Clinical trials: Disabilities, Intellectual
Prader-Willi Syndrome
A Pharmacokinetic and Safety Study of CSTI-500 in Subjects With Prader-Willi Syndrome
The purpose of this Phase 1 study is to evaluate the pharmacokinetics (PK) and safety of a single dose of CSTI-500 10 mg in subjects with Prader-Willi syndrome (PWS) between 13 and 50 years of age with a genetically confirmed…
- Trial name
- NCT05504395
- Testing
- CSTI-500
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- ConSynance Therapeutics
Cerebral Palsy, Cerebral Palsy (CP)
Assessing Intellectual and Motor Outcomes in High-risk Infants
Cerebral palsy (CP) is a condition when a baby has a brain injury that affects their movement and muscle tone. Some people with CP can have other developmental issues, like learning impairments, but many do not and have isolated…
- Conditions
- Cerebral Palsy, Cerebral Palsy (CP), Cerebral Palsy Children, High-risk Infants
- Trial name
- AIM-High (NCT06857539)
- Where
- 4 sites in Ireland
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- University College Cork
Down Syndrome, Subclinical Hypothyroidism
Treatment Trial of Subclinical Hypothyroidism in Down Syndrome
The purpose of this research study is to learn about the effects of treating subclinical hypothyroidism (SCH) with thyroid hormone replacement in children and adolescents with Down syndrome (DS).
- Trial name
- NCT01832753
- Testing
- Levothyroxine
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
Intellectual Disabilities (F70-F79), Problem;Behaviour;Adult
Mental Health and Healthcare in Adults With Intellectual Disabilities
Intellectual disability (ID) is a diagnosis characterized by significant limitations both in intellectual functioning and in adaptive behavior as expressed in conceptual, social and practical adaptive skills.
- Conditions
- Intellectual Disabilities (F70-F79), Problem;Behaviour;Adult, Mental Health Issue, Employment
- Trial name
- NCT05703503
- Where
- Norway
- Volunteers
- 214 people, healthy volunteers welcome
- Sponsor
- Norwegian University of Science and Technology
Neurodevelopmental Disorders, Intellectual Disability
Longitudinal Study of Neurogenetic Disorders
The purpose of this study is to analyze patterns in individuals with hnRNP (and other) genetic variants, including their neurological comorbidities, other medical problems and any treatment.
- Conditions
- Neurodevelopmental Disorders, Intellectual Disability, Developmental Delay, Autism Spectrum Disorder
- Trial name
- NCT03492060
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Columbia University
Problem Behavior, Intellectual Disability
Improving Treatment for Children With Intellectual and Developmental Disabilities and Problem Behavior in Schools
Children with intellectual and developmental disabilities (IDDs) often engage in problem behavior, and functional communication training (FCT) is a commonly used treatment for problem behavior in clinical settings.
- Conditions
- Problem Behavior, Intellectual Disability, Developmental Disorder
- Trial name
- NCT05445596
- Testing
- Functional communication training
- Where
- United States
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- Western Kentucky University
Epilepsy, Mental Retardation
Ketogenic Diet vs.Antiepileptic Drug Treatment in Drug Resistant Epilepsy
This is an open randomized controlled study in children with mental retardation and refractory epilepsy in which treatment with ketogenic diet (KD) is compared with treatment with the antiepileptic drug (AED), not tried by the…
- Trial name
- NCT00552526
- Testing
- Ketogenic diet, Antiepileptic drug (AED)
- Where
- Norway
- Volunteers
- 60 people
- Sponsor
- Oslo University Hospital
Mucopolysaccharidosis II
A Study of JR-141 in Patients With Mucopolysaccharidosis II
A Phase II/ III multicenter, open-label, single-group, designed to evaluate the efficacy and safety of study drug for the treatment of the MPS II.
- Trial name
- NCT03568175
- Testing
- JR-141
- Where
- 19 sites in Japan
- Volunteers
- 28 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Prader-Willi Syndrome
CBDV vs Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)
This study aims to examine the feasibility and safety of cannabidivarin (CBDV) as a treatment for children and young adults with PWS.
- Trial name
- NCT03848481
- Testing
- CBDV Compound
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Eric Hollander
Intellectual Disability
Search for Phenotype-modifying Genes in Patients With Intellectual Disabilities.
Each form of intellectual disability under study is a rare disease in its own right, and it is therefore difficult to study the variability of its expression.
