Hydroxychloroquine Administration for Reduction of Pexophagy

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.

Timeline

Start

2019-01-11

Primary completion

2020-05-05

Completion

2020-05-05

Drugs

Indications