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/Actively Recruiting
Led by Children's Hospital of Fudan University · Updated on 2026-03-16
60
Participants Needed
1
Research Sites
26 weeks
Total Duration
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 HCQs role in treatment. Participants are randomly assigned to one of two groups one group receives hydroxychloroquine orally at a dose of 10 mgkg 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
Hydroxychloroquine in Children's Interstitial Lung Diseases With Genetic Causes
L
Liling Qian, Doctor
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Children's Hospital of Fudan University
Lead Sponsor
You may qualify if you...
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Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
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
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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Lead Sponsor