151TiP TANGENT: A phase III, global, randomized, double-blind, placebo-controlled trial to assess the efficacy and safety of emactuzumab in patients with tenosynovial giant cell tumor

ESMO Open, vol. 10, pp. 104464

Abstract

follow up, n¼6, 46% experienced rash; n¼6, 46% diarrhea; n¼3, 23% required phosphate supplement.Delayed AEs included a case of hidradenitis.Among the 4 female pts, 3 (75%) experienced ovarian toxicity.The 3 pts who underwent dose reductions did so due to rash, diarrhea and fatigue; and diarrhea, respectively.All pts tolerated the reduced dose of 100 mg BID.At the most recent follow-up, 8 pts (62%) had reported improved pain from baseline. 2 out of 5 pts (40%) with intrabdominal tumors had RECIST 1.1 partial response.Conclusions: Nirogacestat was well tolerated, with management of AEs at first month associated with dose interruptions but infrequent dose reductions.Our findings support the recommended starting dose of 150 mg twice daily (BID) in real-world experience.

Authors 9

1. Emanuela Palmerini

   Istituto Ortopedico Rizzoli

   Affiliation as printed

   DIMES - Experimental, Diagnostic and Specialty Medicine Dept, Istituto Ortopedico Rizzoli, Bologna, Italy

2. Suzanne H. C. Veltkamp
   Affiliation as printed

   Oncology Dept., SynOx Therapeutics UK Limited, Oxford, United Kingdom

3. H. Gelderblom Aachen Medical Oncology Department

   Leiden University Medical Center

   Affiliation as printed

   Medical Oncology Dept., LUMC - Leids Universitair Medisch Centrum, Leiden, Netherlands

4. Javier Martín‐Broto

   Hospital Universitario Fundación Jiménez Díaz

   Affiliation as printed

   Medical Oncology Dept., Hospital Universitario Fundacion Jimenez Diaz, Madrid, Spain

5. A. Sebio García

   Hospital de Sant Pau

   Affiliation as printed

   Medical Oncology, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain

6. Gabriel Tinoco

   The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute

   Affiliation as printed

   Medical Oncology, OSUCCC - The Ohio State University Comprehensive Cancer Center - James, Columbus, United States of America

7. Elyse Seltzer
   Affiliation as printed

   Oncology Dept., SynOx Therapeutics UK Limited, Oxford, United Kingdom

8. Matthew A. Davies
   Affiliation as printed

   Clinical Operations, SynOx Therapeutics UK Limited, Oxford, United Kingdom

9. Jean‐Yves Blay

   Centre Léon Bérard

   Affiliation as printed

   Medicine Department, Centre Léon Bérard, Lyon, France