Clinical trials: Multiple Disability
Prader-Willi Syndrome
Characterization of Serum Proteome in Subjects With Prader Willi Syndrome (PROTEOMARKER)
The lack of serum markers that can be used to identify the levels of steatosis in obese subjects, or that can indicate a rapid progression of the metabolic disease, in which it is very often difficult to perform analyzes with…
- Trial name
- PROTEOMARKER (NCT05761184)
- Testing
- Serum collection for proteomic analysis
- Where
- Italy
- Volunteers
- 52 people
- Sponsor
- Istituto Auxologico Italiano
Myotonic Dystrophy 1
Chinese Multicenter Clinical Outcome Cohort Study of Myotonic Dystrophy Type 1 (C-DMCOS-DM1)
Myotonic dystrophy type 1 (DM1) is an autosomal dominant multisystem disorder caused by an expanded CTG trinucleotide repeat in the 3' untranslated region of the DMPK gene.
- Trial name
- C-DMCOS-DM1 (NCT06101940)
- Testing
- MRI scan, Electrocardiography, Pulmonary function test
- Where
- 22 sites in China
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Huashan Hospital
Profound Multiple Intellectual Disabilities
Development of a Visio-behavioral Scale to Evaluate Visual Abilities of Patients With Profound Multiple Intellectual Disabilities (PLH-Vision)
The main objective of this study is to develop and validate a visio-behavioral scale of visual abilities for subjects with profound multiple intellectual disabilities.
- Trial name
- PLH-Vision (NCT03040609)
- Testing
- Visual-behavioral Scale at day 1, Test-retest at the end of 2 weeks, Test at 6 months
- Where
- France
- Volunteers
- 232 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Amyloid Plaque, Beta-Amyloid
A Study to Assess the Effects of ACI-24.060 in Alzheimer's Disease and in Down Syndrome (ABATE Study)
The purpose of this study is to assess the safety, tolerability, immunogenicity and pharmacodynamic effects of ACI-24.060 in subjects with prodromal Alzheimer's disease and in non-demented adults with Down syndrome.
- Conditions
- Amyloid Plaque, Beta-Amyloid, DSAD, Prodromal Alzheimer's Disease
- Trial name
- NCT05462106
- Testing
- ACI-24.060 at Dose A in Study Part 1a, ACI-24.060 at Dose B in Study Part 1a, ACI-24.060 at Dose C in Study Part 1a
- Where
- 26 sites in United States, Spain and 1 more
- Volunteers
- 304 people
- Sponsor
- AC Immune SA
Down Syndrome
Gait Parameters of Persons With Down Syndrome With and Without Orthotics Using GaitRite
This is a research study of persons with Down Syndrome between 2 and 80 years old who are independently walking. The purpose of this study is to see what effect orthotics have on how people with Down Syndrome walk.
- Trial name
- NCT01390558
- Testing
- custom orthotics
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Oregon Health and Science University
Holoprosencephaly, HPE
Clinical and Genetic Studies on Holoprosencephaly
This study will examine how holoprosencephaly (HPE) affects people, how they change over time, and what genes may be involved in the cause of the disorder.
- Conditions
- Holoprosencephaly, HPE, Developmental Delay Disorders, Brain Disorders
- Trial name
- NCT00088426
- Where
- United States
- Volunteers
- 256 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Neurodevelopmental Disorders, Autism Spectrum Disorder
Patterns of Neurodevelopmental Disorders
The purpose of this study is to systematically evaluate the results of medical investigations to identify symptom and biological patterns and common etiologies of neurodevelopmental disorders.
- Conditions
- Neurodevelopmental Disorders, Autism Spectrum Disorder, Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal Infection, Pediatric Acute-Onset Neuropsychiatric Syndrome
- Trial name
- NCT06213090
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Richard Frye
Nutritional Deficiencies, Developmental Delay
Project Grow Smart: Intervention Trial of Multiple Micronutrients and Early Learning Among Infants in India
Project Grow Smart evaluates the impact fortification with multiple micronutrient powders (MNP) vs. placebo (one vitamin) on child development (primary outcome) and on micronutrient status, growth, and morbidity (secondary…
- Trial name
- GrowSmart (NCT01660958)
- Testing
- MNP, Early Learning
- Where
- India
- Volunteers
- 834 people, healthy volunteers welcome
- Sponsor
- University of Maryland, Baltimore
Prader-Willi Syndrome
Mitochondrial Complex I Dysfunction in PWS
Prader-Willi Syndrome (PWS) is characterized by profound infantile hypotonia, growth delay, cognitive impairment, muscle weakness and exercise intolerance.
