NAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double-Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients with Retinitis Pigmentosa
- Sponsor: Johns Hopkins University
- Principal Investigator: Kimberly Stepien, MD
- Study Coordinator: Nickie Stangel
Study Objective:
To determine if taking N-acetylcysteine (NAC) for several years provides benefit in patients with retinitis pigmentosa (RP).
Study Design:
Eligible patients will be randomized 2:1 to NAC 1800 mg bid versus placebo for 45 months.
Inclusion Criteria:
- Age ≥ 18 and ≤65 years at time of signing Informed Consent Form
- For candidates of childbearing potential: willingness to use a method of contraception
- Agreement not to take supplements other than vitamin A
- Both eyes must exhibit the RP phenotype with evidence of loss of night vision, gradual constriction of visual fields, and maintenance of visual acuity;
- In addition, an eye must meet the following criteria to be included in the study:
- Gradable EZ on a horizontal SD-OCT scan through the fovea center with width ≤ 8000 µm and ≥1500 µm and with well-defined truncation at both the nasal and temporal sides;
- BCVA ≥ ETDRS letter score of 61 (20/60 Snellen equivalent);
- Sufficiently clear ocular media and adequate pupillary dilation to allow good quality images sufficient for analysis and grading by central reading center.
Exclusion Criteria:
- History of other disease, physical examination finding, or clinical laboratory finding giving reasonable suspicion that oral NAC may be contraindicated or that follow up may be jeopardized
- Evidence of cone-rod dystrophy or pattern dystrophy including focal areas of atrophy or pigmentary changes in the central macula
- Glaucoma or other optic nerve disease causing visual field loss or reduced visual acuity
- Any prior macular laser photocoagulation
- Intraocular surgery within 3 months prior to screening
- High myopia with spherical equivalent refractive error > 8 diopters. If an eye has had cataract surgery or refractive surgery, a pre-operative refractive error spherical equivalent > 8 diopters is an exclusion
- Any concurrent ocular condition that might affect interpretation of results
Contact Nickie Stangel with questions, (608) 263-8783
For more information about this study, visit clinicaltrials.gov: Oral N-acetylcysteine for Retinitis Pigmentosa – Full Text View – ClinicalTrials.gov