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Natural Disease Progression in Participants With Choroideremia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04795206
Recruitment Status : Completed
First Posted : March 12, 2021
Last Update Posted : September 8, 2021
Sponsor:
Information provided by (Responsible Party):
Biogen

Study Description
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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.

Condition or disease Intervention/treatment
Choroideremia Other: No Intervention

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.
Study Design
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Study Type : Observational
Actual Enrollment : 1178 participants
Observational Model: Cohort
Time Perspective: Retrospective
Official Title: A Retrospective Cohort Analysis of the Natural Disease Progression of Patients With Choroideremia in Real-World Clinical Practice
Actual Study Start Date : August 26, 2020
Actual Primary Completion Date : May 7, 2021
Actual Study Completion Date : August 10, 2021

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Cohort 1: Main Cohort
All eligible participants with CHM in IRIS Registry will be included.
 Other: No Intervention
Administered as specified in the treatment arm.
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.
 Other: No Intervention
Administered as specified in the treatment arm.


Outcome Measures
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Primary Outcome Measures :
  1. Percentage of Participants with Progression of Best Corrected Visual Acuity (BCVA) in Relation to Age [ Time Frame: Up to 6 years ]
  2. Percentage of Participants with Progression of BCVA Over Time After the Baseline BCVA [ Time Frame: Up to 6 years ]

Secondary Outcome Measures :
  1. Percentage of Participants with Demographics Described at Baseline [ Time Frame: Up to 6 years ]
    The demographics will include age, sex, race, geographic region and treating provider specialty.

  2. Matching Participants with CHM in the IRIS® Registry to Biogen's IST Study Population Using Propensity Score (PS) Matching [ Time Frame: Up to 6 years ]
    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.


Eligibility Criteria
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Ages Eligible for Study:   10 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04795206


Locations
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United States, California
Research Site
San Francisco, California, United States, 94107
Sponsors and Collaborators
Biogen
Investigators
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Study Director: Medical Director Biogen
More Information
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Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT04795206    
Other Study ID Numbers: US-CHM-11761
First Posted: March 12, 2021    Key Record Dates
Last Update Posted: September 8, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/
URL: https://vivli.org/

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Biogen:
X-linked inherited retinal disorder
Rare disease
Bilateral disease
Additional relevant MeSH terms:
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Choroideremia
Disease Progression
Disease Attributes
Pathologic Processes
Eye Diseases, Hereditary
 Eye Diseases
Choroid Diseases
Uveal Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked