NCT06190626 Longitudinal Prospective Natural History Study of Retinopathy in Zellweger Spectrum Disorder
| NCT ID | NCT06190626 |
| Status | Recruiting |
| Phase | — |
| Sponsor | McGill University Health Centre/Research Institute of the McGill University Health Centre |
| Condition | Zellweger Spectrum Disorder |
| Study Type | OBSERVATIONAL |
| Enrollment | 30 participants |
| Start Date | 2023-12-18 |
| Primary Completion | 2029-01-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 30 participants in total. It began in 2023-12-18 with a primary completion date of 2029-01-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The goal of this observational study is to define the course of the retinal degeneration in a ZSD patient cohort. The objective of this study is to gather information so the investigators can: 1. define the course of the retinal degeneration in a ZSD patient cohort with retinal degeneration 2. define what tests best monitor the progression of the retinal degeneration 3. generate prognostic information about vision loss in ZSD. At each yearly visit, the participants will answer a functional vision questionnaire, have a physical evaluation, blood test, and participate in a variety of vision tests. The investigators will also collect pertinent medical history. Participants will travel to study site. The study will provide financial support for board and travel.
Eligibility Criteria
Inclusion Criteria: * confirmed ZSD with deleterious variants in PEX genes identified * confirmed or expected retinal involvement Exclusion Criteria: * unable to perform a minimum of one vision test * severe ZSD disease
Contact & Investigator
Nancy E Braverman, MD, MS
PRINCIPAL INVESTIGATOR
McGill University Health Centre/Research Institute of the McGill University Health Centre
Frequently Asked Questions
Who can join the NCT06190626 clinical trial?
This trial is open to participants of all sexes, studying Zellweger Spectrum Disorder. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06190626 currently recruiting?
Yes, NCT06190626 is actively recruiting participants. Contact the research team at nancy.braverman@mcgill.ca for enrollment information.
Where is the NCT06190626 trial being conducted?
This trial is being conducted at Rochester, United States, Edmonton, Canada, Montreal, Canada.
Who is sponsoring the NCT06190626 clinical trial?
NCT06190626 is sponsored by McGill University Health Centre/Research Institute of the McGill University Health Centre. The principal investigator is Nancy E Braverman, MD, MS at McGill University Health Centre/Research Institute of the McGill University Health Centre. The trial plans to enroll 30 participants.