Clinical trials: Disabilities, Intellectual
Hunter Syndrome, Mucopolysaccharidosis II
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
The objective of this study is to determine the safety of once weekly dosing of idursulfase 0.5 mg/kg administered by intravenous (IV) infusion for male Hunter syndrome patients ≤ 5 years old.
- Conditions
- Hunter Syndrome, Mucopolysaccharidosis II, MPS II
- Trial name
- NCT00607386
- Testing
- Idursulfase
- Where
- 3 sites in Brazil, Poland and 1 more
- Volunteers
- 28 people
- Sponsor
- Shire
Prader-Willi Syndrome
A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS
The goal of the VNS4PWS clinical study is to test the efficacy, safety, and acceptability of transcutaneous vagus nerve stimulation (tVNS) treatment in people with PWS.
- Trial name
- VNS4PWS (NCT06144645)
- Testing
- tVNS, intermittent stimulation, tVNS, continuous stimulation
- Where
- 18 sites in United States
- Volunteers
- 102 people
- Sponsor
- Foundation for Prader-Willi Research
Down Syndrome
Effects of Lower Limb Fitness Program in Children With DS
Down Syndrome is a congenital disorder resulting from the trisomy of the human chromosome 21, which will determine, among other characteristics, intellectual disability and growth retardation.
- Trial name
- NCT06407895
- Testing
- Lower Limb Fitness Program, Routine Therapy
- Where
- Pakistan
- Volunteers
- 28 people
- Sponsor
- Riphah International University
X-Linked Adrenoleukodystrophy
Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.
- Trial name
- NCT03196765
- Testing
- Sobetirome (NV1205)
- Where
- 13 sites in Argentina, Australia and 6 more
- Sponsor
- NeuroVia, Inc.
Neurodevelopmental Disorders
Clinical Testing of a Real-Time Behavior Measurement Tool: Measuring Outcomes for CHAnge
The efficacy of clinical trials addressing behavioral issues in individuals with intellectual/developmental disabilities (IDD) has traditionally been hampered by lack of objective and sensitive measures.
- Trial name
- MOCHA (NCT03229928)
- Testing
- MOCHA
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
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.
Therapeutic Recreation, Intellectual Disability
The Effects of Therapeutic Recreation Activities on Aberrant Behaviours of Individuals With Intellectual Disabilities
The aim of this study was to determine the effect of Therapeutic Recreation Activities on Aberrant Behavior of Adolescents With Intellectual Disabilities.
- Conditions
- Therapeutic Recreation, Intellectual Disability, Recreational Activities, Aberrant Behaviors
- Trial name
- NCT04546464
- Testing
- Therapeutic Recreation Program
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Selcuk University
Mucopolysaccharidosis II
An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Multicenter, open-label, single-group, designed to evaluate the long term efficacy and safety of study drug for the treatment of the MPS II.
- Trial name
- NCT04348136
- Testing
- JR-141
- Where
- 20 sites in Japan
- Volunteers
- 27 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Rett Syndrome
A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome
The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).
- Trial name
- NCT07503444
- Testing
- fenfluramine hydrochloride
- Where
- 40 sites in United States, Belgium and 10 more
- Volunteers
- 200 people
- Sponsor
- UCB BIOSCIENCES, Inc.
Trisomy 13, Trisomy 18
Prenatal Non-invasive Aneuploidy Test Utilizing SNPs Trial
This prospective blinded study will assess the diagnostic capability of an informatics enhanced SNP based technology (Parental Support) to identify pregnant women who are carrying a fetus with an aneuploidy from fee floating DNA…
- Conditions
- Trisomy 13, Trisomy 18, Trisomy 21, Aneuploidy
- Trial name
- PreNATUS (NCT01545674)
- Testing
- Blood Draw
- Where
- 29 sites in United States, Canada and 4 more
- Volunteers
- 937 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Prader-Willi Syndrome
An Open-Label Study of DCCR Tablet in Patients With PWS
The primary objective of this study is to monitor the long-term safety of DCCR in PWS patients.
- Trial name
- NCT04086810
- Testing
- DCCR
- Sponsor
- Soleno Therapeutics, Inc.
Pyruvate Dehydrogenase Complex Deficiency
Expanded Access Treatment Protocol With DCA for Patients With PDCD
Expanded Access (EA) will provide a transition to continue therapy for those patients who are currently in the open label extension of the Phase III study, SL 1009-01, while also allowing new patients diagnosed with PDCD who meet…
- Trial name
- NCT06931262
- Testing
- Dichloroacetate (DCA)
- Sponsor
- Saol Therapeutics Inc
Down Syndrome, Aneuploidy
Noninvasive Screening for Affected Pregnancies: Assay Development & Optimization in Affected Pregnancies
To collect samples for the purpose of developing and optimizing an in vitro noninvasive prenatal diagnostic (NIPD) test. The NIPD test employs circulating cell free (ccff) DNA extracted from whole blood samples collected from…
- Trial name
- CHARMM-AP (NCT01052688)
- Testing
- Maternal blood draw of 30 to 50ml
- Where
- 10 sites in United States
- Volunteers
- 150 people
- Sponsor
- Sequenom, Inc.
Mucopolysaccharidosis II
A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21)
A Phase II open-label, randomized, parallel group, 2 sites (Brazil), designed to evaluate the safety and efficacy of 3 doses of study drug for the treatment of the MPS II.
- Trial name
- NCT03359213
- Testing
- JR-141
- Where
- 2 sites in Brazil
- Volunteers
- 18 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Oral Health, Intellectual Disability
Supervised Brushing Programme for Intellectual Disabled Students
Intellectual disabled persons faced more dental problems. According to a medical review paper of international and local studies, this group has poorer dental hygiene, more severe gum disease and more untreated caries.
- Conditions
- Oral Health, Intellectual Disability, Randomized Controlled Trial
- Trial name
- NCT03234231
- Testing
- 3-month supervised tooth brushing programme, oral health education talk
- Volunteers
- 602 people
- Sponsor
- MD Equation
Education
Effectiveness of an Early Reading Intervention for Young Children With Intellectual Disabilities Who Need or Use Augmentative and Alternative Communication
The goal of this 2x2 factorial randomized controlled trial is to evaluate the effectiveness of two literacy interventions delivered at different time points, in kindergarten and in first grade, on the development of literacy…
- Trial name
- NCT07116200
- Testing
- Early Literacy Intervention in Kindergarten, Literacy Intervention in First Grade
- Where
- Norway
- Volunteers
- 120 people
- Sponsor
- Ostfold University College
Global Developmental Delay, Cerebral Palsy
Predicting Developmental Disability Type and Mental Retardation Level in Children With General Developmental Delay
The purpose of this study is to find the relationship between the stage and quality of developmental delay during infancy and toddler age, and the final diagnosis that the child gets a few years later (MR, type of PDD, CP or…
- Conditions
- Global Developmental Delay, Cerebral Palsy, Mental Retardation, Pervasive Developmental Disorders
- Trial name
- NCT01430845
- Where
- Israel
- Volunteers
- 150 people
- Sponsor
- Sheba Medical Center
Vaccine Refusal, Compliance, Patient
Vaccinations and People With Disabilities
The goal of this observational study is to learn about vaccinations hesitancy, delay or avoidance in children with chronic diseases, congenital anomalies or disabilities.
- Conditions
- Vaccine Refusal, Compliance, Patient, Disability, Intellectual, Congenital Disorders
- Trial name
- NCT05749419
- Where
- Israel
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Hadassah Medical Organization
Obstructive Sleep Apnea, Hypertonia, Muscle
Self-Supporting Nasopharyngeal Airway (ssNPA) Treating Upper Airway Obstruction in Hypotonia
The researchers are investigating if the Self-Supporting Nasopharyngeal Airway (ssNPA) device can be used in the treatment of obstructive sleep apnea in children with Hypotonic Upper Airway Obstruction (HUAO).
- Conditions
- Obstructive Sleep Apnea, Hypertonia, Muscle, Nasal Airway Obstruction, Tolerance
- Trial name
- NCT05527652
- Testing
- Self-Supporting Nasopharyngeal Airway (ssNPA)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- University of Michigan
Down Syndrome
The Effects Of "Minds In Motion The Maze" on Balance and Gate In Children With Down Syndrome
This study explores the impact of "Minds In Motion: The Maze" on balance and gait in children with Down syndrome-a population often facing motor and coordination challenges.
- Trial name
- NCT06955052
- Testing
- "Minds In Motion The Maze", conventional treatment
- Where
- Pakistan
- Volunteers
- 20 people
- Sponsor
- Riphah International University