Can an antioxidant pill slow blindness in usher syndrome?
NCT ID NCT07710196
First seen Jul 17, 2026 · Last updated Jul 17, 2026
Summary
This phase 3 trial tests whether an experimental drug called NPI-001 can slow the loss of light-sensing cells in the eyes of adults with Usher syndrome, a genetic condition that causes both hearing and vision loss. About 80 participants take either NPI-001 or a placebo twice daily for 24 months. Researchers measure changes in the area of the retina's ellipsoid zone to see if the drug preserves vision.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- an experimental oral drug called NPI-001 (N-acetyl cysteine amide)
- What this could lead to
- If it works, this could point toward a treatment that slows vision loss in people with Usher syndrome.
- What could go wrong
- This is a relatively small trial, and many promising drugs fail to show benefit in larger studies. The drug may not slow vision loss or could cause side effects.
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
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 80 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Aug 2026
An estimate. Start dates often move.
- Expected to finish
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Feb 2029
An estimate. End dates often move.
- Lead sponsor
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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
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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. Able to comprehend and willing to sign an informed consent form (OCF) and adhere to the study protocol. 2. Diagnosed with RP associated with Usher syndrome. 3. EZ width ≥ 500 microns, which includes the fovea in each eye at Visit 1. 4. All edges of the EZ area in both eyes can be visualized at Visit 1 (Screening). 5. Have at least 20 detectable points on the MAIA grid in at least one eye at the Screening and Baseline visits (same eye for both visits). 6. On stable dose of medications associated with other conditions for at least one month. 7. Willing to use contraception during study (for anyone of childbearing potential). Exclusion Criteria: 1. Concurrent retinal pathologies that result in vision loss or inability to fixate. 2. Intraocular surgery within the last two months or capsulotomy within the last month. 3. Current or history of uveitis, Coat's disease, diabetic retinopathy, glaucoma, herpes simplex of the eye, or currently has a cataract that prevents visualization of the posterior pole. 4. Likely to require cataract surgery within the next 12 months. 5. Unstable fixation during microperimetry in either eye at either Screening or Baseline visits. 6. Use of any other investigational new drug, or participation in another clinical trial within 12 weeks before the start of study treatment. 7. Use of N-acetylcysteine containing products in the previous 30 days prior to the baseline visit or unwilling to refrain from such supplements for the duration of the study. 8. Chronic liver or kidney disease, cystic fibrosis, severe asthma, or chronic obstructive pulmonary disease (COPD), history of thrombocytopenia not due to a reversible cause, or other blood dyscrasia. 9. Suspected liver dysfunction determined by having alanine aminotransferase (ALT), aspartate aminotransferase (AST), or bilirubin values \> 1.5 X the upper limit of normal (ULN) at screening. 10. Platelet or hemoglobin values \< 100 at screening. 11. History of known sensitivity to N-acetylcysteine or similar thiol compounds or any ingredients of NPI-001. 12. History of hypersensitivity to any medication or food resulting in systemic symptoms. 13. History of cancer (other than non-melanoma skin cancer) diagnosed or requiring treatment within the past 2 years. 14. Pregnant women or women planning to become pregnant in the next 25 months or men with partners planning to become pregnant in the next 25 months. 15. Lactating women who are breast-feeding. 16. Potential participant lives in the same household as a current participant in this study. 17. Subjects who are unwilling or unable to refrain from donation of blood from 30 days prior to Screening, plasma from 2 weeks prior to Screening, or platelets from 6 weeks prior to Screening until 56 days following the last study drug dose administered. 18. Inability to provide blood samples, including difficulty with venous access. 19. Any reason, in the opinion of the Principal Investigator, the participant should not participate.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
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Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
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