A Natural History Study to Evaluate Functional and Anatomical Progression in Retinitis Pigmentosa

NCT04558983 | Terminated

• 1 other identifier: IRB00227603
• Study Type: observational
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

This study will assess the progression of RP as seen on newer modalities including spectral-domain optical coherence (SD-OCT) and macular assessment integrity (MAIA) microperimetry to evaluate disease status. Understanding the natural history of the disease is not only essential to monitoring and comparing patient populations in clinical trials. It is also fundamental in the predevelopment phase in order to optimize the study duration needed to observe a statistically significant outcome. Furthermore, since the progression of RP is usually slow, relying on traditional tests can take an unfeasible length of time to observe any meaningful changes and assess therapeutic efficacy for new drugs. Therefore, the results of this study will be beneficial in establishing reliable endpoints and outcome measures for future clinical trials. Such outcome measures may be able to detect treatment response with more precision. More importantly, investigators may be able to detect changes early enough to prevent irreversible vision loss.

Conditions

Retinitis Pigmentosa

Outcome Measures

Primary Outcomes (1)

• Change in mean macular sensitivity (dB) over time as assessed by microperimetry

  Microperimetry (MAIA) will be used to test whether there is a change in sensitivity (dB) in the macula

  Baseline, every six months up to 2 years

Secondary Outcomes (6)

• Change in Best Corrected Visual Acuity (BVCA)

  Baseline, every six months up to 2 years

• Change in Ellipsoid Zone (EZ) width

  Baseline, every six months up to 2 years

• Change in Quality of Life survey metrics

  Baseline, every year up to 2 years

• Change in mean retinal sensitivity

  Baseline and at 2 years

• Correlation between change in visual functional and anatomical measures

  Baseline, every six months up to 2 years

Other Outcomes (5)

• Correlation between baseline functional and anatomical measures

  Baseline, up to 2 years

• Correlation between baseline functional measures and Quality of Life survey metrics

  Baseline, up to 2 years

• Correlation between functional, anatomic and Quality of Life measures

  Baseline, up to 2 years

Study Arms (1)

Retinitis Pigmentosa

Patients with Retinitis Pigmentosa

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

Patients diagnosed with Retinitis Pigmentosa

You may qualify if:

• Age 18 years or older
• Patients diagnosed with Retinitis Pigmentosa
• Ability to provide informed consent
• Ability to authorize use and disclosure of protected health information

You may not qualify if:

• Concomitant ocular pathology that limits central macular function, including but not limited to age-related macular degeneration, diabetic retinopathy, and retinal vein occlusion
• If EZ width ≤200µm

Sponsors & Collaborators

• Johns Hopkins University: lead

Study Sites (1)

Wilmer Eye Institute at Johns Hopkins University

Baltimore, Maryland, 21287, 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 Officials

• Peter A Campochiaro, M.D.

  Johns Hopkins University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 16, 2020
• First Posted: September 22, 2020
• Study Start: June 11, 2020
• Primary Completion: December 20, 2024
• Study Completion: December 20, 2024
• Last Updated: December 27, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)