Using hydroxychloroquine to treat children's lung diseases caused by genetics
Official title: Safety and Efficacy of Hydroxychloroquine in Children's Interstitial Lung Diseases With Genetic Causes: a Randomized Controlled Study
Run by Children's Hospital of Fudan University · NCT04532346 · Data updated 2026-11-12
This study is testing if hydroxychloroquine can help children with genetic lung diseases feel better and improve their health.
Quick facts
| Phase | Early Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 1 Month to 18 Years |
| Sex | All |
| Enrollment | 60 (estimated) |
| Sponsor | Children's Hospital of Fudan University Academic / other |
| Locations | 1 site (Shanghai, Shanghai Municipality) |
| Status | Recruiting |
| Started | September 1, 2024 |
| Main results expected | October 2026 |
| Study ends | April 2027 |
| Registry record updated | March 16, 2026 |
| Trial ID | NCT04532346 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this study are children under 18 years old with a clinical diagnosis of chILD and a confirmed genetic diagnosis.
✕Probably not a fit
Patients with acute severe infections, known hypersensitivity to hydroxychloroquine, or significant renal insufficiency may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This clinical trial aims to evaluate the efficacy and safety of hydroxychloroquine (HCQ) in treating children's interstitial lung diseases (chILD) that have genetic causes. It is a randomized controlled trial involving children under 18 years old who have a clinical diagnosis of chILD and a confirmed genetic diagnosis. The study will assess the impact of HCQ on these patients, who often experience high morbidity and mortality due to their condition. The trial seeks to fill a gap in existing research, as no randomized controlled studies have previously been conducted on this topic.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option for children suffering from genetic interstitial lung diseases.
How similar studies have gone: While hydroxychloroquine has shown promise in case reports, this is the first randomized controlled trial specifically targeting this patient population.
Who can join: the full rules
Show full inclusion / exclusion criteria
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
Where it's running
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Children's hospital of Fudan University
Shanghai, Shanghai Municipality, China Recruiting
Study contacts
- Study coordinator: Liling Qian, Doctor
- Email: llqian@126.com
- Phone: 021-64931913
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.