Anything is better than nothing’: exploring attitudes towards novel therapies in leukodystrophy clinical trials

Background/Aim Leukodystrophies comprise a group of genetic white matter disorders that lead to progressive motor and cognitive impairment. Recent development of novel therapies has led to an increase in clinical trials for leukodystrophies. To enable recruitment of individuals with a leukodystrophy into clinical trials, clinical trial acceptability should be ascertained. We sought therefore, to identify the motivations for and barriers to clinical trial participation in addition to clinical trial features that may be of concern to individuals with a leukodystrophy and/or their carers. Methods Adults with a leukodystrophy and parents/carers of individuals with a leukodystrophy were recruited through the Australian Leukodystrophy Registry and through online advertisements. Qualitative semi-structured interviews were used to explore participants views on what clinical trials involve, the perceived risks and benefits of clinical trials, their desire to participate in clinical trials and their personal experience with leukodystrophy. Thematic analysis of data was performed with co-coding of interview transcripts. Results 5 interviews were held with parents of children with leukodystrophy, 4 with parents of adults with leukodystrophy and 3 with adults diagnosed with leukodystrophy. Motivations for clinical trial enrolment include access to potentially lifesaving novel treatments and improved prognostic outcomes. Participants were concerned about adverse clinical trial outcomes, including side effects and exacerbation of illness. Despite this, majority of participants were willing to try anything in clinical trials, demonstrating a high tolerance for first in human trials and trials utilising invasive treatment options. Conclusions Interviewees communicated a strong desire to participate in interventional clinical trials involving novel therapies. To support enrolment into future leukodystrophy clinical trials we suggest the provision of transparent information regarding clinical trial treatments, consideration of alternative trial control measures, and inclusion of treating clinicians in the trial recruitment process. Clinicians play an integral role in initiating transparent conversations regarding trial risks and adverse outcomes. Ausführliche Beschreibung

Gespeichert in:

Autor*in:	Wilson, Ella [verfasserIn] Leventer, Richard [verfasserIn] Cunningham, Chloe [verfasserIn] de Silva, Michelle G. [verfasserIn] Hodgson, Jan [verfasserIn] Uebergang, Eloise [verfasserIn]

Format:	E-Artikel
Sprache:	Englisch

Erschienen:	2024

Schlagwörter:	Leukodystrophy Clinical-trials Rare disease Attitudes Novel therapies Gene therapies White matter disorders Pediatric neurology Adult neurology

Anmerkung:	© Crown 2024

Übergeordnetes Werk:	Enthalten in: Orphanet journal of rare diseases - BioMed Central, 2006, 19(2024), 1 vom: 05. Sept.
Übergeordnetes Werk:	volume:19 ; year:2024 ; number:1 ; day:05 ; month:09

Links:	Volltext

DOI / URN:	10.1186/s13023-024-03320-9

Katalog-ID:	SPR057229872

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520			\|a Background/Aim Leukodystrophies comprise a group of genetic white matter disorders that lead to progressive motor and cognitive impairment. Recent development of novel therapies has led to an increase in clinical trials for leukodystrophies. To enable recruitment of individuals with a leukodystrophy into clinical trials, clinical trial acceptability should be ascertained. We sought therefore, to identify the motivations for and barriers to clinical trial participation in addition to clinical trial features that may be of concern to individuals with a leukodystrophy and/or their carers. Methods Adults with a leukodystrophy and parents/carers of individuals with a leukodystrophy were recruited through the Australian Leukodystrophy Registry and through online advertisements. Qualitative semi-structured interviews were used to explore participants views on what clinical trials involve, the perceived risks and benefits of clinical trials, their desire to participate in clinical trials and their personal experience with leukodystrophy. Thematic analysis of data was performed with co-coding of interview transcripts. Results 5 interviews were held with parents of children with leukodystrophy, 4 with parents of adults with leukodystrophy and 3 with adults diagnosed with leukodystrophy. Motivations for clinical trial enrolment include access to potentially lifesaving novel treatments and improved prognostic outcomes. Participants were concerned about adverse clinical trial outcomes, including side effects and exacerbation of illness. Despite this, majority of participants were willing to try anything in clinical trials, demonstrating a high tolerance for first in human trials and trials utilising invasive treatment options. Conclusions Interviewees communicated a strong desire to participate in interventional clinical trials involving novel therapies. To support enrolment into future leukodystrophy clinical trials we suggest the provision of transparent information regarding clinical trial treatments, consideration of alternative trial control measures, and inclusion of treating clinicians in the trial recruitment process. Clinicians play an integral role in initiating transparent conversations regarding trial risks and adverse outcomes.
