Collaboration for Alzheimer s Prevention Data and sample sharing in preclinical AD trials

Transcription

1 Collaboration for Alzheimer s Prevention Data and sample sharing in preclinical AD trials Stacie Weninger, Maria C. Carrillo, Billy Dunn, Paul S. Aisen, Randall J. Bateman, Joanne D. Kotz, Jessica B. Langbaum, Eric McDade, Susan L. Mills, Eric M. Reiman, Reisa Sperling, Anna M. Santacruz, Pierre N. Tariot, Kathleen A. Welsh-Bohmer CAMD 2017

2 Mission of CAP CAP was established in 2011 Initially included 3 academic-led prevention trials: A4, DIAN-TU and API; expanded to include TOMMORROW trial, with plans to continue to expand to include other prevention trials (requires a commitment to data sharing) Facilitated by FBRI, AA, NIA, and the FDA To date has been a forum for academic trial investigators to discuss key issues as these groups plan and implement preclinical AD treatment trials

3 CAP Discussions Include: What are the most informative aspects of the various trial designs, and how do we best harmonize fluid and imaging biomarkers and cognitive endpoints? How do we maximize access to data and samples from trials, and how do we ensure that lessons learned are generalized as quickly as possible to inform the field? For 5 years, met regularly to address these and other topics

4 The urgency Sharing data and biological samples from preclinical AD trials as soon as possible is critical to ensure that knowledge gained through individual trials will enable progress of the field as a whole. Data and samples from preclinical AD trials will help to: inform our understanding of the natural history of AD inform the size and design of future trials clarify the utility of biomarker and cognitive measurements accelerate the evaluation of preclinical treatments for AD

5 Potential constraints We recognize that there may be constraints on data and sample sharing: maintaining the scientific integrity of the trial, including preservation of blinding, is essential sharing must not compromise the ability of the study to withstand independent scientific scrutiny, including regulatory review maintaining the confidentiality of participants in these trials is of the utmost importance and may pose a particular challenge in certain populations such as those at risk for carrying a dominant mutation causing familial early-onset AD

6 A proposed framework

7 Facilitating data sharing as early as possible Where possible, standardized data acquisition techniques and assessments should be included to enhance the ability to compare data between trials. Measurement of multiple potential biomarkers should be included in trial designs to facilitate the identification of biomarkers of disease evolution and treatment response that could be used in future trials. Screening and prerandomization baseline data should be made available to the scientific community within 12 months of enrollment completion. All study data should be made available to the scientific community after the earlier of either regulatory approval of the tested treatment or 18 months after the completion or early termination of the trial.

8 Sharing biological samples Sample sharing is particularly important, as analyzing samples using new technologies or based on other new developments may offer an opportunity for a breakthrough in target or biomarker identification. Sponsors should ensure that the informed consent process clearly describes the intent to share samples and should also provide for the long-term storage of samples. The first priority for sample use is proper conduct of the study, which includes appropriate retention of samples in sufficient quantities for analyses during ongoing trials as well as for confirmatory testing after trial completion. Remaining study samples should be made available to the scientific community at the time that the associated data are released.

9 Agreement to CAP principles For the [Study Name], we agree to adhere to CAP principles for data and sample sharing. Sponsors and companies that have agreed to this statement: Lilly: DIAN-TU, A4 Roche/Genetech: DIAN, API ADAD Novartis: API Generation Amgen: API Generation Takeda: TOMMORROW 9

10 Sharing for Prerandomization Data Trial Public Presentation Prerandomization Data Prerandomization Data Sharing Analyses and sharing with collaborators General availability (through approved data request) A4 Already occurring will continue at CTAD 2016 As soon as enrollment is complete and data are QC'd Within 1 year after enrollment is complete EARLY Agree in principle Agree in principle Agree in principle A3 Will seek agreement to adhere to principles Will seek agreement to adhere to principles Will seek agreement to adhere to principles TOMMORROW Already occurring- Presented screening data at AAIC 2016 will continue with presentation at CTAD 2016 principles principles

11 Trial DIAN-TU Sola Gant NexGen Sharing for Prerandomization Data Public Presentation Prerandomization Data Ongoing AMP meetings, AAIC 2016 (CAP FRS), AAIC 2017 ongoing AMP meetings, AAIC 2016 (CAP FRS), AAIC 2017 Will seek agreement to adhere to principles Analyses and sharing with collaborators 1Q2017 (after QC is complete) 1Q2017 (after QC is complete) Will seek agreement to adhere to principles Prerandomization Data Sharing General availability (through approved data request) 2Q2017 2Q2017 Will seek agreement to adhere to principles API ADAD Colombia principles principles (~1Q2018) principles (~1Q2018) API Generation (APOE4 HM) Study Ongoing for genetic testing & disclosure data; agree to adhere to principles for other data principles (~1Q2019) principles (~1Q2019)

12 Trial Sharing of Data After Trial Completion Analyses and sharing with collaborators Data After Trial Completion General availability (through approved data request) A4 Once double blind is completed; may depend on extension trial plans Current language is "once regulatory process is complete, will depend on plans for extension trial and accelerated approval mechanism as we will maintain the blind but working on potential solutions with triple blinding data to make double blind data available as soon as possible EARLY Agree in principle Agree in principle A3 Will seek agreement to adhere to principles Will seek agreement to adhere to principles TOMMORROW principles principles

13 DIAN-TU Sola Sharing of Data After Trial Completion Trial Gant Analyses and sharing with collaborators (timing may change w/ open label extension) (timing may change w/ open label extension) Data After Trial Completion General availability (through approved data request) (timing may change w/ open label extension) (timing may change w/ open label extension) NexGen principles principles API ADAD Colombia API Generation (APOE4 HM) Study principles (~2023+) principles (~2024+) Data will be shared following the earlier of (a) approval by the FDA for commercialization of the Study Drug for any indication or (b) 18 months following completion or early termination of the Study. Data will be shared following the earlier of: (a) a Joint Publication, (b) the FDA has approved for commercialization the Study Drug to which the proposed presentation or publication relates, or (c) eighteen (18) months have elapsed following the completion or early termination of the Study.

14 SUMMARY We call on sponsors and investigators of all Alzheimer s trials to adopt these guidelines and implement them into ongoing and planned trials. CAP welcomes new partners who agree to CAP sharing principles. Annual forum at AAIC for all who have signed onto these principles.