Anything is better than nothing’: exploring attitudes towards novel therapies in leukodystrophy clinical trials
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.
Authors
- M. De Silva (ORCID: https://orcid.org/0000-0002-2304-3834)
- E. Wilson (ORCID: https://orcid.org/0009-0009-0347-1143)
- Jan Hodgson (ORCID: https://orcid.org/0000-0001-7821-9617)
- Richard Jacob Leventer (ORCID: https://orcid.org/0000-0003-0362-5607)
- Eloise Uebergang (ORCID: https://orcid.org/0000-0003-0305-7822)
- C.A. Cunningham
Institutions
- Royal Children's Hospital (AU)
- The University of Melbourne (AU)
- Victorian Clinical Genetics Services (AU)
- Murdoch Children's Research Institute (AU)
- Melbourne Genomics Health Alliance (AU)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2024-09-05
- DOI
- https://doi.org/10.1186/s13023-024-03320-9
- Citations
- 1
- Primary Topic
- RNA regulation and disease
- Type
- article
- Field-Weighted Citation Impact
- 0.23