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allfields	10.1186/s13023-024-03320-9 doi (DE-627)SPR057229872 (SPR)s13023-024-03320-9-e DE-627 ger DE-627 rakwb eng 610 VZ Wilson, Ella verfasserin (orcid)0009-0009-0347-1143 aut Anything is better than nothing’: exploring attitudes towards novel therapies in leukodystrophy clinical trials 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Crown 2024 Background/Aim Leukodystrophies comprise a group of genetic white matter disorders that lead to progressive motor and cognitive impairment. Recent development of novel therapies has led to an increase in clinical trials for leukodystrophies. To enable recruitment of individuals with a leukodystrophy into clinical trials, clinical trial acceptability should be ascertained. We sought therefore, to identify the motivations for and barriers to clinical trial participation in addition to clinical trial features that may be of concern to individuals with a leukodystrophy and/or their carers. Methods Adults with a leukodystrophy and parents/carers of individuals with a leukodystrophy were recruited through the Australian Leukodystrophy Registry and through online advertisements. Qualitative semi-structured interviews were used to explore participants views on what clinical trials involve, the perceived risks and benefits of clinical trials, their desire to participate in clinical trials and their personal experience with leukodystrophy. Thematic analysis of data was performed with co-coding of interview transcripts. Results 5 interviews were held with parents of children with leukodystrophy, 4 with parents of adults with leukodystrophy and 3 with adults diagnosed with leukodystrophy. Motivations for clinical trial enrolment include access to potentially lifesaving novel treatments and improved prognostic outcomes. Participants were concerned about adverse clinical trial outcomes, including side effects and exacerbation of illness. Despite this, majority of participants were willing to try anything in clinical trials, demonstrating a high tolerance for first in human trials and trials utilising invasive treatment options. Conclusions Interviewees communicated a strong desire to participate in interventional clinical trials involving novel therapies. To support enrolment into future leukodystrophy clinical trials we suggest the provision of transparent information regarding clinical trial treatments, consideration of alternative trial control measures, and inclusion of treating clinicians in the trial recruitment process. Clinicians play an integral role in initiating transparent conversations regarding trial risks and adverse outcomes. Leukodystrophy (dpeaa)DE-He213 Clinical-trials (dpeaa)DE-He213 Rare disease (dpeaa)DE-He213 Attitudes (dpeaa)DE-He213 Novel therapies (dpeaa)DE-He213 Gene therapies (dpeaa)DE-He213 White matter disorders (dpeaa)DE-He213 Pediatric neurology (dpeaa)DE-He213 Adult neurology (dpeaa)DE-He213 Leventer, Richard verfasserin aut Cunningham, Chloe verfasserin aut de Silva, Michelle G. verfasserin aut Hodgson, Jan verfasserin aut Uebergang, Eloise verfasserin aut Enthalten in Orphanet journal of rare diseases BioMed Central, 2006 19(2024), 1 vom: 05. Sept. (DE-627)50900637X (DE-600)2225857-7 1750-1172 nnns volume:19 year:2024 number:1 day:05 month:09 https://dx.doi.org/10.1186/s13023-024-03320-9 X:SPRINGER Resolving-System kostenfrei Volltext SYSFLAG_0 GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 19 2024 1 05 09
spelling	10.1186/s13023-024-03320-9 doi (DE-627)SPR057229872 (SPR)s13023-024-03320-9-e DE-627 ger DE-627 rakwb eng 610 VZ Wilson, Ella verfasserin (orcid)0009-0009-0347-1143 aut Anything is better than nothing’: exploring attitudes towards novel therapies in leukodystrophy clinical trials 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Crown 2024 Background/Aim Leukodystrophies comprise a group of genetic white matter disorders that lead to progressive motor and cognitive impairment. Recent development of novel therapies has led to an increase in clinical trials for leukodystrophies. To enable recruitment of individuals with a leukodystrophy into clinical trials, clinical trial acceptability should be ascertained. We sought therefore, to identify the motivations for and barriers to clinical trial participation in addition to clinical trial features that may be of concern to individuals with a leukodystrophy and/or their carers. Methods Adults with a leukodystrophy and parents/carers of individuals with a leukodystrophy were recruited through the Australian Leukodystrophy Registry and through online