- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05674058
Function and Imaging Assessments for G1961E-associated Stargardt Disease (FIRSTORBIT)
December 21, 2022 updated by: Institute of Molecular and Clinical Ophthalmology Basel
Function and Imaging Assessments for G1961E-associated Stargardt Disease: Outcome Measure Ranking as Basis for Interventional Trials
Some phenotypes of Stargardt disease are rather distinct.
This includes the 'bull's eye maculopathy' phenotype associated with the frequent ABCA4 G1961E variant.
In anticipation of a treatment trial, this natural history study aims to compare functional and structural outcome measures systematically.
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Study Type
Observational
Enrollment (Anticipated)
40
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Maximilian Pfau, MD
- Phone Number: +41 79 525 92 47
- Email: Maximilian.Pfau@usb.ch
Study Locations
-
-
Basel-Stadt
-
Basel, Basel-Stadt, Switzerland, CH-4031
- Recruiting
- University Hospital Basel
-
Contact:
- Kristina Pfau, MD
- Phone Number: +41 79 357 36 93
- Email: Kristina.Pfau@usb.ch
-
Contact:
- Maximilian Pfau, MD
- Phone Number: +41 79 525 92 47
- Email: Maximilian.Pfau@usb.ch
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Non-Probability Sample
Study Population
The study will include subjects with G1961E-associated Stargardt disease.
Description
Inclusion Criteria:
- Adult patients with G1961E-associated Stargardt disease
- Defined by (1.) the presence of two pathogenic ABCA4 variants with at least one ABCA4G1961E variant, (2.) a Stargardt disease phenotype, and (3.) the absence of pathogenic variants in PRPH2, PROM1, and ELOVL4
Exclusion Criteria:
- Inability to give informed consent
- Prior surgery (other than anti-VEGF injections, cataract surgery, YAG laser capsulotomy, or laser retinopexy) that - according to the investigator's judgment - may affect visual function assessments (e.g., retinal detachment surgery, glaucoma filtration surgery, or a history of a corneal transplant in the study-eye)
- Concurrent ophthalmic conditions in the study eye that (according to the investigator's judgment) may contribute to loss of vision such as clinically significant opacification of the ocular media (corneal dystrophies, cataract), other retinal diseases, or disease of the optic nerve head and visual pathway (including amblyopia)
- Major surgery planned or other events that (according to the investigator's judgment) could hinder follow-up examinations (e.g., knee replacement)
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Progression of photoreceptor outer and inner segment loss
Time Frame: Two years from Baseline
|
Rate of change for the (square-root transformed) EZ loss area (in mm/yr)
|
Two years from Baseline
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
General Publications
- Pfau M, Cukras CA, Huryn LA, Zein WM, Ullah E, Boyle MP, Turriff A, Chen MA, Hinduja AS, Siebel HE, Hufnagel RB, Jeffrey BG, Brooks BP. Photoreceptor degeneration in ABCA4-associated retinopathy and its genetic correlates. JCI Insight. 2022 Jan 25;7(2):e155373. doi: 10.1172/jci.insight.155373.
- Pfau M, Holz FG, Muller PL. Retinal light sensitivity as outcome measure in recessive Stargardt disease. Br J Ophthalmol. 2021 Feb;105(2):258-264. doi: 10.1136/bjophthalmol-2020-316201. Epub 2020 Apr 28.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
November 1, 2022
Primary Completion (Anticipated)
October 31, 2025
Study Completion (Anticipated)
October 31, 2025
Study Registration Dates
First Submitted
December 21, 2022
First Submitted That Met QC Criteria
December 21, 2022
First Posted (Estimate)
January 6, 2023
Study Record Updates
Last Update Posted (Estimate)
January 6, 2023
Last Update Submitted That Met QC Criteria
December 21, 2022
Last Verified
December 1, 2022
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- FIRSTORBIT
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Undecided
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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