Adaptive multi-interventional trial platform to improve patient care for fibrotic interstitial lung diseases
© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ..
BACKGROUND: Fibrotic interstitial lung diseases (fILDs) are a heterogeneous group of lung diseases associated with significant morbidity and mortality. Despite a large increase in the number of clinical trials in the last 10 years, current regulatory-approved management approaches are limited to two therapies that prevent the progression of fibrosis. The drug development pipeline is long and there is an urgent need to accelerate this process. This manuscript introduces the concept and design of an innovative research approach to drug development in fILD: a global Randomised Embedded Multifactorial Adaptive Platform in fILD (REMAP-ILD).
METHODS: Description of the REMAP-ILD concept and design: the specific terminology, design characteristics (multifactorial, adaptive features, statistical approach), target population, interventions, outcomes, mission and values, and organisational structure.
RESULTS: The target population will be adult patients with fILD, and the primary outcome will be a disease progression model incorporating forced vital capacity and mortality over 12 months. Responsive adaptive randomisation, prespecified thresholds for success and futility will be used to assess the effectiveness and safety of interventions. REMAP-ILD embraces the core values of diversity, equity, and inclusion for patients and researchers, and prioritises an open-science approach to data sharing and dissemination of results.
CONCLUSION: By using an innovative and efficient adaptive multi-interventional trial platform design, we aim to accelerate and improve care for patients with fILD. Through worldwide collaboration, novel analytical methodology and pragmatic trial delivery, REMAP-ILD aims to overcome major limitations associated with conventional randomised controlled trial approaches to rapidly improve the care of people living with fILD.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2024 |
|---|---|
| Erschienen: |
2024 |
| Enthalten in: |
Zur Gesamtaufnahme - year:2024 |
|---|---|
| Enthalten in: |
Thorax - (2024) vom: 06. März |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Kawano-Dourado, Leticia [VerfasserIn] |
|---|
| Links: |
|---|
| Themen: |
Connective tissue disease associated lung disease |
|---|
| Anmerkungen: |
Date Revised 06.03.2024 published: Print-Electronic Citation Status Publisher |
|---|
| doi: |
10.1136/thorax-2023-221148 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM369379888 |
|---|
| LEADER | 01000naa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM369379888 | ||
| 003 | DE-627 | ||
| 005 | 20240307232934.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 240307s2024 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1136/thorax-2023-221148 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1319.xml |
| 035 | |a (DE-627)NLM369379888 | ||
| 035 | |a (NLM)38448221 | ||
| 035 | |a (PII)thorax-2023-221148 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Kawano-Dourado, Leticia |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Adaptive multi-interventional trial platform to improve patient care for fibrotic interstitial lung diseases |
| 264 | 1 | |c 2024 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Revised 06.03.2024 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status Publisher | ||
