- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
- Interventions
- QR-421a, Sham-procedure (dose cohort 1&2 only)
- Drug · Other
- Lead sponsor
- ProQR Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 4
- States / cities
- Boston, Massachusetts • Ann Arbor, Michigan • Portland, Oregon + 1 more
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Showing 1–8
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- 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
- 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
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
- Interventions
- QR-421a, Sham-procedure
- Drug · Other
- Lead sponsor
- Laboratoires Thea
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 2
- States / cities
- Dallas, Texas • Madison, Wisconsin
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2
- Interventions
- RNA antisense oligonucleotide for intravitreal injection
- Drug
- Lead sponsor
- Laboratoires Thea
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 4
- States / cities
- Boston, Massachusetts • Ann Arbor, Michigan • Portland, Oregon + 1 more
- 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
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
- Interventions
- Intravitreal Injection of Ultevursen, No intervention, will not receive any active study intervention
- Drug · Other
- Lead sponsor
- Laboratoires Thea
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 81 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 10
- States / cities
- San Francisco, California • Miami, Florida • Atlanta, Georgia + 7 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