (Diagnosis)
Integration of New Generation Multi-omics Analyses for the Diagnosis of Genetic Neurodevelomental Disorders
Neurodevelopmental disorders (NDDs), including intellectual disability (ID), represent the most common indication for genetic testing.
- Conditions
- Intellectual Disability, Neurodevelopmental Disorder (Diagnosis), srGS, lrGS
- Trial name
- NEXTOMIX (NCT07755098)
- Testing
- Re-analysis of available srGS data, Re-analysis of available srGS data, followed by complementary analyses (lrGS sequencing, mRNA, and GMOs), srGS sequencing
- Where
- France
- Volunteers
- 132 people
- Sponsor
- Centre Hospitalier Universitaire Dijon
Chronic Troublesome Sialorrhea, Cerebral Palsy
Clinical Study to Investigate the Efficacy and Safety of NT 201 Compared to Placebo in the Treatment of Chronic Troublesome Drooling Associated With Neurological Disorders and/or Intellectual Disability
The objective of this study is to investigate the efficacy and safety of NT 201 compared with placebo for the treatment of chronic troublesome sialorrhea associated with neurological disorders (e.g.
- Conditions
- Chronic Troublesome Sialorrhea, Cerebral Palsy, Stroke, Traumatic Brain Injury
- Trial name
- SIPEXI (NCT02270736)
- Testing
- NT 201
- Where
- 31 sites in Georgia, Hungary and 4 more
- Volunteers
- 256 people
- Sponsor
- Merz Pharmaceuticals GmbH
Healthy, Fragile X Syndrome
Aromatase Activity and Ovarian Growth Factors in African-American Versus Caucasian Women
The purpose of the study is to understand how the ovarian follicle (the fluid filled structure in the ovary that contains the egg) makes estrogen and other hormones during normal aging, in women with different ethnic backgrounds…
- Trial name
- NCT00334971
- Testing
- Follicle Aspiration
- Where
- United States
- Volunteers
- 116 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Down Syndrome (Trisomy 21)
AEF0217 in Participants With Down Syndrome
The goal of this clinical trial is to identify if AEF0217 show an improvement in adaptive behaviors (daily life activities) in adults and older adolescents with Down Syndrome. It will also learn about the safety of AEF0217.
- Trial name
- NCT07334912
- Testing
- AEF0217 100 µg, AEF0217 300 µg
- Where
- 10 sites in France, Italy and 1 more
- Volunteers
- 188 people
- Sponsor
- Aelis Farma
Rett Syndrome
An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome
To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls with Rett syndrome
- Trial name
- DAFFODIL™ (NCT04988867)
- Testing
- Trofinetide
- Where
- 7 sites in United States
- Volunteers
- 15 people
- Sponsor
- ACADIA Pharmaceuticals Inc.
Rett Syndrome
Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls and women with Rett syndrome
- Trial name
- LILAC™ (NCT04279314)
- Testing
- Trofinetide
- Where
- 21 sites in United States
- Volunteers
- 154 people
- Sponsor
- ACADIA Pharmaceuticals Inc.
Down Syndrome (DS), Supplementation
Effects of Chronic Creatine and Beta-Hydroxy-Beta-Methylbutyrate Supplementation on Oxidative Stress, Inflammation, Muscle Strength, and Body Composition in Individuals With Down Syndrome
Purpose: This study will test whether 8 weeks of creatine and HMB supplementation can improve muscle strength, body composition, balance, and overall health in people with Down syndrome.
- Trial name
- NCT07213063
- Testing
- Nutrition education, Supplement
- Where
- Spain
- Volunteers
- 32 people
- Sponsor
- Universidad de Burgos
Adrenomyeloneuropathy Without Cerebral Involvement
Epidural Spinal Cord Stimulation for Lower-limb Impairment in Adrenomyeloneuropathy
Adrenal spinal neuropathy (AMN) is a rare X-linked genetic disease caused by mutations in the ABCD1 gene, and belongs to a special type of adrenal leukodystrophy.
- Trial name
- EPIC-AMN (NCT06796920)
- Testing
- Spinal cord stimulation
- Where
- 4 sites in China
- Volunteers
- 10 people
- Sponsor
- Third Military Medical University
Rett Syndrome
Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome
Rett syndrome (RTT) is a serious neurodevelopmental disorder that has a significant impact on patients and their families. Patients suffer from severe social dysfunction and poor quality of life, and there is currently no…
- Trial name
- NCT06856759
- Testing
- Intrathecal injection of AAV-MECP2 for the treatment of Rett syndrome
- Where
- China
- Volunteers
- 8 people
- Sponsor
- Guangzhou Women and Children's Medical Center
Down Syndrome
A Study of RO5186582 in Down Syndrome Among Children 6 to 11 Years of Age
This study will evaluate the safety, tolerability, efficacy, and pharmacokinetic and pharmacodynamic activity of 3 different dosages of RO5186582 compared with placebo.
- Trial name
- NCT02484703
- Testing
- RO5186582
- Where
- 9 sites in United States
- Volunteers
- 45 people
- Sponsor
- Hoffmann-La Roche
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