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Randomized controlled phase 2 trial of hydroxychloroquine in childhood interstitial lung disease.

Matthias GrieseMatthias KapplerFlorian StehlingJohannes SchulzeWinfried BadenCordula Koerner-RettbergJulia CarlensFreerk PrenzelLutz NährlichAndreas ThalmeierDaniela SebahKai KronfeldHans RockChristian Ruckesnull nullMartin WetzkeElias SeidlNicolaus Schwerk
Published in: Orphanet journal of rare diseases (2022)
Acknowledging important shortcomings of the study, including a small study population, the treatment duration, lack of outcomes like lung function testing below age of 6 years, the small effect size of HCQ treatment observed requires careful reassessments of prescriptions in everyday practice (EudraCT-Nr.: 2013-003714-40, www.clinicaltrialsregister.eu , registered 02.07.2013). Registration The study was registered on 2 July 2013 (Eudra-CT Number: 2013-003714-40), whereas the approval by BfArM was received 24.11.2014, followed by the approval by the lead EC of the University Hospital Munich on 20.01.2015. At clinicaltrials.gov the trial was additionally registered on November 8, 2015 (NCT02615938).
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