Status:
RECRUITING
Hydroxychloroquin (HCQ) in chILD of Genetic Defect
Lead Sponsor:
Children's Hospital of Fudan University
Conditions:
Surfactant Dysfunction
Eligibility:
All Genders
1-18 years
Phase:
NA
Brief 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 mu...
Detailed 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 mortali...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
July 1 2017
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
July 30 2026
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT03822780
Start Date
July 1 2017
End Date
July 30 2026
Last Update
May 13 2024
Active Locations (1)
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1
Children's hospital of Fudan University
Shanghai, Shanghai Municipality, China, 201102