A Prospective, Observational Study in Adults With Retinitis Pigmentosa (RP)

NCT06517940 | Active Not Recruiting

• 1 other identifier: DE101111
• Study Type: observational
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

The natural history in individuals with severe retinitis pigmentosa (RP) is variable and there remains an unmet need to better understand disease progression in this population. The goal of this study is to determine which visual assessments individuals with RP and low visual acuity can reliably perform and to evaluate the annual decline of visual function in severe RP.

Conditions

Retinitis Pigmentosa

Keywords

Retinitis Pigmentosa. Natural history

Outcome Measures

Primary Outcomes (1)

• Reliability (test-retest variability within subjects)

  Reliability (test-retest variability within subjects) on: * Total visual area (degrees squared) on kinetic perimetry using III-4e stimulus (excluding temporal islands) (Time frame: 12 months) * Early Treatment of Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA) Log of Minimum Angle of Resolution (LogMAR) equivalent (or Low Vision Test, as applicable) (Time frame: 12 months) * Retinal light sensitivity on Full-field Stimulus Threshold test (Time frame: 12 months) * Retinal sensitivity and fixation stability on microperimetry (Time frame: 12 months)

  12 months

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Retinitis pigmentosa subjects with low visual acuity

You may qualify if:

• Subject is able to provide written informed consent and is willing and able to comply with the protocol.
• Subject is ≥ 18 years of age at the time of informed consent.
• Subject has a clinical diagnosis of nonsyndromic retinitis pigmentosa.
• Subject is willing to consent to genetic testing, if not already done.
• Subject has BCVA in the worse eye between hand motion and 35 Early Treatment of Diabetic Retinopathy Study (ETDRS) letter score (approximately ≤ 20/200 Snellen equivalent), inclusive at Screening.
• For subjects who are able to reliably perform kinetic perimetry in the opinion of the Investigator and the assessment provides accurate information, the central visual field diameter (ie, excluding temporal islands) of any visual field diameter is \< 20 degrees in the worse eye as measured with the III-4e stimulus using Octopus 900 perimetry at Screening.
• On OCT, inner retinal layers are observed and are retained in the worse eye.

You may not qualify if:

• Subject has an eye disease or visual disorder other than retinitis pigmentosa that impairs visual function.
• Subject has any other eye condition, which in the opinion of the Investigator, would preclude an accurate evaluation at any time during the study.
• On OCT, subject has cystoid macular edema in the worse eye in the opinion of the Investigator or is currently receiving treatment for cystoid macular edema in the worse eye.
• Subject has unstable intraocular lens.
• Subject has a clinically significant epiretinal membrane based on the Investigator's judgment that will interfere with the assessments of visual function.
• Subject with a prior vitrectomy in the worse eye will be evaluated on a case-by-case basis by the Investigator to determine if the subject is eligible for the study.
• Subject has any clinically significant unstable medical condition or any clinically significant chronic disease that in the opinion of the Investigator, would limit the subject's ability to complete and/or participate in the study.
• Subject has received treatment with any nonapproved, experimental, or investigational therapy in either eye (systemic, topical, intravitreal) and/or received treatment in an interventional clinical trial for an eye disease or disorder within 90 days or 5 half-lives, whichever is longer, prior to Screening or anticipates the need to receive treatment in an interventional clinical trial during their participation in this study.
• Subject has previously received cell therapy, gene augmentation therapy, genome editing therapy, or any subretinal administered therapy for an eye disease or disorder or anticipates the need for these therapies during their participation in this study.
• Subject has previously received LUXTURNA (voretigene neparvovec-rzyl) or anticipates the need for this therapy during their participation in this study.
• Subject is in the opinion of the Investigator unsuitable in any other way to participate in this study.

Sponsors & Collaborators

• Sumitomo Pharma America, Inc.: lead

Study Sites (1)

Mass Eye and Ear

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 18, 2024
• First Posted: July 24, 2024
• Study Start: October 3, 2024
• Primary Completion: April 24, 2026
• Study Completion: April 24, 2026
• Last Updated: February 27, 2026

Record last verified: 2026-02

Locations

US (1)