NCT03856866 (HARP) is a Phase 2 clinical trial of Hydroxychloroquine in Zellweger Syndrome, sponsored by The Hospital for Sick Children.

Who is sponsoring NCT03856866?

NCT03856866 is sponsored by The Hospital for Sick Children.

What drugs are being tested in NCT03856866?

Interventions in NCT03856866: Hydroxychloroquine, Placebo.

What condition does NCT03856866 study?

NCT03856866 studies Zellweger Syndrome, Peroxisome Biogenesis Disorders.

Is NCT03856866 still recruiting?

NCT03856866 is currently completed. Last updated 17 December 2020.

Last reviewed 2020-12-17 · How we verify

NCT03856866: HARP

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Hydroxychloroquine Administration for Reduction of Pexophagy

Completed Phase 2 Last updated 17 December 2020

What this trial tests

Phase 2 trial testing Hydroxychloroquine in Zellweger Syndrome in 3 participants. Completed in 5 May 2020.

Timeline

11 January 2019

Primary endpoint
5 May 2020

5 May 2020

Quick facts

Lead sponsor	The Hospital for Sick Children
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	crossover
Masking	quadruple
Primary purpose	treatment
Enrollment	3
Start date	11 January 2019
Primary completion	5 May 2020
Estimated completion	5 May 2020
Sites	1 location across Canada

Drugs / interventions tested

Hydroxychloroquine (HYDROXYCHLOROQUINE) — full drug profile →
Placebo

Conditions studied

Zellweger Syndrome — all drugs for Zellweger Syndrome →
Peroxisome Biogenesis Disorders — all drugs for Peroxisome Biogenesis Disorders →

Sponsor

The Hospital for Sick Children

Who can join

Adults 6 Months to 40, any sex, with Zellweger Syndrome or Peroxisome Biogenesis Disorders. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

Selective autophagy as a therapeutic target for neurological diseases.
Xu W, Ocak U, Gao L, Tu S, et al · · 2021 · cited 61× · PMID 33067655 · DOI 10.1007/s00018-020-03667-9
Peroxisome and pexophagy in neurological diseases.
Xu W, Yan J, Shao A, Lenahan C, et al · · 2024 · PMID 39734532 · DOI 10.1016/j.fmre.2023.04.016

Verify or expand the search:

Other trials of Hydroxychloroquine

Trials testing the same drug.

NCT05842174 — Targeting Ischemia-Induced Autophagy Dependence in Hepatocellular Carcinoma · Phase 1, PHASE2 · recruiting
NCT07343635 — The Efficacy and Safety of Anti-inflammation Treatment (Hirudoid Introduction Followed by Yellow Light Therapy) Combined · NA · recruiting
NCT07352566 — Utilization of a Microdevice for Psoriasis and Atopic Dermatitis · Phase 4 · not yet recruiting
NCT06971744 — Autophagy Maintenance (AUTOMAIN) · Phase 2 · recruiting
NCT05841758 — Hydroxychloroquine as a Steroid-sparing Agent in Extrapulmonary Sarcoidosis · Phase 4 · recruiting

Other recruiting trials for Zellweger Syndrome

Currently open trials in the same condition.

NCT03047369 — The Myelin Disorders Biorepository Project · recruiting
NCT02171104 — MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis · Phase 2 · active not recruiting

Other The Hospital for Sick Children trials

Trials by the same sponsor.

NCT07454681 — MRI Assessment of Lung Airways in Cystic Fibrosis: Evaluate MRI's Ability to Detect Changes in Airway Structure . · not yet recruiting
NCT07452575 — 3D Ultrasound in Hemophilic Ankles · NA · not yet recruiting
NCT07508826 — AI-assisted Endotracheal Intubation · NA · not yet recruiting
NCT07345819 — Dexamethasone vs. Placebo in Children and Youth Hospitalized for Orbital Cellulitis · NA · not yet recruiting
NCT07497178 — MRgFUS for Childhood Epilepsy · NA · not yet recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT03856866 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by The Hospital for Sick Children
Last refreshed: 17 December 2020

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03856866.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing