RecruitingEarly Phase 1

Hydroxychloroquine in Children's Interstitial Lung Diseases With Genetic Causes

China60 participantsStarted 2024-09-01

Plain-language summary

The aim of this proposed study is to evaluate the efficacy and safety of hydroxychloroquine (HCQ) in children's interstitial lung diseases(chILD) with genetic causes. This study is a randomized controlled clinical trial.

Who can participate

Age range

1 Month – 18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * A clinical diagnosis of chILD with age\<18 years * Genetically diagnosed (e.g. SFTPC, SFTPB, ABCA3, NKX2-1, CSF2RA, CSF2RB, IARS, MARS, COPA, SLC7A7, LRBA) * Patients have to be clinically stable with no major changes in their medication in the last 4 weeks * No HCQ treatment in the last 12 weeks * Signed and dated informed consent of the subject (if subject has the ability) and the representatives (of underaged children) must be available before start of any specific trial procedures Exclusion Criteria: * Acute severe infectious exacerbations * Known hypersensitivity to HCQ, or other ingredients of the tablets * Proven retinopathy or maculopathy * Renal insufficiency at screening, defined as glomerular filtration rate (GFR)\< 40 mL/min/1.73 m2 in patients aged 3 to 8 weeks\< 60 mL/min/1.73 m2 in patients ≥ 8 weeks of age * Participation in other clinical trials during the present clinical trial

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Oxygenation status

Timeframe: at first month, at 3rd month, at 6th month, at 12th month

Trial details

NCT IDNCT04532346

SponsorChildren's Hospital of Fudan University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-10

Contact for this trial

Liling Qian, Doctor

021-64931913 llqian@126.com

View on ClinicalTrials.gov More Interstitial Lung Disease trials

Who can participate

Age range

1 Month – 18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.