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ClinicalTrials.gov public records Last synced May 22, 2026, 12:15 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Eye Diseases Hereditary Clear all
Completed Phase 1 Interventional
View directory record Official record
Conditions
Dry Age-related Macular Degeneration, Geographic Atrophy, Macular Degeneration, Retinal Disease, Retinal Degeneration
Interventions
GEM103
Biological
Lead sponsor
Gemini Therapeutics, Inc.
Industry
Eligibility
50 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
12
States / cities
Phoenix, Arizona • Bakersfield, California • Beverly Hills, California + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 10, 2021 · Synced May 22, 2026, 12:15 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Nasal Type Extranodal NK/T-cell Lymphoma, Anaplastic Large Cell Lymphoma, Angioimmunoblastic T-cell Lymphoma, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Burkitt Lymphoma, Childhood Chronic Myelogenous Leukemia, Childhood Myelodysplastic Syndromes, Childhood Nasal Type Extranodal NK/T-cell Lymphoma, Cutaneous B-cell Non-Hodgkin Lymphoma, de Novo Myelodysplastic Syndromes, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Hematopoietic/Lymphoid Cancer, Hepatosplenic T-cell Lymphoma, Intraocular Lymphoma, Nodal Marginal Zone B-cell Lymphoma, Peripheral T-cell Lymphoma, Post-transplant Lymphoproliferative Disorder, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Burkitt Lymphoma, Recurrent Adult Diffuse Large Cell Lymphoma, Recurrent Adult Diffuse Mixed Cell Lymphoma, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Grade III Lymphomatoid Granulomatosis, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Immunoblastic Large Cell Lymphoma, Recurrent Adult Lymphoblastic Lymphoma, Recurrent Adult T-cell Leukemia/Lymphoma, Recurrent Childhood Anaplastic Large Cell Lymphoma, Recurrent Childhood Grade III Lymphomatoid Granulomatosis, Recurrent Childhood Large Cell Lymphoma, Recurrent Childhood Lymphoblastic Lymphoma, Recurrent Childhood Small Noncleaved Cell Lymphoma, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Grade 3 Follicular Lymphoma, Recurrent Mantle Cell Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Mycosis Fungoides/Sezary Syndrome, Recurrent Small Lymphocytic Lymphoma, Recurrent/Refractory Childhood Hodgkin Lymphoma, Refractory Chronic Lymphocytic Leukemia, Refractory Multiple Myeloma, Relapsing Chronic Myelogenous Leukemia, Secondary Myelodysplastic Syndromes, Small Intestine Lymphoma, Splenic Marginal Zone Lymphoma, Stage II Multiple Myeloma, Stage III Adult Burkitt Lymphoma, Stage III Adult Diffuse Large Cell Lymphoma, Stage III Adult Diffuse Mixed Cell Lymphoma, Stage III Adult Diffuse Small Cleaved Cell Lymphoma, Stage III Adult Hodgkin Lymphoma, Stage III Adult Immunoblastic Large Cell Lymphoma, Stage III Adult Lymphoblastic Lymphoma, Stage III Adult T-cell Leukemia/Lymphoma, Stage III Childhood Hodgkin Lymphoma, Stage III Chronic Lymphocytic Leukemia, Stage III Cutaneous T-cell Non-Hodgkin Lymphoma, Stage III Grade 1 Follicular Lymphoma, Stage III Grade 2 Follicular Lymphoma, Stage III Grade 3 Follicular Lymphoma, Stage III Mantle Cell Lymphoma, Stage III Marginal Zone Lymphoma, Stage III Multiple Myeloma, Stage III Mycosis Fungoides/Sezary Syndrome, Stage III Small Lymphocytic Lymphoma, Stage IV Adult Burkitt Lymphoma, Stage IV Adult Diffuse Large Cell Lymphoma, Stage IV Adult Diffuse Mixed Cell Lymphoma, Stage IV Adult Diffuse Small Cleaved Cell Lymphoma, Stage IV Adult Hodgkin Lymphoma, Stage IV Adult Immunoblastic Large Cell Lymphoma, Stage IV Adult Lymphoblastic Lymphoma, Stage IV Adult T-cell Leukemia/Lymphoma, Stage IV Childhood Hodgkin Lymphoma, Stage IV Chronic Lymphocytic Leukemia, Stage IV Cutaneous T-cell Non-Hodgkin Lymphoma, Stage IV Grade 1 Follicular Lymphoma, Stage IV Grade 2 Follicular Lymphoma, Stage IV Grade 3 Follicular Lymphoma, Stage IV Mantle Cell Lymphoma, Stage IV Marginal Zone Lymphoma, Stage IV Mycosis Fungoides/Sezary Syndrome, Stage IV Small Lymphocytic Lymphoma, Testicular Lymphoma, Waldenström Macroglobulinemia
Interventions
