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Showing 1–9 of 9 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Retinitis Pigmentosa, Leber Congenital Amaurosis
Interventions
OCU400 Low Dose, OCU400 Med Dose, OCU400 High Dose, OCU400 Second Eye Dosing
Drug
Lead sponsor
Ocugen
Industry
Eligibility
6 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
7
States / cities
Phoenix, Arizona • La Jolla, California • Miami, Florida + 4 more
Recruiting No phase listed Observational

ARPKD Database Study

NCT01401998
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
Conditions
Blindness, Leber Congenital Amaurosis 10, Vision Disorders, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease, Retinal Degeneration
Interventions
Not listed
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
3 Years to 99 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
4
States / cities
Miami, Florida • Boston, Massachusetts • Ann Arbor, Michigan + 1 more
Conditions
Leber's Congenital Amaurosis
Interventions
QR-110
Drug
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
2
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders, Vision Disorder, Neurological Manifestations, Eye Diseases, Hereditary, Eye Diseases, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Placebo IVT
Drug · Other
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
San Francisco, California • Miami, Florida • Iowa City, Iowa + 2 more
Completed Not applicable Interventional Accepts healthy volunteers

Use of an External Erectile Device in Transgender Man Following Phalloplasty

NCT03823014
Conditions
Transsexualism, Surgery, Sex Behavior, Erectile Dysfunction
Interventions
Elator
Device
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years to 64 Years
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease, Retinal Degeneration, Vision Disorders, Eye Disorders Congenital
Interventions
EDIT-101
Drug
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
3 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
5
States / cities
Miami, Florida • Boston, Massachusetts • Ann Arbor, Michigan + 2 more
Terminated Phase 1Phase 2 Interventional Results available

Extension Study to Study PQ-110-001 (NCT03140969)

NCT03913130
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
Interventions
QR-110
Drug
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
2
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Sham
Drug · Other
Lead sponsor
ProQR Therapeutics
Industry
Eligibility
8 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Iowa City, Iowa