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

NCT04355689 | Active Not Recruiting Phase 1 DMC

• 1 other identifier: C-18-04
• Study Type: interventional
• Target: 49
• Locations: 1 country
• Sites: 4

Brief 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.

Conditions

Usher Syndromes

Keywords

retinitis pigmentosa Usher vision NPI-001 tablet

Outcome Measures

Primary Outcomes (1)

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

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

  24 months

Study Arms (2)

NPI-001

EXPERIMENTAL

NPI-001 Tablet, 250 mg, BID

Drug: NPI-001

Placebo

PLACEBO COMPARATOR

Placebo Tablet, BID

Other: Placebo

Interventions

NPI-001 DRUG

oral tablet

NPI-001

Placebo OTHER

Placebo tablets

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• Ocular:
• 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.
• Non-Ocular:
• 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.
• Suspected liver dysfunction determined by having alanine aminotransferase (ALT), aspartate aminotransferase (AST), or bilirubin values \> 1.5 X the upper limit of normal (ULN).
• Platelet or hemoglobin values that are below the lower limit of normal at screening (subjects with normal hemoglobin and mean corpuscular volume below the lower limit of normal should have iron studies performed to ensure that they are iron replete before taking part in the study), or neutrophils or white cell count which is above the upper limit of normal.
• Presence of more than + proteinuria on urinalysis at screening or (confirmed by abnormal albumin creatinine ratio).
• Presence of hematuria on urinalysis at screening. (If hematuria is detected on urinalysis, then the specimen should be subjected to microscopy, and subject should be excluded if more than 10 X 106 red blood cells/L.) If the subject is a female in whom the hematuria may be due to menses, then the urinalysis can be repeated after a few days.
• C-reactive protein (CRP) value above 10 mg/L.

Sponsors & Collaborators

• Nacuity Pharmaceuticals, Inc.: lead
• Foundation Fighting Blindness: collaborator

Study Sites (4)

Queensland Eye Institute

Brisbane, Australia

Location

CERA

Melbourne, Australia

Location

Lions Eye Institute

Perth, Australia

Location

Sydney Eye Hospital / Save Sight Institute

Sydney, Australia

Location

MeSH Terms

Conditions

Usher Syndromes

Condition Hierarchy (Ancestors)

Deaf-Blind Disorders; Deafness; Hearing Loss; Hearing Disorders; Ear Diseases; Otorhinolaryngologic Diseases; Hearing Loss, Sensorineural; Sensation Disorders; Neurologic Manifestations; Nervous System Diseases; Blindness; Vision Disorders; Retinitis Pigmentosa; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Eye Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Eye Diseases, Hereditary; Genetic Diseases, Inborn; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Lee Anderson, MD

  Nacuity Pharmaceuticals, Inc.

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double-masked
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: NPI-001 Tablets versus Placebo

Study Record Dates

• First Submitted: April 17, 2020
• First Posted: April 21, 2020
• Study Start: September 3, 2020
• Primary Completion: June 20, 2025
• Study Completion: April 1, 2026
• Last Updated: December 23, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

AU (4)