advertisements. Qualitative semi-structured interviews were used to explore participants views on what clinical trials involve, the perceived risks and benefits of clinical trials, their desire to participate in clinical trials and their personal experience with leukodystrophy. Thematic analysis of data was performed with co-coding of interview transcripts. Results 5 interviews were held with parents of children with leukodystrophy, 4 with parents of adults with leukodystrophy and 3 with adults diagnosed with leukodystrophy. Motivations for clinical trial enrolment include access to potentially lifesaving novel treatments and improved prognostic outcomes. Participants were concerned about adverse clinical trial outcomes, including side effects and exacerbation of illness. Despite this, majority of participants were willing to try anything in clinical trials, demonstrating a high tolerance for first in human trials and trials utilising invasive treatment options. Conclusions Interviewees communicated a strong desire to participate in interventional clinical trials involving novel therapies. To support enrolment into future leukodystrophy clinical trials we suggest the provision of transparent information regarding clinical trial treatments, consideration of alternative trial control measures, and inclusion of treating clinicians in the trial recruitment process. Clinicians play an integral role in initiating transparent conversations regarding trial risks and adverse outcomes. Leukodystrophy (dpeaa)DE-He213 Clinical-trials (dpeaa)DE-He213 Rare disease (dpeaa)DE-He213 Attitudes (dpeaa)DE-He213 Novel therapies (dpeaa)DE-He213 Gene therapies (dpeaa)DE-He213 White matter disorders (dpeaa)DE-He213 Pediatric neurology (dpeaa)DE-He213 Adult neurology (dpeaa)DE-He213 Leventer, Richard verfasserin aut Cunningham, Chloe verfasserin aut de Silva, Michelle G. verfasserin aut Hodgson, Jan verfasserin aut Uebergang, Eloise verfasserin aut Enthalten in Orphanet journal of rare diseases BioMed Central, 2006 19(2024), 1 vom: 05. Sept. (DE-627)50900637X (DE-600)2225857-7 1750-1172 nnns volume:19 year:2024 number:1 day:05 month:09 https://dx.doi.org/10.1186/s13023-024-03320-9 X:SPRINGER Resolving-System kostenfrei Volltext SYSFLAG_0 GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 19 2024 1 05 09
allfields_unstemmed	10.1186/s13023-024-03320-9 doi (DE-627)SPR057229872 (SPR)s13023-024-03320-9-e DE-627 ger DE-627 rakwb eng 610 VZ Wilson, Ella verfasserin (orcid)0009-0009-0347-1143 aut Anything is better than nothing’: exploring attitudes towards novel therapies in leukodystrophy clinical trials 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Crown 2024 Background/Aim Leukodystrophies comprise a group of genetic white matter disorders that lead to progressive motor and cognitive impairment. Recent development of novel therapies has led to an increase in clinical trials for leukodystrophies. To enable recruitment of individuals with a leukodystrophy into clinical trials, clinical trial acceptability should be ascertained. We sought therefore, to identify the motivations for and barriers to clinical trial participation in addition to clinical trial features that may be of concern to individuals with a leukodystrophy and/or their carers. Methods Adults with a leukodystrophy and parents/carers of individuals with a leukodystrophy were recruited through the Australian Leukodystrophy Registry and through online advertisements. Qualitative semi-structured interviews were used to explore participants views on what clinical trials involve, the perceived risks and benefits of clinical trials, their desire to participate in clinical trials and their personal experience with leukodystrophy. Thematic analysis of data was performed with co-coding of interview transcripts. Results 5 interviews were held with parents of children with leukodystrophy, 4 with parents of adults with leukodystrophy and 3 with adults diagnosed with leukodystrophy. Motivations for clinical trial enrolment include access to potentially lifesaving novel treatments and improved prognostic outcomes. Participants were concerned about adverse clinical trial outcomes, including side effects and exacerbation of illness. Despite this, majority of participants were willing to try anything in clinical trials, demonstrating a high tolerance for first in human trials and trials utilising invasive treatment options. Conclusions Interviewees communicated a strong desire to participate in interventional clinical trials involving novel therapies. To support enrolment into future leukodystrophy clinical trials we suggest the provision