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NCT06891443 · ClinicalTrials.gov registry record · Phase 3

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

A Phase 3 study of Eye Diseases and Retinal Disease, sponsored by Laboratoires Thea.

Recruiting
Registry status
Phase 3
Development phase
32
Enrollment target
14
Study locations

NCT06891443: Recruiting Phase 3 study of Eye Diseases and Retinal Disease, sponsored by Laboratoires Thea.

NCT06891443 is a Phase 3 study of Eye Diseases and Retinal Disease that is actively recruiting participants, run by Laboratoires Thea. The registered enrollment target is 32 participants, below the 741,302-participant average among 27 other Eye Diseases trials with a reported enrollment target (100% lower). The trial reports 14 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT06891443, a Phase 3 study of Eye Diseases and Retinal Disease, is actively recruiting participants, sponsored by Laboratoires Thea.

RECRUITING
Registry status
Phase 3
Development phase
32 participants
Enrollment target
14
Study locations

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.

Primary Outcome

Change from baseline in BCVA based on the Freiburg Acuity and Contrast Test (FrACT) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs)

Interventions

  • DRUG sepofarsen
  • OTHER Placebo IVT

Study Locations (14)

Other

  • Universitair Ziekenhuis Gent (UZ) - Ghent
  • Centre de maladies rares CHNO des Quinze Vingt - Paris
  • Justus-Liebig Universität - Department of Ophthalmology - Giessen
  • Klinikum der Ludwig-Maximilian Universität München - München
  • University of Tuebingen - Inst. for Ophthalmic Research - Tübingen
  • Radboud Universitair Medisch Centrum - Nijmegen
  • Moorfields Eye Hospital NHS Foundation Trust - London

California

  • UCSF Wayne and Gladys Valley Center for Vision - San Francisco

Florida

  • University of Miami - Bascom Palmer Eye Institute - Miami

Iowa

  • University of Iowa - Iowa City

Minnesota

  • University of Minnesota Medical School - Minneapolis

Pennsylvania

  • University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics - Philadelphia

Alberta

  • University of Alberta - Edmonton

Ontario

  • The Hospital for Sick Children - SickKids - Toronto

Trial Details

FieldValue
Enrollment Target 32 participants
Start Date 2025-06-04
Est. Completion 2028-10
Phase Phase 3
Laboratoires Thea

11 total trials

What NCT06891443 shows while recruiting

NCT06891443 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 32 participants, a relatively small participant target, below the 741,302-participant average among 27 other Eye Diseases trials with a reported enrollment target (100% lower).

The record links to 10 conditions, with Eye Diseases appearing as the primary indexed condition, and to 2 interventions - of which sepofarsen is the first listed.

NCT06891443 lists 14 locations in 8 states (Other, California, Florida).

Frequently Asked Questions

What is clinical trial NCT06891443 about?

NCT06891443 is a clinical study titled "Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)". 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.

What is the current status of trial NCT06891443?

This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 32 participants. The study started on 2025-06-04. Estimated completion is 2028-10.

What conditions does trial NCT06891443 study?

This clinical trial studies the following conditions: Eye Diseases, Retinal Disease, Eye Diseases, Hereditary, Blindness, Sensation Disorders.

What interventions are being tested in trial NCT06891443?

The interventions under investigation include: sepofarsen (DRUG), Placebo IVT (OTHER).

Who is sponsoring clinical trial NCT06891443?

This trial is sponsored by Laboratoires Thea, which has 11 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT06891443 being conducted?

This trial has 14 study locations across California, Florida, Iowa, Minnesota, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Similar trials for Eye Diseases

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT06891443's enrollment target sits among peer trials

32 21st of 27 higher than 7 of 27 other Eye Diseases trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Eye Diseases trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT06891443, the US trial registry maintained by the National Library of Medicine. NCT06891443 (small enrollment · multi site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.