Clinical trials: Disabilities Multiple
Down Syndrome
Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children
Evaluation of the following in very young children with Down syndrome: the efficacy of systematic treatment with L-thyroxine at controlled doses (clinically and by ultrasensitive thyreostimulating hormone (TSH) assay), the…
- Trial name
- ACTHYF (NCT01576705)
- Testing
- thyroid hormone and folinic acid
- Where
- France
- Volunteers
- 175 people
- Sponsor
- Institut Jerome Lejeune
Hypofibrinogenemia, Afibrinogenemia
Human Fibrinogen Concentrate in Pediatric Cardiac Surgery
The goal of the study is to determine whether the use of Human Fibrinogen Concentrate (RiaSTAP) will decrease blood loss and the need for component blood therapy in neonates and infants undergoing cardiopulmonary bypass.
- Conditions
- Hypofibrinogenemia, Afibrinogenemia, Bleeding Disorders
- Trial name
- RiaSTAP (NCT02822599)
- Testing
- RiaStAP, Saline
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Nicklaus Children's Hospital f/k/a Miami Children's Hospital
Down Syndrome, Trisomy 21
Down Syndrome Metabolic Health Study
The purpose of this research study is to determine which measures best capture cardiovascular disease (CVD) risk and type 2 diabetes (T2DM) risk in children and adolescents with Down syndrome (DS).
- Trial name
- NCT01821300
- Where
- 2 sites in United States
- Volunteers
- 257 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Smith-Lemli-Opitz Syndrome
Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome
The purpose of this study is to determine whether supplementation with an oil-based cholesterol suspension will correct the biochemical abnormalities in cholesterol and its precursors in individuals with the Smith-Lemli-Opitz…
- Trial name
- NCT00272844
- Testing
- crystalline cholesterol oil-based suspension
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Boston Children's Hospital
Down Syndrome
Rivastigmine Study in Adolescents With Down Syndrome
The purpose of this study is to determine if short term use of rivastigmine can improve functional abilities (for example, language, memory, and executive function) in adolescents with Down syndrome.
- Trial name
- DS-Riv (NCT01084135)
- Testing
- Rivastigmine
- Where
- 2 sites in United States
- Volunteers
- 42 people
- Sponsor
- Duke University
Prader-Willi Syndrome
Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome
The primary objective of this study is to assess the efficacy of cannabidiol oral solution on hyperphagia-related behavior in patients with Prader-Willi Syndrome (PWS).
- Trial name
- NCT02844933
- Testing
- Cannabidiol
- Where
- 7 sites in United States
- Volunteers
- 7 people
- Sponsor
- Radius Pharmaceuticals, Inc.
PTEN Gene Mutation, PTEN Hamartoma Tumor Syndrome
RAD001 and Neurocognition in PTEN Hamartoma Tumor Syndrome
Phosphatase and TENsin homolog (PTEN) gene germline mutations are associated with a spectrum of clinical manifestations characterized by neurocognitive deficits, intellectual disability, autism symptomatology, skin lesions…
- Trial name
- NCT02991807
- Testing
- RAD001
- Where
- 3 sites in United States
- Volunteers
- 46 people
- Sponsor
- Boston Children's Hospital
Prader Willi Syndrome
Evaluation of Tolerance, Suckling and Food Intake After Repeated Nasals Administrations of Oxytocin in PWS Infants
The Prader-Willi syndrome (PWS) includes severe neonatal hypotonia with impaired suckling leading to failure to thrive in the most severe cases, subsequently followed by an early onset of morbid obesity with hyperphagia and…
- Trial name
- OTBB2 (NCT02205034)
- Testing
- oxytocin
- Where
- France
- Volunteers
- 18 people
- Sponsor
- University Hospital, Toulouse
Down Syndrome, Postural Balance
Balance Bike Training in Down Syndrome
Background: Children with Down syndrome (DS) present with hypotonia, impaired balance and poor gross motor proficiency which lead to difficulty learning new motor skills, decreased engagement in physical activity, and limited…
- Trial name
- NCT03677245
- Testing
- Strider Balance Bike
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- University of Mississippi Medical Center
Intellectual Disability, Autism
Enhancing Genomic Laboratory Reports to Enhance Communication and Empower Patients
Current lab reports are designed to communicate results from the laboratory to the provider; they are not designed to be accessible to patients.
