30 Participants Needed

N-acetylcysteine for Retinitis Pigmentosa

Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: Johns Hopkins University
Must be taking: N-acetylcysteine
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Trial Summary

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

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 had better visual function, and in animal models, NAC helped preserve eye cell structure and function over time.12345

Is N-acetylcysteine safe for humans?

N-acetylcysteine (NAC) is generally considered safe for humans, but it can cause some side effects like stomach issues, which usually go away on their own or with a lower dose. It has been used for various conditions and has a good safety profile, making it feasible for long-term treatment.12367

How does the drug N-acetylcysteine differ from other treatments for retinitis pigmentosa?

N-acetylcysteine (NAC) is unique because it works by reducing oxidative stress, which helps protect and improve the function of cone cells in the eye, potentially slowing vision loss in retinitis pigmentosa. Unlike other treatments, NAC is taken orally as effervescent tablets, making it a convenient option for long-term use.12348

What is the purpose of this trial?

This trial is testing N-acetylcysteine (NAC) tablets to help people with Retinitis Pigmentosa (RP), a disease that causes vision loss. NAC is an antioxidant that protects eye cells from damage. The goal is to see if NAC can slow down or stop the progression of vision loss in RP patients.

Research Team

PC

Peter Campochiaro

Principal Investigator

Johns Hopkins University

Eligibility Criteria

This trial is for adults over 18 with Retinitis Pigmentosa (RP), a condition leading to vision loss. Participants must consent to use their health information and understand the study's details. It excludes those with other eye diseases affecting central vision, active eye infections, or uncontrolled high blood pressure.

Inclusion Criteria

I have been diagnosed with retinitis pigmentosa.
Authorization of use and disclosure of protected health information
Informed consent

Exclusion Criteria

I have an eye condition affecting the center of my vision.
My blood pressure is not controlled even with medication.
I currently have an eye infection.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive N-Acetylcysteine (NAC) at a dose of 1800 mg twice a day

Up to 2 years
Visits every three months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may continue receiving NAC to assess long-term safety and tolerability

Long-term

Treatment Details

Interventions

  • NAC effervescent tablets
Trial Overview The FIGHT-RP 1 Extension Study tests NAC effervescent tablets as a potential treatment for RP. The study builds on previous findings that suggest antioxidants like NAC could help preserve eyesight in RP by reducing oxidative damage in the retina.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Experimental ArmExperimental Treatment1 Intervention
All participants to receive study intervention.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Johns Hopkins University

Lead Sponsor

Trials
2,366
Recruited
15,160,000+

Zambon SpA

Industry Sponsor

Trials
31
Recruited
6,300+

Findings from Research

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]
Oral N-acetylcysteine (NAC) is safe and well tolerated in patients with moderately advanced retinitis pigmentosa (RP), with a maximum tolerated dose of 1800 mg twice daily, despite some gastrointestinal side effects that resolved with dose adjustments.
The treatment showed significant improvements in best-corrected visual acuity (BCVA) over 24 weeks, particularly in the higher dose cohort (1800 mg), suggesting NAC may enhance the function of compromised cone photoreceptors in RP.
Oral N-acetylcysteine improves cone function in retinitis pigmentosa patients in phase I trial.Campochiaro, PA., Iftikhar, M., Hafiz, G., et al.[2020]
Oral administration of N-acetylcysteine (NAC) significantly reduced cone cell death and preserved cone function in two mouse models of retinitis pigmentosa (RP) for at least 6 months, indicating its potential as a long-term treatment.
Topical application of NAC also effectively reduced oxidative damage in the retina, suggesting that both oral and topical forms of NAC could be safe and feasible options for prolonged treatment in humans with RP.
N-Acetylcysteine promotes long-term survival of cones in a model of retinitis pigmentosa.Lee, SY., Usui, S., Zafar, AB., et al.[2013]

References

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]
Continued use of dorzolamide for the treatment of cystoid macular oedema in patients with retinitis pigmentosa. [2022]
Locus-Level Changes in Macular Sensitivity in Patients with Retinitis Pigmentosa Treated with Oral N-acetylcysteine. [2022]
N-acetylcysteine Pharmacology and Applications in Rare Diseases-Repurposing an Old Antioxidant. [2023]
N-acetylcysteine in handgrip exercise: plasma thiols and adverse reactions. [2021]
Protection of human retinal pigment epithelial cells from oxidative damage using cysteine prodrugs. [2021]
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