Natural History of the Progression of Choroideremia Study (NIGHT)

NightstaRx

Status: Completed

Conditions: Choroideremia

Study type: Observational

Funder types: Other

Identifiers

NCT03359551

273CH001

NSR-CHM-OS1 (Other Identifier)

Details and patient eligibility

About

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.

Full description

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

Enrollment: 319 patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.