Hydroxychloroquine Administration for Reduction of Pexophagy
Status:
Completed
Trial end date:
2020-05-05
Target enrollment:
Participant gender:
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.