Randomized controlled phase 2 trial of hydroxychloroquine in childhood interstitial lung disease.
Hydroxychloroquine
Interstitial lung diseases
Randomized-controlled trial
chILD
Journal
Orphanet journal of rare diseases
ISSN: 1750-1172
Titre abrégé: Orphanet J Rare Dis
Pays: England
ID NLM: 101266602
Informations de publication
Date de publication:
23 07 2022
23 07 2022
Historique:
received:
24
04
2022
accepted:
11
06
2022
entrez:
23
7
2022
pubmed:
24
7
2022
medline:
27
7
2022
Statut:
epublish
Résumé
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. 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 treatment, assessed as oxygenation (calculated from a change in transcutaneous O 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).
Sections du résumé
BACKGROUND
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.
RESULTS
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 treatment, assessed as oxygenation (calculated from a change in transcutaneous O
CONCLUSIONS
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).
Identifiants
pubmed: 35871071
doi: 10.1186/s13023-022-02399-2
pii: 10.1186/s13023-022-02399-2
pmc: PMC9308121
doi:
Substances chimiques
Hydroxychloroquine
4QWG6N8QKH
Banques de données
ClinicalTrials.gov
['NCT02615938']
EudraCT
['2013-003714-40']
Types de publication
Clinical Trial, Phase II
Journal Article
Multicenter Study
Randomized Controlled Trial
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
289Investigateurs
Margarete Olivier
(M)
Stefan Zielen
(S)
Azadeh Bagheri-Potthof
(A)
Ulrich Thome
(U)
Julia Gebhardt
(J)
Anna Mehl
(A)
Susanne Gabriele Lau
(SG)
Utz Philipp
(U)
Matthias Kopp
(M)
Guido Stichtenoth
(G)
Olaf Sommerburg
(O)
Mirjam Stahl
(M)
Richard Kitz
(R)
Christoph Rietschel
(C)
Philippe Stock
(P)
Frank Ahrens
(F)
Helge Hebestreit
(H)
Florian Segerer
(F)
Folke Brinkmann
(F)
Schlegtendal Anne
(S)
Claudia Eismann
(C)
Dörthe Neuner
(D)
Sabine Witt
(S)
Meike Hengst
(M)
Maria Feilcke
(M)
Jürgen Babl
(J)
Gabriele Stauffer
(G)
Tanja Nickolay
(T)
Stanislav Gorbulev
(S)
Gisela Anthony
(G)
Linda Stöhr
(L)
Laura Vieweg
(L)
Anke Strenge-Hesse
(A)
Informations de copyright
© 2022. The Author(s).
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