ACTIVE_NOT_RECRUITING

Oral N-acetylcysteine for Retinitis Pigmentosa

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Conditions

Retinitis PigmentosaN-acetylcysteineEllipsoid zoneMacular sensitivityBest corrected visual acuityEllipsoid zone widthEllipsoid zone areaOxidative damageUsher SyndromeAntioxidants

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

Retinitis pigmentosa (RP) is an inherited retinal degeneration caused by one of several mistakes in the genetic code. Such mistakes are called mutations. The mutations cause degeneration of rod photoreceptors which are responsible for vision in dim illumination resulting in night blindness. After rod photoreceptors are eliminated, gradual degeneration of cone photoreceptors occurs resulting in gradual constriction of side vision that eventually causes tunnel vision. Oxidative stress contributes to cone degeneration. N-acetylcysteine (NAC) reduces oxidative stress and in animal models of RP it slowed cone degeneration. In a phase I clinical trial in patients with RP, NAC taken by month for 6 months caused some small improvements in two different vision tests suggesting that long-term administration of NAC might slow cone degeneration in RP. NAC Attack is a clinical trial being conducted at many institutions in the US, Canada, and Europe designed to determine if taking NAC for several years provides benefit in patients with RP.

Official Title

NAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients With Retinitis Pigmentosa

Quick Facts

Study Start:2023-10-11
Study Completion:2029-05
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05537220

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 65 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Ability and willingness to provide informed consent
  2. * Age ≥ 18 and ≤65 years at time of signing Informed Consent Form
  3. * Ability and willingness to comply with the study protocol and to participate in all study visits and assessments in the investigator's judgement
  4. * For candidates of childbearing potential: willingness to use a method of contraception
  5. * Agreement not to take supplements other than vitamin A
  6. * Both eyes must exhibit the RP phenotype with evidence of loss of night vision, gradual constriction of visual fields, and maintenance of visual acuity;
  7. * In addition, an eye must meet the following criteria to be included in the study:
  8. * Gradable EZ on 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;
  9. * BCVA ≥ ETDRS letter score of 61 (20/60 Snellen equivalent);
  10. * Sufficiently clear ocular media and adequate pupillary dilation to allow good quality images sufficient for analysis and grading by central reading center.
  1. * Active cancer within the past 12 months, except for appropriately treated carcinoma in situ of the cervix, non-melanoma skin carcinoma, or prostate cancer with Gleason score ≤ 6 and stable prostate specific antigen for \> 12 months
  2. * Renal failure requiring renal transplant, hemodialysis, peritoneal dialysis, or anticipated to require hemodialysis or peritoneal dialysis during the study
  3. * Liver disease, cystic fibrosis, asthma, or chronic obstructive pulmonary disease (COPD), history of thrombocytopenia not due to a reversible cause or other blood dyscrasia
  4. * Uncontrolled blood pressure (defined as systolic \> 180 and/or diastolic \> 100 mmHg while at rest) at screening. If a patient's initial measurement exceeds these values, a second reading may be taken 30 or more minutes later. If the patient's blood pressure must be controlled by antihypertensive medication, the patient may become eligible if medication is taken continuously for at least 30 days.
  5. * History of other disease, physical examination finding, or clinical laboratory finding giving reasonable suspicion that oral NAC may be contraindicated or that follow up may be jeopardized
  6. * Cerebrovascular accident or myocardial infarction within 6 months of screening
  7. * Participation in an investigational study that involves treatment with any drug or device within 6 months of screening
  8. * Three relatives already enrolled in study
  9. * Pregnant, breast feeding, or intending to become pregnant during the study treatment period. Women of childbearing potential who have not had tubal ligation must have a urine pregnancy test at screening.
  10. * Known history of allergy to NAC
  11. * Having taken NAC in any form in the past 4 months
  12. * Phenylketonuria
  13. * Fructose intolerance
  14. * Glucose-galactose malabsorption
  15. * Sucrase-isomaltase insufficiency
  16. * Abnormal laboratory value including the value of alanine aminotransferase (ALT), aspartate aminotransferase (AST), or bilirubin being greater than 1.5 x the upper limit of normal
  17. * Any major abnormal findings on blood chemistry, hematology, and renal function lab tests that in the opinion of the Site Investigator and/or the Study Chair makes the candidate not suitable to participate in the trial
  18. * HIV or hepatitis B infection
  19. * Evidence of cone-rod dystrophy or pattern dystrophy including focal areas of atrophy or pigmentary changes in the central macula
  20. * Cystoid spaces involving the fovea substantially reducing vision
  21. * Glaucoma or other optic nerve disease causing visual field loss or reduced visual acuity
  22. * Intra ocular pressure \>27 mm Hg from two measurements. If a patient's initial measurement exceeds 27 mm Hg, a second reading must be taken.
  23. * Any retinal disease other than RP causing reduction in visual field or visual acuity
  24. * Any prior macular laser photocoagulation
  25. * Intraocular surgery within 3 months prior to screening
  26. * High myopia with spherical equivalent refractive error \> 8 diopters. If an eye has had cataract surgery or refractive surgery, a pre-operative refractive error spherical equivalent \> 8 diopters is an exclusion
  27. * Any concurrent ocular condition that might affect interpretation of results
  28. * History of uveitis in either eye

Contacts and Locations

Principal Investigator

Peter A Campochiaro, MD
STUDY_CHAIR
Johns Hopkins University
Xiangrong Kong
STUDY_DIRECTOR
Johns Hopkins University

Study Locations (Sites)

University of California - Davis, Department of Ophthalmology & Vision Science
Davis, California, 95817
United States
University of Southern California, Keck School of Medicine
Los Angeles, California, 90089
United States
University of California - San Francisco, Department of Ophthalmology
San Francisco, California, 94158
United States
Stanford University, Byers Eye Institute
Stanford, California, 94303
United States
Vitreo Retinal Associates
Gainesville, Florida, 32607
United States
University of Florida - Jacksonville, UF Health Jacksonville
Jacksonville, Florida, 32209
United States
University of Miami, Bascom Palmer Eye Institute
Miami, Florida, 33136
United States
Emory University, Emory Eye Center
Atlanta, Georgia, 30322
United States
University Of Illinois At Chicago
Chicago, Illinois, 60612
United States
Northwestern University
Evanston, Illinois, 60208
United States
University of Iowa, Carver College of Medicine
Iowa City, Iowa, 52242
United States
Wilmer Eye Institute- Johns Hopkins University
Baltimore, Maryland, 21287
United States
Harvard University, Mass. Eye and Ear
Boston, Massachusetts, 02114
United States
University of Michigan, Kellogg Eye Center
Ann Arbor, Michigan, 48105
United States
University of Minnesota, Department of Ophthalmology and Visual Neurosciences
Minneapolis, Minnesota, 55455
United States
Mayo Clinic, Department of Ophthalmology
Rochester, Minnesota, 55905
United States
University of Oklahoma, Dean McGee Eye Institute
Oklahoma City, Oklahoma, 73104
United States
Scheie Eye Institute
Philadelphia, Pennsylvania, 19104
United States
Vanderbilt University, Vanderbilt Eye Institute
Nashville, Tennessee, 37211
United States
Retina Foundation of the Southwest
Dallas, Texas, 75231
United States
University of Utah, Moran Eye Center
Salt Lake City, Utah, 84132
United States
University of Washington, Department of Ophthalmology
Seattle, Washington, 98109
United States
University of Wisconsin - Madison, McPherson Eye Research Institute
Madison, Wisconsin, 53705
United States
Medical College of Wisconsin, The Eye Institute
Milwaukee, Wisconsin, 53226
United States

Collaborators and Investigators

Sponsor: Johns Hopkins University

  • Peter A Campochiaro, MD, STUDY_CHAIR, Johns Hopkins University
  • Xiangrong Kong, STUDY_DIRECTOR, Johns Hopkins University

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-10-11
Study Completion Date2029-05

Study Record Updates

Study Start Date2023-10-11
Study Completion Date2029-05

Terms related to this study

Keywords Provided by Researchers

  • N-acetylcysteine
  • Ellipsoid zone
  • Macular sensitivity
  • Best corrected visual acuity
  • Ellipsoid zone width
  • Ellipsoid zone area
  • Oxidative damage
  • Usher Syndrome
  • Antioxidants

Additional Relevant MeSH Terms

  • Retinitis Pigmentosa