Willingness of people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor (ETI) to participate in randomized modulator and inhaled antimicrobial clinical trials
•Willingness to enroll in clinical trials was assessed for those on CFTR modulators.•Willingness to enroll was associated with proposed trial length and comparator.•Shorter trials and active comparators were favored over alternatives.•Future studies in people on CFTR modulators will be feasible for...
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| Veröffentlicht in: | Journal of cystic fibrosis 2023-07, Vol.22 (4), p.652-655 |
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| container_title | Journal of cystic fibrosis |
| container_volume | 22 |
| creator | VanDevanter, D.R. Zemanick, E.T. Konstan, M.W. Ren, C.L. Odem-Davis, K. Emerman, I. Young, J. Mayer-Hamblett, N. |
| description | •Willingness to enroll in clinical trials was assessed for those on CFTR modulators.•Willingness to enroll was associated with proposed trial length and comparator.•Shorter trials and active comparators were favored over alternatives.•Future studies in people on CFTR modulators will be feasible for some trial designs.
To assess the feasibility of enrolling people with CF (pwCF) taking the CFTR modulator elexacaftor/tezacaftor/ivacaftor (ETI) in clinical trials of a new modulator.
PwCF receiving ETI at CHEC-SC study (NCT03350828) enrollment were surveyed for interest in 2-week to 6-month placebo- (PC) and active-comparator (AC) modulator studies. Those taking inhaled antimicrobials (inhABX) were surveyed for interest in PC inhABX studies.
Of 1791 respondents, 75% [95% CI 73, 77] would enroll in a 2-week PC modulator study versus 51% [49, 54] for a 6-month study; 82% [81, 84] and 63% [61, 65] would enroll in 2-week and 6 month AC studies; 77% [74, 80] of 551 taking inhABX would enroll in a 2-week PC inhABX study versus 59% [55, 63] for a 6-month study. Previous clinical trial experience increased willingness.
Study designs will affect feasibility of future clinical trials of new modulators and inhABX in people receiving ETI. |
| doi_str_mv | 10.1016/j.jcf.2023.04.007 |
| format | Article |
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To assess the feasibility of enrolling people with CF (pwCF) taking the CFTR modulator elexacaftor/tezacaftor/ivacaftor (ETI) in clinical trials of a new modulator.
PwCF receiving ETI at CHEC-SC study (NCT03350828) enrollment were surveyed for interest in 2-week to 6-month placebo- (PC) and active-comparator (AC) modulator studies. Those taking inhaled antimicrobials (inhABX) were surveyed for interest in PC inhABX studies.
Of 1791 respondents, 75% [95% CI 73, 77] would enroll in a 2-week PC modulator study versus 51% [49, 54] for a 6-month study; 82% [81, 84] and 63% [61, 65] would enroll in 2-week and 6 month AC studies; 77% [74, 80] of 551 taking inhABX would enroll in a 2-week PC inhABX study versus 59% [55, 63] for a 6-month study. Previous clinical trial experience increased willingness.
Study designs will affect feasibility of future clinical trials of new modulators and inhABX in people receiving ETI.</description><identifier>ISSN: 1569-1993</identifier><identifier>ISSN: 1873-5010</identifier><identifier>EISSN: 1873-5010</identifier><identifier>DOI: 10.1016/j.jcf.2023.04.007</identifier><identifier>PMID: 37100705</identifier><language>eng</language><publisher>Netherlands: Elsevier B.V</publisher><subject>Aminophenols - adverse effects ; Anti-Infective Agents ; Benzodioxoles - adverse effects ; Cystic Fibrosis - drug therapy ; Cystic Fibrosis - genetics ; Cystic Fibrosis Transmembrane Conductance Regulator - genetics ; Humans ; Mutation</subject><ispartof>Journal of cystic fibrosis, 2023-07, Vol.22 (4), p.652-655</ispartof><rights>2023</rights><rights>Copyright © 2023. Published by Elsevier B.V.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c3677-823ca410aedc1339c29237b2770267fab9b7eebf386a76beedde6183cbd64aa13</citedby><cites>FETCH-LOGICAL-c3677-823ca410aedc1339c29237b2770267fab9b7eebf386a76beedde6183cbd64aa13</cites><orcidid>0000-0003-4431-0644 ; 0000-0002-7507-9337 ; 0000-0002-9779-2571 ; 0000-0002-0557-7277 ; 0000-0002-5727-8164</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://dx.doi.org/10.1016/j.jcf.2023.04.007$$EHTML$$P50$$Gelsevier$$H</linktohtml><link.rule.ids>230,314,780,784,885,3550,27924,27925,45995</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/37100705$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>VanDevanter, D.R.</creatorcontrib><creatorcontrib>Zemanick, E.T.</creatorcontrib><creatorcontrib>Konstan, M.W.</creatorcontrib><creatorcontrib>Ren, C.L.</creatorcontrib><creatorcontrib>Odem-Davis, K.</creatorcontrib><creatorcontrib>Emerman, I.</creatorcontrib><creatorcontrib>Young, J.</creatorcontrib><creatorcontrib>Mayer-Hamblett, N.</creatorcontrib><creatorcontrib>CHEC-SC Study Group</creatorcontrib><title>Willingness of people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor (ETI) to participate in randomized modulator and inhaled antimicrobial clinical trials</title><title>Journal of cystic fibrosis</title><addtitle>J Cyst Fibros</addtitle><description>•Willingness to enroll in clinical trials was assessed for those on CFTR modulators.•Willingness to enroll was associated with proposed trial length and comparator.•Shorter trials and active comparators were favored over alternatives.•Future studies in people on CFTR modulators will be feasible for some trial designs.
