- 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
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- 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
- 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