of transparent information regarding clinical trial treatments, consideration of alternative trial control measures, and inclusion of treating clinicians in the trial recruitment process. Clinicians play an integral role in initiating transparent conversations regarding trial risks and adverse outcomes. Leukodystrophy (dpeaa)DE-He213 Clinical-trials (dpeaa)DE-He213 Rare disease (dpeaa)DE-He213 Attitudes (dpeaa)DE-He213 Novel therapies (dpeaa)DE-He213 Gene therapies (dpeaa)DE-He213 White matter disorders (dpeaa)DE-He213 Pediatric neurology (dpeaa)DE-He213 Adult neurology (dpeaa)DE-He213 Leventer, Richard verfasserin aut Cunningham, Chloe verfasserin aut de Silva, Michelle G. verfasserin aut Hodgson, Jan verfasserin aut Uebergang, Eloise verfasserin aut Enthalten in Orphanet journal of rare diseases BioMed Central, 2006 19(2024), 1 vom: 05. Sept. (DE-627)50900637X (DE-600)2225857-7 1750-1172 nnns volume:19 year:2024 number:1 day:05 month:09 https://dx.doi.org/10.1186/s13023-024-03320-9 X:SPRINGER Resolving-System kostenfrei Volltext SYSFLAG_0 GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 19 2024 1 05 09
allfieldsGer	10.1186/s13023-024-03320-9 doi (DE-627)SPR057229872 (SPR)s13023-024-03320-9-e DE-627 ger DE-627 rakwb eng 610 VZ Wilson, Ella verfasserin (orcid)0009-0009-0347-1143 aut Anything is better than nothing’: exploring attitudes towards novel therapies in leukodystrophy clinical trials 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Crown 2024 Background/Aim Leukodystrophies comprise a group of genetic white matter disorders that lead to progressive motor and cognitive impairment. Recent development of novel therapies has led to an increase in clinical trials for leukodystrophies. To enable recruitment of individuals with a leukodystrophy into clinical trials, clinical trial acceptability should be ascertained. We sought therefore, to identify the motivations for and barriers to clinical trial participation in addition to clinical trial features that may be of concern to individuals with a leukodystrophy and/or their carers. Methods Adults with a leukodystrophy and parents/carers of individuals with a leukodystrophy were recruited through the Australian Leukodystrophy Registry and through online advertisements. Qualitative semi-structured interviews were used to explore participants views on what clinical trials involve, the perceived risks and benefits of clinical trials, their desire to participate in clinical trials and their personal experience with leukodystrophy. Thematic analysis of data was performed with co-coding of interview transcripts. Results 5 interviews were held with parents of children with leukodystrophy, 4 with parents of adults with leukodystrophy and 3 with adults diagnosed with leukodystrophy. Motivations for clinical trial enrolment include access to potentially lifesaving novel treatments and improved prognostic outcomes. Participants were concerned about adverse clinical trial outcomes, including side effects and exacerbation of illness. Despite this, majority of participants were willing to try anything in clinical trials, demonstrating a high tolerance for first in human trials and trials utilising invasive treatment options. Conclusions Interviewees communicated a strong desire to participate in interventional clinical trials involving novel therapies. To support enrolment into future leukodystrophy clinical trials we suggest the provision of transparent information regarding clinical trial treatments, consideration of alternative trial control measures, and inclusion of treating clinicians in the trial recruitment process. Clinicians play an integral role in initiating transparent conversations regarding trial risks and adverse outcomes. Leukodystrophy (dpeaa)DE-He213 Clinical-trials (dpeaa)DE-He213 Rare disease (dpeaa)DE-He213 Attitudes (dpeaa)DE-He213 Novel therapies (dpeaa)DE-He213 Gene therapies (dpeaa)DE-He213 White matter disorders (dpeaa)DE-He213 Pediatric neurology (dpeaa)DE-He213 Adult neurology (dpeaa)DE-He213 Leventer, Richard verfasserin aut Cunningham, Chloe verfasserin aut de Silva, Michelle G. verfasserin aut Hodgson, Jan verfasserin aut Uebergang, Eloise verfasserin aut Enthalten in Orphanet journal of rare diseases BioMed Central, 2006 19(2024), 1 vom: 05. Sept. (DE-627)50900637X (DE-600)2225857-7 1750-1172 nnns volume:19 year:2024 number:1 day:05 month:09 https://dx.doi.org/10.1186/s13023-024-03320-9 X:SPRINGER Resolving-System kostenfrei Volltext SYSFLAG_0 GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 19 2024 1 05 09
