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ClinicalTrials.gov public records Last synced May 22, 2026, 12:14 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–7 of 7 matching trials from the live ClinicalTrials.gov search.
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Recruiting No phase listed Observational Accepts healthy volunteers
Conditions
Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
Interventions
Not listed
Lead sponsor
Foundation Fighting Blindness
Other
Eligibility
Not listed
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2037
U.S. locations
1
States / cities
Columbia, Maryland
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced May 22, 2026, 12:14 AM EDT
Conditions
Bardet-Biedl Syndrome, POMC Deficiency Obesity, PCSK1 Deficiency Obesity, LEPR Deficiency Obesity
Interventions
Setmelanotide
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 5 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
3
States / cities
Aurora, Colorado • New York, New York • Marshfield, Wisconsin
Source: ClinicalTrials.gov public record
Updated Nov 26, 2024 · Synced May 22, 2026, 12:14 AM EDT
Conditions
Bardet Biedl Syndrome (BBS), Alström Syndrome (AS)
Interventions
Setmelanotide, Placebo
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
6 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
6
States / cities
San Diego, California • Springfield, Massachusetts • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 30, 2023 · Synced May 22, 2026, 12:14 AM EDT
Conditions
Bardet-Biedl Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
194 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 3, 2019 · Synced May 22, 2026, 12:14 AM EDT
Conditions
Bardet-Biedl Syndrome, POMC Deficiency
Interventions
Setmelanotide 2 mg, Setmelanotide 2.5 mg, Setmelanotide 3 mg, Setmelanotide 20 mg, Setmelanotide 25 mg, Setmelanotide 30 mg
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
6 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
2
States / cities
Scottsdale, Arizona • Marshfield, Wisconsin
Source: ClinicalTrials.gov public record
Updated Nov 25, 2024 · Synced May 22, 2026, 12:14 AM EDT
Recruiting No phase listed Observational
Conditions
Hepato/Renal Fibrocystic Disease, Autosomal Recessive Polycystic Kidney Disease, Joubert Syndrome, Bardet Biedl Syndrome, Meckel-Gruber Syndrome, Congenital Hepatic Fibrosis, Caroli Syndrome, Oro-Facial-Digital Syndrome Type I, Nephronophthisis, Glomerulocystic Kidney Disease
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 18 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2030
U.S. locations
6
States / cities
Washington D.C., District of Columbia • Atlanta, Georgia • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 12, 2025 · Synced May 22, 2026, 12:14 AM EDT
Conditions
Bardet-Biedl Syndrome
Interventions
Not listed
Lead sponsor
Marshfield Clinic Research Foundation
Other
Eligibility
Not listed
Enrollment
1,200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2030
U.S. locations
1
States / cities
Marshfield, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 18, 2022 · Synced May 22, 2026, 12:14 AM EDT