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Natural History of the Progression of Choroideremia Study (NIGHT)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03359551
Recruitment Status : Completed
First Posted : December 2, 2017
Last Update Posted : January 27, 2021
Sponsor:
Information provided by (Responsible Party):
Biogen ( NightstaRx Ltd, a Biogen Company )

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.

Condition or disease
Choroideremia

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

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Study Type : Observational
Actual Enrollment : 319 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Natural History of the Progression of Choroideremia Study
Actual Study Start Date : June 30, 2015
Actual Primary Completion Date : October 1, 2020
Actual Study Completion Date : October 1, 2020

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. Change from Baseline in Reading Performance using International Reading Speed Texts (IReST) [ Time Frame: Baseline up to Month 12 ]
    Reading performance will be evaluated prior to pupil dilation.

  2. Change from Baseline in Color Vision [ Time Frame: Baseline up to Month 12 ]
    Colour vision will be tested prior to pupil dilation. Each eye will be tested separately and in the same order.

  3. Change from Baseline in Visual Fields [ Time Frame: Baseline up to Month 12 ]
    The progression of defects in visual fields will be assessed in both eyes using perimetry equipment.

  4. Change from Baseline in Contrast Sensitivity [ Time Frame: Baseline up to Month 12 ]
    Contrast sensitivity will be measured for both eyes, prior to pupil dilation, using a Pelli Robson chart.

  5. Change from Baseline in Retinal Thinning Using Spectral Domain Optical Coherence Tomography (SD-OCT) [ Time Frame: Baseline up to Month 20 ]
    SD-OCT measurements will be performed after dilation of the participant's pupil.

  6. Change from Baseline in Area of Viable Retinal Tissue Using Fundus Autofluorescence [ Time Frame: Baseline up to Month 20 ]
    Fundus Autofluorescence will be performed after dilation of the participant's pupil to assess changes in the area of viable retinal tissue.

  7. Change from Baseline in Retinal Sensitivity Using Microperimetry [ Time Frame: Baseline up to Month 20 ]
    Macular analyser integrity assessment (MAIA) microperimetry will be conducted for both eyes to assess changes in retinal sensitivity within the macula.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Criteria

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03359551


Locations
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United States, California
Research Site
Los Angeles, California, United States, 90095
United States, Florida
Research Site
Miami, Florida, United States, 33136
United States, Maryland
Research Site
Baltimore, Maryland, United States, 21287
United States, New York
Research Site
New York, New York, United States, 10032
United States, Oregon
Research Site
Portland, Oregon, United States, 97232
United States, Texas
Research Site
Dallas, Texas, United States, 75231
United States, Wisconsin
Research Site
Madison, Wisconsin, United States, 53705
Brazil
Research Site
São Paulo, Brazil, 04552-050
Canada, Alberta
Research Site
Edmonton, Alberta, Canada
Canada
Research Site
Montréal, Canada, H3A 0E7
Research Site
Vancouver, Canada, V5Z 3N9
Denmark
Research Site
Glostrup, Denmark
Finland
Research Site
Helsinki, Finland, 00290
France
Research Site
Montpellier, France, 74103
Germany
Research Site
Bonn, Germany, 53127
Research Site
Tübingen, Germany
Netherlands
Research Site
Nijmegen, Netherlands
United Kingdom
Research Site
London, United Kingdom, EC1V 2PD
Research Site
Manchester, United Kingdom, M13 9WL
Research Site
Oxford, United Kingdom, OX3 9DU
Sponsors and Collaborators
NightstaRx Ltd, a Biogen Company
Investigators
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Study Director: Medical Director Biogen
Publications:
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Responsible Party: NightstaRx Ltd, a Biogen Company
ClinicalTrials.gov Identifier: NCT03359551    
Other Study ID Numbers: 273CH001 (NSR-CHM-OS1)
First Posted: December 2, 2017    Key Record Dates
Last Update Posted: January 27, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Biogen ( NightstaRx Ltd, a Biogen Company ):
REP1
Retina
Retinal Degeneration
Retinal Dystrophy
Natural History
Additional relevant MeSH terms:
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Choroideremia
Eye Diseases, Hereditary
Eye Diseases
Choroid Diseases
Uveal Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked