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Showing 1–24 of 172 matching trials from the live ClinicalTrials.gov search.
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Completed No phase listed Observational

Pediatric Patients With Metabolic or Other Genetic Disorders

NCT02769949
View directory record Official record
Conditions
Genetic Disorder, Asperger Disorder, Autism Spectrum Disorder, Fragile X Syndrome, Developmental Delay
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Year to 99 Years
Enrollment
96 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 1, 2025 · Synced Jul 21, 2026, 10:09 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Evaluation of Computerized Template Entry for Compensation and Pension (C&P) Examinations

NCT00988546
View directory record Official record
Conditions
Joint Diseases
Interventions
Computer based template for exam documentation, Dictation for exam documentation
Other
Lead sponsor
US Department of Veterans Affairs
Federal
Eligibility
Not listed
Enrollment
37 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 1, 2009 · Synced Jul 21, 2026, 10:09 PM EDT
Terminated Phase 2Phase 3 Interventional Results available

Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)

NCT01433354
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
AFQ056
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
12 Years to 18 Years
Enrollment
119 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
7
States / cities
Sacramento, California • Decatur, Georgia • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2016 · Synced Jul 21, 2026, 10:09 PM EDT
Completed No phase listed Observational

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers

NCT01971957
View directory record Official record
Conditions
Sjogren-Larsson Syndrome (SLS)
Interventions
Not listed
Lead sponsor
University of Nebraska
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
2
States / cities
Omaha, Nebraska • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 7, 2024 · Synced Jul 21, 2026, 10:09 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

RASopathy Biorepository

NCT04395495
View directory record Official record
Conditions
RAS Mutation, Neurofibromatosis 1, Noonan Syndrome, Noonan Syndrome With Multiple Lentigines, Noonan Neurofibromatosis Syndrome, Cardiofaciocutaneous Syndrome, Costello Syndrome, Legius Syndrome, Smith-Kingsmore Syndrome, MTOR Gene Mutation, GATOR-1 Gene Mutation, SYNGAP1-Related Intellectual Disability, DLG4, MAPK1 Gene Mutation
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2065
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced Jul 21, 2026, 10:09 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Rivastigmine Study in Adolescents With Down Syndrome

NCT01084135
View directory record Official record
Conditions
Down Syndrome
Interventions
Rivastigmine, Liquid Placebo
Drug · Other
Lead sponsor
Duke University
Other
Eligibility
10 Years to 17 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
2
States / cities
Baltimore, Maryland • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 5, 2015 · Synced Jul 21, 2026, 10:09 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

NCT03566043
View directory record Official record
Conditions
Mucopolysaccharidosis Type II (MPS II)
Interventions
RGX-121
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
4 Months to 5 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
4
States / cities
Oakland, California • New Brunswick, New Jersey • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 27, 2025 · Synced Jul 21, 2026, 10:09 PM EDT
Completed Not applicable Interventional Results available

North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2

NCT03548779
View directory record Official record
Conditions
Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability, Autism Spectrum Disorder, Hypotonia, Inborn Errors of Metabolism, Movement Disorders, Genetic Disease, Development Delay, Chromosome Abnormality, Hearing Loss, Dysmorphic Features, Skeletal Dysplasia, Congenital Abnormality, Microcephaly, Macrocephaly
Interventions
Pre-visit prep, usual care + exome seq
Behavioral · Diagnostic Test
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
0 Years and older
Enrollment
548 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
3
States / cities
Asheville, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina
Source: ClinicalTrials.gov public record
Updated May 12, 2025 · Synced Jul 21, 2026, 10:09 PM EDT
Completed Phase 2 Interventional

Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome

NCT05322096
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
RGH-706, Placebo
Drug
Lead sponsor
Gedeon Richter Plc.
Industry
Eligibility
17 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
6
States / cities
San Diego, California • Chicago, Illinois • Brooklyn, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2024 · Synced Jul 21, 2026, 10:09 PM EDT
Completed Phase 4 Interventional

Vanderbilt University Spasticity Management Program Evaluation Plan

NCT00179114
View directory record Official record
Conditions
Spasticity
Interventions
Botulinum Toxin Type A, Intrathecal baclofen administered by the Medtronic SyncroMed(TM) pump (ITB)
Drug
Lead sponsor
Vanderbilt University
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2006
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Aug 19, 2007 · Synced Jul 21, 2026, 10:09 PM EDT
Terminated Phase 2 Interventional Results available

Evaluating The Safety Of Donepezil Hydrochloride (Aricept) For Up To 1 Year In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome - Follow-Up To A 10-Week, Double-Blind, Placebo-Controlled Trial

NCT00675025
View directory record Official record
Conditions
Down Syndrome
Interventions
Donepezil hydrochloride (Aricept)
Drug
Lead sponsor
Eisai Inc.
Industry
Eligibility
10 Years to 17 Years
Enrollment
117 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
31
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 24 more
Source: ClinicalTrials.gov public record
Updated Mar 28, 2021 · Synced Jul 21, 2026, 10:09 PM EDT
Completed Phase 4 Interventional Results available

Efficacy and Safety of Memantine Hydrochloride in Enhancing the Cognitive Abilities of Young Adults With Down Syndrome

NCT01112683
View directory record Official record
Conditions
Down Syndrome
Interventions
Memantine, Placebo
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years to 32 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jan 31, 2013 · Synced Jul 21, 2026, 10:09 PM EDT
Completed No phase listed Observational

