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 range6 Months – 40 Years

SexALL

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.

What they're measuring

Electroretinogram (ERG) voltage changes.

Timeframe: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.

Change in the red blood cell levels of plasmalogen.

Timeframe: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.

Change in the plasma levels of phytanic acid.

Timeframe: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.

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.

Trial details

NCT IDNCT03856866

SponsorThe Hospital for Sick Children

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2020-05-05

View on ClinicalTrials.gov More Zellweger Syndrome trials

Plain-language summary

Who can participate

Age range6 Months – 40 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Electroretinogram (ERG) voltage changes.

Timeframe: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.

Change in the red blood cell levels of plasmalogen.

Timeframe: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.

Change in the plasma levels of phytanic acid.

Timeframe: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.

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.