biostudies-literatureGriese ME-RareFP7Deutsches Zentrum für Lungenforschung289https://www.ebi.ac.uk/biostudies/studies/S-EPMC9308121biostudies-literatureUnknown17(1)<h4>Background</h4>No results of controlled trials are available for any of the few treatments offered to children with interstitial lung diseases (chILD). We evaluated hydroxychloroquine (HCQ) in a phase 2, prospective, multicentre, 1:1-randomized, double-blind, placebo-controlled, parallel-group/crossover trial. HCQ (START arm) or placebo were given for 4 weeks. Then all subjects received HCQ for another 4 weeks. In the STOP arm subjects already taking HCQ were randomized to 12 weeks of HCQ or placebo (= withdrawal of HCQ). Then all subjects stopped treatment and were observed for another 12 weeks.<h4>Results</h4>26 subjects were included in the START arm, 9 in the STOP arm, of these four subjects participated in both arms. The primary endpoint, presence or absence of a response to treatOrphanet journal of rare diseasesRandomized controlled phase 2 trial of hydroxychloroquine in childhood interstitial lung disease.PMC930812182DZLG13A2No. 30565301GM1702Ruckes CFeilcke MPrenzel FOlivier MKappler MGriese MSeidl ECarlens JNeuner DHebestreit HBaden WStichtenoth GAhrens FStohr LEismann CHengst MSebah DStahl MHCQ-study groupStauffer GRietschel CBrinkmann FSchulze JGebhardt JVieweg LNahrlich LZielen SStock PKoerner-Rettberg CNickolay TLau SGGorbulev SWetzke MSchwerk NSegerer FKitz RPhilipp UKronfeld KRock HWitt SAnne SBagheri-Potthof AThalmeier ASommerburg OStrenge-Hesse AStehling FBabl JMehl AThome UAnthony GKopp MfalseRandomized controlled phase 2 trial of hydroxychloroquine in childhood interstitial lung disease.<h4>Background</h4>No results of controlled trials are available for any of the few treatments offered to children with interstitial lung diseases (chILD). We evaluated hydroxychloroquine (HCQ) in a phase 2, prospective, multicentre, 1:1-randomized, double-blind, placebo-controlled, parallel-group/crossover trial. HCQ (START arm) or placebo were given for 4 weeks. Then all subjects received HCQ for another 4 weeks. In the STOP arm subjects already taking HCQ were randomized to 12 weeks of HCQ or placebo (= withdrawal of HCQ). Then all subjects stopped treatment and were observed for another 12 weeks.<h4>Results</h4>26 subjects were included in the START arm, 9 in the STOP arm, of these four subjects participated in both arms. The primary endpoint, presence or absence of a response to treat2022-01-01T00:00:00Z2022 Jul2025-04-25T18:12:06.867Z2022-08-06T22:39:41.659ZS-EPMC93081213587107110.1186/s13023-022-02399-2