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Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
NCT06891443 · View on ClinicalTrials.gov ↗
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.
Conditions Studied
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
| Field | Value |
|---|---|
| Enrollment Target | 32 participants |
| Start Date | 2025-06-04 |
| Est. Completion | 2028-10 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06891443
The ClinicalTrials.gov registry entry for NCT06891443 describes a study currently listed as recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 32 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Laboratoires Thea, which has 32 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06891443 reports 14 study locations spanning 8 distinct geographic areas — top geographies include Other, California, Florida. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06891443?
The interventions under investigation include: sepofarsen (DRUG), Placebo IVT (OTHER). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06891443?
This trial is sponsored by Laboratoires Thea, which has 32 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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