Hydroxychloroquin (HCQ) in chILD of Genetic Defect

Launched by CHILDREN'S HOSPITAL OF FUDAN UNIVERSITY · Jan 28, 2019

Keywords

ClinConnect Summary

This clinical trial is studying the use of a medication called hydroxychloroquine (HCQ) to see if it is safe and effective for children with a specific lung condition known as chILD, which is caused by certain genetic mutations affecting lung surfactant. Surfactant is a substance that helps keep the lungs open, and when it doesn't work properly, it can lead to serious breathing problems. The trial is currently looking for participants who are clinically stable and fall into the age group of mature newborns (at least 37 weeks old) to children under 18 years old, who have a confirmed genetic diagnosis related to surfactant dysfunction.

To join the study, potential participants must have not received HCQ treatment in the past three months and should be able to understand the trial's details, with consent provided by themselves or their guardians. However, certain conditions, like developmental disorders or specific infections, may exclude individuals from participating. Those who do take part can expect close monitoring to assess the treatment's effects on their condition. This trial could be important for discovering new treatment options for children facing these challenging lung issues.

Gender

ALL

Eligibility 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