Actively Recruiting

Early Phase 1
Age: 1Month - 18Years
All Genders
ID04532346

Safety and Efficacy of Hydroxychloroquine in Children's Interstitial Lung Diseases With Genetic Causes: a Randomized Controlled Study

Led by Children's Hospital of Fudan University · Updated on 2026-03-16

60

Participants Needed

1

Research Sites

26 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This research aims to evaluate the effectiveness and safety of hydroxychloroquine (HCQ) in children with interstitial lung diseases (chILD) caused by genetic factors. chILD is a rare group of chronic lung disorders with high health risks, often linked to gene variations affecting surfactant proteins. This randomized controlled trial focuses on children under 18 years with genetically confirmed chILD to better understand HCQ's role in treatment. Participants are randomly assigned to one of two groups: one group receives hydroxychloroquine orally at a dose of 10 mg/kg twice daily for 12 months, with a maximum daily dose of 400 mg. The other group serves as a control and does not receive hydroxychloroquine. This study spans a full year of treatment to observe potential changes and effects. During the study, researchers will monitor participants' oxygenation status at 1, 3, 6, and 12 months. They will also evaluate improvements in clinical symptoms at the same intervals. Participants are expected to be clinically stable before joining, with no recent medication changes or HCQ use. Safety and health monitoring will continue throughout the trial, which is planned to end in April 2027.

CONDITIONS

Brief Title

Hydroxychloroquine in Children's Interstitial Lung Diseases With Genetic Causes

Who Can Participate

Age: 1Month - 18Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Clinical diagnosis of children's interstitial lung disease under 18 years of age
  • Genetic diagnosis involving relevant genes (e.g., SFTPC, SFTPB, ABCA3, NKX2-1, CSF2RA, CSF2RB, IARS, MARS, COPA, SLC7A7, LRBA)
  • Clinically stable with no major medication changes in the last 4 weeks
  • No hydroxychloroquine treatment in the last 12 weeks
  • Signed informed consent from participant or legal representative before trial procedures
Not Eligible

You will not qualify if you...

  • Acute severe infectious exacerbations
  • Known allergy or hypersensitivity to hydroxychloroquine or tablet ingredients
  • Proven retinopathy or maculopathy
  • Renal insufficiency at screening (GFR <40 mL/min/1.73 m2 for 3 to 8 weeks old; <60 mL/min/1.73 m2 for patients 8 weeks and older)
  • Participation in other clinical trials during this study

AI-Screening

AI-Powered Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - 12 months

Participants receive hydroxychloroquine or no treatment and are monitored for efficacy and safety over 12 months.

Visits at 1 month, 3 months, 6 months, and 12 months

Trial Site Locations

Total: 1 location

1

Children's hospital of Fudan University

Shanghai, Shanghai Municipality, China, 201102

Actively Recruiting

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Research Team

L

Liling Qian, Doctor

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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Published Research Related To This Trial

An official American Thoracic Society clinical practice guideline: classification, evaluation, and management of childhood interstitial lung disease in infancy.

Geoffrey Kurland, Robin R Deterding, James S Hagood...

https://pubmed.ncbi.nlm.nih.gov/23905526

Mutations in methionyl-tRNA synthetase gene in a Chinese family with interstitial lung and liver disease, postnatal growth failure and anemia.

Yu Sun, Guorui Hu, Jihang Luo...

https://pubmed.ncbi.nlm.nih.gov/28148924