cyclophosphamide, fludarabine phosphate, tacrolimus, mycophenolate mofetil, polymerase chain reaction, fluorescence in situ hybridization, polymorphism analysis, gene expression analysis, total-body irradiation, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation
Drug · Genetic · Radiation + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Not listed
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2014
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated May 16, 2017 · Synced May 22, 2026, 12:15 AM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
Stargardt Disease, Diabetic Retinopathy, Macular Degeneration (Age Related)
Interventions
Anterior Chamber (AC) Tap
Other
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 100 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 25, 2022 · Synced May 22, 2026, 12:15 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Cognitive/Functional Effects, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
Not listed
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
65 Years and older
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2030
U.S. locations
7
States / cities
Boone, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2025 · Synced May 22, 2026, 12:15 AM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Gyrate Atrophy
Interventions
Gene therapy
Procedure
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 22, 2026, 12:15 AM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Spin exercise
Other
Lead sponsor
Emory University
Other
Eligibility
18 Years to 60 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 25, 2018 · Synced May 22, 2026, 12:15 AM EDT
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 22, 2026, 12:15 AM 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 22, 2026, 12:15 AM EDT
Not listed Phase 1 Interventional
View directory record Official record
Conditions
Retinoblastoma
Interventions
Episcleral Topotecan
Drug
Lead sponsor
Targeted Therapy Technologies, LLC
Industry
Eligibility
Up to 18 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Oct 30, 2023 · Synced May 22, 2026, 12:15 AM 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 22, 2026, 12:15 AM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Retinoblastoma
Interventions
filgrastim, carboplatin, cisplatin, cyclophosphamide, etoposide, mesna, vincristine sulfate
Biological · Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Up to 17 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2001
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 27, 2013 · Synced May 22, 2026, 12:15 AM 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 22, 2026, 12:15 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Uveal Melanoma, Cutaneous Melanoma, BAP1 Gene Mutation, Renal Cell Carcinoma, Mesothelioma, Hepatocellular Carcinoma, Cholangiocarcinoma, Meningioma Atypical
Interventions
Not listed
Lead sponsor
Mohamed Abdel-Rahman
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2026
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 8, 2026 · Synced May 22, 2026, 12:15 AM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Retinoblastoma
Interventions
Carboplatin, Maxitrol®, Focal Therapy, Plaque Radiotherapy
Drug · Procedure
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 17 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 19, 2020 · Synced May 22, 2026, 12:15 AM 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 22, 2026, 12:15 AM 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 22, 2026, 12:15 AM 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 22, 2026, 12:15 AM 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 22, 2026, 12:15 AM 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 22, 2026, 12:15 AM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Eye Disease, Achromatopsia
Interventions
NT-501 CNTF-releasing implant
Biological
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 20, 2016 · Synced May 22, 2026, 12:15 AM 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 22, 2026, 12:15 AM 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 22, 2026, 12:15 AM 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 22, 2026, 12:15 AM 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 22, 2026, 12:15 AM EDT