Clinical trial NCT04795206: Natural Disease Progression in Participants With Choroideremia
Official title: A Retrospective Cohort Analysis of the Natural Disease Progression of Patients With Choroideremia in Real-World Clinical Practice
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Choroideremia
Keywords: X-linked inherited retinal disorder
Rare disease
Bilateral disease
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2021-03-12
Enrollment: 1178
Sex: ALL
Minimum age (years): 10
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Main cohort will include all eligible CHM participants in IRIS registry. Trial-matched cohort will include only male CHM participants from Cohort 1 to match Biogen's IST study population using propensity score matching.

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.

Eligibility 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.

Primary outcomes:
measure: Percentage of Participants with Progression of Best Corrected Visual Acuity (BCVA) in Relation to Age
timeFrame: Up to 6 years
measure: Percentage of Participants with Progression of BCVA Over Time After the Baseline BCVA
timeFrame: Up to 6 years
Secondary outcomes:
measure: Percentage of Participants with Demographics Described at Baseline
timeFrame: Up to 6 years
measure: Matching Participants with CHM in the IRIS® Registry to Biogen's IST Study Population Using Propensity Score (PS) Matching
timeFrame: Up to 6 years
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: Research Site
state: California
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: