Hydroxychloroquine in Children's Interstitial Lung Diseases With Genetic Causes
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age1-18
SponsorChildren's Hospital of Fudan University
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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
Trial design
Design model
Parallel
Treatments tested in this trial
Hydroxychloroquine
DrugHydroxychloroquine 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.
Treatment groups
60 Participants
are divided into 2 treatment groupsGroup A: HydroxychloroquineExperimental treatment 1 intervention
Group B: controlNo intervention 0 interventions
Trial outcomes
Primary outcomes
1
Oxygenation status
It is a repeated measurement variable. It is a binary variable (1/0). In a patient without supplemental O2, increase≥5% in O2 saturation or decrease in respiratory rate≥20% means significant change or responder to hydroxychloroquine and the varibale would be setted into "1". In a patient with supplemental O2, increase≥5% in O2 saturation or decrease in respiratory rate≥20% or withdrawal of O2 means significant change or responder to hydroxychloroquine and the varibale would be setted into "1". If no supplemental O2 is necessary, the O2 saturation and respiratory rate are measured in an awake patient after 5 min at rest. If the patient needs supplement O2 , the supplementation is withdrawn after 5 min at rest and the O2 saturation and respiratory rate are measured.
Time frame
at first month, at 3rd month, at 6th month, at 12th monthSecondary outcomes
1
Improvement in clinical manifestation
It is a repeated measurement variable. It is a binary variable (1/0). If coughing resolved, intercostal retraction disappeared and weight for height back to normal is observed in a patient or improvement in chest radiography and pulmonary function tests is present, it means improvement in clinical manifestation and the variable would be setted into "1".
Time frame
at first month, at 3rd month, at 6th month, at 12th monthSponsors and contacts
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Source ClinicalTrials.gov