Ignite Creation Date:
2024-05-06 @ 4:04 PM
Last Modification Date:
2024-10-26 @ 2:03 PM
Study NCT ID:
NCT04864496
Status:
UNKNOWN
Last Update Posted:
2021-04-29
First Post:
2021-04-21
Brief Title:
Effects of Treatment With N- Acetylcysteine on Visual Outcomes in Patients With Retinitis Pigmentosa
Sponsor:
Shahid Beheshti University of Medical Sciences
Organization:
Shahid Beheshti University of Medical Sciences
Study Overview
Official Title:
Effects of Oral N- Acetylcysteine on Macular Function in Retinitis Pigmentosa a Phase 2 Randomized Controlled Trial
Status:
UNKNOWN
Status Verified Date:
2021-04
Last Known Status:
ACTIVE_NOT_RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Retinitis pigmentosa RP is an inherited retinal disease with great heterogeneity RP comprises a large group of genetic disorders causing progressive loss of vision Despite many suggested treatments there is actually no effective therapy for most types of RP at present Mutations that cause RP initially lead to rod cell death After rod photoreceptors death cone photoreceptors also gradually die There are several hypotheses as to why mutation-induced rod photoreceptor cell death invariably leads to gradual dysfunction and death of cone photoreceptors resulting in severe visual acuity loss and blindness Rods constitute 95 percent of cells in the outer retina As they degenerate oxygen consumption is reduced and the level of tissue oxygen markedly increases After rods degeneration several markers of oxidative damage appear in cones This oxidative stress over time may lead to cone dysfunction and death Antioxidants reduce markers of oxidative damage and promote cone function and survival In RP cone death occurs as a result of the death of rods rather than as the result of the pathogenic mutations and therefore treatment with antioxidants may have the potential to be applied to all patients with RP irrespective of the disease-causing mutation
N-acetylcysteine is a derivative of L cysteine that plays a role in the biosynthesis of glutathione and neutralizes reactive oxygen species It also has a direct antioxidant activity via its reactive sulfhydryl agent Its systemic use shows an acceptable safety profile It has been shown that the use of systemic N-acetylcysteine provides significant intraocular concentration and antioxidant activity that may lead to the promotion of cone function and survival
In a recent phase 1 randomized clinical trial RCT it was revealed that oral N-acetylcysteine NAC was safe and well-tolerated in patients with moderately advanced RP and might improve sub-optimally functioning macular cones The authors concluded that a randomized placebo-controlled trial is needed to determine if oral NAC can provide long-term stabilization andor improvement in visual function in patients with RP In this phase 2 RCT eligible patients with the diagnosis of moderately advanced RP are randomly divided into two groups treatment group N-acetylcysteine tablets and controls placebo Each group will be treated for 6 months In this study we will investigate if the use of oral N- acetylcysteine as a potent antioxidant agent can slow down or reverse the disease process in RP patients with prior moderate loss of vision It may potentially demonstrate a treatment modality regardless of the genetic type of RP The primary outcome measure will be the stability or improvement of the best-corrected visual acuity BCVA The secondary outcome measures will be changes in color vision electroretinogram visual field structural OCT indices after 6 months The same parameters will be re-evaluated 3 months after discontinuation of treatment at month 9
N-acetylcysteine is a derivative of L cysteine that plays a role in the biosynthesis of glutathione and neutralizes reactive oxygen species It also has a direct antioxidant activity via its reactive sulfhydryl agent Its systemic use shows an acceptable safety profile It has been shown that the use of systemic N-acetylcysteine provides significant intraocular concentration and antioxidant activity that may lead to the promotion of cone function and survival
In a recent phase 1 randomized clinical trial RCT it was revealed that oral N-acetylcysteine NAC was safe and well-tolerated in patients with moderately advanced RP and might improve sub-optimally functioning macular cones The authors concluded that a randomized placebo-controlled trial is needed to determine if oral NAC can provide long-term stabilization andor improvement in visual function in patients with RP In this phase 2 RCT eligible patients with the diagnosis of moderately advanced RP are randomly divided into two groups treatment group N-acetylcysteine tablets and controls placebo Each group will be treated for 6 months In this study we will investigate if the use of oral N- acetylcysteine as a potent antioxidant agent can slow down or reverse the disease process in RP patients with prior moderate loss of vision It may potentially demonstrate a treatment modality regardless of the genetic type of RP The primary outcome measure will be the stability or improvement of the best-corrected visual acuity BCVA The secondary outcome measures will be changes in color vision electroretinogram visual field structural OCT indices after 6 months The same parameters will be re-evaluated 3 months after discontinuation of treatment at month 9
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None