- Conditions
- Intellectual Disability, Autism, Multiple Congenital Anomalies
- Trial name
- NCT02504502
- Testing
- enhanced genomic report
- Volunteers
- 52 people
- Sponsor
- Geisinger Clinic
Down Syndrome
Gait in Children With Down Syndrome While Wearing Orthoses
On average, Down syndrome (DS) occurs once in every 700 live births and results in life-long disability and increased risk for comorbidities.1 Individuals with DS are also susceptible to secondary physical impairments and…
- Trial name
- NCT01598350
- Testing
- Supramalleolar Orthoses (Cascade)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Puget Sound
Acute Lymphoblastic Leukemia, Adult B Lymphoblastic Lymphoma
Risk-Adapted Chemotherapy in Treating Younger Patients With Newly Diagnosed Standard-Risk Acute Lymphoblastic Leukemia or Localized B-Lineage Lymphoblastic Lymphoma
This partially randomized phase III trial studies the side effects of different combinations of risk-adapted chemotherapy regimens and how well they work in treating younger patients with newly diagnosed standard-risk acute…
- Conditions
- Acute Lymphoblastic Leukemia, Adult B Lymphoblastic Lymphoma, Ann Arbor Stage I B Lymphoblastic Lymphoma, Ann Arbor Stage II B Lymphoblastic Lymphoma
- Trial name
- NCT01190930
- Testing
- Cyclophosphamide, Cytarabine, Dexamethasone
- Where
- 243 sites in United States, Australia and 5 more
- Volunteers
- 9,350 people
- Sponsor
- Children's Oncology Group
Hypothyroidism, Trisomy 21
Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients
Children with levothyroxine (T21) have developmental delay and other functional gastrointestinal (GI) issues that may negatively affect L-T4 tolerability and absorption.
- Trial name
- NCT04747275
- Testing
- Liquid stable levothyroxine (L-T4) Tirosint-SOL, Oral tablet levothyroxine (L-T4)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Children's Mercy Hospital Kansas City
Prader-Willi Syndrome
Intranasal Oxytocin for Infants With Prader-Willi Syndrome
The purpose of this study is to compare the change in suck and swallow competency from baseline to morning of day 6 with intranasal oxytocin spray vs placebo in infants/children with Prader-Willi Syndrome who are in nutritional…
- Trial name
- NCT03245762
- Testing
- Oxytocin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- University of Florida
Angelman Syndrome, Nervous System Diseases
Dietary Supplements for the Treatment of Angelman Syndrome
Angelman syndrome (AS) is a complex genetic disorder that affects the nervous system. The purpose of this study is to determine the effectiveness of certain dietary supplements in treating the symptoms of AS.
- Trial name
- NCT00348933
- Testing
- Betaine, Creatine, Metafolin
- Where
- 4 sites in United States
- Volunteers
- 90 people
- Sponsor
- University of California, San Diego
Prader-Willi Syndrome
A Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Participants With Prader-Willi Syndrome
The objective of this study is to assess the long-term safety and tolerability of Cannabidiol Oral Solution (CBD) in participants with Prader-Willi Syndrome.
- Trial name
- NCT03458416
- Testing
- Cannabidiol Oral Solution
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Radius Pharmaceuticals, Inc.
Down Syndrome
Assessing Malleability of Spatial Abilities in Down Syndrome
Based on available literature and our own preliminary research, the researchers have concluded that persons with Down syndrome (DS) exhibit difficulties in utilizing the specific spatial abilities of mental rotation and…
- Trial name
- NCT05332912
- Testing
- Spatial Ability Experience
- Where
- United States
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- University of Alabama, Tuscaloosa
Respiratory Syncytial Virus Infection
Synagis® Liquid 50mg, 100mg for Intramuscular Injection Special Investigation in Immunocompromised Children With Synagis®
This post marketing observational study (PMOS) was conducted in Japan during the 2013-2014 and 2014-2015 Respiratory Syncytial Virus (RSV) seasons to assess the safety and effectiveness of palivizumab for the prevention of…
- Trial name
- NCT02016690
- Volunteers
- 312 people
- Sponsor
- AbbVie
Prader-Willi Syndrome
A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome
The purpose of this is study is to evaluate the effects of DCCR (diazoxide choline controlled release tablets) in children and adults with Prader-Willi syndrome.
- Trial name
- NCT03440814
- Testing
- DCCR
- Where
- 29 sites in United States, United Kingdom
- Volunteers
- 127 people
- Sponsor
- Soleno Therapeutics, Inc.
Down Syndrome, Cognitive Dysfunction
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 11 To 17
The purpose of this study is to determine the efficacy and safety of donepezil hydrochloride (Aricept) in the treatment of the cognitive dysfunction shown by children with Down syndrome, aged 11 to 17.
- Trial name
- NCT00754052
- Testing
- Aricept (donepezil hydrochloride)
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Eisai Inc.