N-acetylcysteine for Retinitis Pigmentosa
(NAC Attack Trial)
Trial Summary
What is the purpose of this trial?
This trial is testing if N-acetylcysteine (NAC) can help slow down vision loss in people with Retinitis Pigmentosa (RP). RP is a genetic condition that causes gradual vision loss. NAC reduces damage caused by too much oxygen around eye cells, potentially protecting vision. N-acetylcysteine (NAC) has been shown to reduce oxidative damage and increase cone function/survival in studies related to RP.
Do I need to stop my current medications to join the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, you cannot take supplements other than vitamin A, and your blood pressure must be controlled if you are on antihypertensive medication.
What data supports the effectiveness of the drug N-acetylcysteine for retinitis pigmentosa?
Research shows that N-acetylcysteine (NAC) can improve vision and protect eye cells in people with retinitis pigmentosa by reducing oxidative stress (damage caused by harmful molecules). In a study, patients taking NAC experienced improved visual function, and animal studies showed that NAC helped preserve eye cell structure and function over time.12345
How does the drug N-acetylcysteine differ from other treatments for retinitis pigmentosa?
Research Team
Peter A Campochiaro, MD
Principal Investigator
Johns Hopkins University
Xiangrong Kong
Principal Investigator
Johns Hopkins University
Eligibility Criteria
Adults aged 18-65 with Retinitis Pigmentosa (RP), willing to consent and follow the study protocol, not taking supplements other than vitamin A, and using contraception if of childbearing potential. Excluded are those with uncontrolled blood pressure, recent participation in another drug/device trial, certain genetic disorders like phenylketonuria or fructose intolerance, active cancer within a year (with exceptions), eye conditions that could affect results, severe myopia over -8 diopters, liver disease, cystic fibrosis, asthma/COPD or renal failure.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive 1800 mg of N-acetylcysteine or placebo twice a day for 45 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- N-acetylcysteine
- Placebo
N-acetylcysteine is already approved in United States, European Union for the following indications:
- Acetaminophen overdose
- Chronic bronchitis
- Cystic fibrosis
- Mucolytic agent
- Paracetamol overdose
- Chronic bronchitis
- Cystic fibrosis
- Mucolytic agent
Find a Clinic Near You
Who Is Running the Clinical Trial?
Johns Hopkins University
Lead Sponsor
McGill University
Collaborator
Medical University of Graz
Collaborator
Radboud University Medical Center
Collaborator
Universität Tübingen
Collaborator
Retina Foundation of the Southwest
Collaborator
University of Florida
Collaborator
Stanford University
Collaborator
Massachusetts Eye and Ear Infirmary
Collaborator
Northwestern University
Collaborator