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Completed

NCT Number: NCT03359551

Natural History of the Progression of Choroideremia Study

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.

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Key information

Conditions

Choroideremia Choroid Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Genetic Diseases, X-Linked Retinal Degeneration Retinal Diseases Retinal Dystrophies Uveal Diseases

Age range

18 year and older

Sex eligibility

Male

Study type

Observational

Primary location

Research Site, São Paulo, Brazil

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About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

NightstaRx Ltd, a Biogen Company

Other

Registry information

Acronym: NIGHT

Important dates

Study start
2015
Primary completion
2020
Study completion
2020
First posted
Dec 2, 2017
Registry last updated
Feb 10, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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