Hydroxychloroquine in Children's Interstitial Lung Diseases With Genetic Causes
Safety and Efficacy of Hydroxychloroquine in Children's Interstitial Lung Diseases With Genetic Causes: a Randomized Controlled Study
Children's Hospital of Fudan University
60 participants
Sep 1, 2024
INTERVENTIONAL
Conditions
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.
Eligibility
Inclusion Criteria5
- 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 Criteria5
- 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
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Interventions
Hydroxychloroquine Sulfate is an anti-malarial and anti-rheumatic drug. hydroxychloroquine has been reported to improve the clinical status of chILD cases wtih genetic causes. The exact mechanism of action of hydroxychloroquine is unknown. In additon to having anti-inflammatory properties, hydroxychloroquine has been shown to affect intracellular processing of surfactant protein.
Locations(1)
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NCT04532346