Hydroxychloroquine in Children's Interstitial Lung Diseases With Genetic Causes

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

EARLY_PHASE1

Total Enrollment

60 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-09-01

Study Completion Date

2027-04-30

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Interstitial Lung Disease Surfactant Dysfunction

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Hydroxychloroquine

Hydroxychloroquine in a dose of 10 mg/kg\*d, p.o., bid for 12 months. The maximum daily dose is 400mg.

Group Type EXPERIMENTAL

Hydroxychloroquine

Intervention Type DRUG

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.

control

control group which do not take hydroxychloroquine for treatment.

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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Hydroxychloroquine

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.

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

1 Month

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No