Natural Disease Progression in Participants With Choroideremia

Choroideremia Clinical Trial

Official title:

A Retrospective Cohort Analysis of the Natural Disease Progression of Patients With Choroideremia in Real-World Clinical Practice

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04795206
• Other study ID #: US-CHM-11761
• Status: Completed
• 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
• 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) IRIS® 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.

Related Conditions & MeSH terms

• Choroideremia
• Disease Progression

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