Prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases: Study protocol for an investigator-initiated, randomized controlled, parallel-group clinical trial
Abstract Background Interstitial lung diseases in children (chILD) are rare and consist of many different entities that affect the parenchyma of the lungs, leading to a chronic lung disease. The natural course of many of these diseases is connected with a high morbidity and significant mortality. Sy...
| Main Authors: | , , , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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BMC
2020-04-01
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| Series: | Trials |
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| Online Access: | http://link.springer.com/article/10.1186/s13063-020-4188-4 |
| _version_ | 1818213986456305664 |
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| author | Matthias Griese Meike Köhler Sabine Witt Daniela Sebah Matthias Kappler Martin Wetzke Nicolaus Schwerk Nagehan Emiralioglu Nural Kiper Kai Kronfeld Christian Ruckes Hans Rock Gisela Anthony Elias Seidl |
| author_facet | Matthias Griese Meike Köhler Sabine Witt Daniela Sebah Matthias Kappler Martin Wetzke Nicolaus Schwerk Nagehan Emiralioglu Nural Kiper Kai Kronfeld Christian Ruckes Hans Rock Gisela Anthony Elias Seidl |
| author_sort | Matthias Griese |
| collection | DOAJ |
| description | Abstract Background Interstitial lung diseases in children (chILD) are rare and consist of many different entities that affect the parenchyma of the lungs, leading to a chronic lung disease. The natural course of many of these diseases is connected with a high morbidity and significant mortality. Symptomatic treatment consists of oxygen supplementation, adequate nutrition adapted to the high energy demand generated by the disease due to the increased breathing effort required, as well as immunization against respiratory pathogens to prevent exacerbations through respiratory infections. No proven pharmacological treatments are available to date. This placebo-controlled study aims to evaluate the efficacy and safety of the mid-term use of hydroxychloroquine in chILD. Methods and design The study is an explorative, prospective, randomized, double-blind, placebo-controlled investigation of hydroxychloroquine (HCQ) in chILD. Patients can be included into the trial when diagnosed with a chronic (≥ 3 weeks’ duration) diffuse parenchymal lung disease (chILD) (1) genetically defined, (2) histologically defined or (3) diagnosed with idiopathic pulmonary hemorrhage (hemosiderosis). The study contains of two different study blocks, a START and a STOP block, which can be initiated in any sequence. Each patient can participate in each block only once. In the START block subjects are randomized to parallel groups for 4 weeks treatment, then the placebo group is switched to the active drug. In the STOP block, subjects taking HCQ are randomized into parallel groups treated with placebo or HCQ. Discussion This study is the first international, investigator-initiated, prospective and controlled investigation of a pharmacological treatment in chILD. The block design was selected as it has the advantage of accommodating patients who are initiating or withdrawing from HCQ therapy, thus allowing the participation of those who were previously started on off-label HCQ. The cross-over design and selected outcome parameters enables us to include appropriate numbers of patients of all age groups from neonates to adults suffering from these rare diseases. Trial registration This is an exploratory, Phase 2a, randomized, double-blind, placebo-controlled, parallel-group, multinational study investigating the initiation or withdrawal of hydroxychloroquine in subjects with chILD. Study title: Hydroxychloroquine in pediatric ILD: START randomized controlled in parallel groups, then switch placebo to the active drug, and STOP randomized controlled in parallel groups to evaluate the efficacy and safety of hydroxychloroquine (HCQ). Short title: HCQ in pediatric ILD, particularly 4surfdefect. EudraCT, ID: 2013–003714-40. Registered on 2 July 2013. ClinicalTrials.gov, ID: NCT02615938 . Registered on 8 November 2015. IZKS trial code: 2013–006; Sponsor: University Hospital, Ludwig-Maximilians University of Munich. Responsible Party: Prof. Dr. med. Matthias Griese, University Hospital, Ludwig-Maximilians University of Munich, Germany. |
| first_indexed | 2024-12-12T06:13:00Z |
| format | Article |
| id | doaj.art-7f0f68745bd744c9a1f5e7cee447adcd |
| institution | Directory Open Access Journal |
| issn | 1745-6215 |
| language | English |
| last_indexed | 2024-12-12T06:13:00Z |
| publishDate | 2020-04-01 |
| publisher | BMC |
| record_format | Article |
| series | Trials |
| spelling | doaj.art-7f0f68745bd744c9a1f5e7cee447adcd2022-12-22T00:35:06ZengBMCTrials1745-62152020-04-012111910.1186/s13063-020-4188-4Prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases: Study protocol for an investigator-initiated, randomized controlled, parallel-group clinical trialMatthias Griese0Meike Köhler1Sabine Witt2Daniela Sebah3Matthias Kappler4Martin Wetzke5Nicolaus Schwerk6Nagehan Emiralioglu7Nural Kiper8Kai Kronfeld9Christian Ruckes10Hans Rock11Gisela Anthony12Elias Seidl13Department of Pediatrics, Dr. von Hauner Children´s Hospital, University Hospital, LMU Munich, German Center for Lung Research (DZL)Department of Pediatrics, Dr. von Hauner Children´s Hospital, University Hospital, LMU Munich, German Center for Lung Research (DZL)Department of Pediatrics, Dr. von Hauner Children´s Hospital, University Hospital, LMU Munich, German Center for Lung Research (DZL)Department of Pediatrics, Dr. von Hauner Children´s