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Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome | Clinical Trials | Clareo Health

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Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome

Safety and Efficacy of NPI-001 Tablets Versus Placebo for Treatment of Retinitis Pigmentosa Associated With Usher Syndrome

NCT04355689•Nacuity Pharmaceuticals, Inc.

View on ClinicalTrials.gov

Summary

This study will examine the safety and efficacy of NPI-001 Tablets as compared to placebo for 24 months in subjects with vision loss due to RP associated with Usher syndrome.

Detailed Description

This study will examine the safety and efficacy of oral NPI-001 Tablets as compared to oral placebo tablets for 24 months in subjects with vision loss due to RP associated with Usher syndrome.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Male or female, age ≥18 years.
•2. Able to comprehend and willing to sign an informed consent form (ICF) and to adhere to the study protocol.
•3. Diagnosed with Usher syndrome.
•4. EZ zone with width ≥500 microns, which includes the fovea in each eye at Visit 2, (Screen B).
•5. Have at least 20 detectable points on the MAIA grid.
•6. On stable dose of medications associated with other conditions for at least one month.
•7. Both female participants of childbearing potential and male participants able to father children must have (or have a partner who has) had a bilateral oophorectomy, hysterectomy or bilateral salpingectomy; must abstain from intercourse; or must agree to practice 2 acceptable methods of contraception throughout the course of the study and 4 weeks after the last visit. Acceptable methods of contraception include hormonal contraception (i.e., birth control pills, injected hormones, dermal patch or vaginal ring), intrauterine device, barrier methods (diaphragm, condom) with spermicide, tubal ligation, or vasectomy.

Exclusion Criteria

•Ocular:
•1. All edges of the EZ area in both eyes cannot be visualized at Visit 2 (Screen B).
•2. Concurrent retinal pathologies that result in vision loss or inability to fixate, including but not limited to, choroideremia, retinal vein occlusion, and neovascular age-related macular degeneration.
•3. Intraocular surgery within the last two months or capsulotomy within the last month.
•4. 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.
•5. Unstable fixation during microperimetry in either eye at either screening or baseline visits.
•Non-Ocular:
•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. Liver or kidney disease, cystic fibrosis, asthma or chronic obstructive pulmonary disease (COPD), history of thrombocytopenia not due to a reversible cause, or other blood dyscrasia.
+14 more criteria

Locations (4)

Queensland Eye Institute

Brisbane, Australia

CERA

Melbourne, Australia

Lions Eye Institute

Perth, Australia

Sydney Eye Hospital / Save Sight Institute

Sydney, Australia

Key Dates

Start Date

September 3, 2020

Primary Completion Date

June 20, 2025

Completion Date

April 1, 2026

Last Updated

December 23, 2025

Enrollment

ACTUAL participants

Conditions

Usher Syndromes

Interventions

NPI-001

DRUG

Placebo

OTHER

Natural History Study of Usher Syndrome ( Light4Deaf )

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Usher Syndromes

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UNKNOWN

Phenotype Correlates Genotype of Inherited Retina Dystrophies, Retinitis Pigmentosa, Con>Rod Dystrophies.

NCT03990727

Retinitis PigmentosaCone Dystrophy+2 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: December 23, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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