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Completed

NCT Number: NCT04795206

Natural Disease Progression in Participants With Choroideremia

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.

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Key information

Conditions

Choroideremia Choroid Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disease Attributes Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Genetic Diseases, X-Linked Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases Uveal Diseases

Age range

10 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Research Site

San Francisco, California, 94107, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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:
  • ICD-9: 363.55
  • ICD-10: H31.2
  • 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:
  • ICD-9: 363.55
  • ICD-10: H31.21
  • SNOMED-CT: 75241009

Key Exclusion Criteria:

  • N/A

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

No intervention

Other

Administered as specified in the treatment arm.

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

Biogen

Industry

Registry information

Official study title

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

Important dates

Study start
2020
Primary completion
2021
Study completion
2021
First posted
Mar 12, 2021
Registry last updated
Sep 8, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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