EXPLORE XLRP 2
Research type
Research Study
Full title
Retrospective Chart Review Study Combined with Cross-sectional Patient and Caregiver Surveys to Document and Describe the Disease-related Clinical, Individual, Sociodemographic Characteristics of Patients with X-linked Retinitis Pigmentosa (XLRP) and the Impact of XLRP on Patients and Their Caregivers
IRAS ID
307690
Contact name
Susan Downes
Contact email
Sponsor organisation
Janssen-Cilag International NV
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
0 years, 9 months, 4 days
Research summary
Research Summary
X-linked retinitis pigmentosa (XLRP) is among the most severe forms of retinitis pigmentosa (RP), an inherited retinal disease. RP is characterized by the gradual degeneration of the photoreceptors (rods and cones) of the retina. The symptoms caused by the progressive degeneration of the rods and the cones usually start in childhood with nyctalopia (night blindness), leading to central vision loss and legal blindness by the 4th decade. There are limited data available on the disease pathway and the patient and societal burden of this orphan disease. In addition to the potential burden of XLRP for patients themselves, those caring for such patients, such as family members or friends, may also experience a significant burden encompassing physical, emotional, financial and social problems, rising from dealing with the potential dependence of the patient on the caregiver. The data from this study will extend the understanding of XLRP using data from clinical practices, with the aim of improving care and enhancing access to innovative treatments for patients with XLRP. The collected information may also support the understanding of the unmet medical need in clinical practice. Approximately 150 to 200 patients with a clinical diagnosis of XLRP, with genetic verification, will be included in the study. Data will be collected retrospectively from patients’ medical records as well as from cross-sectional patient and caregiver surveys. Data obtained from medical records will include the patients’ socio-demographics, comorbidities (ophthalmologic and non-ophthalmologic), clinical parameters, diagnostic tools used/outcomes of the assessment, consultations, and medical resource use. Patient and caregiver enrollment involvement is planned remotely, including where possible remote consent. The surveys will be managed via a telephone interview.REC name
London - Surrey Research Ethics Committee
REC reference
21/PR/1584
Date of REC Opinion
26 Jan 2022
REC opinion
Further Information Favourable Opinion