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Natural History of the Progression of Choroideremia Study (NIGHT)

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ClinicalTrials.gov Identifier: NCT03359551
Recruitment Status : Recruiting
First Posted : December 2, 2017
Last Update Posted : August 8, 2018
Sponsor:
Information provided by (Responsible Party):
Nightstar Therapeutics

Brief Summary:
Choroideremia (CHM) is a rare, X-linked recessive, degenerative disease of the retina which begins in childhood with loss of night vision and gradually progresses to legal blindness by the fifth decade. There are no approved treatements for CHM and the disease is poorly characterised given its rare nature and scarscity of available data. The objective of this study is to collect natural history data from a large cohort of CHM patients in order to evaluate possible efficacy measures that could be utilised in future interventional trials of novel therapies.

Condition or disease
Choroideremia

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Study Type : Observational
Estimated Enrollment : 300 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Natural History of the Progression of Choroideremia Study
Actual Study Start Date : June 2015
Estimated Primary Completion Date : November 2019
Estimated Study Completion Date : November 2019

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  1. Change in best-corrected visual acuity using the Early Treatment Diabetic Retinopathy Study (EDTRS) protocol. [ Time Frame: 20 months ]

Secondary Outcome Measures :
  1. Change in mean sensitivity using microperimetry. [ Time Frame: 20 months ]
  2. Change in area of viable retina using fundus autofluorescence [ Time Frame: 20 months ]
  3. Change in retinal thickness using SD-OCT. [ Time Frame: 20 months ]


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
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
The study population consists of up to 300 Choroideremia patients.
Criteria

Inclusion Criteria:

  • Are willing and able to provide informed consent for participation in the study.
  • Are male and ≥18 years of age.
  • 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 Early Treatment Diabetic Retinopathy Study [ETDRS] letters) in at least one eye.

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.

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


Contacts
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Contact: Nightstar Therapeutics +44 (0)207 062 2777 researchenquiries@nightstartx.com

Locations
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United States, California
Study Site Recruiting
Los Angeles, California, United States, 90095
United States, Florida
Study Site Recruiting
Miami, Florida, United States, 33136
United States, Maryland
Study Site Recruiting
Baltimore, Maryland, United States, 21287
United States, New York
Study Site Recruiting
New York, New York, United States, 10032
United States, Oregon
Study Site Recruiting
Portland, Oregon, United States, 97232
United States, Texas
Study Site Recruiting
Dallas, Texas, United States, 75231
United States, Wisconsin
Study Site Recruiting
Madison, Wisconsin, United States, 53705
Brazil
Study Site Recruiting
São Paulo, Brazil, 04552-050
Canada
Study Site Recruiting
Québec, Canada, H3A 0E7
Study Site Recruiting
Vancouver, Canada, V5Z 3N9
Finland
Study Site Recruiting
Helsinki, Finland, 00290
France
Study Site Recruiting
Montpellier, France, 74103
Germany
Study Site Recruiting
Bonn, Germany, 53127
Study Site Recruiting
Tübingen, Germany
Netherlands
Study Site Recruiting
Nijmegen, Netherlands
United Kingdom
Study Site Recruiting
London, United Kingdom, EC1V 2PD
Study Site Recruiting
Manchester, United Kingdom, M13 9WL
Study Site Recruiting
Oxford, United Kingdom, OX3 9DU
Sponsors and Collaborators
Nightstar Therapeutics

Publications:
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Responsible Party: Nightstar Therapeutics
ClinicalTrials.gov Identifier: NCT03359551     History of Changes
Other Study ID Numbers: NSR-CHM-OS1
First Posted: December 2, 2017    Key Record Dates
Last Update Posted: August 8, 2018
Last Verified: August 2018
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 Nightstar Therapeutics:
Choroideremia
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