Hydroxychloroquine in Children's Interstitial Lung Diseases With Genetic Causes

Last updated: February 18, 2025
Sponsor: Children's Hospital of Fudan University
Overall Status: Active - Recruiting

Phase

1

Condition

Lung Disease

Treatment

Hydroxychloroquine

Clinical Study ID

NCT04532346
QLL202008
  • Ages 1-18
  • All Genders

Study 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 Criteria

Inclusion

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 inthe last 4 weeks

  • No HCQ treatment in the last 12 weeks

  • Signed and dated informed consent of the subject (if subject has the ability) andthe representatives (of underaged children) must be available before start of anyspecific trial procedures

Exclusion

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)< 40mL/min/1.73 m2 in patients aged 3 to 8 weeks< 60 mL/min/1.73 m2 in patients ≥ 8weeks of age

  • Participation in other clinical trials during the present clinical trial

Study Design

Total Participants: 60
Treatment Group(s): 1
Primary Treatment: Hydroxychloroquine
Phase: 1
Study Start date:
September 01, 2024
Estimated Completion Date:
April 30, 2027

Study Description

Children Interstitial lung disease (chILD) is a heterogeneous group of rare respiratory disorders of known and unknown etiologies that are mostly chronic and associated with high morbidity and mortality. Genetic factors are important contributors to chILD. Genetic variations have been mainly described in genes encoding (or interacting with) the surfactant proteins (SP): SP-C (SFTPC) and the ATP-binding cassette-family A-member 3 (ABCA3) (ABCA3), and less frequently in the genes encoding NKX homeobox 2 (NKX2)-1 (NKX2-1), SP-B (SFTPB), SP-A (SFTPA) and other genes.

Hydroxychloroquine has been reported to be useful in cases or case series of chILD including those with genetic causes alone or in combination with systemic steroids. However, the efficacy is highly variable and no randomized controlled study has been reported. The study is a randomized controlled investigation aiming to evaluate the efficacy and safety of hydroxychloroquine in chILD with genetic causes.

Connect with a study center

  • Children's hospital of Fudan University

    Shanghai, Shanghai 201102
    China

    Active - Recruiting

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