allfieldsSound	10.1186/s13023-024-03320-9 doi (DE-627)SPR057229872 (SPR)s13023-024-03320-9-e DE-627 ger DE-627 rakwb eng 610 VZ Wilson, Ella verfasserin (orcid)0009-0009-0347-1143 aut Anything is better than nothing’: exploring attitudes towards novel therapies in leukodystrophy clinical trials 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Crown 2024 Background/Aim Leukodystrophies comprise a group of genetic white matter disorders that lead to progressive motor and cognitive impairment. Recent development of novel therapies has led to an increase in clinical trials for leukodystrophies. To enable recruitment of individuals with a leukodystrophy into clinical trials, clinical trial acceptability should be ascertained. We sought therefore, to identify the motivations for and barriers to clinical trial participation in addition to clinical trial features that may be of concern to individuals with a leukodystrophy and/or their carers. Methods Adults with a leukodystrophy and parents/carers of individuals with a leukodystrophy were recruited through the Australian Leukodystrophy Registry and through online advertisements. Qualitative semi-structured interviews were used to explore participants views on what clinical trials involve, the perceived risks and benefits of clinical trials, their desire to participate in clinical trials and their personal experience with leukodystrophy. Thematic analysis of data was performed with co-coding of interview transcripts. Results 5 interviews were held with parents of children with leukodystrophy, 4 with parents of adults with leukodystrophy and 3 with adults diagnosed with leukodystrophy. Motivations for clinical trial enrolment include access to potentially lifesaving novel treatments and improved prognostic outcomes. Participants were concerned about adverse clinical trial outcomes, including side effects and exacerbation of illness. Despite this, majority of participants were willing to try anything in clinical trials, demonstrating a high tolerance for first in human trials and trials utilising invasive treatment options. Conclusions Interviewees communicated a strong desire to participate in interventional clinical trials involving novel therapies. To support enrolment into future leukodystrophy clinical trials we suggest the provision of transparent information regarding clinical trial treatments, consideration of alternative trial control measures, and inclusion of treating clinicians in the trial recruitment process. Clinicians play an integral role in initiating transparent conversations regarding trial risks and adverse outcomes. Leukodystrophy (dpeaa)DE-He213 Clinical-trials (dpeaa)DE-He213 Rare disease (dpeaa)DE-He213 Attitudes (dpeaa)DE-He213 Novel therapies (dpeaa)DE-He213 Gene therapies (dpeaa)DE-He213 White matter disorders (dpeaa)DE-He213 Pediatric neurology (dpeaa)DE-He213 Adult neurology (dpeaa)DE-He213 Leventer, Richard verfasserin aut Cunningham, Chloe verfasserin aut de Silva, Michelle G. verfasserin aut Hodgson, Jan verfasserin aut Uebergang, Eloise verfasserin aut Enthalten in Orphanet journal of rare diseases BioMed Central, 2006 19(2024), 1 vom: 05. Sept. (DE-627)50900637X (DE-600)2225857-7 1750-1172 nnns volume:19 year:2024 number:1 day:05 month:09 https://dx.doi.org/10.1186/s13023-024-03320-9 X:SPRINGER Resolving-System kostenfrei Volltext SYSFLAG_0 GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 19 2024 1 05 09
language	English
source	Enthalten in Orphanet journal of rare diseases 19(2024), 1 vom: 05. Sept. volume:19 year:2024 number:1 day:05 month:09
sourceStr	Enthalten in Orphanet journal of rare diseases 19(2024), 1 vom: 05. Sept. volume:19 year:2024 number:1 day:05 month:09
format_phy_str_mv	Article
institution	findex.gbv.de
topic_facet	Leukodystrophy Clinical-trials Rare disease Attitudes Novel therapies Gene therapies White matter disorders Pediatric neurology Adult neurology
dewey-raw	610
isfreeaccess_bool	true
container_title	Orphanet journal of rare diseases
authorswithroles_txt_mv	Wilson, Ella @@aut@@ Leventer, Richard @@aut@@ Cunningham, Chloe @@aut@@ de Silva, Michelle G. @@aut@@ Hodgson, Jan @@aut@@ Uebergang, Eloise @@aut@@
publishDateDaySort_date	2024-09-05T00:00:00Z
hierarchy_top_id	50900637X
dewey-sort	3610
id	SPR057229872
language_de	englisch
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author	Wilson, Ella
spellingShingle	Wilson, Ella ddc 610 misc Leukodystrophy misc Clinical-trials misc Rare disease misc Attitudes misc Novel therapies misc Gene therapies misc White matter disorders misc Pediatric neurology misc Adult neurology Anything is better than nothing’: exploring attitudes towards novel therapies in leukodystrophy clinical trials
