Natural Disease Progression in Participants With Choroideremia

Completed

• Conditions: Choroideremia
• Interventions: Other: No Intervention

• Registration Number: NCT04795206
• Lead Sponsor: Biogen

Brief Summary

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.

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.

Study Timeline

• Study Start: 2020-08-26
• Study First Submitted: 2021-03-09
• Study First Submitted QC: 2021-03-09
• Study First Posted: 2021-03-12
• Primary Completion: 2021-05-07
• Study Completion: 2021-08-10
• Status Verified: 2021-09
• Last Update Submitted: 2021-09-07
• Last Update Posted: 2021-09-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 1178

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Cohort 1: Main Cohort	No Intervention	All eligible participants with CHM in IRIS Registry will be included.
Cohort 2: Trial-Matched Cohort	No Intervention	Only male participants with CHM from Cohort 1 to match Biogen's IST study population using propensity score matching will be included.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Percentage of Participants with Demographics Described at Baseline	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	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.

Trial Locations

Locations (1)

Research Site; 🇺🇸 San Francisco, California, United States