Using hydroxychloroquine to treat genetic lung disease in children
Hydroxychloroquine in Pediatric ILD With Genetic Surfactant Dysfunction Disorders: Cross-control, Prospective Study
NA · Children's Hospital of Fudan University · NCT03822780
This study is testing if hydroxychloroquine can help children with a genetic lung disease feel better and manage their symptoms.
Quick facts
| Phase | NA |
|---|---|
| Study type | Interventional |
| Enrollment | 25 (estimated) |
| Ages | 1 Month to 18 Years |
| Sex | All |
| Sponsor | Children's Hospital of Fudan University (other) |
| Locations | 1 site (Shanghai, Shanghai) |
| Trial ID | NCT03822780 on ClinicalTrials.gov |
What this trial studies
This research investigates the efficacy and safety of hydroxychloroquine sulfate (HCQ) for treating pediatric interstitial lung disease (chILD) caused by mutations in pulmonary surfactant-associated genes. The study focuses on children with genetically diagnosed surfactant dysfunction disorders, which are often chronic and associated with high morbidity. Current treatments are limited and primarily involve corticosteroids, which have variable efficacy. The study aims to explore HCQ's potential anti-inflammatory properties as an alternative treatment option.
Who should consider this trial
Good fit: Ideal candidates include clinically stable children aged 2 months to 18 years with genetically diagnosed surfactant dysfunction disorders.
Not a fit: Patients who have received hydroxychloroquine treatment in the last 3 months may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for children suffering from severe lung diseases caused by genetic mutations.
How similar studies have performed: While there have been case reports suggesting positive responses to hydroxychloroquine in similar conditions, this approach is still relatively novel and untested in a formal clinical trial setting.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients should be clinically stable for inclusion into the study * Mature newborn ≥ 37 weeks of gestation, Infants and children (≥2month and \< 18y) or previously preterm (≤ 37 weeks of gestation) babies or children(≥2month and \<18y) if chILD genetically diagnosed * chILD genetically diagnosed surfactant dysfunction disorders including patients with mutations in SFTPC, SFTPB, ABCA3, TTF1 (Nkx2-1), FOXF1 further extremely rare entities with specific mutations, for example in TBX4, NPC2, NPC1, NPB, COPA, LRBA and other genes * no HCQ treatment in the last 3 months * Ability of subject or/and legal representatives to understand character and individual consequences of clinical trial * 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: Subjects presenting with any of the following criteria will not be included in the trial: * chILD primarily related to developmental disorders * chILD primarily related to growth abnormalities reflecting deficient alveolarization * chILD related to chronic aspiration * chILD related to immunodeficiency * chILD related to abnormalities in lung vessel structure * chILD related to organ transplantation/organ rejection/GvHD * chILD related to recurrent infections * Acute severe infectious exacerbations * Known hypersensitivity to HCQ, or other ingredients of the tablets * Proven retinopathy or maculopathy * Glucose-6-phosphate-dehydrogenase deficiency resulting in favism or hemolytic anemia * Myasthenia gravis * Hematopoetic disorders * Participation in other clinical trials during the present clinical trial or not beyond the time of 4 half-lives of the medication used, at least one week * Hereditary galactose intolerance, lactase deficiency or glucose-galactose- malabsorption * Simultaneous prescription of other potentially nephrotoxic or hepatotoxic medication at the discretion of the treating physician
Where this trial is running
Shanghai, Shanghai
- Children's hospital of Fudan University — Shanghai, Shanghai, China (RECRUITING)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Surfactant Dysfunction, Interstitial lung disease, Hydroxychloroquine