What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Retinitis Pigmentosa that focus on reducing oxidative damage to cone photoreceptors similar to N-acetylcysteine (NAC). Current research, including the FIGHT-RP 1 Study, is exploring the potential of NAC due to its antioxidant properties and ability to promote cone survival and function. Other treatments under investigation include various antioxidants and nutritional supplements aimed at slowing the progression of RP.

What other types of research exist?

Promising alternatives to N-acetylcysteine for Retinitis Pigmentosa patients include high-dose vitamin A and E supplementation, particularly in conditions like abetalipoproteinemia. Other potential treatments could involve emerging pharmacotherapies targeting oxidative stress pathways, although specific novel agents were not detailed in the provided context.

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Antioxidant

N-acetylcysteine for Retinitis Pigmentosa

Q: What side effects are associated with this type of intervention?

Common treatments for Retinitis Pigmentosa, particularly antioxidants like N-acetylcysteine (NAC), can have several side effects. NAC is generally well-tolerated but can cause gastrointestinal issues such as vomiting, diarrhea, stomatitis, abdominal pain, and nausea. Less frequent but serious side effects include hypersensitivity reactions like anaphylactic shock, dyspnea, bronchospasm, angioedema, tachycardia, urticaria, rash, and pruritus. Other reported side effects include headache, tinnitus, pyrexia, decreased blood pressure, face edema, and hemorrhage. Patients should discuss these potential side effects with their healthcare provider when considering treatment options.

Phase 1

Waitlist Available

Led By Peter Campochiaro

Research Sponsored by Johns Hopkins University

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients diagnosed with RP

Age 18 years

Must not have

Patients with a concomitant ocular pathology that limits central macular function, including but not limited to: age-related macular degeneration, diabetic retinopathy, and retinal vein occlusion

Patients with uncontrolled hypertension (defined as diastolic blood pressure > 95 mm Hg or systolic blood pressure > 160 mm Hg despite medical therapy)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2 years

Awards & highlights

No Placebo-Only Group

All Individual Drugs Already Approved

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

NAC may cause gastrointestinal issues such as vomiting, diarrhea, and abdominal pain. Less common are hypersensitivity reactions like anaphylactic shock or skin rashes. Other possible side effects include headache, tinnitus, fever, low blood pressure, facial swelling, and bleeding.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been diagnosed with retinitis pigmentosa.

Select...

I am 18 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have an eye condition affecting the center of my vision.

Select...

My blood pressure is not controlled even with medication.

Select...

I currently have an eye infection.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Blood Pressure (mmHg)

Tolerability of N-Acetylcysteine as assessed by drug-related symptoms

Tolerability of N-Acetylcysteine as assessed by number of times medication is taken per day

+1 more

Secondary study objectives

Change in aqueous reduced to oxidized glutathione ratio (GSH/GSSG)

Change in best corrected visual acuity (BCVA)

Change in central retinal sensitivity as assessed by microperimetry

+1 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Experimental ArmExperimental Treatment1 Intervention

All participants to receive study intervention.

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Retinitis Pigmentosa (RP) is characterized by progressive loss of photoreceptors, primarily due to oxidative stress. Antioxidants like N-acetylcysteine (NAC) play a crucial role in reducing oxidative damage to cone photoreceptors, thereby promoting their survival and function. NAC works by replenishing intracellular glutathione levels, a key antioxidant that neutralizes reactive oxygen species (ROS) and reduces oxidative stress. This mechanism is particularly important for RP patients as it helps slow the progression of vision loss and maintains visual function. Other treatments, such as gene therapy, aim to correct genetic defects, but antioxidants like NAC offer a more immediate approach to managing oxidative damage in RP.

Efficacy of various antioxidants in the protection of the retinal pigment epithelium from oxidative stress.