| 520 | |a © Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. | ||
| 520 | |a BACKGROUND: Fibrotic interstitial lung diseases (fILDs) are a heterogeneous group of lung diseases associated with significant morbidity and mortality. Despite a large increase in the number of clinical trials in the last 10 years, current regulatory-approved management approaches are limited to two therapies that prevent the progression of fibrosis. The drug development pipeline is long and there is an urgent need to accelerate this process. This manuscript introduces the concept and design of an innovative research approach to drug development in fILD: a global Randomised Embedded Multifactorial Adaptive Platform in fILD (REMAP-ILD) | ||
| 520 | |a METHODS: Description of the REMAP-ILD concept and design: the specific terminology, design characteristics (multifactorial, adaptive features, statistical approach), target population, interventions, outcomes, mission and values, and organisational structure | ||
| 520 | |a RESULTS: The target population will be adult patients with fILD, and the primary outcome will be a disease progression model incorporating forced vital capacity and mortality over 12 months. Responsive adaptive randomisation, prespecified thresholds for success and futility will be used to assess the effectiveness and safety of interventions. REMAP-ILD embraces the core values of diversity, equity, and inclusion for patients and researchers, and prioritises an open-science approach to data sharing and dissemination of results | ||
| 520 | |a CONCLUSION: By using an innovative and efficient adaptive multi-interventional trial platform design, we aim to accelerate and improve care for patients with fILD. Through worldwide collaboration, novel analytical methodology and pragmatic trial delivery, REMAP-ILD aims to overcome major limitations associated with conventional randomised controlled trial approaches to rapidly improve the care of people living with fILD | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Review | |
| 650 | 4 | |a Connective tissue disease associated lung disease | |
| 650 | 4 | |a Hypersensitivity pneumonitis | |
| 650 | 4 | |a Idiopathic pulmonary fibrosis | |
| 650 | 4 | |a Interstitial Fibrosis | |
| 650 | 4 | |a Rheumatoid lung disease | |
| 700 | 1 | |a Kulkarni, Tejaswini |e verfasserin |4 aut | |
| 700 | 1 | |a Ryerson, Christopher J |e verfasserin |4 aut | |
| 700 | 1 | |a Rivera-Ortega, Pilar |e verfasserin |4 aut | |
| 700 | 1 | |a Baldi, Bruno Guedes |e verfasserin |4 aut | |
| 700 | 1 | |a Chaudhuri, Nazia |e verfasserin |4 aut | |
| 700 | 1 | |a Funke-Chambour, Manuela |e verfasserin |4 aut | |
| 700 | 1 | |a Hoffmann-Vold, Anna-Maria |e verfasserin |4 aut | |
| 700 | 1 | |a Johannson, Kerri A |e verfasserin |4 aut | |
| 700 | 1 | |a Khor, Yet Hong |e verfasserin |4 aut | |
| 700 | 1 | |a Montesi, Sydney B |e verfasserin |4 aut | |
| 700 | 1 | |a Piccari, Lucilla |e verfasserin |4 aut | |
| 700 | 1 | |a Prosch, Helmut |e verfasserin |4 aut | |
| 700 | 1 | |a Molina-Molina, María |e verfasserin |4 aut | |
| 700 | 1 | |a Sellares Torres, Jacobo |e verfasserin |4 aut | |
| 700 | 1 | |a Bauer-Ventura, Iazsmin |e verfasserin |4 aut | |
| 700 | 1 | |a Rajan, Sujeet |e verfasserin |4 aut | |
| 700 | 1 | |a Jacob, Joseph |e verfasserin |4 aut | |
| 700 | 1 | |a Richards, Duncan |e verfasserin |4 aut | |
| 700 | 1 | |a Spencer, Lisa G |e verfasserin |4 aut | |
