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Peroxisome Biogenesis Disorders clinical trials

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NCT ID: NCT03856866 Completed - Zellweger Syndrome Clinical Trials

Hydroxychloroquine Administration for Reduction of Pexophagy

HARP
Start date: January 11, 2019
Phase: Phase 2
Study type: Interventional

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.