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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| No Intervention | Other | Administered as specified in the treatment arm. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants with Progression of Best Corrected Visual Acuity (BCVA) in Relation to Age | Up to 6 years | |
| Percentage of Participants with Progression of BCVA Over Time After the Baseline BCVA | Up to 6 years |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants with Demographics Described at Baseline | The demographics will include age, sex, race, geographic region and treating provider specialty. | Up to 6 years |
| Matching Participants with CHM in the IRIS® Registry to Biogen's IST Study Population Using Propensity Score (PS) Matching |
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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:
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:
Key Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
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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.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Biogen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | San Francisco | California | 94107 | United States |
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/
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| ID | Term |
|---|---|
| D015794 | Choroideremia |
| D035583 | Rare Diseases |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D015862 | Choroid Diseases |
| D014603 | Uveal Diseases |
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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. |
| Up to 6 years |
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |