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| ID | Type | Description | Link |
|---|---|---|---|
| NSR-CHM-OS1 | Other Identifier | NightstaRx Ltd, a Biogen Company |
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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.
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | Up to Month 20 |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in Reading Performance using International Reading Speed Texts (IReST) | Reading performance will be evaluated prior to pupil dilation. | Baseline up to Month 12 |
| Change from Baseline in Color Vision |
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Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Biogen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Los Angeles | California | 90095 | United States | ||
| Research Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28499705 | Background | Hariri AH, Velaga SB, Girach A, Ip MS, Le PV, Lam BL, Fischer MD, Sankila EM, Pennesi ME, Holz FG, MacLaren RE, Birch DG, Hoyng CB, MacDonald IM, Black GC, Tsang SH, Bressler NM, Larsen M, Gorin MB, Webster AR, Sadda SR; Natural History of the Progression of Choroideremia (NIGHT) Study Group. Measurement and Reproducibility of Preserved Ellipsoid Zone Area and Preserved Retinal Pigment Epithelium Area in Eyes With Choroideremia. Am J Ophthalmol. 2017 Jul;179:110-117. doi: 10.1016/j.ajo.2017.05.002. Epub 2017 May 10. |
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In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/
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Colour vision will be tested prior to pupil dilation. Each eye will be tested separately and in the same order.
| 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. | 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. | 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. | 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. | 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. | Baseline up to Month 20 |
| Miami |
| Florida |
| 33136 |
| United States |
| Research Site | Baltimore | Maryland | 21287 | United States |
| Research Site | New York | New York | 10032 | United States |
| Research Site | Portland | Oregon | 97232 | United States |
| Research Site | Dallas | Texas | 75231 | United States |
| Research Site | Madison | Wisconsin | 53705 | United States |
| Research Site | São Paulo | 04552-050 | Brazil |
| Research Site | Edmonton | Alberta | Canada |
| Research Site | Montreal | H3A 0E7 | Canada |
| Research Site | Vancouver | V5Z 3N9 | Canada |
| Research Site | Glostrup Municipality | Denmark |
| Research Site | Helsinki | 00290 | Finland |
| Research Site | Montpellier | 74103 | France |
| Research Site | Bonn | 53127 | Germany |
| Research Site | Tübingen | Germany |
| Research Site | Nijmegen | Netherlands |
| Research Site | London | EC1V 2PD | United Kingdom |
| Research Site | Manchester | M13 9WL | United Kingdom |
| Research Site | Oxford | OX3 9DU | United Kingdom |
| ID | Term |
|---|---|
| D015794 | Choroideremia |
| D012162 | Retinal Degeneration |
| D058499 | Retinal Dystrophies |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D015862 | Choroid Diseases |
| D014603 | Uveal Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
| D012164 | Retinal Diseases |
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