Natural History of the Progression of Choroideremia Study

Status:	Recruiting
Healthy:	No
Age Range:	18 - Any
Updated:	8/10/2018
Start Date:	June 2015
End Date:	November 2019
Contact:	Nightstar Therapeutics
Email:	researchenquiries@nightstartx.com
Phone:	+44 (0)207 062 2777

Choroideremia (CHM) is a rare, X-linked recessive, degenerative disease of the retina which
begins in childhood with loss of night vision and gradually progresses to legal blindness by
the fifth decade. There are no approved treatements for CHM and the disease is poorly
characterised given its rare nature and scarscity of available data. The objective of this
study is to collect natural history data from a large cohort of CHM patients in order to
evaluate possible efficacy measures that could be utilised in future interventional trials of
novel therapies.

Inclusion Criteria:

- Are willing and able to provide informed consent for participation in the study.

- Are male and ≥18 years of age.

- 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 Early
Treatment Diabetic Retinopathy Study [ETDRS] letters) in at least one eye.

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.

We found this trial at

sites

Clinical Trial Facility in Miami Florida

Miami, Florida 33136

from

Miami, FL