Alport Syndrome, X-LinkedPhase 2CHXLASNCT04937907

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

A Phase 2 study testing Hydroxychloroquine Sulfate 100 milligram (mg) Tab and Benazepril hydrochloride 10 milligram (mg) Tab in people with Alport Syndrome, X-Linked, run by Shanghai Children's Hospital at 1 site in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 3 to 18 years
Conditions the study is for: Alport Syndrome, X-Linked
What is being tested?: Hydroxychloroquine Sulfate 100 milligram (mg) Tab and Benazepril hydrochloride 10 milligram (mg) Tab
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 50 participants
Where: 1 siteChina
Study dates: 8 Sep 2021to 31 Dec 2024
Phase: Phase 2
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 3 to 18 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

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;

Who cannot join

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.

Trial locations and contacts

1 site in 1 country.

China1 site

Shanghai Children's Hospital
Shanghai, Shanghai Municipality, 200062

Other trials in China

About this study

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

Read the detailed description

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: Alport Syndrome, X-Linked
Being tested: Hydroxychloroquine Sulfate 100 milligram (mg) Tab, Benazepril hydrochloride 10 milligram (mg) Tab
Keywords: Alport Syndrome, Hydroxychloroquine, Treatment
Medical subject headings: Nephritis, Hereditary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Hydroxychloroquine Sulfate 100 milligram (mg) TabDrug
Also called HCQ
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) TabDrug
Also called Benazepril
Patients administered Benazepril by oral at a dose of 5mg or 10mg once a day at least 6 months.

Study groups in NCT04937907
Study group	Role	What they receive
Hydroxychloroquine Cohort	Experimental	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). Receives: Drug: Hydroxychloroquine Sulfate 100 milligram (mg) Tab; Drug: Benazepril hydrochloride 10 milligram (mg) Tab
Comparator Cohort	Sham comparator	During treatment with HCQ, Patients randomized to Comparator Cohort only received enalapril(5-10mg qd). Receives: Drug: Benazepril hydrochloride 10 milligram (mg) Tab

What is being measured

The primary outcome is the main question the study is designed to answer.

Change in urinary erythrocyte count(/HP)
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.
Measured: Baseline to maximum 48 weeks

Secondary outcomes (6)

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

8 Sep 202131 Dec 2024

First posted24 Jun 2021
Study start8 Sep 2021
Primary completion31 Dec 2024
Study completion31 Dec 2024

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (5)Papers the sponsor has linked to this study.
Other trials for Alport Syndrome, X-LinkedStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 50 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04937907
Sponsor's protocol ID: 2021SPNR001
Other IDs: 2019YLYM06
Acronym: CHXLAS
Status last verified: Feb 2026
First submitted: 17 Jun 2021
Last updated: 2 Mar 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Publications

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.
PubMed 25388007
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.
PubMed 31754267
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.
PubMed 32034323
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.
PubMed 31382914
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.
PubMed 30922594

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 2
EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive
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Early Check: Expanded Screening in Newborns
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All Alport Syndrome, X-Linked trials

Study at a glance

From the registry record, last updated by the sponsor on 2 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Efficacy and Safety of Hydroxychloroquine in Patients With X-linked Alport Syndrome in China (CHXLAS)
ClinicalTrials.gov ID: NCT04937907
Acronym / sponsor ID: CHXLAS / 2021SPNR001
Conditions: Alport Syndrome, X-Linked
Keywords: Alport Syndrome, Hydroxychloroquine, Treatment
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 50 participants (actual)
Lead sponsor: Shanghai Children's HospitalUniversity, hospital or other
Study start: 8 Sep 2021 (actual)
Primary completion: 31 Dec 2024 (actual)
Study completion: 31 Dec 2024 (actual)
First posted: 24 Jun 2021 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Patients in the Phase 2 cohort will be randomized 1:1 to either Hydroxychloroquine Cohort or Comparator Cohort.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Hydroxychloroquine Cohort	Experimental	Drug: Hydroxychloroquine Sulfate 100 milligram (mg) Tab, Drug: Benazepril hydrochloride 10 milligram (mg) TabPatients 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).
Comparator Cohort	Sham comparator	Drug: Benazepril hydrochloride 10 milligram (mg) TabDuring treatment with HCQ, Patients randomized to Comparator Cohort only received enalapril(5-10mg qd).

Who may qualify

Ages: 3 to 18 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (7 listed)

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;

Key exclusion criteria (7 listed)

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;

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Hydroxychloroquine Sulfate 100 milligram (mg) TabAlso: HCQPatients administered HCQ by oral at a dose of 6.5mg per kilogram twice a day at least 6 months.	Drug	Hydroxychloroquine Cohort
Benazepril hydrochloride 10 milligram (mg) TabAlso: BenazeprilPatients administered Benazepril by oral at a dose of 5mg or 10mg once a day at least 6 months.	Drug	Comparator Cohort, Hydroxychloroquine Cohort

Primary outcome

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.

6 secondary outcomes

Change in 24-hour urinary protein quantity
Time frame: Baseline to maximum 48 weeks
Change in urinary albumin characterization
Time frame: Baseline to maximum 48 weeks
Change in urinary albumin to creatinine ratio
Time frame: Baseline to maximum 48 weeks
Change in urinary erythrocyte count(urinary sediment analyzer)
Time frame: Baseline to maximum 48 weeks
Change in eGFR from baseline
Time frame: Baseline to maximum 48 weeks
Number of participants with treatment-related adverse events
Time frame: Baseline to maximum 48 weeks

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2026.
Background: PubMed 25388007Hertz 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.
Background: PubMed 31754267Daga 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.
Background: PubMed 32034323Schrezenmeier 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.
Background: PubMed 31382914Yang 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.
Background: PubMed 30922594Liu 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.
Individual participant dataSharing planned: No

Related trials for Alport Syndrome, X-Linked

RecruitingPhase 2
EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
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RecruitingObservational study
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Wen-yan Huang, PhD study director

Shanghai Children's Hospital

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 2 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in urinary erythrocyte count(/HP) (time frame: Baseline to maximum 48 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04937907, which also lists 5 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Mar 2026.