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ClinicalTrials.gov public records Last synced Sep 17, 2026, 8:38 PM EDT

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Showing 1–24 of 74 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Trisomy 21, Trisomy 18, Trisomy 13, Sex Chromosome Abnormalities, Microdeletion Syndromes
Interventions
Not listed
Lead sponsor
Natera, Inc.
Industry
Eligibility
18 Years and older
Enrollment
1,059 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2020
U.S. locations
7
States / cities
San Carlos, California • San Gabriel, California • Washington D.C., District of Columbia + 4 more
Recruiting Phase 1Phase 2 Interventional Accepts healthy volunteers

A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment

NCT06239116
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
Prader-Willi Syndrome
Interventions
DCCR, Placebo
Drug
Lead sponsor
Essentialis, Inc.
Industry
Eligibility
10 Years to 22 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Orange, California
Conditions
Prader-Willi Syndrome
Interventions
RAD011, Placebo
Drug
Lead sponsor
Radius Pharmaceuticals, Inc.
Industry
Eligibility
8 Years to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
8
States / cities
Los Angeles, California • San Diego, California • Iowa City, Iowa + 5 more
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Parent and Infant Inter(X)Action Intervention (PIXI)

NCT03836300
Conditions
Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome, Duchenne Muscular Dystrophy, Phelan-McDermid Syndrome, Rett Syndrome, Smith Magenis Syndrome, Williams Syndrome, Turner Syndrome, Klinefelter Syndrome, Chromosome 22q11.2 Deletion Syndrome, Tuberous Sclerosis, Down Syndrome
Interventions
Parent-Infant Inter(X)action Intervention (PIXI)
Behavioral
Lead sponsor
RTI International
Other
Eligibility
Up to 99 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2026
U.S. locations
1
States / cities
Research Triangle Park, North Carolina
Conditions
Prader-Willi Syndrome
Interventions
Setmelanotide, Placebo
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
16 Years to 65 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
5
States / cities
Irvine, California • Gainesville, Florida • Kansas City, Kansas + 2 more
Terminated Phase 2 Interventional Accepts healthy volunteers Results available

CBDV vs Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)

NCT03848481
Conditions
Prader-Willi Syndrome
Interventions
CBDV Compound, Placebo
Drug
Lead sponsor
Eric Hollander
Other
Eligibility
5 Years to 30 Years
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2024
U.S. locations
1
States / cities
The Bronx, New York
Terminated Not applicable Interventional

Home-based SSP on Individuals With PWS

NCT03871751
Conditions
Prader-Willi Syndrome
Interventions
Safe and Sound Protocol
Behavioral
Lead sponsor
Indiana University
Other
Eligibility
3 Years to 17 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019
U.S. locations
1
States / cities
Aurora, Colorado
Completed No phase listed Observational Accepts healthy volunteers

Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW

NCT02670694
Conditions
Rett Syndrome, Prader-Willi Syndrome, Angelman Syndrome, Sleep Problems
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Year to 18 Years
Enrollment
804 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
9
States / cities
Birmingham, Alabama • Irvine, California • San Diego, California + 6 more
Conditions
Prader-Willi Syndrome, Excessive Daytime Sleepiness, Hyperphagia, Body Weight, Mood, Behavior
Interventions
Sham Light, Bright Light Therapy
Other
Lead sponsor
Maimonides Medical Center
Other
Eligibility
6 Years to 88 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Brooklyn, New York
Conditions
Metabolism and Nutrition Disorder, Obesity
Interventions
liraglutide, placebo
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
6 Years to 18 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
5
States / cities
Santa Monica, California • Baltimore, Maryland • Buffalo, New York + 2 more
Conditions
Prader-Willi Syndrome, Hypothalamic Obesity, Obesity/Therapy
Interventions
Tirzepatide
Drug
Lead sponsor
Grace Kim
Other
Eligibility
18 Years to 26 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
3
States / cities
Saint Paul, Minnesota • Nashville, Tennessee • Seattle, Washington
Conditions
Quality of Life
Interventions
B. lactis B94, Placebo
Dietary Supplement
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 75 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
3
States / cities
Gainesville, Florida
Conditions
Angelman Syndrome, Prader-Willi Syndrome
Interventions
Newborn Screening Assay
Diagnostic Test
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
Not listed
Enrollment
11 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
1
States / cities
Madison, Wisconsin
Conditions
Hyperphagia, Prader-Willi Syndrome, Hyperphagia in Prader-Willi Syndrome
Interventions
ARD-101
Drug
Lead sponsor
Aardvark Therapeutics, Inc.
Industry
Eligibility
13 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
16
States / cities
Birmingham, Alabama • Orange, California • Palo Alto, California + 13 more
Conditions
Prader-Willi Syndrome
Interventions
Regulating Together group therapy
Behavioral
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
13 Years to 17 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027
U.S. locations
1
States / cities
Kansas City, Missouri
Conditions
Prader-Willi Syndrome
Interventions
Cannabidiol Oral Solution
Drug
Lead sponsor
Radius Pharmaceuticals, Inc.
Industry
Eligibility
8 Years to 17 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Tacoma, Washington
Completed Not applicable Interventional Accepts healthy volunteers

Treatment of Self-Injurious Behavior in Individuals With Prader-Willi Syndrome

NCT00065923
Conditions
Prader-Willi Syndrome, Self-Injurious Behavior
Interventions
Topiramate
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years to 66 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2002
U.S. locations
1
States / cities
Gainesville, Florida
Conditions
Prader-Willi Syndrome
Interventions
Nutropin AQ
Drug
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
16 Years to 60 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
1
States / cities
Kansas City, Missouri
Conditions
Hyperphagia, Prader-Willi Syndrome
Interventions
Placebo, ARD-101
Drug
Lead sponsor
Aardvark Therapeutics, Inc.
Industry
Eligibility
7 Years and older
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
17
States / cities
Birmingham, Alabama • Encinitas, California • Orange, California + 14 more
Conditions
Prader-Willi Syndrome
Interventions
NNZ-2591
Drug
Lead sponsor
Neuren Pharmaceuticals Limited
Industry
Eligibility
4 Years to 12 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
4
States / cities
San Diego, California • Atlanta, Georgia • Chevy Chase, Maryland + 1 more
Conditions
Prader-Willi Syndrome, Hyperphagia
Interventions
Intranasal Oxytocin (IN-OXT), Matched Placebo
Drug · Other
Lead sponsor
Montefiore Medical Center
Other
Eligibility
5 Years to 18 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
The Bronx, New York
Conditions
Prader-Willi Syndrome
Interventions
Not listed
Lead sponsor
Foundation for Prader-Willi Research
Other
Eligibility
5 Years and older
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
1
States / cities
Walnut, California
Conditions
Prader-Willi Syndrome
Interventions
DCCR, Placebo for DCCR
Drug
Lead sponsor
Soleno Therapeutics, Inc.
Industry
Eligibility
4 Years and older
Enrollment
115 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
20
States / cities
Orange, California • Palo Alto, California • San Diego, California + 17 more