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Hydroxychloroquine Administration for Reduction of Pexophagy

NCT03856866 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2020-12-17

No results posted yet for this study

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.

Conditions

  • Zellweger Syndrome
  • Peroxisome Biogenesis Disorders

Interventions

DRUG

Hydroxychloroquine

Hydroxychloroquine: 4mg/kg/day, divided bid.

DRUG

Placebo

Liquid suspension compounded to mimic the active hydroxycholoquine interventional agent.

Sponsors & Collaborators

  • The Hospital for Sick Children

    lead OTHER

Principal Investigators

  • Neal Sondheimer, MD · The Hospital for Sick Children

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
6 Months
Max Age
40 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-01-11
Primary Completion
2020-05-05
Completion
2020-05-05
FDA Drug
Yes

Countries

  • Canada

Study Locations

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Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03856866 on ClinicalTrials.gov