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Experimental eye drug hopes to restore sight in rare blindness

NCT ID NCT06891443

Disease control Sponsor: Laboratoires Thea Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 trial tests an experimental drug called sepofarsen in 32 people with Leber congenital amaurosis (LCA), a rare genetic condition that causes severe vision loss from birth. The drug is injected into one eye, while the other eye gets a placebo, to see if it safely improves vision. Participants must have a specific CEP290 gene mutation and be at least 6 years old.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
sepofarsen (an RNA-based drug injected into the eye)
What this could lead to
If it works, this could improve vision in people with a rare genetic form of blindness.
What could go wrong
This is a small, early-stage trial with only 32 people. The drug may not improve vision or could cause side effects like eye irritation or infection.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 32 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2025

Expected to finish

Oct 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

blindness (disorder) Eye Abnormalities Eye Diseases, Hereditary eye disorder Leber congenital amaurosis Leber congenital amaurosis 10 Leber hereditary optic neuropathy Meckel Syndrome, Type 4 Neurologic Manifestations retinal disorder Sensation Disorders vision disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Blindness Eye diseases Eye diseases, hereditary Eye disorders congenital Leber congenital amaurosis Leber congenital amaurosis 10 Neurological manifestations Retinal disease Sensation disorders Vision disorder

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    17 sites in 9 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Centre de maladies rares CHNO des Quinze Vingt

    RECRUITING

    Paris, 75012, France

  • Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP)

    RECRUITING

    São Paulo, São Paulo, 04023-062, Brazil

  • Hospital Sant Joan de Déu (SJD Barcelona Children's Hospital)

    RECRUITING

    Barcelona, 08950, Spain

  • INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte

    RECRUITING

    Belo Horizonte, Minas Gerais, 30150270, Brazil

  • Justus-Liebig Universität - Department of Ophthalmology

    RECRUITING

    Giessen, 35392, Germany

  • Klinikum der Ludwig-Maximilian Universität München

    RECRUITING

    München, 81377, Germany

  • Moorfields Eye Hospital NHS Foundation Trust

    RECRUITING

    London, EC1V 2PD, United Kingdom

  • Radboud Universitair Medisch Centrum

    RECRUITING

    Nijmegen, 6525 GA, Netherlands

  • The Hospital for Sick Children - SickKids

    RECRUITING

    Toronto, Ontario, M5G 2L3, Canada

  • UCSF Wayne and Gladys Valley Center for Vision

    RECRUITING

    San Francisco, California, 94158, United States

  • Universitair Ziekenhuis Gent (UZ)

    RECRUITING

    Ghent, 9000, Belgium

  • University of Alberta

    RECRUITING

    Edmonton, Alberta, T6G 2C8, Canada

  • University of Iowa

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Miami - Bascom Palmer Eye Institute

    RECRUITING

    Miami, Florida, 33156, United States

  • University of Minnesota Medical School

    RECRUITING

    Minneapolis, Minnesota, 55455, United States

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

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • University of Tuebingen - Inst. for Ophthalmic Research

    RECRUITING

    Tübingen, 72076, Germany

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