Active — Not RecruitingPhase 1/2

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

Australia49 participantsStarted 2020-09-03

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Male or female, age ≥18 years.
✓. Able to comprehend and willing to sign an informed consent form (ICF) and to adhere to the study protocol.
✓. Diagnosed with Usher syndrome.
✓. EZ zone with width ≥500 microns, which includes the fovea in each eye at Visit 2, (Screen B).
✓. Have at least 20 detectable points on the MAIA grid.
✓. On stable dose of medications associated with other conditions for at least one month.
✓. 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

✕. All edges of the EZ area in both eyes cannot be visualized at Visit 2 (Screen B).
✕. 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.
✕. Intraocular surgery within the last two months or capsulotomy within the last month.
✕. 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.
✕. Unstable fixation during microperimetry in either eye at either screening or baseline visits.
✕. Use of any other investigational new drug, or participation in another clinical trial within 12 weeks before the start of study treatment.
✕. 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.
✕. 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.

What they're measuring

Evaluate change from baseline for retinal sensitivity assessed by microperimetry of active versus placebo

Timeframe: 24 months

Trial details

NCT IDNCT04355689

SponsorNacuity Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-06-20

View on ClinicalTrials.gov More Usher Syndromes trials