Clinical trials: Disability Evaluation
Hunter Syndrome
Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
This long-term study will provide Elaprase treatment to children enrolled in this study and will utilize data from both enrolled patients and Hunter Outcome Survey (HOS) patient registry data to conduct the primary growth…
- Trial name
- NCT02455622
- Testing
- Elaprase for intravenous (IV) infusion
- Where
- 8 sites in United States, Dominican Republic and 6 more
- Volunteers
- 21 people
- Sponsor
- Shire
Down Syndrome
An Online Health Tool About Down Syndrome: Down Syndrome Clinic to You
The investigators conducted a national two-arm, randomized controlled trial of caregivers of individuals with DS to assess the efficacy of DSC2U in assuring adherence to evidence-based guidelines.
- Trial name
- DSC2U (NCT04227197)
- Testing
- Down Syndrome Clinic to You (DSC2U)
- Where
- United States
- Volunteers
- 281 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
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
Hearing Loss
Thai-version Five Minute Hearing Test
This study is aimed to test the sensitivity and specificity of Thai-version Five Minutes Hearing test against the audiometry to screen hearing problem in community.
- Trial name
- Thai_FMHT (NCT01408992)
- Testing
- Audiometry, Thai-Five Minute Hearing Test
- Where
- Thailand
- Volunteers
- 558 people, healthy volunteers welcome
- Sponsor
- Khon Kaen University
Obsessive-Compulsive Disorder, Autism Spectrum Disorder
Riluzole to Treat Child and Adolescent Obsessive-Compulsive Disorder With or Without Autism Spectrum Disorders
This study will examine the effectiveness of riluzole for treating Obsessive-Compulsive Disorder in Youth, Including those with Autism Spectrum Disorders.
- Conditions
- Obsessive-Compulsive Disorder, Autism Spectrum Disorder, Autism, Asperger Disorder
- Trial name
- NCT00251303
- Testing
- Riluzole
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- National Institute of Mental Health (NIMH)
CAT Intervention Group, Control Group
Development and Evaluation CAT and Youth
This R21 provides a multidisciplinary One Health approach to developing and evaluating a novel Cat Assisted Training (CAT) animal assisted intervention (AAI) for early adolescents with developmental disabilities (DD) and their…
- Trial name
- CAT (NCT05950672)
- Testing
- CAT Intervention Group
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Oregon State University
Hunter Syndrome
A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India
The main aim of this study is to learn more about the safety profile of Elaprase in Indian children and adults with hunter syndrome.
- Trial name
- NCT05058391
- Testing
- Elaprase
- Where
- 5 sites in India
- Volunteers
- 5 people
- Sponsor
- Takeda
Growth & Development, Child Development
Resident Surveillance of Pediatric Patient Developmental Status at the Two Month Preventive Care Visit
The purpose of this study is to evaluate the efficacy of individualized performance feedback coupled with an educational module in improving resident performance of the physical and developmental examination component of…
- Conditions
- Growth & Development, Child Development, Internship and Residency, Education, Medical
- Trial name
- NCT00465361
- Testing
- performance feedback with an educational module
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Children's Mercy Hospital Kansas City
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
AMN, AMN Gene Mutation
A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)
This is a Phase 1/2 randomized, blinded, dose-escalation study to evaluate the safety and efficacy of intrathecal (IT) administration of SBT101, a recombinant adeno-associated virus serotype 9 (AAV9) containing a functional copy…
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Trial name
- PROPEL (NCT05394064)
- Testing
- SBT101 Treatment
- Where
- 2 sites in United States, Netherlands
- Volunteers
- 9 people
- Sponsor
- SwanBio Therapeutics, Inc.
Attendance at Treatment, PTSD, Substance Use, and Attendance at Treatment
Evaluation and Treatment of Substance Abuse in Veterans With PTSD Disability Claims
Veterans can apply for compensation and pension (C \& P) benefits for a disability related to military service. The application exam for these benefits provides an opportunity for Veterans returning from service to access VA…
- Trial name
- SBIRT-PTSD (NCT01597856)
- Testing
- SBIRT
- Where
- 2 sites in United States
- Volunteers
- 346 people, healthy volunteers welcome
- Sponsor
- VA Office of Research and Development
Infantile Refsum's Disease, Zellweger Syndrome
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
OBJECTIVES: I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism II.
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders
- Trial name
- NCT00007020
- Testing
- Cholic Acids
- Where
- United States
- Volunteers
- 85 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Prader-Willi Syndrome
Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome
The purpose of this study was to evaluate the effects of a once daily subcutaneous injectable formulation of setmelanotide in obese participants with Prader-Willi syndrome on tolerability, weight loss, and hyperphagia-related…
- Trial name
- NCT02311673
- Testing
- Setmelanotide
- Where
- 5 sites in United States
- Volunteers
- 40 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
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.
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
Smith-Lemli-Opitz Syndrome
Simvastatin Therapy in Smith-Lemli-Opitz Syndrome
This study will evaluate the safety and effectiveness of simvastatin in treating children with Smith-Lemli-Opitz syndrome (SLOS).
- Trial name
- NCT00064792
- Testing
- Simvastatin Susp., OraPlus
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Forbes Porter, M.D.
Down Syndrome, Intellectual Disability
Down Syndrome Memantine Follow-up Study
The purpose of this research study is to learn if the medication Memantine Hydrochloride (the study medication) can help adolescents and young adults with Down syndrome. Dr.
- Trial name
- NCT02304302
- Testing
- Memantine
- Where
- 2 sites in United States, Brazil
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- University Hospitals Cleveland Medical Center
Down Syndrome
Efficacy and Safety of Memantine Hydrochloride in Enhancing the Cognitive Abilities of Young Adults With Down Syndrome
The purpose of this 16-week research study is to determine whether a drug called memantine hydrochloride (memantine) has the potential to help improve memory and other cognitive abilities of young adults with Down syndrome (DS).
- Trial name
- NCT01112683
- Testing
- Memantine
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- University of Colorado, Denver
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
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.