Clinical trials: Disabilities, Intellectual
Prader-Willi Syndrome, Hyperphagia
Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome
This Phase 2b/3 double-blind, placebo-controlled study will evaluate the safety, tolerability, and effects of livoletide on food-related behaviors in patients with Prader-Willi Syndrome (PWS).
- Trial name
- ZEPHYR (NCT03790865)
- Testing
- Livoletide
- Where
- 37 sites in United States, Australia and 6 more
- Volunteers
- 158 people
- Sponsor
- Millendo Therapeutics SAS
Lesch-Nyhan Disease
Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease
The purpose of this study is to determine the effects of ecopipam in patients with Lesch-Nyhan Disease (LND). Ecopipam specifically blocks the actions of one chemical in the brain that helps nerves talk to one another; ecopipam…
- Trial name
- NCT01065558
- Testing
- Ecopipam
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Psyadon Pharma
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
Lysosomal Storage Disease, Peroxisomal Disorder
Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
This study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) in patients with high-risk lysosomal and peroxisomal disorders using a novel…
- Trial name
- NCT01626092
- Testing
- Campath-1H, Clofarabine, Melphalan
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Prader-Willi Syndrome
A Study of GLWL-01 in Patients With Prader-Willi Syndrome
The aim of this study is to evaluate efficacy, safety, and pharmacokinetics of GLWL-01 in the treatment of patients with Prader-Willi Syndrome (PWS).
- Trial name
- NCT03274856
- Testing
- GLWL-01
- Where
- 7 sites in United States, Canada
- Volunteers
- 19 people
- Sponsor
- GLWL Research Inc.
Communicable Diseases, Prevention
SAFE Workplace Intervention for People With IDD
The purpose of this study is to examine the feasibility and efficacy of a systematic training approach targeting behaviors to increase safety and prevention of airborne diseases such as COVID-19 in the workplace for individuals…
- Conditions
- Communicable Diseases, Prevention, Workplace Intervention
- Trial name
- NCT06112249
- Testing
- SAFE Employment Training
- Where
- 2 sites in United States
- Volunteers
- 67 people
- Sponsor
- Temple University
Prader-Willi Syndrome
CBDV vs Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)
This study aims to examine the feasibility and safety of cannabidivarin (CBDV) as a treatment for children and young adults with PWS.
- Trial name
- NCT03848481
- Testing
- CBDV Compound
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Eric Hollander
Down Syndrome
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (HCl) (Aricept) In Treating Cognitive Dysfunction Exhibited By Children With Down Syndrome
The purpose of this study is to determine whether donepezil HCl is effective and safe in improving cognitive dysfunction exhibited by children and adolescents with Down syndrome (DS).
- Trial name
- NCT00570128
- Testing
- Donepezil HCl
- Where
- 31 sites in United States
- Volunteers
- 129 people
- Sponsor
- Eisai Inc.
Fragile X Syndrome (FXS)
Decisional Capacity and Informed Consent in Fragile X Syndrome
The Decisional Capacity and Informed Consent in Fragile X Syndrome (FXS) project is for the Eunice Kennedy Shriver National Institute of Child Health \& Human Development, 1R01HD071987-01A1, and will provide the first…
- Trial name
- NCT02465931
- Testing
- Paper and Pencil Informed Consent, Digital Informed Consent Tool
- Where
- United States
- Volunteers
- 152 people
- Sponsor
- RTI International
Fragile X Syndrome
Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome
This Phase IIb study is designed to assess whether 3 doses of AFQ056 are safe and effective in treating the behavioral symptoms of Fragile X Syndrome.
- Trial name
- NCT01357239
- Testing
- AFQ056
- Where
- 38 sites in United States, Australia and 14 more
- Volunteers
- 139 people
- Sponsor
- Novartis Pharmaceuticals
Fragile X Syndrome
Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome
The study objective is to explore the efficacy, safety and tolerability of STX209 for treatment of irritability in subjects with FSX.
- Trial name
- NCT00788073
- Testing
- STX209
- Where
- 12 sites in United States
- Volunteers
- 63 people
- Sponsor
- Seaside Therapeutics, Inc.
Down Syndrome, Mild Cognitive Impairment
Nicotine Treatment of Cognitive Decline in Down Syndrome
This study will ascertain whether nicotine is safe and tolerable in DS patients, help with dose-ranging of nicotine in DS, look for evidence of enhancements in cognitive functioning, and establish evidence for biological and…
- Trial name
- NCT01778946
- Testing
- Low Dose Nicotine (7mg), Moderate Dose Nicotine (14mg)
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Vanderbilt University Medical Center
Fragile X Syndrome
Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
The purpose of this study is to generate long-term safety, tolerability and efficacy data for AFQ056 in eligible adult patients with FXS who have participated in the CAFQ056A2212 (NCT01253629).study and patients who have…
- Trial name
- NCT01348087
- Testing
- AFQ056
- Where
- 28 sites in United States, Australia and 8 more
- Volunteers
- 148 people
- Sponsor
- Novartis Pharmaceuticals
Learning Disability
Investigating the Clinical and Cost Effectiveness of Lycra Splinting
People with learning disabilities (LDs) experience high levels of health problems, including cerebral palsy, other problems associated with posture, movement and function, and injury (falls are the commonest cause of injury in…
- Trial name
- NCT02345512
- Testing
- Lycra Splinting Garment
- Where
- United Kingdom
- Volunteers
- 11 people, healthy volunteers welcome
- Sponsor
- Glasgow Caledonian University
Mucopolysaccharidosis, Hurler Syndrome
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Rationale: Chemotherapy administration before a donor stem cell transplant is necessary to stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome
- Trial name
- NCT01043640
- Testing
- Campath-1H, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Down Syndrome
Safety, Tolerability and Immunogenicity of ACI-24 Vaccine in Adults With Down Syndrome
The purpose of this study is to test in adults with Down Syndrome the safety, tolerability and immunogenicity of a vaccine, ACI-24.
- Trial name
- 3-Star (NCT02738450)
- Testing
- ACI-24 low dose, ACI-24 high dose
- Where
- 4 sites in United States
- Volunteers
- 20 people
- Sponsor
- AC Immune SA
Obstructive Sleep Apnea, Down Syndrome
Medications for Obstructive Sleep Apnea In Children With Down Syndrome
This is a randomized, double blind, cross-over study of the combination of atomoxetine and oxybutynin (ato-oxy) in children with DS and OSA documented by polysomnography (PSG).
- Trial name
- MOSAIC (NCT04115878)
- Testing
- Atomoxetine and oxybutynin (ato-oxy)
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- University of Arizona
Down Syndrome, Health, Subjective
Developing a Down Syndrome Health Instrument
Although over 200,000 individuals with DS live in the United States, studies to date have focused on outcomes apart from health.
- Trial name
- NCT04631237
- Testing
- Observational, no intervention
- Where
- United States
- Volunteers
- 758 people
- Sponsor
- Massachusetts General Hospital
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
Hunter Syndrome
An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
The objective of this study is to evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do…
- Trial name
- NCT00882921
- Testing
- Idursulfase
- Where
- 6 sites in United States, Brazil and 1 more
- Volunteers
- 26 people
- Sponsor
- Shire