Natural Disease Progression in Participants With Choroideremia

NCT04795206 | Completed

• 1 other identifier: US-CHM-11761
• Study Type: observational
• Target: 1,178
• Locations: 1 country
• Sites: 1

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.

Conditions

Choroideremia

Keywords

X-linked inherited retinal disorder; Rare disease; Bilateral disease

Outcome Measures

Primary Outcomes (2)

• 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

Secondary Outcomes (2)

• Percentage of Participants with Demographics Described at Baseline

  Up to 6 years

• Matching Participants with CHM in the IRIS® Registry to Biogen's IST Study Population Using Propensity Score (PS) Matching

  Up to 6 years

Study Arms (2)

Cohort 1: Main Cohort

All eligible participants with CHM in IRIS Registry will be included.

Other: No Intervention

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

Interventions

No Intervention OTHER

Administered as specified in the treatment arm.

Cohort 1: Main Cohort; Cohort 2: Trial-Matched Cohort

Eligibility Criteria

• Age: 10 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• N/A

Sponsors & Collaborators

• Biogen: lead

Study Sites (1)

Research Site

San Francisco, California, 94107, United States

Location

MeSH Terms

Conditions

Choroideremia; Rare Diseases

Condition Hierarchy (Ancestors)

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

Study Officials

• Medical Director

  Biogen

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 9, 2021
• First Posted: March 12, 2021
• Study Start: August 26, 2020
• Primary Completion: May 7, 2021
• Study Completion: August 10, 2021
• Last Updated: September 8, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will share

Locations

US (1)