Actively Recruiting
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
Led by Laboratoires Thea · Updated on 2026-03-17
32
Participants Needed
14
Research Sites
173 weeks
Total Duration
On this page
Sponsors
L
Laboratoires Thea
Lead Sponsor
S
Sepul Bio
Collaborating Sponsor
AI-Summary
What this Trial Is About
The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A\>G (p.Cys998X) mutation in the CEP290.
CONDITIONS
Official Title
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of LCA10 with homozygous or compound heterozygous c.2991+1655A>G mutation in CEP290
- Age 18 years or older for adults; age 6 to under 18 years for minors
- Best-corrected visual acuity (BCVA) equal to or worse than logMAR +0.4 to +2.9 based on FrACT test; light perception subjects with prior better vision also eligible
- Symmetrical vision between eyes with BCVA difference within 0.2 logMAR at baseline
- Detectable outer nuclear layer in the macula area as determined at screening
You will not qualify if you...
- Mutations in genes other than CEP290 linked to other inherited retinal diseases or syndromes
- Any eye condition that could prevent comparison of the two eyes
- Presence of unstable cystoid macular edema or recent changes in carbonic anhydrase inhibitor treatment within 3 months prior to enrollment; stable CME allowed
- Significant lens opacities or cataracts as per AREDS lens grading scale
- Previous genetic or stem-cell therapy for eye or other diseases, including sepofarsen
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 14 locations
1
UCSF Wayne and Gladys Valley Center for Vision
San Francisco, California, United States, 94158
Actively Recruiting
2
University of Miami - Bascom Palmer Eye Institute
Miami, Florida, United States, 33156
Actively Recruiting
3
University of Iowa
Iowa City, Iowa, United States, 52242
Actively Recruiting
4
University of Minnesota Medical School
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
5
University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
6
Universitair Ziekenhuis Gent (UZ)
Ghent, Belgium, 9000
Actively Recruiting
7
University of Alberta
Edmonton, Alberta, Canada, T6G 2C8
Actively Recruiting
8
The Hospital for Sick Children - SickKids
Toronto, Ontario, Canada, M5G 2L3
Actively Recruiting
9
Centre de maladies rares CHNO des Quinze Vingt
Paris, France, 75012
Actively Recruiting
10
Justus-Liebig Universität - Department of Ophthalmology
Giessen, Germany, 35392
Actively Recruiting
11
Klinikum der Ludwig-Maximilian Universität München
München, Germany, 81377
Actively Recruiting
12
University of Tuebingen - Inst. for Ophthalmic Research
Tübingen, Germany, 72076
Actively Recruiting
13
Radboud Universitair Medisch Centrum
Nijmegen, Netherlands, 6525 GA
Actively Recruiting
14
Moorfields Eye Hospital NHS Foundation Trust
London, United Kingdom, EC1V 2PD
Actively Recruiting
Research Team
S
Sepul Bio Patient Advocacy Director
CONTACT
S
Sepul Bio Chief Medical Officer
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
7
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