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Poster display - Cocktail

959 - Phase 1 Investigator’s Perceptions to ‘Supersized Seamless Trials in Oncology'

Date

24 Nov 2018

Session

Poster display - Cocktail

Presenters

Brigette Ma

Citation

Annals of Oncology (2018) 29 (suppl_9): ix23-ix27. 10.1093/annonc/mdy430

Authors

B.B.Y. Ma1, N. van der Velden2, F. Mo3, H.H. Loong1, L. Siu4, B.C. Goh5, Y. Bang6, C. Lin7, J. Desai8, M.P. Lolkema2

Author affiliations

  • 1 Clinical Oncology, The Chinese University of Hong Kong, NT - Shatin/HK
  • 2 Medical Oncology, Erasmus MC Cancer Institute, 3025 - Rotterdam/NL
  • 3 Department Of Clinical Oncology, The Chinese University of Hong Kong, 000 - Hong Kong/HK
  • 4 Medical Oncology, Princess Margaret Hospital, M5G 2M9 - Toronto/CA
  • 5 Haematology-oncology, National University Health System, Singapore, 119228 - Singapore/SG
  • 6 Internal Medicine (medical Oncology), Seoul National University Hospital, 110-744 - Seoul/KR
  • 7 Department Of Oncology, National Taiwan University Hospital, 100 - Taipei city/TW
  • 8 Cancer Medicine, Peter MacCallum Cancer Centre, 3000 - Melbourne/AU
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Resources

Abstract 959

Background

The Adaptive phase (ph) 1/2 trial design with larger-sized expansion cohorts (ExC) (abbreviated, ‘SUMO’ design) has gained popularity in oncology. We examined the perceptions of ph 1 investigators toward this design.

Methods

Subjects were identified by expert nomination or via PubMed search of authors who have published > 3 ph1 or ph 1/2 cancer drug trials from 2010-17. Invitations were sent by emails containing an e-link to a survey with 4 domains: (1) ph 1 experience; (2) perception on utility, (3) safety & scientific validity, & (4) operational challenges. Responses were graded as Likert scores.

Results

534 invitations were sent & 98 responses (18.3%) were returned. Of the 80 respondents who completed all questions, 76.5% are male & 92.8% are academic physicians in university teaching hospitals from North America (31.7%), Asia (30.5%), Europe (25.6%) & Australia (12.2%). They are mainly experienced principal investigators (PI): 70% are PI of > 5 active ph 1 trials, 60% have been PI for > 10 yrs, 53% graduated > 20yrs. Over 80% felt that SUMO design can expedite drug development, but opinions are divided over if it should be preferred over other designs (46.3% Yes, vs 53.7% otherwise, o/w). When selecting ExC, 57.1% felt that strong scientific rationale based on preclinical data is the key, but 31.2% felt it was not necessary. Divergent opinions are reported over the appropriateness of waiving standard statistical methods like pre-defining type 1/2 error (45.5% OK to waive, vs 54.5% o/w), early stopping rule (52% OK to waive, vs 48% o/w) & a ‘control arm’ (26.7% OK to waive vs 73.3% o/w) in randomized cohorts. Most respondents (68%) felt that the SUMO design for first-in-human trials does not put patients at greater risk. Most agreed with increasing size & number of ExC, yet, 74.7% reported ‘some’ to ’significant’ operational challenges at their respective institutions with SUMO trials. Detailed correlations will be presented.

Conclusions

Although the SUMO design is perceived to be a safe & efficient way to develop cancer drugs by most experienced Ph 1 trialists, this survey has highlighted some pressing issues in its application: the need to optimize the scientific & statistical justification of ExCo selection, to recognize & improve the operational challenges posed by such design.

Editorial acknowledgement

Clinical trial identification

Legal entity responsible for the study

Brigette Ma.

Funding

Has not received any funding.

Disclosure

All authors have declared no conflicts of interest.

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