- Trial name
- NCT03831425
- Testing
- Coenzyme Q10
- Where
- Canada
- Sponsor
- The Hospital for Sick Children
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
- Trial name
- NCT06430385
- Testing
- ION440
- Where
- 12 sites in United States, Austria and 3 more
- Volunteers
- 48 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Severe Stroke, Acute Stroke
Optimizing Early Enteral Nutrition in Severe Stroke
The purpose of this academic lead study is to explore the ideal nutritional support strategy for patient with acute severe stroke.
- Conditions
- Severe Stroke, Acute Stroke, Dysphagia
- Trial name
- OPENS (NCT02982668)
- Testing
- Initial enteral feeding, metoclopramide or mosapride
- Where
- 4 sites in China
- Volunteers
- 306 people
- Sponsor
- Xijing Hospital
Prader-Willi Syndrome
Optimizing the Social Engagement System in Prader-Willi Syndrome: Insights From the Polyvagal Theory
The Polyvagal Theory focuses on how function and structure changed in the vertebrate autonomic nervous system during evolution. The theory is named for the vagus, a major cranial nerve that regulates bodily state.
- Trial name
- NCT03101826
- Testing
- Listening Project Protocol
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Indiana University
Down Syndrome
Effects of Plyometric Exercises on Static and Dynamic Balance of Children With Down,s Syndrome.
Down syndrome is a condition in which a person has an extra chromosome. Typically, a baby is born with 46 chromosomes. Down syndrome is a genetic condition that causes mild to serious physical and developmental problems.
- Trial name
- NCT05951543
- Testing
- plyometric exercises, dynamic balance group control group
- Where
- Pakistan
- Volunteers
- 20 people
- Sponsor
- Riphah International University
Older Adults, ICU
Prognostic Modelling for Prediction of Mortality and Functional Disability in Critically-ill Elderly Patients
Prospective observational study recruiting elderly patients of 60 years and above admitted to Intensive Care Unit (ICU), to study multiple domains of biomarkers ability to predict mortality of patients during intensive care unit…
- Conditions
- Older Adults, ICU, Mortality Prediction, Functional Disability After ICU
- Trial name
- MYELDERLYICU (NCT06163976)
- Where
- 4 sites in Malaysia
- Volunteers
- 329 people
- Sponsor
- Clinical Research Centre, Malaysia
Hunter Syndrome
Hunter Outcome Survey (HOS)
The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.
- Trial name
- HOS (NCT03292887)
- Where
- United States
- Volunteers
- 1,443 people
- Sponsor
- Shire
Down's Syndrome
Treadmill Training With and Without Weighted Ankle Cuffs on Gait Parameters
Down syndrome is a condition in which a person has an extra chromosome. Chromosomes are small "packages" of genes in the body. Down syndrome (DS), also known as trisomy 21, is caused by the presence of all or part of a third copy…
- Trial name
- NCT06176755
- Testing
- Treadmill training with weighted ankle cuffs, Treadmill training
- Where
- Pakistan
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- Riphah International University
Translocation Down Syndrome, Chromosomal Abnormalities
Prevalence of Translocation Phenotyping in Children With Down Syndrome and Their Parents
This research study will investigate a special type of Down syndrome called translocation Down syndrome. While most children with Down syndrome have an extra copy of chromosome 21, about 3-4% have this extra chromosome material…
- Trial name
- PTP-DS (NCT07175168)
- Testing
- No medical or experimental intervention will be administered
- Volunteers
- 230 people
- Sponsor
- Assiut University
Down Syndrome, Obstructive Sleep Apnea Syndrome
Down Syndrome and Continuous Positive Pressure Therapy
The purpose of this study is to evaluate the efficacy and tolerability of Continuous Positive Pressure for SAOS in Down Syndrome patients.
- Trial name
- Morphee (NCT00394290)
- Testing
- Continuous Positive Pressure
- Where
- France
- Volunteers
- 31 people
- Sponsor
- Institut Jerome Lejeune
Kinesthetic Illusion
Animal Fun Program and Kinesthetic Development in Down Syndrome
The purpose of the study is that Animal-fun activities capture children's attention, making them a powerful tool for enhancing kinesthetic awareness and gross motor skills.
- Trial name
- NCT07579559
- Testing
- Animal fun program
- Where
- Pakistan
- Volunteers
- 25 people
- Sponsor
- Riphah International University
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.