N-acetylcysteine for Retinitis Pigmentosa

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Retinitis Pigmentosa+1 More
N-acetylcysteine - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This trial will test if N-acetylcysteine can help treat retinitis pigmentosa, an inherited retinal degeneration caused by mutations.

Eligible Conditions
  • Retinitis Pigmentosa

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

1 Primary · 2 Secondary · Reporting Duration: Baseline, 4.5 months, 9 months, 18 months, 27 months, 36 months, 40.5 months, 45 months

45 months
Safety of oral NAC as assessed by adverse events
Baseline and 45 months
Change in best-corrected visual acuity
Change in mean macular sensitivity measured by microperimetry (MP)
Cumulative change of EZ area assessed as the area above curve (AAC)
Progressive change of ellipsoid zone (EZ) width
Month 45
Change in patient reported outcome assessed using NEI-VFQ 25
Month 36
Change in BCVA
Change in mean macular sensitivity measured by MP
Month 45
Eye
Eye
Month 45
Change in cone reflectivity measured by AOSLO
Change in cone regularity measured by AOSLO
Change in cone spacing measured by adaptive optics-scanning laser ophthalmoscopy (AOSLO)

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Trial Design

2 Treatment Groups

Group 1 - N-acetylcysteine
1 of 2
Group 2 - Placebo
1 of 2

Experimental Treatment

Non-Treatment Group

438 Total Participants · 2 Treatment Groups

Primary Treatment: N-acetylcysteine · Has Placebo Group · Phase 3

Group 1 - N-acetylcysteine
Drug
Experimental Group · 1 Intervention: N-acetylcysteine · Intervention Types: Drug
Group 2 - Placebo
Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
N-acetylcysteine
2008
Completed Phase 4
~1290

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: baseline, 4.5 months, 9 months, 18 months, 27 months, 36 months, 40.5 months, 45 months

Who is running the clinical trial?

