Oral N-acetylcysteine for Retinitis Pigmentosa

Inclusion Criteria

• •General
• •Ability and willingness to provide informed consent
• •Age ≥ 18 and ≤65 years at time of signing Informed Consent Form
• •Ability and willingness to comply with the study protocol and to participate in all study visits and assessments in the investigator's judgement
• •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.
• •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
• •Cerebrovascular accident or myocardial infarction within 6 months of screening
• •Participation in an investigational study that involves treatment with any drug or device within 6 months of screening
• •Three relatives already enrolled in study
• •Pregnant, breast feeding, or intending to become pregnant during the study treatment period. Women of childbearing potential who have not had tubal ligation must have a urine pregnancy test at screening.
• •Known history of allergy to NAC
• •Having taken NAC in any form in the past 4 months
• •Phenylketonuria
• •Fructose intolerance
• •Glucose-galactose malabsorption
• •Sucrase-isomaltase insufficiency
• •Abnormal laboratory value including the value of alanine aminotransferase (ALT), aspartate aminotransferase (AST), or bilirubin being greater than 1.5 x the upper limit of normal
• •Any major abnormal findings on blood chemistry, hematology, and renal function lab tests that in the opinion of the Site Investigator and/or the Study Chair makes the candidate not suitable to participate in the trial
• •HIV or hepatitis B infection