Hydroxychloroquine in Children's Interstitial Lung Diseases With Genetic Causes

Launched by CHILDREN'S HOSPITAL OF FUDAN UNIVERSITY · Aug 26, 2020

Keywords

ClinConnect Summary

This clinical trial is studying the use of a medication called hydroxychloroquine (HCQ) to see if it can help children with interstitial lung diseases (chILD) that have genetic causes. Interstitial lung diseases make it difficult for the lungs to function properly, and this trial aims to determine whether HCQ is safe and effective for these young patients. The study is currently looking for participants aged 18 years and younger who have been genetically diagnosed with specific conditions related to their lung disease and have been stable without major medication changes recently.

If a child is eligible to participate, they will need to provide consent before starting any trial procedures. Participants will be monitored closely throughout the study to assess how well they respond to the treatment and to ensure their safety. It’s important to note that children who have certain health issues, such as severe infections or kidney problems, will not be able to join the trial. Overall, this research could provide valuable insights into new treatment options for children struggling with these challenging lung diseases.

Gender

ALL

Eligibility 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