Natural History of the Progression of Choroideremia Study
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Choroideremia
- Sponsor
- NightstaRx Ltd, a Biogen Company
- Enrollment
- 319
- Locations
- 1
- Primary Endpoint
- Change in Best-Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (EDTRS)
- Status
- Completed
- Last Updated
- 3 years ago
Overview
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.
Investigators
Eligibility Criteria
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.
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.
Outcomes
Primary Outcomes
Change in Best-Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (EDTRS)
Time Frame: Up to Month 20
BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If \<20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). An increase in the number of letters read correctly means that vision has improved.
Secondary Outcomes
- Change from Baseline in Reading Performance using International Reading Speed Texts (IReST)(Baseline up to Month 12)
- Change from Baseline in Visual Fields(Baseline up to Month 12)
- Change from Baseline in Retinal Thinning Using Spectral Domain Optical Coherence Tomography (SD-OCT)(Baseline up to Month 20)
- Change from Baseline in Area of Viable Retinal Tissue Using Fundus Autofluorescence(Baseline up to Month 20)
- Change from Baseline in Contrast Sensitivity(Baseline up to Month 12)
- Change from Baseline in Color Vision(Baseline up to Month 12)
- Change from Baseline in Retinal Sensitivity Using Microperimetry(Baseline up to Month 20)