Hydroxychloroquin (HCQ) in chILD of Genetic Defect

• Status: Recruiting
• Trial end date: 2026-07-30
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this proposed research is to investigate the efficacy and safety of hydroxychloroquine sulfate (HCQ, Quensyl) for pediatric ILD(chILD) caused by pulmonary surfactant-associated genes mutations.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital of Fudan University
• Treatments: Hydroxychloroquine

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