Status:

COMPLETED

Natural History of the Progression of Choroideremia Study

Lead Sponsor:

NightstaRx Ltd, a Biogen Company

Conditions:

Choroideremia

Eligibility:

MALE

18+ years

Brief Summary

The objective of this natural history study is to gain a better understanding of the progression of choroideremia (CHM) and add to the knowledge base for this rare disease.

Detailed Description

This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.

Eligibility Criteria

Inclusion

  • Key
  • Are willing and able to provide informed consent for participation in the study.
  • Have a clinical phenotype and confirmed genetic diagnosis of CHM.
  • Have active disease clinically visible within the macular region.
  • Are willing and able to undergo ophthalmic examinations once every 4 months for up to 20 months.
  • Have a BCVA better than or equal to 6/60 (20/200; decimal 0.1; LogMAR 1.0; 34-38 ETDRS letters) in at least one eye.
  • Key

Exclusion

  • Have a history of amblyopia in the eligible eye.
  • Have any other significant ocular or non-ocular disease/disorder in the eligible eye which, in the opinion of the investigator, may put the subject at risk because of participation in the study, influence the results of the study or influence the subject's ability to participate in the study.
  • Have participated in an interventional research study in the past 6 months.
  • NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Key Trial Info

Start Date :

June 30 2015

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

October 1 2020

Estimated Enrollment :

319 Patients enrolled

Trial Details

Trial ID

NCT03359551

Start Date

June 30 2015

End Date

October 1 2020

Last Update

February 10 2023

Active Locations (20)

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Page 1 of 5 (20 locations)

1

Research Site

Los Angeles, California, United States, 90095

2

Research Site

Miami, Florida, United States, 33136

3

Research Site

Baltimore, Maryland, United States, 21287

4

Research Site

New York, New York, United States, 10032