Natural Disease Progression in Participants With Choroideremia

Overview

The primary objective of this study is to quantify disease progression measured by best corrected visual acuity (BCVA) in the real-world setting among Choroideremia (CHM) participants. The secondary objectives are to describe demographic and baseline clinical characteristics among CHM participants and to match CHM participants in the Intelligent Research in Sight (IRIS®) Registry to Biogen's investigator sponsored trial (IST) study population using propensity score matching.

Full Title of Study: “A Retrospective Cohort Analysis of the Natural Disease Progression of Patients With Choroideremia in Real-World Clinical Practice”

Study Type

  • Study Type: Observational
  • Study Design
    • Time Perspective: Retrospective
  • Study Primary Completion Date: May 7, 2021

Detailed Description

This study is a retrospective cohort study of CHM eyes and participants which will include all eligible participants and eyes with the first documented diagnosis of CHM from July 1st, 2013 to December 31st, 2019 that meet the study criteria. This study will be conducted using data from American Academy of Ophthalmology's (AAO) IRIS® Registry which captures CHM participant's demographics, clinical characteristics and clinical outcomes from 2013.

Interventions

  • Other: No Intervention
    • Administered as specified in the treatment arm.

Arms, Groups and Cohorts

  • Cohort 1: Main Cohort
    • All eligible participants with CHM in IRIS Registry will be included.
  • Cohort 2: Trial-Matched Cohort
    • Only male participants with CHM from Cohort 1 to match Biogen’s IST study population using propensity score matching will be included.

Clinical Trial Outcome Measures

Primary Measures

  • Percentage of Participants with Progression of Best Corrected Visual Acuity (BCVA) in Relation to Age
    • Time Frame: Up to 6 years
  • Percentage of Participants with Progression of BCVA Over Time After the Baseline BCVA
    • Time Frame: Up to 6 years

Secondary Measures

  • Percentage of Participants with Demographics Described at Baseline
    • Time Frame: Up to 6 years
    • The demographics will include age, sex, race, geographic region and treating provider specialty.
  • Matching Participants with CHM in the IRIS® Registry to Biogen’s IST Study Population Using Propensity Score (PS) Matching
    • Time Frame: Up to 6 years
    • A propensity score matching approach will be performed to select eligible male CHM participants from IRIS Registry to match Biogen’s IST study population. With the approach, the probability of participating in the IST study given the observed baseline participant characteristics will be estimated for each participant. Propensity score will be estimated using logistic regression and pre-specified baseline covariates including participants demographics and clinical characteristics. The propensity match will be considered adequate if all the variables between the PS-matched CHM cohort and IST study cohort have an absolute value of the standardized mean difference (SMD) in PS-score that is less than 0.1.

Participating in This Clinical Trial

Key Inclusion Criteria:

Cohort 1: All CHM Participants

  • Participants with a documented diagnosis of CHM identified by the presence of International Classification of Diseases (ICD)-9/10 or SNOMED coding: 1. ICD-9: 363.55 2. ICD-10: H31.2 3. SNOMED-CT: 75241009 Cohort 2: Trial-Matched CHM Participants – Male participants with a documented diagnosis of CHM identified by the presence of ICD-9/10 or SNOMED coding: 1. ICD-9: 363.55 2. ICD-10: H31.21 3. SNOMED-CT: 75241009 Key Exclusion Criteria:

  • N/A NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Gender Eligibility: All

Minimum Age: 10 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Biogen
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Medical Director, Study Director, Biogen

Clinical trials entries are delivered from the US National Institutes of Health and are not reviewed separately by this site. Please see the identifier information above for retrieving further details from the government database.

At TrialBulletin.com, we keep tabs on over 200,000 clinical trials in the US and abroad, using medical data supplied directly by the US National Institutes of Health. Please see the About and Contact page for details.