- Conditions
- Rett Syndrome
- Interventions
- Emerald
- Device
- Lead sponsor
- Rett Syndrome Research Trust
- Other
- Eligibility
- Female only
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Smith-Lemli-Opitz Syndrome
- Interventions
- Simvastatin Susp., OraPlus
- Drug
- Lead sponsor
- Forbes Porter, M.D.
- NIH
- Eligibility
- 4 Years to 18 Years
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2010
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- PTEN Gene Mutation, PTEN Hamartoma Tumor Syndrome
- Interventions
- RAD001, Placebo
- Drug
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 5 Years to 45 Years
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 3
- States / cities
- Palo Alto, California • Boston, Massachusetts • Cleveland, Ohio
- Conditions
- Fragile X Syndrome
- Interventions
- acamprosate, Placebo
- Drug
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- 5 Years to 23 Years
- Enrollment
- 46 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2018
- U.S. locations
- 2
- States / cities
- Chicago, Illinois • Cincinnati, Ohio
- Conditions
- Intellectual Disability, Mild, Intellectual Disability, Mild to Moderate, Developmental Disability
- Interventions
- Cheek swab for PGx testing, Medication safety review
- Other
- Lead sponsor
- GalenusRx, Inc.
- Industry
- Eligibility
- 21 Years and older
- Enrollment
- 700 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 2
- States / cities
- Montverde, Florida • The Bronx, New York
- Conditions
- Prader Willi Syndrome
- Interventions
- Intranasal oxytocin, Placebo
- Drug · Other
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 5 Years to 11 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015
- U.S. locations
- 3
- States / cities
- Orange, California • Gainesville, Florida • Kansas City, Kansas
- 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
- Conditions
- Alzheimer Disease, Down Syndrome
- Interventions
- ION269
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 35 Years to 55 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 5
- States / cities
- Indianapolis, Indiana • Kansas City, Kansas • Lexington, Kentucky + 2 more
- Conditions
- Obstructive Sleep Apnea, Down Syndrome
- Interventions
- Atomoxetine and Oxybutynin (ato-oxy)
- Drug
- Lead sponsor
- University of Arizona
- Other
- Eligibility
- 6 Years to 17 Years
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- Tucson, Arizona
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease, Sandhoffs Disease, Wolman Disease, I-Cell Disease, Sanfilippo Syndrome, GM1 Gangliosidosis
- Interventions
- Clofarabine, Total body Irradiation, Melphalan, Hematopoietic Stem Cell Transplantation, Alemtuzumab, mycophenylate mofetil, Cyclosporine A, Hydroxyurea
- Drug · Procedure · Biological + 1 more
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 70 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2014
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Hypothalamic Obesity, Prader-Willi Syndrome, PWS
- Interventions
- Part A: RM-718 or placebo (matched to specific RM-718 dose cohort), Part B: RM-718 or placebo (matched to specific RM-718 dose cohort), Part C: RM-718, Part D: RM-718
- Drug
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.
- Industry
- Eligibility
- 12 Years to 65 Years
- Enrollment
- 150 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2024 – 2028
- U.S. locations
- 7
- States / cities
- Birmingham, Alabama • Chicago, Illinois • Boston, Massachusetts + 3 more
- Conditions
- Down Syndrome
- Interventions
- No Intervention
- Other
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 6 Years to 11 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2016
- U.S. locations
- 3
- States / cities
- Tucson, Arizona • Boston, Massachusetts • Durham, North Carolina
- Conditions
- Intellectual Disability, Autism Spectrum Disorder, Self-Injurious Behavior, Communication Disabilities, Communication, Nonverbal
- Interventions
- PrefFCT, NonPrefFCT
- Behavioral
- Lead sponsor
- Joel E. Ringdahl
- Other
- Eligibility
- 2 Years to 90 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2031
- U.S. locations
- 2
- States / cities
- Athens, Georgia • Iowa City, Iowa
- Conditions
- Self-Injurious Behavior, Mental Retardation
- Interventions
- Naltrexone hydrochloride, Transcutaneous sensory nerve stimulation
- Drug · Procedure
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 4 Years to 25 Years
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1997 – 2002
- U.S. locations
- 2
- States / cities
- Chapel Hill, North Carolina • Morganton, North Carolina
- Conditions
- Prader-Willi Syndrome
- Interventions
- tVNS, intermittent stimulation, tVNS, continuous stimulation
- Device
- Lead sponsor
- Foundation for Prader-Willi Research
- Other
- Eligibility
- 10 Years to 40 Years
- Enrollment
- 102 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 18
- States / cities
- Palo Alto, California • San Diego, California • Aurora, Colorado + 14 more
- Conditions
- Fragile X Syndrome
- Interventions
- MG01CI extended-release tablet
- Drug
- Lead sponsor
- Alcobra Ltd.
- Industry
- Eligibility
- 15 Years to 55 Years
- Enrollment
- 62 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 11
- States / cities
- Phoenix, Arizona • Sacramento, California • Aurora, Colorado + 8 more
- Conditions
- Down Syndrome
- Interventions
- Exercise
- Other
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 10 Years to 17 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Williams Syndrome, Supravalvular Aortic Stenosis, Cardiovascular Disease
- Interventions
- Not listed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 3 Years to 85 Years
- Enrollment
- 159 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2024
- U.S. locations
- 2
- States / cities
- Bethesda, Maryland • St Louis, Missouri
- Conditions
- Hunter Syndrome
- Interventions
- Idursulfase-IT, Elaprase
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- Up to 18 Years · Male only
- Enrollment
- 56 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2024
- U.S. locations
- 3
- States / cities
- Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina
- Conditions
- Mental Retardation, Spasticity
- Interventions
- Not listed
- Lead sponsor
- Vanderbilt University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2007
- U.S. locations
- 1
- States / cities
- Davidson County, Tennessee
- Conditions
- Fragile X Syndrome, Fragile X Mental Retardation Syndrome, Mental Retardation, X Linked, Genetic Diseases, X-Linked, Trinucleotide Repeat Expansion, Fra(X) Syndrome, Intellectual Disability, FXS, Neurobehavioral Manifestations, Sex Chromosome Disorders
- Interventions
- Placebo Medication, Metformin
- Drug
- Lead sponsor
- University of California, Davis
- Other
- Eligibility
- 6 Years to 25 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2024
- U.S. locations
- 1
- States / cities
- Sacramento, California
- Conditions
- Down Syndrome
- Interventions
- muscle strength
- Other
- Lead sponsor
- State University of New York - Upstate Medical University
- Other
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- Started 2008
- U.S. locations
- 1
- States / cities
- Syracuse, New York
- Conditions
- Down Syndrome, Down Syndrome, Partial Trisomy 21, Alzheimer Disease
- Interventions
- [18F]-FEOBV Radiotracer
- Drug
- Lead sponsor
- Vanderbilt University Medical Center
- Other
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2027
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee
- Conditions
- Down Syndrome
- Interventions
- Combined exercise intervention, Usual care / Sham intervention
- Behavioral
- Lead sponsor
- University of Nevada, Las Vegas
- Other
- Eligibility
- 18 Years to 35 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2024
- U.S. locations
- 1
- States / cities
- Las Vegas, Nevada