ChoroideremiaNIGHTNCT03359551
Natural History of the Progression of Choroideremia Study
An observational study of Choroideremia, run by NightstaRx Ltd, a Biogen Company at 20 sites in 9 countries.
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. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Choroideremia
- How many people
- 319 participants
- Where
- 20 sitesin 9 countries
- Study dates
- 30 Jun 2015to 1 Oct 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
20 sites in 9 countries.
20 sites in 9 countries
United States7 sites
Research Site
Baltimore, Maryland, 21287
Research Site
Dallas, Texas, 75231
Research Site
Los Angeles, California, 90095
Research Site
Madison, Wisconsin, 53705
Research Site
Miami, Florida, 33136
Research Site
New York, New York, 10032
Research Site
Portland, Oregon, 97232
Canada3 sites
Research Site
Edmonton, Alberta
Research Site
Montreal, H3A 0E7
Research Site
Vancouver, V5Z 3N9
United Kingdom3 sites
Research Site
London, EC1V 2PD
Research Site
Manchester, M13 9WL
Research Site
Oxford, OX3 9DU
About this 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.
Read the detailed description
This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.
- Conditions
- Choroideremia
- Keywords
- REP1, Retina, Retinal Degeneration, Retinal Dystrophy, Natural History
- Medical subject headings
- Choroideremia, Retinal Degeneration, Retinal Dystrophies
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to Month 20
Secondary outcomes (7)
Change from Baseline in Reading Performance using International Reading Speed Texts (IReST)
Reading performance will be evaluated prior to pupil dilation.
Measured: Baseline up to Month 12
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline up to Month 20
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2017
- Study start30 Jun 2015
- Primary completion1 Oct 2020
- Study completion1 Oct 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for ChoroideremiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 319 participants (actual)
Sponsor and investigators
- Lead sponsor
- NightstaRx Ltd, a Biogen Company
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03359551
- Sponsor's protocol ID
- 273CH001
- Other IDs
- NSR-CHM-OS1
- Acronym
- NIGHT
- Status last verified
- Feb 2023
- First submitted
- 24 Nov 2017
- Last updated
- 10 Feb 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/
Publications
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.
PubMed 28499705
Study at a glance
From the registry record, last updated by the sponsor on 10 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Natural History of the Progression of Choroideremia Study
- ClinicalTrials.gov ID
- NCT03359551
- Acronym / sponsor ID
- NIGHT / 273CH001
- Conditions
- Choroideremia
- Keywords
- REP1, Retina, Retinal Degeneration, Retinal Dystrophy, Natural History
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 319 participants (actual)
- Lead sponsor
- NightstaRx Ltd, a Biogen CompanyUniversity, hospital or other
- Study start
- 30 Jun 2015 (actual)
- Primary completion
- 1 Oct 2020 (actual)
- Study completion
- 1 Oct 2020 (actual)
- First posted
- 2 Dec 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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 (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
7 secondary outcomes
Change from Baseline in Reading Performance using International Reading Speed Texts (IReST)
Time frame: Baseline up to Month 12
Change from Baseline in Color Vision
Time frame: Baseline up to Month 12
Change from Baseline in Visual Fields
Time frame: Baseline up to Month 12
Change from Baseline in Contrast Sensitivity
Time frame: Baseline up to Month 12
Change from Baseline in Retinal Thinning Using Spectral Domain Optical Coherence Tomography (SD-OCT)
Time frame: Baseline up to Month 20
Change from Baseline in Area of Viable Retinal Tissue Using Fundus Autofluorescence
Time frame: Baseline up to Month 20
Change from Baseline in Retinal Sensitivity Using Microperimetry
Time frame: Baseline up to Month 20
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Feb 2023.
- Background: PubMed 28499705Hariri 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.
- Individual participant dataSharing planned: Yes. In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/
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Choroideremia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Biogen
20 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Choroideremia, run by NightstaRx Ltd, a Biogen Company at 20 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States, Canada, United Kingdom, Germany, Brazil and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Best-Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (EDTRS) (time frame: Up to Month 20). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03359551, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Feb 2023.