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topic_title	610 VZ Anything is better than nothing’: exploring attitudes towards novel therapies in leukodystrophy clinical trials Leukodystrophy (dpeaa)DE-He213 Clinical-trials (dpeaa)DE-He213 Rare disease (dpeaa)DE-He213 Attitudes (dpeaa)DE-He213 Novel therapies (dpeaa)DE-He213 Gene therapies (dpeaa)DE-He213 White matter disorders (dpeaa)DE-He213 Pediatric neurology (dpeaa)DE-He213 Adult neurology (dpeaa)DE-He213
topic	ddc 610 misc Leukodystrophy misc Clinical-trials misc Rare disease misc Attitudes misc Novel therapies misc Gene therapies misc White matter disorders misc Pediatric neurology misc Adult neurology
topic_unstemmed	ddc 610 misc Leukodystrophy misc Clinical-trials misc Rare disease misc Attitudes misc Novel therapies misc Gene therapies misc White matter disorders misc Pediatric neurology misc Adult neurology
topic_browse	ddc 610 misc Leukodystrophy misc Clinical-trials misc Rare disease misc Attitudes misc Novel therapies misc Gene therapies misc White matter disorders misc Pediatric neurology misc Adult neurology
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title	Anything is better than nothing’: exploring attitudes towards novel therapies in leukodystrophy clinical trials
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title_full	Anything is better than nothing’: exploring attitudes towards novel therapies in leukodystrophy clinical trials
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author_browse	Wilson, Ella Leventer, Richard Cunningham, Chloe de Silva, Michelle G. Hodgson, Jan Uebergang, Eloise
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title_sort	anything is better than nothing’: exploring attitudes towards novel therapies in leukodystrophy clinical trials
title_auth	Anything is better than nothing’: exploring attitudes towards novel therapies in leukodystrophy clinical trials
abstract	Background/Aim Leukodystrophies comprise a group of genetic white matter disorders that lead to progressive motor and cognitive impairment. Recent development of novel therapies has led to an increase in clinical trials for leukodystrophies. To enable recruitment of individuals with a leukodystrophy into clinical trials, clinical trial acceptability should be ascertained. We sought therefore, to identify the motivations for and barriers to clinical trial participation in addition to clinical trial features that may be of concern to individuals with a leukodystrophy and/or their carers. Methods Adults with a leukodystrophy and parents/carers of individuals with a leukodystrophy were recruited through the Australian Leukodystrophy Registry and through online advertisements. Qualitative semi-structured interviews were used to explore participants views on what clinical trials involve, the perceived risks and benefits of clinical trials, their desire to participate in clinical trials and their personal experience with leukodystrophy. Thematic analysis of data was performed with co-coding of interview transcripts. Results 5 interviews were held with parents of children with leukodystrophy, 4 with parents of adults with leukodystrophy and 3 with adults diagnosed with leukodystrophy. Motivations for clinical trial enrolment include access to potentially lifesaving novel treatments and improved prognostic outcomes. Participants were concerned about adverse clinical trial outcomes, including side effects and exacerbation of illness. Despite this, majority of participants were willing to try anything in clinical trials, demonstrating a high tolerance for first in human trials and trials utilising invasive treatment options. Conclusions Interviewees communicated a strong desire to participate in interventional clinical trials involving novel therapies. To support enrolment into future leukodystrophy clinical trials we suggest the provision of transparent information regarding clinical trial treatments, consideration of alternative trial control measures, and inclusion of treating clinicians in the trial recruitment process. Clinicians play an integral role in initiating transparent conversations regarding trial risks and adverse outcomes. © Crown 2024
