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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT03856866 |
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Date of registration:
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05/11/2018 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Hydroxychloroquine Administration for Reduction of Pexophagy
HARP |
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Scientific title:
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Hydroxychloroquine Administration for Reduction of Pexophagy |
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Date of first enrolment:
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January 11, 2019 |
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Target sample size:
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5 |
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Recruitment status: |
Recruiting |
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URL:
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https://clinicaltrials.gov/show/NCT03856866 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 2
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Countries of recruitment
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Canada
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Contacts
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Name:
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Neal Sondheimer, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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The Hospital for Sick Children |
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Name:
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Neal Sondheimer, MD |
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Address:
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Telephone:
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4168137654 |
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Email:
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neal.sondheimer@sickkids.ca |
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Affiliation:
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Name:
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Bushra Momtaz |
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Address:
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Telephone:
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Email:
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bushra.momtaz@sickkids.ca |
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Affiliation:
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Diagnosed with a peroxisomal defect due to PEX1, PEX6 or PEX26 through a SCC or
CLIA-certified clinical genetic testing laboratory.
- Abnormal plasma very-long-chain fatty acid levels.
- All therapies available in Canada have been considered and ruled out, have failed or
were justified as being unsuitable for the patient. We note that there are no
therapies available.
- At least 84 days from last HCQ dose
Exclusion Criteria:
- Known sensitivity to HCQ.
- Known Glucose-6-phosphate dehydrogenase deficiency.
- Expected survival is less than six months.
- The patient does not provide informed consent.
- The patient is participating in another interventional clinical trial.
Age minimum:
6 Months
Age maximum:
40 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Peroxisome Biogenesis Disorders
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Zellweger Syndrome
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Intervention(s)
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Drug: Placebo
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Drug: Hydroxychloroquine
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Primary Outcome(s)
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Change in the red blood cell levels of plasmalogen.
[Time Frame: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.]
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Change in the plasma levels of phytanic acid.
[Time Frame: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.]
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Change in the plasma levels of very-long chain fatty acids.
[Time Frame: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.]
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Electroretinogram (ERG) voltage changes.
[Time Frame: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.]
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Secondary Outcome(s)
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Pediatric Inventory for Parents (PIP) following the treatment arms.
[Time Frame: 36 week. Measurements following each treatment arm.]
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Eye examination: Optical Coherence Tomography
[Time Frame: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.]
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Eye examination: Visual Acuity
[Time Frame: 12 week. Measurements at Day 0, Day 84(+/-7 days) of each treatment arm.]
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Secondary ID(s)
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1000061385
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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