Clinical trials: Disabilities, Intellectual
Brain Disorders, Aicardi Syndrome
Brain Development Research Program
Dr. Elliott Sherr and his collaborators at University of California, San Francisco (UCSF) are studying the genetic causes of disorders of cognition and epilepsy, in particular disorders of brain development that affect the corpus…
- Trial name
- NCT00305305
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- University of California, San Francisco
Fragile X Syndrome, Metformin
Metformin in Children With Fragile X Syndrome
This study is a controlled trial of metformin in children with fragile X syndrome(FXS). The age of FXS children range from 2 to 16 years old.
- Trial name
- NCT05120505
- Testing
- Metformin
- Where
- China
- Volunteers
- 37 people
- Sponsor
- Children's Hospital of Fudan University
Williams Syndrome, Supravalvular Aortic Stenosis
Williams Syndrome (WS) and Supravalvar Aortic Stenosis (SVAS) DNA and Tissue Bank
Williams syndrome is a rare genetic disorder occurring in 1:8000-12,000 individuals. It is caused by the deletion of 25-27 coding genes, including elastin (ELN) on the 7th human chromosome.
- Conditions
- Williams Syndrome, Supravalvular Aortic Stenosis, Cardiovascular Disease
- Trial name
- NCT02706639
- Where
- 3 sites in United States
- Volunteers
- 305 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Prader-Willi Syndrome
Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS
This is a multicenter, open label, multi cohort study to evaluate the efficacy and safety of somatropin in a cohort of Japanese participants with PWS.
- Trial name
- NCT04697381
- Testing
- somatropin - GH naïve pediatric cohort, somatropin - GH treated cohort, somatropin - adult cohort
- Where
- 5 sites in Japan
- Volunteers
- 33 people
- Sponsor
- Pfizer
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.
MDS, Anemia, Aplastic
Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells
The main purpose of this investigational (not approved by the FDA) Phase I research is to test whether transplantation of umbilical cord blood cells can be safely supplemented with a transfusion of a portion of these cells that…
- Conditions
- MDS, Anemia, Aplastic, Inborn Errors of Metabolism, Congenital Marrow Failure
- Trial name
- NCT00692926
- Testing
- ALDHbr Umbilical Cord Blood Cells
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Joanne Kurtzberg, MD
Down Syndrome, Adaptive Behavior
Life Skills Improved in Children With Down Syndrome After Using Assistive Technology
Enhancing independence and quality of life are key modifiable outcomes that are short- and long-term goals for children with Down syndrome (DS) and for their parents.
- Conditions
- Down Syndrome, Adaptive Behavior, Quality of Life
- Trial name
- NCT05343468
- Testing
- The MapHabit System
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- MapHabit, Inc.
Intellectual Disability, Healthy Subjects
Bladder and Bowel Functions, Participation and Quality of Life in Children With Intellectual Disabilities
Many neurodevelopmental, psychiatric, and medical disorders are commonly associated with intellectual disability. The presence of neurodevelopmental and psychiatric (NDP) comorbidities has been reported to negatively impact the…
- Trial name
- NCT07059858
- Where
- Turkey (Türkiye)
- Volunteers
- 102 people
- Sponsor
- Abant Izzet Baysal University
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
Down Syndrome, Activity
Activities and Participation of Children With Down Syndrome
The aim of this study is to examine the activity participation levels of Down syndrome(DS) children and the restrictive or supportive factors affecting their participation levels and to investigate their effects on quality of…
- Conditions
- Down Syndrome, Activity, Participation, Quality of Life
- Trial name
- NCT05152251
- Testing
- Evaluation
- Where
- Turkey (Türkiye)
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Down Syndrome
Effect of Whole Body Vibration Versus Core Stability Exercises on Balance and Muscle Strength in Children With Down Syndrome
Children had Down syndrome often have impaired balance and postural control and result as less active than their peers that can lead to reduced quality of life and movement skills.
