Using hydroxychloroquine to treat children's lung diseases caused by genetics
Safety and Efficacy of Hydroxychloroquine in Children's Interstitial Lung Diseases With Genetic Causes: a Randomized Controlled Study
EARLY_PHASE1 · Children's Hospital of Fudan University · NCT04532346
This study is testing if hydroxychloroquine can help children with genetic lung diseases feel better and improve their health.
Quick facts
| Phase | EARLY_PHASE1 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 1 Month to 18 Years |
| Sex | All |
| Sponsor | Children's Hospital of Fudan University (other) |
| Locations | 1 site (Shanghai, Shanghai Municipality) |
| Trial ID | NCT04532346 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the efficacy and safety of hydroxychloroquine (HCQ) in treating children's interstitial lung diseases (chILD) that have genetic causes. It is a randomized controlled trial involving children under 18 years old who have a clinical diagnosis of chILD and a confirmed genetic diagnosis. The study will assess the impact of HCQ on these patients, who often experience high morbidity and mortality due to their condition. The trial seeks to fill a gap in existing research, as no randomized controlled studies have previously been conducted on this topic.
Who should consider this trial
Good fit: Ideal candidates for this study are children under 18 years old with a clinical diagnosis of chILD and a confirmed genetic diagnosis.
Not a fit: Patients with acute severe infections, known hypersensitivity to hydroxychloroquine, or significant renal insufficiency may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option for children suffering from genetic interstitial lung diseases.
How similar studies have performed: While hydroxychloroquine has shown promise in case reports, this is the first randomized controlled trial specifically targeting this patient population.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Shanghai, Shanghai Municipality
- Children's hospital of Fudan University — Shanghai, Shanghai Municipality, China (RECRUITING)
Study contacts
- Study coordinator: Liling Qian, Doctor
- Email: llqian@126.com
- Phone: 021-64931913
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: Interstitial Lung Disease, Surfactant Dysfunction