Hydroxychloroquine (HCQ) in Pediatric Interstitial Lung Disease (ILD)

Summary

This is an exploratory Phase 2, randomized, double-blind, placebo-controlled, parallel-group, multinational study investigating the initiation or withdrawal of hydroxychloroquine in subjects with chILD.

Timeline

Start

2015-08-21

Primary completion

2019-06-27

Completion

2020-09-09

Outcome

Missed primary endpoint

Stopped: “Lack of ressources after inspection and appropriate correcting actions to be taken”

registry analysis (superiority test); Start Placebo, Then HCQ (Group A) vs Start HCQ, Then HCQ (Group B); p = 1; Fisher Exact NCT02615938 ↗

Publications

• Background Griese M, Kohler M, Witt S, Sebah D, Kappler M, Wetzke M, Schwerk N, Emiralioglu N, Kiper N, Kronfeld K, Ruckes C, Rock H, Anthony G, Seidl E. Prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases: Study protocol for an investigator-initiated, randomized controlled, parallel-group clinical trial. Trials. 2020 Apr 3;21(1):307. doi: 10.1186/s13063-020-4188-4.
• Background Braun S, Ferner M, Kronfeld K, Griese M. Hydroxychloroquine in children with interstitial (diffuse parenchymal) lung diseases. Pediatr Pulmonol. 2015 Apr;50(4):410-9. doi: 10.1002/ppul.23133. Epub 2014 Dec 9.
• Results Griese M, Kappler M, Stehling F, Schulze J, Baden W, Koerner-Rettberg C, Carlens J, Prenzel F, Nahrlich L, Thalmeier A, Sebah D, Kronfeld K, Rock H, Ruckes C; HCQ-study group; Wetzke M, Seidl E, Schwerk N. Randomized controlled phase 2 trial of hydroxychloroquine in childhood interstitial lung disease. Orphanet J Rare Dis. 2022 Jul 23;17(1):289. doi: 10.1186/s13023-022-02399-2.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Hydroxychloroquine	Small molecule	6.5 mg/kg	Oral

Indications