To assess the feasibility of enrolling people with CF (pwCF) taking the CFTR modulator elexacaftor/tezacaftor/ivacaftor (ETI) in clinical trials of a new modulator.
PwCF receiving ETI at CHEC-SC study (NCT03350828) enrollment were surveyed for interest in 2-week to 6-month placebo- (PC) and active-comparator (AC) modulator studies. Those taking inhaled antimicrobials (inhABX) were surveyed for interest in PC inhABX studies.
Of 1791 respondents, 75% [95% CI 73, 77] would enroll in a 2-week PC modulator study versus 51% [49, 54] for a 6-month study; 82% [81, 84] and 63% [61, 65] would enroll in 2-week and 6 month AC studies; 77% [74, 80] of 551 taking inhABX would enroll in a 2-week PC inhABX study versus 59% [55, 63] for a 6-month study. Previous clinical trial experience increased willingness.
Study designs will affect feasibility of future clinical trials of new modulators and inhABX in people receiving ETI.</description><subject>Aminophenols - adverse effects</subject><subject>Anti-Infective Agents</subject><subject>Benzodioxoles - adverse effects</subject><subject>Cystic Fibrosis - drug therapy</subject><subject>Cystic Fibrosis - genetics</subject><subject>Cystic Fibrosis Transmembrane Conductance Regulator - genetics</subject><subject>Humans</subject><subject>Mutation</subject><issn>1569-1993</issn><issn>1873-5010</issn><issn>1873-5010</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2023</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp9kcFu1DAQhiMEoqXwAFyQj-WQ1I6TOBEHVFUtVKrEpYij5TiT7qycONjehfaZeMjOarcVXDjNeOab32P_WfZe8EJw0Zyti7Udi5KXsuBVwbl6kR2LVsm85oK_pLxuulx0nTzK3sS45lwortrX2ZFUgmheH2d_fqBzON_NECPzI1vALw7YL0wrZu9jQstG7IOPGFkAC7glmIGD38aaMflwluDhKcXtIWOnl7fXH1nybDGBNHAxCRjOLJh58BM-wMAmP2yc2cFUo97KOKqaOeGENvgejWOWVkNLSQp0jG-zVyMFeHeIJ9n3q8vbi6_5zbcv1xfnN7mVjVJ5W0prKsENDFZI2dmyK6XqS6V42ajR9F2vAPpRto1RTQ8wDNCIVtp-aCpjhDzJPu91l00_kQjMKRinl4CTCffaG9T_dmZc6Tu_1YLXpezqihRODwrB_9xATHrCaME5M4PfRF22vOm6umk5oWKP0qNjDDA-3yO43tms15ps1jubNa80GUczH_5e8HniyVcCPu0BoG_aIgQdLcJsYUByMenB43_kHwG_bL7H</recordid><startdate>20230701</startdate><enddate>20230701</enddate><creator>VanDevanter, D.R.</creator><creator>Zemanick, E.T.</creator><creator>Konstan, M.W.</creator><creator>Ren, C.L.</creator><creator>Odem-Davis, K.</creator><creator>Emerman, I.</creator><creator>Young, J.</creator><creator>Mayer-Hamblett, N.</creator><general>Elsevier B.V</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><scope>5PM</scope><orcidid>https://orcid.org/0000-0003-4431-0644</orcidid><orcidid>https://orcid.org/0000-0002-7507-9337</orcidid><orcidid>https://orcid.org/0000-0002-9779-2571</orcidid><orcidid>https://orcid.org/0000-0002-0557-7277</orcidid><orcidid>https://orcid.org/0000-0002-5727-8164</orcidid></search><sort><creationdate>20230701</creationdate><title>Willingness of people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor (ETI) to participate in randomized modulator and inhaled antimicrobial clinical trials</title><author>VanDevanter, D.R. ; Zemanick, E.T. ; Konstan, M.W. ; Ren, C.L. ; Odem-Davis, K. ; Emerman, I. ; Young, J. ; Mayer-Hamblett, N.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c3677-823ca410aedc1339c29237b2770267fab9b7eebf386a76beedde6183cbd64aa13</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2023</creationdate><topic>Aminophenols - adverse effects</topic><topic>Anti-Infective Agents</topic><topic>Benzodioxoles - adverse effects</topic><topic>Cystic Fibrosis - drug therapy</topic><topic>Cystic Fibrosis - genetics</topic><topic>Cystic Fibrosis Transmembrane Conductance Regulator - genetics</topic><topic>Humans</topic><topic>Mutation</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>VanDevanter, D.R.