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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04937907
Recruitment Status : Recruiting
First Posted : June 24, 2021
Last Update Posted : September 16, 2021
Sponsor:
Information provided by (Responsible Party):
Shanghai Children's Hospital

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Alport Syndrome, X-Linked Drug: Hydroxychloroquine Sulfate 100 milligram (mg) Tab Drug: Benazepril hydrochloride 10 milligram (mg) Tab Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy and Safety of Hydroxychloroquine in Patients With X-linked Alport Syndrome in China (CHXLAS)
Actual Study Start Date : September 8, 2021
Estimated Primary Completion Date : December 31, 2022
Estimated Study Completion Date : June 30, 2023


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 Name: 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 Name: 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 Name: Benazepril




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.



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Ages Eligible for Study:   3 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04937907


Contacts
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Contact: Wen-yan Huang, PhD +8618964025491 huangwenyan@sjtu.edu.cn
Contact: Lei Sun, MD +8618817821787 sunlei@shchildren.com.cn

Locations
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China, Shanghai
Shanghai Children's Hospital Recruiting
Shanghai, Shanghai, China, 200062
Contact: Wen-yan Huang, PHD    +8618964025491    huangwenyan@sjtu.edu.cn   
Sponsors and Collaborators
Shanghai Children's Hospital
Investigators
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Study Director: Wen-yan Huang, PhD Shanghai Children's Hospital
Publications:
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Responsible Party: Shanghai Children's Hospital
ClinicalTrials.gov Identifier: NCT04937907    
Other Study ID Numbers: 2021SPNR001
2019YLYM06 ( Other Grant/Funding Number: Shanghai Children's Hospital )
First Posted: June 24, 2021    Key Record Dates
Last Update Posted: September 16, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Shanghai Children's Hospital:
Alport Syndrome
Hydroxychloroquine
Treatment
Additional relevant MeSH terms:
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Nephritis, Hereditary
Syndrome
Disease
Pathologic Processes
Urogenital Abnormalities
Nephritis
Kidney Diseases
Urologic Diseases
Congenital Abnormalities
Collagen Diseases
Connective Tissue Diseases
Hydroxychloroquine
Benazepril
Antimalarials
Antiprotozoal Agents
Antiparasitic Agents
Anti-Infective Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antirheumatic Agents
Angiotensin-Converting Enzyme Inhibitors
Protease Inhibitors
Antihypertensive Agents