Clinical trials: Neurodevelopmental Disability
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Neurodevelopmental Disorders, Autism Spectrum Disorder (ASD)
Single Session Support for Parents of Children With Neurodevelopmental Disabilities Struggling With Anxious Avoidance
This study will inform the development of a brief online program to help parents better understand and respond to anxiety-related behaviors in children with neurodevelopmental disorders (NDDs) such as autism spectrum disorder…
- Conditions
- Neurodevelopmental Disorders, Autism Spectrum Disorder (ASD), Attention-Deficit/Hyperactivity Disorder (ADHD), Intellectual Disability
- Trial name
- EMPOWER-ND (NCT07898592)
- Testing
- EMPOWER-ND
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Boston University Charles River Campus
Neuropsychiatric Disorder, Neurological Disorder
Studying Childhood-onset Behavioral, Psychiatric, and Developmental Disorders
Background: Many psychiatric, behavioral, and developmental disorders are genetic. This means that they tend to run in families. Some begin in childhood, while others do not appear until adulthood.
- Conditions
- Neuropsychiatric Disorder, Neurological Disorder, Neurodevelopmental Disorder, Sleep
- Trial name
- NCT01778504
- Where
- United States
- Volunteers
- 237 people
- Sponsor
- National Institute of Mental Health (NIMH)
Neurodevelopmental Disorders, Intellectual Disability
Multi-Omics Genetic Investigation Strategies to Enhance the Diagnostic Workflow for Rare Diseases
LEOPARD is a multicenter study aimed at improving the genetic diagnosis of patients with neurodevelopmental disorders (NDDs) of unknown etiology, particularly those with moderate-to-severe intellectual disability.
- Conditions
- Neurodevelopmental Disorders, Intellectual Disability, Autism Spectrum Disorder, Genetic Diseases, Inborn
- Trial name
- LEOPARD (NCT07884227)
- Where
- Italy
- Volunteers
- 200 people
- Sponsor
- Istituto Giannina Gaslini
CLIFAHDD, Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay
Aprepitant for the Treatment of CLIFAHDD
There are no drug treatments for individuals with Congenital Contractures of the Limb and Face, Hypotonia, and Developmental Delay (CLIFAHDD), an ultra-rare and severe neurodevelopmental disease.
- Conditions
- CLIFAHDD, Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay, NALCN Channelopathy, Ataxia - Other
- Trial name
- NCT07845825
- Testing
- Aprepitant Powder for Oral Suspension
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- The University of Texas Health Science Center at San Antonio
Autism Spectrum Disorder, Attention Deficit Hyperactivity Disorder
Pai.ACT - An Artificial Intelligence Driven Chatbot Assisted ACT
Limited psychological support for parents of children with special needs in Hong Kong can profoundly impact the child rehabilitation process and the well-being of parent-child dyads.
- Conditions
- Autism Spectrum Disorder, Attention Deficit Hyperactivity Disorder, Neurodevelopmental Disorders, Intellectual Disability
- Trial name
- NCT06086951
- Testing
- Pai.ACT Group, Control Group
- Where
- 2 sites in Hong Kong
- Volunteers
- 60 people
- Sponsor
- Chinese University of Hong Kong
Rett Syndrome
Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome
The primary objectives of this study are to evaluate the safety, tolerability and preliminary efficacy of a single intrathecal (IT) dose of TSHA-102 in pediatric females with typical Rett syndrome.
- Trial name
- ASPIRE (NCT07480564)
- Testing
- TSHA-102
- Where
- 3 sites in United States
- Volunteers
- 4 people
- Sponsor
- Taysha Gene Therapies, Inc.
Intellectual Disability
Effects of Exercise Snacks on Physical Health and Cognitive Function in Individuals With Intellectual Disabilities
Intellectual disability (ID) is a neurodevelopmental disorder accompanied by sedentary lifestyles, which worsens cognitive function and physical health.
- Trial name
- NCT07759232
- Testing
- High-Intensity Interval Training, Exercise Snacks
- Where
- China
- Volunteers
- 24 people
- Sponsor
- University of Macau
Urologic Diseases, Gastrointestinal Diseases
PACT Programme for Parents of Children With SHCN
This randomised controlled trial aims to determine the efficacy of a 12-week, smartphone-based Prosocial-orientated Acceptance and Commitment Training (PACT) programme plus age-appropriate positive parenting advice on the…
- Conditions
- Urologic Diseases, Gastrointestinal Diseases, Vascular Diseases, Cleft Lip and Palate
- Trial name
- NCT05467527
- Testing
- Prosocial-orientated Acceptance and Commitment Training plus positive parenting advice, Positive parenting advice
- Where
- 2 sites in Hong Kong
- Volunteers
- 196 people
- Sponsor
- Chinese University of Hong Kong
Autism Spectrum Disorder, Attention Deficit Hyperactivity Disorder
FAB Programme for Parents of Children With NDD
The study aims to examine the effectiveness of a psychotherapy approach called Facilitator-guided Acceptance and Commitment Bibliotherapy (FAB) in improving the psychological health of parents of young children with…
- Conditions
- Autism Spectrum Disorder, Attention Deficit Hyperactivity Disorder, Neurodevelopmental Disorders, Intellectual Disability
- Trial name
- NCT05872737
- Testing
- Facilitator-guided Acceptance and Commitment Bibliotherapy (FAB), General Parenting Education and Advice
- Where
- 2 sites in Hong Kong
- Volunteers
- 154 people
- Sponsor
- Chinese University of Hong Kong
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
Fragile X Syndrome (FXS), Fragile X Syndrome
Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome
The purpose of this Phase IIa study is to evaluate the safety, tolerability, and effectiveness of CTH120 in adult males with Fragile X syndrome.
