Natural Disease Progression in Participants With Choroideremia

Biogen

Status: Completed

Conditions: Choroideremia

Treatments: Other: No Intervention

Study type: Observational

Funder types: Industry

Identifiers

NCT04795206

US-CHM-11761

Details and patient eligibility

About

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 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.

Enrollment: 1,178 patients

Sex: All

Ages: 10+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

1,178 participants in 2 patient groups

Cohort 1: Main Cohort

Description:

All eligible participants with CHM in IRIS Registry will be included.

Treatment:

Other: No Intervention

Cohort 2: Trial-Matched Cohort

Description:

Only male participants with CHM from Cohort 1 to match Biogen's IST study population using propensity score matching will be included.

Treatment:

Other: No Intervention