Clinical trials: Disabilities, Intellectual
Down Syndrome, Gross Motor Development Delay
Investigating Trunk Control in Young Children With Down Syndrome
Overall, the purpose of this dissertation research is to investigate trunk control in young children with DS. This will be done in three studies: 1.
- Trial name
- NCT03544840
- Testing
- Physical Therapy Home Program
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Texas Woman's University
Cerebral Palsy, Autism
Rehabilitation of Children With Multiple Disabilities
The objective of the study is to compare the usual care and treatment of children and youth (0-19 years) with multiple developmental delays and disabilities and their families in Simcoe York with a co-ordinated, navigated…
- Conditions
- Cerebral Palsy, Autism, Down's Syndrome, Brain Injury
- Trial name
- NCT01379443
- Testing
- CTN model of care for children and their families
- Where
- Canada
- Volunteers
- 445 people
- Sponsor
- Children's Treatment Network
Rett Syndrome, Preserved Speech Variant, Mecp2 Duplication Syndrome
Rare Diseases Clinical Research Network: Neurophysiological Correlates
The overall purpose of this project is to advance understanding of the neurophysiological features of Rett syndrome (RTT), MECP2 Duplication (MECP2 Dup) and RTT-related disorders (CDKL5, FOXG1) to gain insight into disease…
- Conditions
- Rett Syndrome, Preserved Speech Variant, Mecp2 Duplication Syndrome, Rett-related Disorders
- Trial name
- NCT03077308
- Testing
- Auditory and Visual Event-related Potentials and EEG
- Where
- 5 sites in United States
- Volunteers
- 185 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
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
Fragile X Syndrome, Fragile X Syndrome (FXS)
Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome
This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of cannabidiol administered as ZYN002 for the treatment of children, adolescent, and young adult patients with…
- Trial name
- RECONNECT (NCT04977986)
- Testing
- ZYN002
- Where
- 27 sites in United States, Australia and 3 more
- Volunteers
- 257 people
- Sponsor
- Harmony Biosciences Management, Inc.
Pyruvate Dehydrogenase Complex Deficiency, Pyruvate Dehydrogenase E1 Alpha Deficiency
Natural History Study of Pyruvate Dehydrogenase Deficiency
Pyruvate dehydrogenase (PDH) deficiency is one of the most common mitochondrial disorders. Patients with this genetic condition have difficulty utilising carbohydrates to produce energy and develop a combination of problems…
- Conditions
- Pyruvate Dehydrogenase Complex Deficiency, Pyruvate Dehydrogenase E1 Alpha Deficiency, Pyruvate Dehydrogenase E1-Beta Deficiency, Pyruvate Dehydrogenase E2 Deficiency
- Trial name
- NCT05257005
- Where
- United Kingdom
- Volunteers
- 50 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Intellectual Disability, Mild to Moderate, Sexual Abuse
Behavioural Skills Training (BST) for Teaching Sexual Abuse Prevention Skills for Children With Intellectual Disabilities
The main goal of the study was to see if Behavioural Skills Training (BST) could improve knowledge about sexual abuse and the ability to resist it among children with intellectual disabilities.
- Trial name
- BST (NCT06732479)
- Testing
- Behavioural Skill Training Programme
- Volunteers
- 120 people
- Sponsor
- University of Canberra
Mild Intellectual Disability
Effects of Modified Pilates on Improving Trunk Control and Balance in Children With Mild Intellectual Disability
This randomized controlled trial will be conducted at Spectrum Clinic, Lahore, over a duration of 8 weeks to assess the impact of Modified Pilates Exercises on trunk control in children with Intellectual Disability (ID).
- Trial name
- NCT06987188
- Testing
- Modified Pilate Exercises, General Physiotherapy Plan
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- Riphah International University
Williams Syndrome, Cardiovascular Disease
WS-SAVE Study (Williams Syndrome Skin and Vessel Elasticity Study)
Background: Williams Syndrome (WS) is a genetic disorder. People with WS have less of a protein that allows parts of the body to stretch than other individuals.
- Conditions
- Williams Syndrome, Cardiovascular Disease, Multisystem Developmental Disorder, Elastin Gene Deletion
- Trial name
- NCT02692846
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sjögren-Larsson Syndrome
A Study of Topical NS2 Cream to Treat Ichthyosis in Sjögren-Larsson Syndrome (SLS)
This is a multi-center, randomized, double-blind, vehicle-controlled, parallel-group study designed to evaluate the safety, pharmacokinetic (PK), and exploratory activity of topically-applied NS2 dermatologic cream administered…
- Trial name
- NCT02402309
- Testing
- Active topical NS2 1% dermatologic cream
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Aldeyra Therapeutics, Inc.
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.
