Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
Recruiting
Phase 3
Interventional Study
Leber Congenital Amaurosis 10
Blindness
Leber Congenital Amaurosis
Sensation Disorders
Vision Disorder
Neurological Manifestations
Eye Diseases, Hereditary
Eye Diseases
Eye Disorders Congenital
Retinal Disease
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 6 and older
- Sex
- Any
- Trial phase
- Phase 3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 32 (estimated)
- Sponsor
- Laboratoires Thea · Industry
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About This Trial
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.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
1. Confirmed clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A\>G mutation in CEP290.
2. Adults: \>=18 years / Minors: 6 to \<18 years.
3. BCVA (FrACT) equal to or worse than logMAR +0.4 (approximate Snellen equ…
Contacts
Sepul Bio Chief Medical Officer
CONTACT