- Trial name
- FXS-CTH120-01 (NCT07654114)
- Testing
- CTH120
- Where
- 2 sites in Spain
- Volunteers
- 30 people
- Sponsor
- Connecta Therapeutics, S.L.
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.
Rett Syndrome, Rett Syndrome, Atypical
Rett Syndrome Registry
The Rett Syndrome Registry is a longitudinal observational study of individuals with MECP2 mutations and a diagnosis of Rett syndrome.
- Conditions
- Rett Syndrome, Rett Syndrome, Atypical, Genetic Disease, Genetic Diseases, X-Linked
- Trial name
- RSR (NCT05432349)
- Where
- 19 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- International Rett Syndrome Foundation
Rett Syndrome
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.
- Trial name
- NCT05898620
- Testing
- NGN-401
- Where
- 16 sites in United States, Australia and 1 more
- Volunteers
- 33 people
- Sponsor
- Neurogene Inc.
Neurodevelopmental Disorders, Intellectual Developmental Disorder
High Depth Exome Sequencing on DNA From a Salivary Sample by Mouth Smear.
Despite technological advances, a genetic etiology has been identified in only about 50% to 60% of patients with Neurodevelopmental disorders (NDDs), with a higher diagnostic yield in the syndromic NDD and IDD subgroups.
- Trial name
- SEPASA (NCT07492199)
- Testing
- genetic
- Volunteers
- 50 people
- Sponsor
- Centre Hospitalier Universitaire de Besancon
Neurodevelopmental Disorders, Neurodevelopmental Disorders (NDD)
Intensive Multimodal Neurorehabilitation Targeting Neuroplasticity in Pediatric Neurodevelopmental and Chromosomal Disorders
This observational study evaluates functional and developmental outcomes in pediatric participants undergoing a two week intensive multimodal neurorehabilitation program.
- Conditions
- Neurodevelopmental Disorders, Neurodevelopmental Disorders (NDD), Neurodevelopmental Disorders and Developmental Abnormalities, Developmental Delay (Disorder)
- Trial name
- GEN-HOPE (NCT07493096)
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Healing Hope International
Neurodevelopmental Disorders, Attention Deficit Hyperactivity Disorder
Videoconferencing-based Focused Acceptance and Commitment Therapy for Parents of SHCN Children
The purpose of the proposed non-randomized waitlist-controlled design study is to evaluate the feasibility, acceptability, and potential effectiveness of using a Videoconferencing-based Individual Focused Acceptance and…
- Conditions
- Neurodevelopmental Disorders, Attention Deficit Hyperactivity Disorder, Autism Spectrum Disorder, Development Delay
- Trial name
- NCT05803252
- Testing
- Focused Acceptance and Commitment Therapy, Waitlist Control
- Where
- 5 sites in Hong Kong
- Volunteers
- 104 people
- Sponsor
- Chinese University of Hong Kong
Intellectual Disability, Neurodevelopmental Disorders
Physical Activity and Community EmPOWERment Project
Purpose: Conduct a wait-list randomized controlled trial (RCT) of an inclusive physical activity program called PACE for adults with intellectual disability (ID) who are not yet showing signs of Alzheimer's Disease…
- Conditions
- Intellectual Disability, Neurodevelopmental Disorders, Autism Spectrum Disorder, Down Syndrome
- Trial name
- PACE (NCT06740162)
- Testing
- PACE Program
- Where
- 2 sites in United States
- Volunteers
- 376 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
GNAO1, Dystonia
Prospective Pilot Trial to Address Feasibility and Safety of Oral Zinc in GNAO1 Associated Disorders
The goal of this clinical trial is to investigate feasibility and safety of an oral therapy with zinc in patients affected by Guanine nucleotide-binding protein G(o) subunit alpha (GNAO1) associated disorders.
- Conditions
- GNAO1, Dystonia, Epilepsy, Development Delay
- Trial name
- ZINCGNAO1 (NCT06412653)
- Testing
- Zinc Acetate Dihydrate
- Where
- Germany
- Volunteers
- 13 people
- Sponsor
- Children's University Hospital Cologne, Germany