Clinical Trial Details
— Status: Completed
Administrative data
NCT number |
NCT03856866 |
Other study ID # |
1000061385 |
Secondary ID |
|
Status |
Completed |
Phase |
Phase 2
|
First received |
|
Last updated |
|
Start date |
January 11, 2019 |
Est. completion date |
May 5, 2020 |
Study information
Verified date |
December 2020 |
Source |
The Hospital for Sick Children |
Contact |
n/a |
Is FDA regulated |
No |
Health authority |
|
Study type |
Interventional
|
Clinical Trial 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.
Description:
HARP is a phase II/III, double-blind, placebo-controlled, randomized, crossover series N-of-1
study of the effect of hydroxychloroquine (HCQ) in patients with peroxisomal biogenesis
disorders (PBD-ZSD). Patients eligible for the study must have a laboratory diagnosis of
PEX1, PEX6 or PEX26 dependent PBD-ZSD from a CLIA or SCC-certified clinical laboratory, a
history of abnormal VLCFA levels, and must be at least 84 days from their last HCQ dose.
Patients will be excluded for known sensitivity to HCQ, known glucose-6-phosphate
dehydrogenase deficiency, if they have an expected survival of less than 9 months or if they
are participating in another interventional clinical trial.
HCQ will be administered at a dose of 4mg/kg/day divided into two doses, as a liquid
suspension that can be given orally or through nasogastric or gastric tube. Within the study,
HCQ or placebo will be given for 84 days, followed by a washout period of 84 days followed by
an 84 day crossover to the alternative therapy to assess the effect the study measures.
Study measures will be completed at four intervals (initiation, end of period 1, start of
period 2, end of trial). Ophthalmological monitoring of patients has three components,
electroretinogram (ERG), visual acuity testing and optical coherence tomography (OCT). Plasma
levels of very long-chain fatty acids (VLCFA), plasmalogen and phytanic acid will be
assessed. Parents will also be administered The Pediatric Inventory for Parents (PIP), a
questionnaire that was developed to evaluate the stress associated with parenting a seriously
ill child, at the end of period 1 and period 2.