Hydroxychloroquine Administration for Reduction of Pexophagy

• Status: Completed
• Trial end date: 2020-05-05
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: The Hospital for Sick Children
• Treatments: Hydroxychloroquine

Criteria

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.