Natural History of the Progression of Choroideremia Study

Choroideremia Clinical Trial

— NIGHT  

Official title:

Natural History of the Progression of Choroideremia Study

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03359551
• Other study ID #: 273CH001
• Secondary ID: NSR-CHM-OS1
• Status: Completed
• Start date: June 30, 2015
• Est. completion date: October 1, 2020

Study information

• Verified date: February 2023
• Source: Biogen
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 319
• Est. completion date: October 1, 2020
• Est. primary completion date: October 1, 2020
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Choroideremia

Locations

Country	Name	City	State
Brazil	Research Site	São Paulo
Canada	Research Site	Edmonton	Alberta
Canada	Research Site	Montréal
Canada	Research Site	Vancouver
Denmark	Research Site	Glostrup
Finland	Research Site	Helsinki
France	Research Site	Montpellier
Germany	Research Site	Bonn
Germany	Research Site	Tübingen
Netherlands	Research Site	Nijmegen
United Kingdom	Research Site	London
United Kingdom	Research Site	Manchester
United Kingdom	Research Site	Oxford
United States	Research Site	Baltimore	Maryland
United States	Research Site	Dallas	Texas
United States	Research Site	Los Angeles	California
United States	Research Site	Madison	Wisconsin
United States	Research Site	Miami	Florida
United States	Research Site	New York	New York
United States	Research Site	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
NightstaRx Ltd, a Biogen Company

Countries where clinical trial is conducted

United States,  Brazil,  Canada,  Denmark,  Finland,  France,  Germany,  Netherlands,  United Kingdom, 

References & Publications (1)

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Secondary	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
Secondary	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.	Baseline up to Month 12
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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