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Clinical Trials in the USA / NCT06891443
Recruiting Phase 3

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

NCT06891443 · tracked via the Priya Life Science USA tracker
Sponsor
Laboratoires Thea
Phase
Phase 3
Started
2025-06-04
Last updated
2026-06-25

Condition(s) studied

Leber Congenital Amaurosis 10BlindnessLeber Congenital AmaurosisSensation DisordersVision DisorderNeurological ManifestationsEye Diseases, HereditaryEye DiseasesEye Disorders CongenitalRetinal Disease

Investigational drug(s) / intervention(s)

sepofarsenPlacebo IVT

sepofarsen: RNA antisense oligonucleotide for intravitreal injection

Placebo IVT: Placebo with identical appearance to sepofarsen

Study summary

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.

Eligibility

Sex
ALL
Min age
6 Years
Max age
Healthy volunteers
No
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 equivalent 20/50) to +2.9 logMAR based on quantifiable, reliable FrACT. LP subjects with documented evidence of prior better vision eligible. 4. Symmetrical disease between the two eyes as defined by a BCVA (FrACT) within 0.2 logMAR at baseline. 5. Detectable ONL in the macular area as determined by the CRC at Screening. Exclusion Criteria: 1. Mutations in genes other than the CEP290 gene associated with other IRD diseases or syndromes. 2. Presence of any ocular pathology in either eye that may make comparison of the eyes not feasible. 3. Presence of unstable concurrent CME, or subject started on (or changed dose of) topical or systemic carbonic anhydrase inhibitor treatment in the 3 months prior to enrollment. CME is allowed if stable for 3 months (with or without treatment). 4. Presence of any clinically significant lens opacities/cataracts based on the AREDS lens grading scale. 5. Any prior receipt of genetic (RNA or DNA therapy) or stem-cell therapy for ocular or non-ocular disease, including sepofarsen.

Primary outcome measure(s)

Trial sites (17)

FacilityCityRegionStatus
UCSF Wayne and Gladys Valley Center for Vision San Francisco California Recruiting
University of Miami - Bascom Palmer Eye Institute Miami Florida Recruiting
University of Iowa Iowa City Iowa Recruiting
University of Minnesota Medical School Minneapolis Minnesota Recruiting
University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics Philadelphia Pennsylvania Recruiting
Universitair Ziekenhuis Gent (UZ) Ghent Belgium Recruiting
INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte Belo Horizonte Minas Gerais Recruiting
Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP) São Paulo São Paulo Recruiting
University of Alberta Edmonton Alberta Recruiting
The Hospital for Sick Children - SickKids Toronto Ontario Recruiting
Centre de maladies rares CHNO des Quinze Vingt Paris France Recruiting
Justus-Liebig Universität - Department of Ophthalmology Giessen Germany Recruiting
Klinikum der Ludwig-Maximilian Universität München München Germany Recruiting
University of Tuebingen - Inst. for Ophthalmic Research Tübingen Germany Recruiting
Radboud Universitair Medisch Centrum Nijmegen Netherlands Recruiting
Hospital Sant Joan de Déu (SJD Barcelona Children's Hospital) Barcelona Spain Recruiting
Moorfields Eye Hospital NHS Foundation Trust London United Kingdom Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06891443 on ClinicalTrials.gov ↗ ← All trials in the USA