University of MinnesotaOTHER
1,239 Previous Clinical Trials
1,460,159 Total Patients Enrolled
1 Trials studying Retinitis Pigmentosa
105 Patients Enrolled for Retinitis Pigmentosa
University of MiamiOTHER
787 Previous Clinical Trials
383,833 Total Patients Enrolled
Duke UniversityOTHER
2,156 Previous Clinical Trials
3,179,298 Total Patients Enrolled
Massachusetts Eye and Ear InfirmaryOTHER
99 Previous Clinical Trials
11,830 Total Patients Enrolled
1 Trials studying Retinitis Pigmentosa
49 Patients Enrolled for Retinitis Pigmentosa
Vitreo Retinal Associates, PAUNKNOWN
University of FloridaOTHER
1,237 Previous Clinical Trials
698,015 Total Patients Enrolled
University of MichiganOTHER
1,618 Previous Clinical Trials
6,410,351 Total Patients Enrolled
3 Trials studying Retinitis Pigmentosa
137 Patients Enrolled for Retinitis Pigmentosa
Johns Hopkins UniversityLead Sponsor
2,057 Previous Clinical Trials
31,002,363 Total Patients Enrolled
9 Trials studying Retinitis Pigmentosa
509 Patients Enrolled for Retinitis Pigmentosa
Emory UniversityOTHER
1,502 Previous Clinical Trials
2,677,149 Total Patients Enrolled
1 Trials studying Retinitis Pigmentosa
4 Patients Enrolled for Retinitis Pigmentosa
Universität TübingenOTHER
21 Previous Clinical Trials
3,487 Total Patients Enrolled
1 Trials studying Retinitis Pigmentosa
20 Patients Enrolled for Retinitis Pigmentosa
University Hospital, Basel, SwitzerlandOTHER
875 Previous Clinical Trials
2,099,353 Total Patients Enrolled
Vanderbilt UniversityOTHER
682 Previous Clinical Trials
6,131,023 Total Patients Enrolled
Radboud University Medical CenterOTHER
1,001 Previous Clinical Trials
510,250 Total Patients Enrolled
1 Trials studying Retinitis Pigmentosa
36 Patients Enrolled for Retinitis Pigmentosa
University of HoustonOTHER
123 Previous Clinical Trials
38,728 Total Patients Enrolled
University College London HospitalsOTHER
174 Previous Clinical Trials
459,130 Total Patients Enrolled
University of WashingtonOTHER
1,587 Previous Clinical Trials
1,566,124 Total Patients Enrolled
McGill UniversityOTHER
351 Previous Clinical Trials
972,333 Total Patients Enrolled
University of OklahomaOTHER
419 Previous Clinical Trials
96,862 Total Patients Enrolled
University of Southern CaliforniaOTHER
821 Previous Clinical Trials
1,361,979 Total Patients Enrolled
University of UtahOTHER
991 Previous Clinical Trials
1,768,470 Total Patients Enrolled
National Eye Institute (NEI)NIH
515 Previous Clinical Trials
1,072,498 Total Patients Enrolled
22 Trials studying Retinitis Pigmentosa
13,695 Patients Enrolled for Retinitis Pigmentosa
University of IowaOTHER
404 Previous Clinical Trials
787,472 Total Patients Enrolled
University of Wisconsin, MadisonOTHER
1,061 Previous Clinical Trials
2,563,371 Total Patients Enrolled
Medical University of GrazOTHER
437 Previous Clinical Trials
219,088 Total Patients Enrolled
University of AmsterdamOTHER
32 Previous Clinical Trials
21,047 Total Patients Enrolled
University of California, DavisOTHER
838 Previous Clinical Trials
5,002,450 Total Patients Enrolled
2 Trials studying Retinitis Pigmentosa
19 Patients Enrolled for Retinitis Pigmentosa
Northwestern UniversityOTHER
1,433 Previous Clinical Trials
769,110 Total Patients Enrolled
Centro Medico ABCOTHER
1 Previous Clinical Trials
6 Total Patients Enrolled
Retina Foundation of the SouthwestOTHER
14 Previous Clinical Trials
1,477 Total Patients Enrolled
3 Trials studying Retinitis Pigmentosa
164 Patients Enrolled for Retinitis Pigmentosa
University of Illinois at ChicagoOTHER
529 Previous Clinical Trials
1,548,764 Total Patients Enrolled
Mayo ClinicOTHER
2,894 Previous Clinical Trials
3,698,289 Total Patients Enrolled
1 Trials studying Retinitis Pigmentosa
4 Patients Enrolled for Retinitis Pigmentosa
Medical College of WisconsinOTHER
554 Previous Clinical Trials
1,151,734 Total Patients Enrolled
Stanford UniversityOTHER
2,148 Previous Clinical Trials
35,678,566 Total Patients Enrolled
Xiangrong KongStudy DirectorJohns Hopkins University
Peter A Campochiaro, MDStudy ChairJohns Hopkins University
10 Previous Clinical Trials
384 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You are able to comply with the study protocol and to participate in all study visits and assessments in the investigator's judgement.
You are of childbearing potential.
You have RP with evidence of loss of night vision, gradual constriction of visual fields, and maintenance of visual acuity.
You have a horizontal SD-OCT scan through the fovea center with width ≤ 8000 µm and ≥1500 µm and with well-defined truncation at both the nasal and temporal sides.
You have a BCVA of 61 (20/60 Snellen equivalent) or better.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 3rd, 2021

Last Reviewed: November 26th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

Who else is applying?

What state do they live in?
California66.7%
Texas33.3%
How old are they?
18 - 65100.0%
What site did they apply to?
Stanford University, Byers Eye Institute50.0%
Retina Foundation of the Southwest50.0%
What portion of applicants met pre-screening criteria?
Met criteria100.0%

How responsive is this trial?

Average response time
  • < 2 Days
Typically responds via
Email50.0%
Phone Call50.0%
Most responsive sites:
  1. Stanford University, Byers Eye Institute: < 48 hours
  2. Retina Foundation of the Southwest: < 48 hours
References