Status:
COMPLETED
Hydroxychloroquine Administration for Reduction of Pexophagy
Lead Sponsor:
The Hospital for Sick Children
Conditions:
Zellweger Syndrome
Peroxisome Biogenesis Disorders
Eligibility:
All Genders
6-40 years
Phase:
PHASE2
Brief 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 liqui...
Detailed Description
HARP is a phase II/III, double-blind, placebo-controlled, randomized, crossover series N-of-1 study of the effect of hydroxychloroquine (HCQ) in patients with peroxisomal biogenesis disorders (PBD-ZSD...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
January 11 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 5 2020
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT03856866
Start Date
January 11 2019
End Date
May 5 2020
Last Update
December 17 2020
Active Locations (1)
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1
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G1X8