Study of Hydroxychloroquine in Patients With X-linked Alport Syndrome in China (CHXLAS) (CHXLAS)

December 6, 2023 updated by: Shanghai Children's Hospital

Efficacy and Safety of Hydroxychloroquine in Patients With X-linked Alport Syndrome in China (CHXLAS)

This Phase 2 randomized controlled trial will study the safety, tolerability, and efficacy of Hydroxychloroquine in qualified patients with Alport syndrome. The trial will be open-label, randomized, controlled and will enroll up to 50 patients.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This Phase 2 randomized controlled trial will study the safety, tolerability, and efficacy of Hydroxychloroquine in qualified patients with Alport syndrome. The trial will be open-label, randomized, controlled and will enroll up to 50 patients.

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.

Study Type

Interventional

Enrollment (Estimated)

50

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
    • Shanghai
      • Shanghai, Shanghai, China, 200062
        • Recruiting
        • Shanghai Children's Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Male or female;
  • 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:

  • Causes of chronic kidney disease aside from Alport syndrome (including but not limited to other heritable disorders leading to chronic kidney disease, diabetic nephropathy, hypertensive nephropathy, lupus nephritis, IgA nephropathy);
  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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).
Drug: 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.
Other Names:
  • HCQ
Drug: 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 Names:
  • Benazepril
Sham Comparator: Comparator Cohort
During treatment with HCQ, Patients randomized to Comparator Cohort only received enalapril(5-10mg qd).
Drug: 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 Names:
  • Benazepril

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in urinary erythrocyte count(/HP)
Time Frame: Baseline to maximum 48 weeks
To assess the change in urinary erythrocyte count(/HP) from baseline to week 48 for patients receiving active drug, compared to patients in Comparator Cohort.
Baseline to maximum 48 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in 24-hour urinary protein quantity
Time Frame: Baseline to maximum 48 weeks
To assess the change in 24-hour urinary protein quantity from baseline to week 48 for patients receiving active drug, compared to patients in Comparator Cohort.
Baseline to maximum 48 weeks
Change in urinary albumin characterization
Time Frame: Baseline to maximum 48 weeks
To assess the change in urinary albumin characterization from baseline to week 48 for patients receiving active drug, compared to patients in Comparator Cohort.
Baseline to maximum 48 weeks
Change in urinary albumin to creatinine ratio
Time Frame: Baseline to maximum 48 weeks
To assess the change in urinary albumin to creatinine ratio from baseline to week 48 for patients receiving active drug, compared to patients in Comparator Cohort.
Baseline to maximum 48 weeks
Change in urinary erythrocyte count(urinary sediment analyzer)
Time Frame: Baseline to maximum 48 weeks
To assess the change in urinary erythrocyte count(urinary sediment analyzer) from baseline to week 48 for patients receiving active drug, compared to patients in Comparator Cohort.
Baseline to maximum 48 weeks
Change in eGFR from baseline
Time Frame: Baseline to maximum 48 weeks
To assess the increase in eGFR from baseline to week 48 for patients receiving active drug, compared to patients in Comparator Cohort.
Baseline to maximum 48 weeks
Number of participants with treatment-related adverse events
Time Frame: Baseline to maximum 48 weeks
Safety will be assessed by monitoring adverse events, physical examinations and clinical laboratory test through 48 weeks.
Baseline to maximum 48 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Shanghai Children's Hospital

Investigators

  • Study Director: Wen-yan Huang, PhD, Shanghai Children's Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 8, 2021

Primary Completion (Estimated)

December 31, 2024

Study Completion (Estimated)

December 31, 2024

Study Registration Dates

First Submitted

June 17, 2021

First Submitted That Met QC Criteria

June 17, 2021

First Posted (Actual)

June 24, 2021

Study Record Updates

Last Update Posted (Estimated)

December 7, 2023

Last Update Submitted That Met QC Criteria

December 6, 2023

Last Verified

December 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2021SPNR001
  • 2019YLYM06 (Other Grant/Funding Number: Shanghai Children's Hospital)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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