June 2021
Volume 62, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2021
The design and patient acceptability of NAC Attack a phase-3 clinical trial for retinitis pigmentosa (RP)
Author Affiliations & Notes
  • Xiangrong Kong
    Wilmer Eye Institute, Johns Hopkins Medicine, Baltimore, Maryland, United States
  • Susie trotochaud
    USHER 2020, Georgia, United States
  • Fahd Naufal
    Wilmer Eye Institute, Johns Hopkins Medicine, Baltimore, Maryland, United States
  • Peter A Campochiaro
    Wilmer Eye Institute, Johns Hopkins Medicine, Baltimore, Maryland, United States
  • Footnotes
    Commercial Relationships   Xiangrong Kong, None; Susie trotochaud, None; Fahd Naufal, None; Peter Campochiaro, ;Allegro (I), ;Merck & Co (I), ;Novartis (I), ;Wave Life Sciences (I), Aerpio Pharmaceuticals (I), Applied Genetic Technologies Corporation (I), Asclepix Therapeutices (F), Exonate Ltd. (I), Genetech/Roche Inc (F), Graybug Vision (F), Oxford Biomedica (F), Perfuse (I), Sanofi/Genezyme (F)
  • Footnotes
    Support  R34EY031429 NEI
Investigative Ophthalmology & Visual Science June 2021, Vol.62, 3543. doi:
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      Xiangrong Kong, Susie trotochaud, Fahd Naufal, Peter A Campochiaro; The design and patient acceptability of NAC Attack a phase-3 clinical trial for retinitis pigmentosa (RP). Invest. Ophthalmol. Vis. Sci. 2021;62(8):3543.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : NAC Attack is a Phase III multicenter, randomized, and placebo-controlled study to evaluate the efficacy and safety of oral N-acetylcysteine (NAC) in patients with RP and is in the planning phase. It aims to enroll 438 eligible patients who will be randomized to either 1800mg/bid NAC or placebo. This study aimed to learn patients attitudes of participating a clinical trial to facilitate the trial design and assess recruitment potential.

Methods : Crowdsourcing is a method that utilizes social media to quickly involve a group of people for ideas. It was used to survey RP patients attitudes toward a study of over 4 years to test an oral medication to slow or stop vision loss caused by RP. The survey questionnaire was developed by the NAC Attack Study Chair and Coordinating Center in collaboration with the USHER 2020 foundation. The survey required no protected health information. It was administered using Google Form by USHER 2020 from Aug to Nov of 2020. The survey link was sent to various email lists of retinal patient advocacy groups, social media, emails through My Retina Tracker of Foundation Fighting Blindness, and was picked up by other RP patients listserve. The study was approved by the JHU IRB.

Results : There were 1473 responses: 24% were of age 18-40, 60% were 40-69, and 15% were >70 years; female was 56%; 20% patients had genetic testing done before but the genetic cause was not found; 96% used a mobile phone and among them 96% received text messages; 61% were very and 18% somewhat enthusiastic about participating in a trial; 70% were very and 28% somewhat willing to have a clinical visit every 4-5 months for 45 months; 83% were very and 15% somewhat willing to have a tele-visit with a doctor between clinical visits. Acceptability of a daily text reminder of medication was 60% Yes, 24% No, and 13% don’t know; 70% were willing to report any missing dosages or problems related to medication using a text message based system daily or weekly.

Conclusions : Enthusiasm was high for participating a clinical trial that may find an effective treatment for RP. Rates of possession of mobile phones and usage of text messages were high. Using SMS messages to keep patients engaged and to encourage and survey their medication adherence is feasible. NAC Attack will have in-clinic follow-up visits every 9 months and in-between every 4.5 months a televisit with site investigator.

This is a 2021 ARVO Annual Meeting abstract.

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