An observational study in Choroideremia, sponsored by NightstaRx Ltd, a Biogen Company. Completed at 20 sites in 9 countries. Open to male participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-02-10.
Sponsored by NightstaRx Ltd, a Biogen Company · Observational
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.
34 studies on the registry are indexed under Choroideremia; 7 are open to participants now.
This study's enrollment of 319 is above the median of 120 across 11 observational studies indexed under Choroideremia.
Browse Choroideremia studies →NightstaRx Ltd, a Biogen Company is the lead sponsor of 3 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Change in Best-Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (EDTRS)
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.
Time frame: Up to Month 20
Change from Baseline in Reading Performance using International Reading Speed Texts (IReST)
Reading performance will be evaluated prior to pupil dilation.
Time frame: Baseline up to Month 12
Change from Baseline in Color Vision
Colour vision will be tested prior to pupil dilation. Each eye will be tested separately and in the same order.
Time frame: Baseline up to Month 12
Change from Baseline in Visual Fields
The progression of defects in visual fields will be assessed in both eyes using perimetry equipment.
Time frame: Baseline up to Month 12
Change from Baseline in Contrast Sensitivity
Contrast sensitivity will be measured for both eyes, prior to pupil dilation, using a Pelli Robson chart.
Time frame: Baseline up to Month 12
Change from Baseline in Retinal Thinning Using Spectral Domain Optical Coherence Tomography (SD-OCT)
SD-OCT measurements will be performed after dilation of the participant's pupil.
Time frame: Baseline up to Month 20
Change from Baseline in Area of Viable Retinal Tissue Using Fundus Autofluorescence
Fundus Autofluorescence will be performed after dilation of the participant's pupil to assess changes in the area of viable retinal tissue.
Time frame: Baseline up to Month 20
Change from Baseline in Retinal Sensitivity Using Microperimetry
Macular analyser integrity assessment (MAIA) microperimetry will be conducted for both eyes to assess changes in retinal sensitivity within the macula.
Time frame: Baseline up to Month 20
Plan to share: Yes — In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/
Data sharing details ↗ · vivli.org
This study is completed, as verified in Feb 2023. You cannot join it, but the record below documents what was studied.
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NightstaRx Ltd, a Biogen Company