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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 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.

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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.

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

Sponsor

Laboratoires Thea

11 total trials

What the Registry Record Tells You About NCT06891443

The ClinicalTrials.gov registry entry for NCT06891443 describes a study currently listed as recruiting, categorized as Phase 3. The registered enrollment target is 32 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 741,302-participant average among 27 other Eye Diseases trials with a reported enrollment target (100% lower). The listed sponsor is Laboratoires Thea, which has 11 total studies on file at ClinicalTrials.gov.

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 reports 14 study locations spanning 8 distinct geographic areas - top geographies include 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.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.