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ClinicalTrials.gov public records Last synced May 21, 2026, 11:39 PM EDT

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

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Eye Disorders Congenital Clear all
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Wolfram Syndrome
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
17 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced May 21, 2026, 11:39 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced May 21, 2026, 11:39 PM EDT
Not listed Phase 2 Interventional
View directory record Official record
Conditions
Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
Interventions
anti-thymocyte globulin, cyclosporine, fludarabine phosphate, melphalan, methylprednisolone, mycophenolate mofetil, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, syngeneic bone marrow transplantation
Biological · Drug · Procedure
Lead sponsor
Herbert Irving Comprehensive Cancer Center
Other
Eligibility
1 Year to 80 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 5, 2014 · Synced May 21, 2026, 11:39 PM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
Adult Langerhans Cell Histiocytosis, Childhood Langerhans Cell Histiocytosis, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
Peripheral Blood Stem Cell Transplantation
Procedure
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 65 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 5, 2014 · Synced May 21, 2026, 11:39 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Retinitis Pigmentosa, Stargardt Macular Dystrophy, Stargardt Disease, Geographic Atrophy From Age-related Macular Degeneration, X-linked Retinoschisis, Retinal Dystrophies
Interventions
Not listed
Lead sponsor
Ray Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Mar 30, 2026 · Synced May 21, 2026, 11:39 PM EDT
Active, not recruiting Phase 1 Interventional
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
CNS10-NPC implantation
Biological
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Beverly Hills, California
Source: ClinicalTrials.gov public record
Updated Aug 18, 2025 · Synced May 21, 2026, 11:39 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Leber Congenital Amaurosis
Interventions
Not listed
Lead sponsor
MeiraGTx UK II Ltd
Industry
Eligibility
3 Years and older
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Aug 13, 2023 · Synced May 21, 2026, 11:39 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Adenovirus, Anesthesia, Anxiety, Anxiolysis, Autism, Autistic Disorder, Bacterial Meningitis, Bacterial Septicemia, Benzodiazepine, Bipolar Disorder, Bone and Joint Infections, Central Nervous System Infections, Convulsions, Cytomegalovirus Retinitis, Early-onset Schizophrenia Spectrum Disorders, Epilepsy, General Anesthesia, Gynecologic Infections, Herpes Simplex Virus, Infantile Hemangioma, Infection, Inflammation, Inflammatory Conditions, Intra-abdominal Infections, Lower Respiratory Tract Infections, Migraines, Pain, Pneumonia, Schizophrenia, Sedation, Seizures, Skeletal Muscle Spasms, Skin and Skin-structure Infections, Treatment-resistant Schizophrenia, Urinary Tract Infections, Withdrawal, Sepsis, Gram-negative Infection, Bradycardia, Cardiac Arrest, Cardiac Arrhythmia, Staphylococcal Infections, Nosocomial Pneumonia, Neuromuscular Blockade, Methicillin Resistant Staphylococcus Aureus, Endocarditis, Neutropenia, Headache, Fibrinolytic Bleeding, Pulmonary Arterial Hypertension, CMV Retinitis, Hypertension, Chronic Kidney Diseases, Hyperaldosteronism, Hypokalemia, Heart Failure, Hemophilia, Heavy Menstrual Bleeding, Insomnia
Interventions
The POPS study is collecting PK data on children prescribed the following drugs of interest per standard of care:
Drug
Lead sponsor
Daniel Benjamin
Other
Eligibility
Up to 21 Years
Enrollment
3,520 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
43
States / cities
Anchorage, Alaska • Little Rock, Arkansas • La Jolla, California + 37 more
Source: ClinicalTrials.gov public record
Updated Sep 5, 2023 · Synced May 21, 2026, 11:39 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Inherited Ophthalmic Diseases, Hypopigmentation Disorder, Corneal Dystrophy, Blue-cone Monochromacy, Best Disease, Aniridia, Albinism
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
1 Day to 120 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2054
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 3, 2026 · Synced May 21, 2026, 11:39 PM EDT
Terminated Phase 2Phase 3 Interventional Results available
View directory record Official record
Conditions
Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions
Ultevursen, Sham-procedure
Drug · Other
Lead sponsor
Laboratoires Thea
Industry
Eligibility
12 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
10
States / cities
San Diego, California • San Francisco, California • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2024 · Synced May 21, 2026, 11:39 PM EDT
Recruiting Phase 1 Interventional
View directory record Official record
Conditions
Retinopathy of Prematurity (ROP)
Interventions
FLQ-101
Drug
Lead sponsor
FELIQS INC.
