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Gene therapy shows promise for rare Blindness-Causing eye disease

NCT ID NCT03374657

Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage study tested a gene therapy called CPK850 in 12 adults with a rare inherited eye disease (RLBP1 retinitis pigmentosa) that causes progressive vision loss and night blindness. The treatment was injected under the retina to deliver a working copy of the faulty gene, aiming to improve the eye's ability to adapt to darkness. The main goals were to check safety and see if vision function could be restored.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

12 people

The number who actually took part.

Started

Aug 2018

Finished

May 2026

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

18 to 70 years

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: * Male and female patients aged 18 to 70 years inclusive. * The visual acuity in the study eye at the screening 1 visit should be no better than 60 ETDRS letters. * Clinical diagnosis of Bothnia dystrophy, Newfoundland rod-cone dystrophy or other progressive retinitis pigmentosa phenotype with mutations in the RLBP1 gene verified by genetic testing. * Visible photoreceptor (outer nuclear) and Retinal Pigment Epithelium (RPE) layers on standard OCT scan in the study eye at the screening 1 visit. Exclusion Criteria: * History of hypersensitivity to the study drug or to drugs of similar classes or to any of the medications required in the perioperative period. * Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints * Any contraindication to the planned surgery or anesthesia as determined by the treating physician (surgeon, anesthesiologist, internist, or designee). * Women who are pregnant, or lactating or women of childbearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for two months after treatment

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Conditions

The condition(s) this trial relates to.

retinitis pigmentosa RLBP1-related retinopathy

As listed by the trial registrant

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

Retinitis pigmentosa

Contacts and locations

Locations

  • Novartis Investigative Site

    Stockholm, SE-112 82, Sweden

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