438 Participants Needed

N-acetylcysteine for Retinitis Pigmentosa

(NAC Attack Trial)

Recruiting at 34 trial locations
MJ
GH
MJ
DW
XK
IW
Overseen ByIzzie Wang, Coordinating Center
Age: 18+
Sex: Any
Trial Phase: Phase 3
Sponsor: Johns Hopkins University
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

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?

N-acetylcysteine (NAC) is unique for retinitis pigmentosa as it is an antioxidant that may help protect retinal cells from oxidative stress, unlike other treatments like gene therapy or carbonic anhydrase inhibitors, which target specific genetic mutations or reduce fluid buildup in the eye.26789

Research Team

PA

Peter A Campochiaro, MD

Principal Investigator

Johns Hopkins University

XK

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

Both of my eyes are affected by RP, losing night vision and field of view but still see clearly.
Ability and willingness to provide informed consent
Ability and willingness to comply with the study protocol and to participate in all study visits and assessments in the investigator's judgement
See 4 more

Exclusion Criteria

My blood pressure is not under control.
I have a history of liver disease, cystic fibrosis, asthma, COPD, low platelet count, or other blood disorders.
I am receiving treatment for kidney failure.
See 12 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 1800 mg of N-acetylcysteine or placebo twice a day for 45 months

45 months
Regular visits at 4.5, 9, 18, 27, 36, 40.5, and 45 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Treatment Details

Interventions

  • N-acetylcysteine
  • Placebo
Trial OverviewThe clinical trial is testing whether long-term use of N-acetylcysteine (NAC) can slow down cone degeneration in patients with RP by reducing oxidative stress. Participants will either receive NAC or a placebo for several years across multiple locations internationally.
Participant Groups
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Group 1 - N-acetylcysteineExperimental Treatment1 Intervention
This is the intervention group. Patients in this group will be receiving 1800 mg of N-acetylcysteine in the form of 3 effervescent 600 mg tablets dissolved in water twice a day for 45 months.
Group II: Group 2 - PlaceboPlacebo Group1 Intervention
Patients in the placebo group will receive identical effervescent tablets lacking active drug.

N-acetylcysteine is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as N-acetylcysteine for:
  • Acetaminophen overdose
  • Chronic bronchitis
  • Cystic fibrosis
  • Mucolytic agent
🇪🇺
Approved in European Union as N-acetylcysteine for:
  • Paracetamol overdose
  • Chronic bronchitis
  • Cystic fibrosis
  • Mucolytic agent

Find a Clinic Near You

Who Is Running the Clinical Trial?

Johns Hopkins University

Lead Sponsor

Trials
2,366
Recruited
15,160,000+

McGill University

Collaborator

Trials
421
Recruited
1,017,000+

Medical University of Graz

Collaborator

Trials
524
Recruited
9,334,000+

Radboud University Medical Center

Collaborator

Trials
1,203
Recruited
1,157,000+

Universität Tübingen

Collaborator

Trials
30
Recruited
14,900+

Retina Foundation of the Southwest

Collaborator

Trials
15
Recruited
1,600+

University of Florida

Collaborator

Trials
1,428
Recruited
987,000+

Stanford University

Collaborator

Trials
2,527
Recruited
17,430,000+

Massachusetts Eye and Ear Infirmary

Collaborator

Trials
115
Recruited
15,000+

Northwestern University

Collaborator

Trials
1,674
Recruited
989,000+

Findings from Research

In a study involving 30 patients with retinitis pigmentosa, higher doses of N-acetylcysteine (NAC) were linked to a reduced risk of sensitivity loss in the macula, suggesting that NAC may help protect vision during treatment.
The research found that loci with low baseline sensitivity were more likely to show both decreases and increases in sensitivity, with foveal regions showing a greater likelihood of improvement, indicating that areas with higher cone density may respond better to NAC treatment.
Locus-Level Changes in Macular Sensitivity in Patients with Retinitis Pigmentosa Treated with Oral N-acetylcysteine.Kong, X., Hafiz, G., Wehling, D., et al.[2022]
In a study involving 8 patients with retinitis pigmentosa, treatment with a topical carbonic anhydrase inhibitor (2% dorzolamide) led to significant reductions in foveal thickness and foveal zone thickness, indicating a positive effect on cystoid macular edema.
Three out of the eight patients experienced clinically significant improvements in visual acuity, suggesting that this treatment may not only reduce edema but also enhance vision in some individuals with retinitis pigmentosa.
Continued use of dorzolamide for the treatment of cystoid macular oedema in patients with retinitis pigmentosa.Fishman, GA., Apushkin, MA.[2022]
In a study involving 79 eyes of 40 patients with chronic posterior blepharitis, N-acetylcysteine (NAC) significantly improved the fluorescein break-up time (FBUT) and mucous fern pattern, indicating enhanced tear stability and eye surface health.
The mechanism of action for NAC in this context is believed to involve the blocking of lipid peroxidation, which may help alleviate symptoms of chronic blepharitis.
N-acetylcysteine in chronic blepharitis.Yalçin, E., Altin, F., Cinhüseyinoglue, F., et al.[2019]

References

Locus-Level Changes in Macular Sensitivity in Patients with Retinitis Pigmentosa Treated with Oral N-acetylcysteine. [2022]
Continued use of dorzolamide for the treatment of cystoid macular oedema in patients with retinitis pigmentosa. [2022]
N-acetylcysteine in chronic blepharitis. [2019]
Oral N-acetylcysteine improves cone function in retinitis pigmentosa patients in phase I trial. [2020]
N-Acetylcysteine promotes long-term survival of cones in a model of retinitis pigmentosa. [2013]
The first gene therapy for RPE65 biallelic dystrophy with voretigene neparvovec-rzyl in Brazil. [2022]
Efficacy and prognostic factors of response to carbonic anhydrase inhibitors in management of cystoid macular edema in retinitis pigmentosa. [2022]
RPE65 c.353G>A, p.(Arg118Lys): A Novel Point Mutation Associated with Retinitis Pigmentosa and Macular Atrophy [2023]
Successful RPE65 gene replacement and improved visual function in humans. [2012]