- Trial name
- GWAS-2DI (NCT06706934)
- Where
- France
- Volunteers
- 1,400 people, healthy volunteers welcome
- Sponsor
- University Hospital, Bordeaux
Rett Syndrome
A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)
The primary objectives of this study are to evaluate the safety of a single intrathecal (IT) dose of TSHA-102 in females with typical Rett syndrome, to select the TSHA-102 dose with the best benefit/risk profile based on the…
- Trial name
- NCT05606614
- Testing
- TSHA-102
- Where
- 9 sites in United States, Canada
- Volunteers
- 17 people
- Sponsor
- Taysha Gene Therapies, Inc.
Weight Loss, Food Habits
School Intervention With Daily Physical Activity and Healthy Food for Students With an Intellectual Disability.
A whole of school intervention with daily physical activity and healthy food for students with intellectual disabilities. The aim is to provide plenty of concrete examples of healthy life style choices and using school personnel…
- Conditions
- Weight Loss, Food Habits, Mental Retardation
- Trial name
- NCT01291238
- Testing
- Healthier, Smarter, Happier
- Where
- Sweden
- Volunteers
- 145 people
- Sponsor
- Karolinska Institutet
Mild Cognitive Impairment
Effects of Shuttle Balance Training on Balance , Gait and Postural Control in Children With Mild Cognitive Impairment
This study aims to investigate the impact of Shuttle Balance Training (SBT) on balance, gait, and postural control among children diagnosed with mild cognitive impairment (MCI).
- Trial name
- NCT06974890
- Testing
- Shuttle Balance Training, Baseline Treatment
- Where
- Pakistan
- Volunteers
- 24 people
- Sponsor
- Riphah International University
Mucopolysaccharidosis II
Mucopolysaccharidosis Type II Observational
This is an observational study planned to document prospectively disease manifestation and neurocognitive course in pediatric patients with a clinical presentation consistent with neuronopathic ("severe") MPS II undergoing…
- Trial name
- NCT04591834
- Testing
- Observational
- Where
- 3 sites in United States, Canada
- Sponsor
- REGENXBIO Inc.
Intellectual Disability, Autism Spectrum Disorder
Harnessing Communication Preferences
The goal of this clinical trial is to evaluate how preference for communication approach (e.g., using a touch talker versus picture cards) impacts treatment maintenance in the context of treatment to reduce challenging behavior…
- Conditions
- Intellectual Disability, Autism Spectrum Disorder, Self-Injurious Behavior, Communication Disabilities
- Trial name
- NCT07278544
- Testing
- PrefFCT, NonPrefFCT
- Where
- 2 sites in United States
- Volunteers
- 60 people
- Sponsor
- Joel E. Ringdahl
Danon Disease
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (rAAV9) vector containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B)…
- Trial name
- NCT06092034
- Testing
- RP-A501
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Borderline Intellectual Functioning (BIF), Intellectual Disability
Co-design of an AI Rehabilitation Program for Children With Borderline Intellectual Disorder
Co-design of an AI Rehabilitation Program for Children With Borderline Intellectual Disorder
- Trial name
- NCT07625592
- Testing
- Tablet-Based AI Cognitive and Language Rehabilitation Program
- Where
- South Korea
- Volunteers
- 30 people
- Sponsor
- Samsung Medical Center
Williams Syndrome, Language
Phonological Constraints on Language Development in Individuals With Williams Syndrome
Williams syndrome (WS) is a neurodevelopmental disease characterized by mild to moderate intellectual disability and an extremely heterogeneous cognitive profile.
- Conditions
- Williams Syndrome, Language, Phonological Short-Term Memory, EEG-NIRS
- Trial name
- SOUNDS (NCT07285720)
- Testing
- EEG-NIRS protocol, Questionnaire
- Where
- France
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire, Amiens
Rett Syndrome
ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome
ANAVEX2-73-RS-003 is a Phase 2/3, double-blind, randomized, placebo-controlled dose escalation safety, tolerability and efficacy study in patients 5-17 years of age with RTT using endpoints including multiple clinical and…
- Trial name
- EXCELLENCE (NCT04304482)
- Testing
- ANAVEX2-73 oral liquid
- Where
- 12 sites in Australia, Canada and 1 more
- Volunteers
- 92 people
- Sponsor
- Anavex Life Sciences Corp.
Fragile-X Syndrome
EXPLAIN -FragilE X Registry: An exPlorative Longitudinal Study for chAracterIzation, Treatment Pathways and patieNt-related Outcomes
This prospective observational study (registry) on Fragile X syndrome (FXS) is designed to evaluate characteristics, management and patient and caregiver-related outcomes the quality of life of Families and patients with FXS and…
- Trial name
- EXPLAIN (NCT01711606)
- Where
- Germany
- Volunteers
- 75 people
- Sponsor
- Novartis Pharmaceuticals