</creatorcontrib><creatorcontrib>Zemanick, E.T.</creatorcontrib><creatorcontrib>Konstan, M.W.</creatorcontrib><creatorcontrib>Ren, C.L.</creatorcontrib><creatorcontrib>Odem-Davis, K.</creatorcontrib><creatorcontrib>Emerman, I.</creatorcontrib><creatorcontrib>Young, J.</creatorcontrib><creatorcontrib>Mayer-Hamblett, N.</creatorcontrib><creatorcontrib>CHEC-SC Study Group</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Journal of cystic fibrosis</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>VanDevanter, D.R.</au><au>Zemanick, E.T.</au><au>Konstan, M.W.</au><au>Ren, C.L.</au><au>Odem-Davis, K.</au><au>Emerman, I.</au><au>Young, J.</au><au>Mayer-Hamblett, N.</au><aucorp>CHEC-SC Study Group</aucorp><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Willingness of people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor (ETI) to participate in randomized modulator and inhaled antimicrobial clinical trials</atitle><jtitle>Journal of cystic fibrosis</jtitle><addtitle>J Cyst Fibros</addtitle><date>2023-07-01</date><risdate>2023</risdate><volume>22</volume><issue>4</issue><spage>652</spage><epage>655</epage><pages>652-655</pages><issn>1569-1993</issn><issn>1873-5010</issn><eissn>1873-5010</eissn><abstract>•Willingness to enroll in clinical trials was assessed for those on CFTR modulators.•Willingness to enroll was associated with proposed trial length and comparator.•Shorter trials and active comparators were favored over alternatives.•Future studies in people on CFTR modulators will be feasible for some trial designs.
To assess the feasibility of enrolling people with CF (pwCF) taking the CFTR modulator elexacaftor/tezacaftor/ivacaftor (ETI) in clinical trials of a new modulator.
PwCF receiving ETI at CHEC-SC study (NCT03350828) enrollment were surveyed for interest in 2-week to 6-month placebo- (PC) and active-comparator (AC) modulator studies. Those taking inhaled antimicrobials (inhABX) were surveyed for interest in PC inhABX studies.
Of 1791 respondents, 75% [95% CI 73, 77] would enroll in a 2-week PC modulator study versus 51% [49, 54] for a 6-month study; 82% [81, 84] and 63% [61, 65] would enroll in 2-week and 6 month AC studies; 77% [74, 80] of 551 taking inhABX would enroll in a 2-week PC inhABX study versus 59% [55, 63] for a 6-month study. Previous clinical trial experience increased willingness.
Study designs will affect feasibility of future clinical trials of new modulators and inhABX in people receiving ETI.</abstract><cop>Netherlands</cop><pub>Elsevier B.V</pub><pmid>37100705</pmid><doi>10.1016/j.jcf.2023.04.007</doi><tpages>4</tpages><orcidid>https://orcid.org/0000-0003-4431-0644</orcidid><orcidid>https://orcid.org/0000-0002-7507-9337</orcidid><orcidid>https://orcid.org/0000-0002-9779-2571</orcidid><orcidid>https://orcid.org/0000-0002-0557-7277</orcidid><orcidid>https://orcid.org/0000-0002-5727-8164</orcidid><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 1569-1993 |
| ispartof | Journal of cystic fibrosis, 2023-07, Vol.22 (4), p.652-655 |
| issn | 1569-1993 1873-5010 1873-5010 |
| language | eng |
| recordid | cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_10523954 |
| source | MEDLINE; Access via ScienceDirect (Elsevier) |
| subjects | Aminophenols - adverse effects Anti-Infective Agents Benzodioxoles - adverse effects Cystic Fibrosis - drug therapy Cystic Fibrosis - genetics Cystic Fibrosis Transmembrane Conductance Regulator - genetics Humans Mutation |
| title | Willingness of people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor (ETI) to participate in randomized modulator and inhaled antimicrobial clinical trials |
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