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Study identifier: NCT06891443 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

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

Condition: Leber Congenital Amaurosis 10 · Blindness · Leber Congenital Amaurosis  ·  Sponsor: Laboratoires Thea

PhasePhase 3
Planned participants32
Who can joinAll sexes, 6 Years to no upper limit
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Sepul Bio Patient Advocacy Director  ·  +31 617060791  ·  contact@sepulbio.com

Sepul Bio Chief Medical Officer

Always discuss trial participation with your own doctor first.

Locations (17)

UCSF Wayne and Gladys Valley Center for VisionSan Francisco, California, United StatesRecruiting
University of Miami - Bascom Palmer Eye InstituteMiami, Florida, United StatesRecruiting
University of IowaIowa City, Iowa, United StatesRecruiting
University of Minnesota Medical SchoolMinneapolis, Minnesota, United StatesRecruiting
University of Pennsylvania - Center for Advanced Retinal & Ocular TherapeuticsPhiladelphia, Pennsylvania, United StatesRecruiting
Universitair Ziekenhuis Gent (UZ)Ghent, BelgiumRecruiting
INRET Clínica/ Santa Casa de Misericórdia de Belo HorizonteBelo Horizonte, Minas Gerais, BrazilRecruiting
Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP)São Paulo, São Paulo, BrazilRecruiting
University of AlbertaEdmonton, Alberta, CanadaRecruiting
The Hospital for Sick Children - SickKidsToronto, Ontario, CanadaRecruiting
Centre de maladies rares CHNO des Quinze VingtParis, FranceRecruiting
Justus-Liebig Universität - Department of OphthalmologyGiessen, GermanyRecruiting

+ 5 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT06891443