- Trial name
- NCT06036069
- Testing
- Conventional physical therapy
- Where
- Egypt
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Ragaee Saeed Mahmoud
Hunter Syndrome
Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment
The study is an extension of two previous studies (HGT-HIT-046 [NCT01506141] and SHP609-302 [NCT02412787]). Participants must have completed one of the previous studies.
- Trial name
- NCT06031259
- Testing
- Idursulfase-IT, Elaprase
- Where
- 4 sites in Canada, France
- Volunteers
- 6 people
- Sponsor
- Takeda
Down Syndrome
Effect Of Brain Gym Exercises On Postural Stability In Children With Down Syndrome
This study will be conducted to detect the effect of brain gym exercises on postural stability in children with down syndrome .
- Trial name
- NCT05769556
- Testing
- Brain Gym Exercises, Traditional physiotherapy
- Where
- Egypt
- Volunteers
- 32 people
- Sponsor
- Aliaa Altohamy Goda
Prader-Willi Syndrome
Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome
This is a single-center, open-label, single-arm study with a double-blind, placebo-controlled, randomized withdrawal extension. Patients are initiated on a DCCR dose of about 1.5 mg/kg (maximum starting dose of 145 mg) and are…
- Trial name
- NCT02034071
- Testing
- DCCR
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Essentialis, Inc.
Down Syndrome
Correlation Between Optic Nerve Vessel Anomalies, Serum Angiogenic Factors and Renal Anomalies in Down Syndrome Children
In approximately half of individuals with Down syndrome, an higher than normal number of vessels cross the optic disc margin. Investigator hypothesize that early retinal vessel branching occurs due to inhibition of angiogenesis…
- Trial name
- DOPANUR (NCT03206957)
- Testing
- Funduscopic examination and retinal photography, Serum levels of endostatin and angiogenesis factors, Renal and low urinary tract Doppler ultrasound
- Where
- Belgium
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Queen Fabiola Children's University Hospital
Learning Disabilities, Cerebral Palsy
Efficacy of AAC for Functional Communication
There is a considerable number children who are unable to speak, and our currently not being provided with any AAC options in their therapeutic facilities.
- Conditions
- Learning Disabilities, Cerebral Palsy, Down Syndrome
- Trial name
- NCT04583332
- Testing
- Rhymes, Individual Items in Rhymes, Objects
- Where
- Pakistan
- Volunteers
- 12 people
- Sponsor
- Riphah International University
Rett Syndrome
Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)
The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome.
- Trial name
- NCT06152237
- Testing
- TSHA-102
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 6 people
- Sponsor
- Taysha Gene Therapies, Inc.
I Cell Disease, Fucosidosis
Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
OBJECTIVES: I. Evaluate bronchoalveolar lavage fluid and serum obtained from pediatric patients with storage disorders prior to allogeneic hematopoietic stem cell transplantation (HSCT) for the presence of proinflammatory…
- Conditions
- I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy, Adrenoleukodystrophy
- Trial name
- NCT00005900
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Fairview University Medical Center
Autism, Mental Retardation
MR Scanning of Very Young Children With Severe Developmental Disorders
The specific is to study the MR morphologic and spectroscopic brain correlates and predictors of development in children with severe developmental disorders (autistic spectrum disorders and/or mental retardation and/or language…
- Conditions
- Autism, Mental Retardation, Language Disorder
- Trial name
- NCT00156221
- Where
- Netherlands
- Sponsor
- UMC Utrecht
Chronic Pain (back / Neck)
Impact of a Pain Neuroscience Education Program on Primary Students with and Without Intellectual Disabilities
This observational study evaluates the impact of a Pain Neuroscience Education (PNE) program on primary school students in Extremadura, Spain.
- Trial name
- NCT06664606
- Testing
- Pain Neuroscience Education Program (PNE)
- Where
- Spain
- Volunteers
- 413 people, healthy volunteers welcome
- Sponsor
- Universidad de Extremadura