Autism Spectrum Disorder, Intellectual Disability
Effectiveness of Music Therapy in Social Skill Intervention for Children With ASD/ID
Autism spectrum disorder (ASD) is a developmental impairment characterized by persistent deficits in social communication and interactions.
- Trial name
- NCT04557488
- Testing
- Music therapy, Behavioral-based social skill training
- Where
- Hong Kong
- Volunteers
- 77 people
- Sponsor
- Education University of Hong Kong
Down Syndrome
Efficacy of Rivastigmine in Patients With Down Syndrome
This study is to understand the efficacy on language and cognitive function in Down syndrome patients who take Rivastigmine.
- Trial name
- NCT00748007
- Testing
- Rivastigmine
- Where
- Taiwan
- Volunteers
- 14 people
- Sponsor
- National Taiwan University Hospital
Down Syndrome, Acute Lymphoblastic Leukemia
ASIA Down Syndrome Acute Lymphoblastic Leukemia 2016
To evaluate the outcome of a prednisolone and low dose methotrexate based protocol in Down syndrome children with ALL (DS-ALL) in an Asia-wide study.
- Conditions
- Down Syndrome, Acute Lymphoblastic Leukemia, Childhood Cancer
- Trial name
- NCT03286634
- Testing
- Daunorubicin, Prednisolone, Vincristine
- Where
- 10 sites in Hong Kong, Japan and 4 more
- Volunteers
- 60 people
- Sponsor
- National Hospital Organization Nagoya Medical Center
X-Linked Adrenoleukodystrophy, Adrenomyeloneuropathy
Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)
The purpose of this study is to assess the safety, tolerance, pharmacokinetics, and pharmacodynamics of sobetirome, a selective thyroid hormone analog, in adult male X-ALD patients.
- Trial name
- NCT01787578
- Testing
- Sobetirome
- Where
- United States
- Sponsor
- Thomas S. Scanlan
Danon Disease
Danon Disease Natural History Study
The goal of this international observational study is to learn about the natural history of Danon disease in male patients (≥8 years of age) and female patients (8 to 50 years of age).
- Trial name
- NCT06214507
- Testing
- No intervention
- Where
- 16 sites in United States, Australia and 5 more
- Volunteers
- 60 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Down Syndrome, Recurrent B Acute Lymphoblastic Leukemia
A Study to Compare Blinatumomab Alone to Blinatumomab With Nivolumab in Patients Diagnosed With First Relapse B-Cell Acute Lymphoblastic Leukemia (B-ALL)
This phase II trial studies the effect of nivolumab in combination with blinatumomab compared to blinatumomab alone in treating patients with B-cell acute lymphoblastic leukemia (B-ALL) that has come back (relapsed).
- Trial name
- NCT04546399
- Testing
- 3-Dimensional Conformal Radiation Therapy, Asparaginase Erwinia chrysanthemi, Biospecimen Collection
- Where
- 227 sites in United States, Australia and 2 more
- Volunteers
- 461 people
- Sponsor
- National Cancer Institute (NCI)
Chromosomal Aneuploidies (e.g., Trisomy 21, Trisomy 18, Trisomy 13); Prenatal Screening Accuracy
Evaluation of Fetal Bladder Emptying During the 11-14 Weeks' Ultrasound Examination as a Negative Predictive Marker for Chromosomal Abnormalities
Template-Based Brief Summary (Sağlık Profesyonelleri İçin) Study Title: The Negative Predictive Value of First-Trimester Fetal Bladder "Rapid Emptying" (≤40 minutes and post-void ≤2 mm) for Major Chromosomal Aneuploidies: A…
- Trial name
- NCT07165743
- Testing
- 40-Minute Real-Time First-Trimester Ultrasonography
- Where
- Turkey (Türkiye)
- Volunteers
- 270 people, healthy volunteers welcome
- Sponsor
- Haseki Training and Research Hospital
Down Syndrome
A Screening Protocol to Assess Adults and Adolescents With Down Syndrome for Eligibility For Upcoming Study of RG1662 (Study BP27832)
This is a single visit study in which people with Down syndrome aged 12-30 will attend the clinical center to undergo assessments to evaluate their eligibility for potential enrollment in the future BP27832 Phase II clinical…
- Trial name
- NCT01920633
- Where
- 22 sites in United States, Argentina and 5 more
- Volunteers
- 135 people
- Sponsor
- Hoffmann-La Roche
Autism Spectrum Disorder
pBFS Guided rTMS Over Cognitive Control Network for ASD
The aim of this trial is to assess the efficacy and safety of precision neuromodulation for alleviating core symptoms in patients with autism spectrum disorder (ASD) who also have intellectual or developmental delay.
- Trial name
- NCT06255535
- Testing
- active iTBS treatment
- Where
- 6 sites in China
- Volunteers
- 215 people
- Sponsor
- Changping Laboratory
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