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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04795206
Other study ID # US-CHM-11761
Secondary ID
Status Completed
Phase
First received
Last updated
Start date August 26, 2020
Est. completion date August 10, 2021

Study information

Verified date September 2021
Source Biogen
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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.


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) IRISĀ® Registry which captures CHM participant's demographics, clinical characteristics and clinical outcomes from 2013.


Recruitment information / eligibility

Status Completed
Enrollment 1178
Est. completion date August 10, 2021
Est. primary completion date May 7, 2021
Accepts healthy volunteers No
Gender All
Age group 10 Years and older
Eligibility 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.

Study Design


Related Conditions & MeSH terms


Intervention

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

Locations

Country Name City State
United States Research Site San Francisco California

Sponsors (1)

Lead Sponsor Collaborator
Biogen

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Percentage of Participants with Progression of Best Corrected Visual Acuity (BCVA) in Relation to Age Up to 6 years
Primary Percentage of Participants with Progression of BCVA Over Time After the Baseline BCVA Up to 6 years
Secondary 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
Secondary Matching Participants with CHM in the IRIS® Registry to Biogen's IST Study Population Using Propensity Score (PS) Matching 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
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Active, not recruiting NCT00427180 - IRIS PILOT - Extended Pilot Study With a Retinal Implant System N/A