Hydroxychloroquine Administration for Reduction of Pexophagy (NCT03856866) | Clinical Trial Compass
CompletedPhase 2
Hydroxychloroquine Administration for Reduction of Pexophagy
Canada3 participantsStarted 2019-01-11
Plain-language summary
A series of N-of-1, crossover, randomized, placebo-controlled, double-blinded trial. Hydroxychloroquine (HCQ) and a crossover to placebo (order is randomized and blinded) will be administered in liquid suspension for 84 days (12 weeks) each with an 84 day washout in between. We hypothesize that HCQ will reduce peroxisomal turnover, which will arrest ongoing injury in PBDs caused by PEX1, PEX6 or PEX26.
Who can participate
Age range
6 Months – 40 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Diagnosed with a peroxisomal defect due to PEX1, PEX6 or PEX26 through a SCC or CLIA-certified clinical genetic testing laboratory.
* Abnormal plasma very-long-chain fatty acid levels.
* All therapies available in Canada have been considered and ruled out, have failed or were justified as being unsuitable for the patient. We note that there are no therapies available.
* At least 84 days from last HCQ dose
Exclusion Criteria:
* Known sensitivity to HCQ.
* Known Glucose-6-phosphate dehydrogenase deficiency.
* Expected survival is less than six months.
* The patient does not provide informed consent.
* The patient is participating in another interventional clinical trial.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Electroretinogram (ERG) voltage changes.
Timeframe: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.
2
Change in the red blood cell levels of plasmalogen.
Timeframe: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.
3
Change in the plasma levels of phytanic acid.
Timeframe: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.
4
Change in the plasma levels of very-long chain fatty acids.
Timeframe: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.