| 700 | 1 | |a Wendelberger, Barbara |e verfasserin |4 aut | |
| 700 | 1 | |a Jensen, Tom |e verfasserin |4 aut | |
| 700 | 1 | |a Quintana, Melanie |e verfasserin |4 aut | |
| 700 | 1 | |a Kreuter, Michael |e verfasserin |4 aut | |
| 700 | 1 | |a Gordon, Anthony C |e verfasserin |4 aut | |
| 700 | 1 | |a Martinez, Fernando J |e verfasserin |4 aut | |
| 700 | 1 | |a Kaminski, Naftali |e verfasserin |4 aut | |
| 700 | 1 | |a Cornelius, Victoria |e verfasserin |4 aut | |
| 700 | 1 | |a Lewis, Roger |e verfasserin |4 aut | |
| 700 | 1 | |a Adams, Wendy |e verfasserin |4 aut | |
| 700 | 1 | |a Jenkins, Gisli |e verfasserin |4 aut | |
| 700 | 0 | |a REMAP-ILD consortium |e verfasserin |4 aut | |
| 700 | 1 | |a Bravery, Amanda |e investigator |4 oth | |
| 700 | 1 | |a Goodwin, Amanda |e investigator |4 oth | |
| 700 | 1 | |a Etges, Ana |e investigator |4 oth | |
| 700 | 1 | |a Boshoff, Ana |e investigator |4 oth | |
| 700 | 1 | |a Günther, Andreas |e investigator |4 oth | |
| 700 | 1 | |a Briggs, Andrew |e investigator |4 oth | |
| 700 | 1 | |a Palmer, Andrew |e investigator |4 oth | |
| 700 | 1 | |a Wilson, Andrew |e investigator |4 oth | |
| 700 | 1 | |a Crawshaw, Anjali |e investigator |4 oth | |
| 700 | 1 | |a Hoffmann-Vold, Anna-Maria |e investigator |4 oth | |
| 700 | 1 | |a Bergeron, Anne |e investigator |4 oth | |
| 700 | 1 | |a Holland, Anne |e investigator |4 oth | |
| 700 | 1 | |a Gordon, Anthony C |e investigator |4 oth | |
| 700 | 1 | |a Prasse, Antje |e investigator |4 oth | |
| 700 | 1 | |a Tzouvelekis, Argyris |e investigator |4 oth | |
| 700 | 1 | |a Trachalaki, Athina |e investigator |4 oth | |
| 700 | 1 | |a Wells, Athol |e investigator |4 oth | |
| 700 | 1 | |a Nair, Avinash Anil |e investigator |4 oth | |
| 700 | 1 | |a Adegunsoye, Ayodeji |e investigator |4 oth | |
| 700 | 1 | |a Wendelberger, Barbara |e investigator |4 oth | |
| 700 | 1 | |a Hope-Gill, Ben |e investigator |4 oth | |
| 700 | 1 | |a Kaul, Bhavika |e investigator |4 oth | |
| 700 | 1 | |a Gooptu, Bibek |e investigator |4 oth | |
| 700 | 1 | |a Baldi, Bruno Guedes |e investigator |4 oth | |
| 700 | 1 | |a Crestani, Bruno |e investigator |4 oth | |
| 700 | 1 | |a Polanczyk, Carisi Anne |e investigator |4 oth | |
| 700 | 1 | |a Vancheri, Carlo |e investigator |4 oth | |
| 700 | 1 | |a Crobal, Carlos |e investigator |4 oth | |
| 700 | 1 | |a Summers, Charlotte |e investigator |4 oth | |
| 700 | 1 | |a Grainge, Chris |e investigator |4 oth | |
| 700 | 1 | |a Ryerson, Chris J |e investigator |4 oth | |
| 700 | 1 | |a Garnier, Christophe von |e investigator |4 oth | |
| 700 | 1 | |a Huntley, Christopher |e investigator |4 oth | |
| 700 | 1 | |a Ravaglia, Claudia |e investigator |4 oth | |
| 700 | 1 | |a Valenzuela, Claudia |e investigator |4 oth | |
| 700 | 1 | |a Hayton, Conal |e investigator |4 oth | |
| 700 | 1 | |a McCarthy, Cormac |e investigator |4 oth | |
| 700 | 1 | |a Chambers, Daniel |e investigator |4 oth | |
| 700 | 1 | |a Babalis, Daphne |e investigator |4 oth | |
| 700 | 1 | |a Thicket, David |e investigator |4 oth | |
| 700 | 1 | |a Turner, David |e investigator |4 oth | |
| 700 | 1 | |a Talwar, Deepak |e investigator |4 oth | |
| 700 | 1 | |a Anand, Devaraj |e investigator |4 oth | |
| 700 | 1 | |a Dhasmana, Devesh |e investigator |4 oth | |
| 700 | 1 | |a Parek, Dhruv |e investigator |4 oth | |
| 700 | 1 | |a Griffiths, Diane |e investigator |4 oth | |