Industry
Eligibility
4 Days to 5 Days
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Apr 20, 2026 · Synced May 21, 2026, 11:39 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Roche Global Development
Other
Eligibility
3 Months to 12 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
8
States / cities
Birmingham, Alabama • Los Angeles, California • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced May 21, 2026, 11:39 PM EDT
Completed No phase listed Observational Results available
View directory record Official record
Conditions
Cerebrotendinous Xanthomatosis (CTX)
Interventions
Not listed
Lead sponsor
Travere Therapeutics, Inc.
Industry
Eligibility
2 Years to 21 Years
Enrollment
442 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
44
States / cities
Birmingham, Alabama • La Jolla, California • Los Angeles, California + 38 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced May 21, 2026, 11:39 PM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
N-acetylcysteine, Placebo
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 65 Years
Enrollment
485 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
24
States / cities
Davis, California • Los Angeles, California • San Francisco, California + 21 more
Source: ClinicalTrials.gov public record
Updated May 3, 2026 · Synced May 21, 2026, 11:39 PM EDT
Not listed Not applicable Interventional
View directory record Official record
Conditions
Eyelid Diseases, Ptosis, Eyelid, Blepharoptosis, Dermatochalasis, Ectropion, Entropion, Eyelid Tumor, Skin Cancer, Eyelid, Floppy Eyelid Syndrome
Interventions
Telemedicine follow-up
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Nov 3, 2020 · Synced May 21, 2026, 11:39 PM EDT
Active, not recruiting Phase 1 Interventional
View directory record Official record
Conditions
Amaurosis of Leber, Retinal Diseases
Interventions
rAAV2-CBSB-hRPE65
Genetic
Lead sponsor
University of Pennsylvania
Other
Eligibility
8 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2026
U.S. locations
2
States / cities
Gainesville, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 4, 2025 · Synced May 21, 2026, 11:39 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
400 µg Brimonidine Tartrate Implant, 200 µg Brimonidine Tartrate Implant, 100 µg Brimonidine Tartrate Implant, Sham (no implant)
Drug · Other
Lead sponsor
Allergan
Industry
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Arlington, Texas
Source: ClinicalTrials.gov public record
Updated Apr 23, 2013 · Synced May 21, 2026, 11:39 PM EDT
Enrolling by invitation Phase 3 Interventional
View directory record Official record
Conditions
Choroideremia, X-Linked Retinitis Pigmentosa
Interventions
BIIB111, BIIB112
Genetic
Lead sponsor
NightstaRx Ltd, a Biogen Company
Other
Eligibility
18 Years and older · Male only
Enrollment
330 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
10
States / cities
Los Angeles, California • Gainesville, Florida • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 11, 2025 · Synced May 21, 2026, 11:39 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Hyper-IgM, DiGeorge Syndrome, Chediak-Higashi Syndrome, Common Variable Immune Deficiency, Immune Dysregulatory Disorders, Hemophagocytic Lymphohistiocytosis, IPEX, Autoimmune Lymphoproliferative Syndrome, X-linked Lymphoproliferative Syndrome
Interventions
Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 21 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Feb 18, 2026 · Synced May 21, 2026, 11:39 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Dermatochalasis, Ptosis, Eyelid
Interventions
Randomized laterality of suture materials for closure (6-0 polypropylene monofilament suture vs fast absorbing gut suture on right eyelid)
Procedure
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
18 Years to 89 Years
Enrollment
23 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 22, 2023 · Synced May 21, 2026, 11:39 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Zidovudine, Sargramostim, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years to 65 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1992
U.S. locations
4
States / cities
Los Angeles, California • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 3, 2021 · Synced May 21, 2026, 11:39 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Retinopathy of Prematurity
Interventions
Ultrasound examination
Procedure
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 27, 2019 · Synced May 21, 2026, 11:39 PM EDT
Completed Not applicable Interventional Results available
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Implant of Argus II Retinal Prosthesis
Device
Lead sponsor
Second Sight Medical Products
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2019
U.S. locations
7
States / cities
Los Angeles, California • San Francisco, California • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 19, 2022 · Synced May 21, 2026, 11:39 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
PHACE Syndrome
Interventions
Not listed
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
Not listed
Enrollment
341 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2022
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Mar 27, 2023 · Synced May 21, 2026, 11:39 PM EDT