Hospital, University Hospital, LMU Munich, German Center for Lung Research (DZL)Department of Pediatrics, Dr. von Hauner Children´s Hospital, University Hospital, LMU Munich, German Center for Lung Research (DZL)Department of Pneumonology, Medical School HannoverDepartment of Pneumonology, Medical School HannoverDepartment of Pediatric Pulmonology, Hacettepe University Faculty of MedicineDepartment of Pediatric Pulmonology, Hacettepe University Faculty of MedicineIZKS, Interdisciplinary Center for Clinical Trials, University Medical Center MainzIZKS, Interdisciplinary Center for Clinical Trials, University Medical Center MainzDepartment of Neurology, University of Marburg, Central Information OfficeDepartment of Neurology, University of Marburg, Central Information OfficeDepartment of Pediatrics, Dr. von Hauner Children´s Hospital, University Hospital, LMU Munich, German Center for Lung Research (DZL)Abstract Background Interstitial lung diseases in children (chILD) are rare and consist of many different entities that affect the parenchyma of the lungs, leading to a chronic lung disease. The natural course of many of these diseases is connected with a high morbidity and significant mortality. Symptomatic treatment consists of oxygen supplementation, adequate nutrition adapted to the high energy demand generated by the disease due to the increased breathing effort required, as well as immunization against respiratory pathogens to prevent exacerbations through respiratory infections. No proven pharmacological treatments are available to date. This placebo-controlled study aims to evaluate the efficacy and safety of the mid-term use of hydroxychloroquine in chILD. Methods and design The study is an explorative, prospective, randomized, double-blind, placebo-controlled investigation of hydroxychloroquine (HCQ) in chILD. Patients can be included into the trial when diagnosed with a chronic (≥ 3 weeks’ duration) diffuse parenchymal lung disease (chILD) (1) genetically defined, (2) histologically defined or (3) diagnosed with idiopathic pulmonary hemorrhage (hemosiderosis). The study contains of two different study blocks, a START and a STOP block, which can be initiated in any sequence. Each patient can participate in each block only once. In the START block subjects are randomized to parallel groups for 4 weeks treatment, then the placebo group is switched to the active drug. In the STOP block, subjects taking HCQ are randomized into parallel groups treated with placebo or HCQ. Discussion This study is the first international, investigator-initiated, prospective and controlled investigation of a pharmacological treatment in chILD. The block design was selected as it has the advantage of accommodating patients who are initiating or withdrawing from HCQ therapy, thus allowing the participation of those who were previously started on off-label HCQ. The cross-over design and selected outcome parameters enables us to include appropriate numbers of patients of all age groups from neonates to adults suffering from these rare diseases. Trial registration This is an exploratory, Phase 2a, randomized, double-blind, placebo-controlled, parallel-group, multinational study investigating the initiation or withdrawal of hydroxychloroquine in subjects with chILD. Study title: Hydroxychloroquine in pediatric ILD: START randomized controlled in parallel groups, then switch placebo to the active drug, and STOP randomized controlled in parallel groups to evaluate the efficacy and safety of hydroxychloroquine (HCQ). Short title: HCQ in pediatric ILD, particularly 4surfdefect. EudraCT, ID: 2013–003714-40. Registered on 2 July 2013. ClinicalTrials.gov, ID: NCT02615938 . Registered on 8 November 2015. IZKS trial code: 2013–006; Sponsor: University Hospital, Ludwig-Maximilians University of Munich. Responsible Party: Prof. Dr. med. Matthias Griese, University Hospital, Ludwig-Maximilians University of Munich, Germany.http://link.springer.com/article/10.1186/s13063-020-4188-4chILDInterstitial lung diseaseHydroxychloroquine |
| spellingShingle | Matthias Griese Meike Köhler Sabine Witt Daniela Sebah Matthias Kappler Martin Wetzke Nicolaus Schwerk Nagehan Emiralioglu Nural Kiper Kai Kronfeld Christian Ruckes Hans Rock Gisela Anthony Elias Seidl Prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases: Study protocol for an investigator-initiated, randomized controlled, parallel-group clinical trial Trials chILD Interstitial lung disease Hydroxychloroquine |
| title | Prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases: Study protocol for an investigator-initiated, randomized controlled, parallel-group clinical trial |
| title_full | Prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases: Study protocol for an investigator-initiated, randomized controlled, parallel-group clinical trial |
| title_fullStr | Prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases: Study protocol for an investigator-initiated, randomized controlled, parallel-group clinical trial |
| title_full_unstemmed | Prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases: Study protocol for an investigator-initiated, randomized controlled, parallel-group clinical trial |
| title_short | Prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases: Study protocol for an investigator-initiated, randomized controlled, parallel-group clinical trial |
| title_sort | prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases study protocol for an investigator initiated randomized controlled parallel group clinical trial |
| topic | chILD Interstitial lung disease Hydroxychloroquine |
| url | http://link.springer.com/article/10.1186/s13063-020-4188-4 |
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