Natural History of the Progression of Choroideremia Study

Natural History of the Progression of Choroideremia Study

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.

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

Key Inclusion Criteria:

• 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 Criteria:

• 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.