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A phase II trial of regorafenib (REGO) in patients (pts) with advanced Ewing sarcoma and related tumors (EWS) of soft tissue and bone: SARC024 trial results.

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Attia, S; Bolejack, V; Ganjoo, KN; George, S; Agulnik, M; Rushing, DA; Loggers, ET; Livingston, MB; Wright, JA; Chawla, SP; Okuno, SH ...
Published in: Journal of Clinical Oncology
May 20, 2017

11005 Background: Pazopanib is approved for soft tissue sarcoma pts after failure of other therapy, but there are few subtype-specific data regarding kinase inhibitor activity. We report on a single arm, phase II trial of REGO in advanced EWS. Methods: EWS pts (age > 18, ECOG 0-2, good organ function) who had at least 1 line of therapy and had PD within 6 mo were eligible. Prior oral kinase inhibitors were not allowed. Initial REGO dose was 160 mg PO QD x21 q28d. Dose reductions were employed for toxicity and AEs. The primary endpoint was PFS at 8 weeks (PFS8w) employing RECIST 1.1. Sample size of 30 allowed determination of the difference between PFS8w of 50% vs 25% with alpha = 0.05 and power of 91%. Results: 30 pts (median age 32, range 19-65; M/F = 20/10; ECOG 0/1/2 = 16/13/1; bone, 12; soft tissue, 18; median prior treatments 5, range 1-10) enrolled at 14 US sites (09/2014-03/2016). Most common grade (G3) toxicities were hypophosphatemia (6), hypertension (2), high ALT (2) and 1 each: fatigue, abd pain, diarrhea, hypokalemia, oral mucositis, neutropenia and rash; no G4 toxicities were noted. 13 pts required ≥1 dose reduction, most commonly hypophosphatemia (n = 7); 2 stopped REGO for toxicity. There was 1 death in the 30 day post study period, not REGO related. Median dose at study end: 140 mg (3.5 tabs, range 80-160 mg) 3 wks on/1wk off. 18/30 pts were without PD at 8 wks. Median PFS: 3.6 mo (95%CI 2.8-3.8 mo). PFS8w by KM was 73% (95%CI 57-89%). Best responses: PR/SD/PD/not evaluable of 3/18/7/2, for RECIST RR 10%. Two pts with PR had EWSR1 translocation by FISH; a third had CIC-DUX4. Median duration of response: 5.5 mo (95%CI 2.9-8.0). Median OS is not reached. Conclusions: The substudy met its primary endpoint. REGO toxicity was similar to that seen previously. Enrollment continues in LPS and OGS cohorts, and is being expanded to further study variant EWS without EWSR1-FLI1 fusion. Study of the existing tissue may elucidate which EWS patients may benefit from REGO. Clinical trial information: NCT02048371.

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Published In

Journal of Clinical Oncology

DOI

EISSN

1527-7755

ISSN

0732-183X

Publication Date

May 20, 2017

Volume

35

Issue

15_suppl

Start / End Page

11005 / 11005

Publisher

American Society of Clinical Oncology (ASCO)

Related Subject Headings

  • Oncology & Carcinogenesis
  • 3211 Oncology and carcinogenesis
  • 1112 Oncology and Carcinogenesis
  • 1103 Clinical Sciences
 

Citation

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Attia, S., Bolejack, V., Ganjoo, K. N., George, S., Agulnik, M., Rushing, D. A., … Maki, R. G. (2017). A phase II trial of regorafenib (REGO) in patients (pts) with advanced Ewing sarcoma and related tumors (EWS) of soft tissue and bone: SARC024 trial results. In Journal of Clinical Oncology (Vol. 35, pp. 11005–11005). American Society of Clinical Oncology (ASCO). https://doi.org/10.1200/jco.2017.35.15_suppl.11005
Attia, Steven, Vanessa Bolejack, Kristen N. Ganjoo, Suzanne George, Mark Agulnik, Daniel A. Rushing, Elizabeth Trice Loggers, et al. “A phase II trial of regorafenib (REGO) in patients (pts) with advanced Ewing sarcoma and related tumors (EWS) of soft tissue and bone: SARC024 trial results.” In Journal of Clinical Oncology, 35:11005–11005. American Society of Clinical Oncology (ASCO), 2017. https://doi.org/10.1200/jco.2017.35.15_suppl.11005.
Attia S, Bolejack V, Ganjoo KN, George S, Agulnik M, Rushing DA, et al. A phase II trial of regorafenib (REGO) in patients (pts) with advanced Ewing sarcoma and related tumors (EWS) of soft tissue and bone: SARC024 trial results. In: Journal of Clinical Oncology. American Society of Clinical Oncology (ASCO); 2017. p. 11005–11005.
Attia, Steven, et al. “A phase II trial of regorafenib (REGO) in patients (pts) with advanced Ewing sarcoma and related tumors (EWS) of soft tissue and bone: SARC024 trial results.Journal of Clinical Oncology, vol. 35, no. 15_suppl, American Society of Clinical Oncology (ASCO), 2017, pp. 11005–11005. Crossref, doi:10.1200/jco.2017.35.15_suppl.11005.
Attia S, Bolejack V, Ganjoo KN, George S, Agulnik M, Rushing DA, Loggers ET, Livingston MB, Wright JA, Chawla SP, Okuno SH, Reinke DK, Riedel RF, Davis LE, Ryan CW, Maki RG. A phase II trial of regorafenib (REGO) in patients (pts) with advanced Ewing sarcoma and related tumors (EWS) of soft tissue and bone: SARC024 trial results. Journal of Clinical Oncology. American Society of Clinical Oncology (ASCO); 2017. p. 11005–11005.

Published In

Journal of Clinical Oncology

DOI

EISSN

1527-7755

ISSN

0732-183X

Publication Date

May 20, 2017

Volume

35

Issue

15_suppl

Start / End Page

11005 / 11005

Publisher

American Society of Clinical Oncology (ASCO)

Related Subject Headings

  • Oncology & Carcinogenesis
  • 3211 Oncology and carcinogenesis
  • 1112 Oncology and Carcinogenesis
  • 1103 Clinical Sciences