| 700 | 1 | |a Villegas, Diego Castillo |e investigator |4 oth | |
| 700 | 1 | |a Richards, Duncan |e investigator |4 oth | |
| 700 | 1 | |a Santucci, Eliana |e investigator |4 oth | |
| 700 | 1 | |a Bendstrup, Elisabeth |e investigator |4 oth | |
| 700 | 1 | |a Balestro, Elisabetta |e investigator |4 oth | |
| 700 | 1 | |a Tsitoura, Eliza |e investigator |4 oth | |
| 700 | 1 | |a Falaschetti, Emanuela |e investigator |4 oth | |
| 700 | 1 | |a Gupta, Ena |e investigator |4 oth | |
| 700 | 1 | |a Farrand, Erica |e investigator |4 oth | |
| 700 | 1 | |a Khan, Fasihul |e investigator |4 oth | |
| 700 | 1 | |a Martinez, Fernando J |e investigator |4 oth | |
| 700 | 1 | |a Bonella, Francesco |e investigator |4 oth | |
| 700 | 1 | |a Lombardi, Francesco |e investigator |4 oth | |
| 700 | 1 | |a Hunninghake, Gary M |e investigator |4 oth | |
| 700 | 1 | |a Saini, Gauri |e investigator |4 oth | |
| 700 | 1 | |a Jenkins, Gisli |e investigator |4 oth | |
| 700 | 1 | |a Gudmundsson, Gunnar |e investigator |4 oth | |
| 700 | 1 | |a Collard, Harold |e investigator |4 oth | |
| 700 | 1 | |a Parfrey, Helen |e investigator |4 oth | |
| 700 | 1 | |a Prosch, Helmut |e investigator |4 oth | |
| 700 | 1 | |a Fainberg, Hernan |e investigator |4 oth | |
| 700 | 1 | |a Adamali, Huzaifa |e investigator |4 oth | |
| 700 | 1 | |a Stewart, Iain |e investigator |4 oth | |
| 700 | 1 | |a Forrest, Ian |e investigator |4 oth | |
| 700 | 1 | |a Glaspole, Ian |e investigator |4 oth | |
| 700 | 1 | |a Bauer-Ventura, Iazsmin |e investigator |4 oth | |
| 700 | 1 | |a Noth, Imre |e investigator |4 oth | |
| 700 | 1 | |a Cox, Ingrid |e investigator |4 oth | |
| 700 | 1 | |a Strambu, Irina |e investigator |4 oth | |
| 700 | 1 | |a Sellares, Jacobo |e investigator |4 oth | |
| 700 | 1 | |a Eaden, James |e investigator |4 oth | |
| 700 | 1 | |a Johnston, Janet |e investigator |4 oth | |
| 700 | 1 | |a Swigris, Jeff |e investigator |4 oth | |
| 700 | 1 | |a Blaikley, John |e investigator |4 oth | |
| 700 | 1 | |a Kim, John S |e investigator |4 oth | |
| 700 | 1 | |a Chung, Jonathan |e investigator |4 oth | |
| 700 | 1 | |a Lasky, Joseph A |e investigator |4 oth | |
| 700 | 1 | |a Jacob, Joseph |e investigator |4 oth | |
| 700 | 1 | |a Lee, Joyce |e investigator |4 oth | |
| 700 | 1 | |a Behr, Juergen |e investigator |4 oth | |
| 700 | 1 | |a Storrer, Karin |e investigator |4 oth | |
| 700 | 1 | |a Lewandowska, Katarzyna |e investigator |4 oth | |
| 700 | 1 | |a Johnson, Kate |e investigator |4 oth | |
| 700 | 1 | |a Antoniou, Katerina |e investigator |4 oth | |
| 700 | 1 | |a Hostettler, Katrin |e investigator |4 oth | |
| 700 | 1 | |a Johannson, Kerri A |e investigator |4 oth | |
| 700 | 1 | |a Hurley, Killian |e investigator |4 oth | |
| 700 | 1 | |a Hett, Kirsty |e investigator |4 oth | |
| 700 | 1 | |a Schwarzkopf, Larissa |e investigator |4 oth | |
| 700 | 1 | |a Fabbri, Laura |e investigator |4 oth | |
| 700 | 1 | |a Price, Laura |e investigator |4 oth | |
| 700 | 1 | |a Pearmain, Laurence |e investigator |4 oth | |
| 700 | 1 | |a Kawano-Dourado, Leticia |e investigator |4 oth | |
| 700 | 1 | |a Galvin, Liam |e investigator |4 oth | |
| 773 | 0 | 8 | |i Enthalten in |t Thorax |d 1946 |g (2024) vom: 06. März |w (DE-627)NLM00010387X |x 1468-3296 |7 nnns |
| 773 | 1 | 8 | |g year:2024 |g day:06 |g month:03 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1136/thorax-2023-221148 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |j 2024 |b 06 |c 03 | ||