Study of Hydroxychloroquine in Patients With X-linked Alport Syndrome in China (CHXLAS)
NCT ID: NCT04937907
Last Updated: 2023-12-07
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
50 participants
INTERVENTIONAL
2021-09-08
2024-12-31
Brief Summary
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Detailed Description
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Patients in the Phase 2 cohort will be randomized 1:1 to either Hydroxychloroquine Cohort or Comparator Cohort.
All patients in the study will follow the same visit and assessment schedule. Following randomization on Day 1, patients will be scheduled to be assessed during treatment at Weeks 4, 12, and 24. Patients will not receive study drug during a 24-week withdrawal period between Weeks 25 and 48. Patients will also be scheduled to be assessed at an in person follow up visit at Week 36, and 48.
Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Hydroxychloroquine Cohort
Patients in the cohort will receive Hydroxychloroquine(HCQ) throughout the study.Patients administered HCQ by oral at a dose of 6.5mg per kilogram twice a day for 6 months. During treatment with HCQ, patients also received enalapril(5-10mg qd).
Hydroxychloroquine Sulfate 100 milligram (mg) Tab
Patients administered HCQ by oral at a dose of 6.5mg per kilogram twice a day at least 6 months.
Benazepril hydrochloride 10 milligram (mg) Tab
Patients administered Benazepril by oral at a dose of 5mg or 10mg once a day at least 6 months.
Comparator Cohort
During treatment with HCQ, Patients randomized to Comparator Cohort only received enalapril(5-10mg qd).
Benazepril hydrochloride 10 milligram (mg) Tab
Patients administered Benazepril by oral at a dose of 5mg or 10mg once a day at least 6 months.
Interventions
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Hydroxychloroquine Sulfate 100 milligram (mg) Tab
Patients administered HCQ by oral at a dose of 6.5mg per kilogram twice a day at least 6 months.
Benazepril hydrochloride 10 milligram (mg) Tab
Patients administered Benazepril by oral at a dose of 5mg or 10mg once a day at least 6 months.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Age 3-18 years old;
* Diagnosis of Alport syndrome by genetic testing (documented mutation in a gene associated with Alport syndrome, including COL4A3, COL4A4, or COL4A5) or histologic assessment using electron microscopy;
* Screening eGFR ≥ 90 mL/min/1.73 m2;
* ACE inhibitor and/or ARB, the dosing regimen should be stable for at least 4 weeks prior to screening;
* No antirheumatic drugs such as hydroxychloroquine have been used;
* Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures;
Exclusion Criteria
* Prior exposure to hydroxychloroquine;
* Ongoing chronic hemodialysis or peritoneal dialysis therapy;
* Renal transplant recipient;
* Any clinically significant illness within 4 weeks before screening or surgical or medical condition (other than Alport syndrome) that could interfere with the subject's study compliance; confound the study results; impact subject safety; or significantly alter the absorption, distribution, metabolism, or excretion of drugs;
* Participation in other interventional clinical studies;
* Known hypersensitivity to any component of the study drug.
3 Years
18 Years
ALL
No
Sponsors
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Shanghai Children's Hospital
OTHER
Responsible Party
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Principal Investigators
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Wen-yan Huang, PhD
Role: STUDY_DIRECTOR
Shanghai Children's Hospital
Locations
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Shanghai Children's Hospital
Shanghai, Shanghai Municipality, China
Countries
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Central Contacts
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Facility Contacts
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Wen-yan Huang, PHD
Role: primary
References
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Hertz JM, Thomassen M, Storey H, Flinter F. Clinical utility gene card for: Alport syndrome - update 2014. Eur J Hum Genet. 2015 Sep;23(9). doi: 10.1038/ejhg.2014.254. Epub 2014 Nov 12. No abstract available.
Daga S, Donati F, Capitani K, Croci S, Tita R, Giliberti A, Valentino F, Benetti E, Fallerini C, Niccheri F, Baldassarri M, Mencarelli MA, Frullanti E, Furini S, Conticello SG, Renieri A, Pinto AM. New frontiers to cure Alport syndrome: COL4A3 and COL4A5 gene editing in podocyte-lineage cells. Eur J Hum Genet. 2020 Apr;28(4):480-490. doi: 10.1038/s41431-019-0537-8. Epub 2019 Nov 21.
Schrezenmeier E, Dorner T. Mechanisms of action of hydroxychloroquine and chloroquine: implications for rheumatology. Nat Rev Rheumatol. 2020 Mar;16(3):155-166. doi: 10.1038/s41584-020-0372-x. Epub 2020 Feb 7.
Yang YZ, Chen P, Liu LJ, Cai QQ, Shi SF, Chen YQ, Lv JC, Zhang H. Comparison of the effects of hydroxychloroquine and corticosteroid treatment on proteinuria in IgA nephropathy: a case-control study. BMC Nephrol. 2019 Aug 5;20(1):297. doi: 10.1186/s12882-019-1488-6.
Liu LJ, Yang YZ, Shi SF, Bao YF, Yang C, Zhu SN, Sui GL, Chen YQ, Lv JC, Zhang H. Effects of Hydroxychloroquine on Proteinuria in IgA Nephropathy: A Randomized Controlled Trial. Am J Kidney Dis. 2019 Jul;74(1):15-22. doi: 10.1053/j.ajkd.2019.01.026. Epub 2019 Mar 25.
Other Identifiers
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2019YLYM06
Identifier Type: OTHER_GRANT
Identifier Source: secondary_id
2021SPNR001
Identifier Type: -
Identifier Source: org_study_id