abstractGer	Background/Aim Leukodystrophies comprise a group of genetic white matter disorders that lead to progressive motor and cognitive impairment. Recent development of novel therapies has led to an increase in clinical trials for leukodystrophies. To enable recruitment of individuals with a leukodystrophy into clinical trials, clinical trial acceptability should be ascertained. We sought therefore, to identify the motivations for and barriers to clinical trial participation in addition to clinical trial features that may be of concern to individuals with a leukodystrophy and/or their carers. Methods Adults with a leukodystrophy and parents/carers of individuals with a leukodystrophy were recruited through the Australian Leukodystrophy Registry and through online advertisements. Qualitative semi-structured interviews were used to explore participants views on what clinical trials involve, the perceived risks and benefits of clinical trials, their desire to participate in clinical trials and their personal experience with leukodystrophy. Thematic analysis of data was performed with co-coding of interview transcripts. Results 5 interviews were held with parents of children with leukodystrophy, 4 with parents of adults with leukodystrophy and 3 with adults diagnosed with leukodystrophy. Motivations for clinical trial enrolment include access to potentially lifesaving novel treatments and improved prognostic outcomes. Participants were concerned about adverse clinical trial outcomes, including side effects and exacerbation of illness. Despite this, majority of participants were willing to try anything in clinical trials, demonstrating a high tolerance for first in human trials and trials utilising invasive treatment options. Conclusions Interviewees communicated a strong desire to participate in interventional clinical trials involving novel therapies. To support enrolment into future leukodystrophy clinical trials we suggest the provision of transparent information regarding clinical trial treatments, consideration of alternative trial control measures, and inclusion of treating clinicians in the trial recruitment process. Clinicians play an integral role in initiating transparent conversations regarding trial risks and adverse outcomes. © Crown 2024
abstract_unstemmed	Background/Aim Leukodystrophies comprise a group of genetic white matter disorders that lead to progressive motor and cognitive impairment. Recent development of novel therapies has led to an increase in clinical trials for leukodystrophies. To enable recruitment of individuals with a leukodystrophy into clinical trials, clinical trial acceptability should be ascertained. We sought therefore, to identify the motivations for and barriers to clinical trial participation in addition to clinical trial features that may be of concern to individuals with a leukodystrophy and/or their carers. Methods Adults with a leukodystrophy and parents/carers of individuals with a leukodystrophy were recruited through the Australian Leukodystrophy Registry and through online advertisements. Qualitative semi-structured interviews were used to explore participants views on what clinical trials involve, the perceived risks and benefits of clinical trials, their desire to participate in clinical trials and their personal experience with leukodystrophy. Thematic analysis of data was performed with co-coding of interview transcripts. Results 5 interviews were held with parents of children with leukodystrophy, 4 with parents of adults with leukodystrophy and 3 with adults diagnosed with leukodystrophy. Motivations for clinical trial enrolment include access to potentially lifesaving novel treatments and improved prognostic outcomes. Participants were concerned about adverse clinical trial outcomes, including side effects and exacerbation of illness. Despite this, majority of participants were willing to try anything in clinical trials, demonstrating a high tolerance for first in human trials and trials utilising invasive treatment options. Conclusions Interviewees communicated a strong desire to participate in interventional clinical trials involving novel therapies. To support enrolment into future leukodystrophy clinical trials we suggest the provision of transparent information regarding clinical trial treatments, consideration of alternative trial control measures, and inclusion of treating clinicians in the trial recruitment process. Clinicians play an integral role in initiating transparent conversations regarding trial risks and adverse outcomes. © Crown 2024
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title_short	Anything is better than nothing’: exploring attitudes towards novel therapies in leukodystrophy clinical trials
url	https://dx.doi.org/10.1186/s13023-024-03320-9
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author2	Leventer, Richard Cunningham, Chloe de Silva, Michelle G. Hodgson, Jan Uebergang, Eloise
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