MatErnal BLood IS Source to Accurately Diagnose Fetal Aneuploidy

NCT01122524
View directory record Official record
Conditions
Pregnant Women, Prenatal Care
Interventions
Not listed
Lead sponsor
Verinata Health, Inc.
Industry
Eligibility
18 Years and older · Female only
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
11
States / cities
Los Angeles, California • San Francisco, California • Fort Collins, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2011 · Synced Jul 21, 2026, 10:09 PM EDT
Enrolling by invitation Not applicable Interventional

Long-term Evaluation of Nasopharyngeal Airway in Hypotonia

NCT06614582
View directory record Official record
Conditions
Hypotonia, Muscle, Obstructive Sleep Apnea, Nasal Airway Obstruction
Interventions
Self-Supporting Nasopharyngeal Airway (ssNPA)
Device
Lead sponsor
University of Michigan
Other
Eligibility
3 Years to 23 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Dec 1, 2025 · Synced Jul 21, 2026, 10:09 PM EDT
Completed Phase 2 Interventional

Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

NCT01253629
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
AFQ056, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 45 Years
Enrollment
175 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
12
States / cities
Phoenix, Arizona • Sacramento, California • Decatur, Georgia + 9 more
Source: ClinicalTrials.gov public record
Updated Dec 22, 2020 · Synced Jul 21, 2026, 10:09 PM EDT
Completed Not applicable Interventional

Macronutrient Regulation of Ghrelin and Peptide YY

NCT02464514
View directory record Official record
Conditions
Obesity, Prader Willi Syndrome
Interventions
High carbohydrate meal, High fat meal
Other
Lead sponsor
Duke University
Other
Eligibility
5 Years to 17 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 9, 2015 · Synced Jul 21, 2026, 10:09 PM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

NCT03633058
View directory record Official record
Conditions
Rett Syndrome
Interventions
Ketamine
Drug
Lead sponsor
Rett Syndrome Research Trust
Other
Eligibility
6 Years to 12 Years · Female only
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
7
States / cities
Birmingham, Alabama • Aurora, Colorado • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated May 27, 2024 · Synced Jul 21, 2026, 10:09 PM EDT
Active, not recruiting Not applicable Interventional

Evaluation of Telehealth Services on Mental Health Outcomes for People With Intellectual and Developmental Disabilities

NCT05336955
View directory record Official record
Conditions
Mental Health Conditions, Developmental Disability
Interventions
Intake and quarterly assessment:, Consultation & coping skills coaching:, 24-hour urgent crisis response and intervention:, Service linkages, referrals, outreach, & training:
Other
Lead sponsor
University of New Hampshire
Other
Eligibility
12 Years to 45 Years
Enrollment
339 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Concord, North Carolina
Source: ClinicalTrials.gov public record
Updated May 10, 2026 · Synced Jul 21, 2026, 10:09 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Stroke and Cerebrovascular Diseases Registry

NCT03281590
View directory record Official record
Conditions
Stroke, Acute Stroke, Acute Brain Injury, Ischemic Stroke, Hemorrhagic Stroke, Transient Ischemic Attack, Subarachnoid Hemorrhage, Cerebral Ischemia, Cerebral Infarction, Cerebral Stroke, Venous Sinus Thrombosis, Cranial
Interventions
Not listed
Lead sponsor
University of New Mexico
Other
Eligibility
1 Year and older
Enrollment
4,594 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2022
U.S. locations
1
States / cities
Albuquerque, New Mexico
Source: ClinicalTrials.gov public record
Updated Aug 21, 2022 · Synced Jul 21, 2026, 10:09 PM EDT
Not listed Not applicable Interventional

Using fMRI to Evaluate Instructional Programs for Children With Developmental Dyslexia

NCT00068835
View directory record Official record
Conditions
Developmental Dyslexia
Interventions
Lindamood-Bell training for dyslexia
Behavioral
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
8 Years to 12 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2005
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 21, 2026, 10:09 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Evaluation of Assessments of Motor Performance, Physical Impairments and Functional Abilities

NCT00747955
View directory record Official record
Conditions
Healthy Volunteers
Interventions
Not listed
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
Not listed
Enrollment
29 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 16, 2019 · Synced Jul 21, 2026, 10:09 PM EDT
Terminated Phase 2 Interventional

An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome

NCT01013480
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Arbaclofen
Drug
Lead sponsor
Seaside Therapeutics, Inc.
Industry
Eligibility
6 Years to 40 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
9
States / cities
Los Angeles, California • Sacramento, California • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Dec 19, 2012 · Synced Jul 21, 2026, 10:09 PM EDT
Not yet recruiting Not applicable Interventional

Evaluation of the Migraceutics® Phase-Specific Dietary Supplement Regimen on Quality of Life, Work Productivity, and Biomarkers in Adult Migraineurs: The Migraceutics® START Study

NCT07634146
View directory record Official record
Conditions
Migraine, Migraine Disease, Migraine Disorder, Migraine Headache, Migraine Disability, Migraine in Adults, Menstrual Migraine, Menstrual Migraine (MM) Headaches
Interventions
Foundational dietary supplement, Luteal phase dietary supplement add-on, Migraine phase dietary supplement add-on, Reference dietary supplement
Dietary Supplement
Lead sponsor
Miracle Wellness LLC
Other
Eligibility
18 Years to 65 Years
Enrollment
920 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Lutherville-Timonium, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 7, 2026 · Synced Jul 21, 2026, 10:09 PM EDT
Recruiting Phase 4 Interventional Accepts healthy volunteers

Evaluating Treatment of ADHD in Children with Down Syndrome

NCT04219280
View directory record Official record
Conditions
Down Syndrome, ADHD
Interventions
Quillivant XR, Placebo
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
6 Years to 17 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2025
U.S. locations
4
States / cities
Sacramento, California • Boston, Massachusetts • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 14, 2025 · Synced Jul 21, 2026, 10:09 PM EDT