Tenosynovial giant cell tumor - null regimens

The purpose of this page is to provide references to "null therapy" such as placebo and observation. These references provide further insight into the historical development of the treatment landscape. See the main TGCT page for regimens that include active anticancer treatment.

All lines of therapy

Placebo

Regimen

Study	Dates of enrollment	Evidence	Comparator	Comparative Efficacy
Tap et al. 2019 (ENLIVEN)	2015-05 to 2016-09	Phase 3 (C)	Pexidartinib	Inferior ORR (primary endpoint)
Gelderblom et al. 2024 (MOTION)	2022-01-21 to 2023-02-21	Phase 3 (C)	Vimseltinib	Inferior ORR (primary endpoint)

No active antineoplastic treatment.

References

1. ENLIVEN: Tap WD, Gelderblom H, Palmerini E, Desai J, Bauer S, Blay JY, Alcindor T, Ganjoo K, Martín-Broto J, Ryan CW, Thomas DM, Peterfy C, Healey JH, van de Sande M, Gelhorn HL, Shuster DE, Wang Q, Yver A, Hsu HH, Lin PS, Tong-Starksen S, Stacchiotti S, Wagner AJ; ENLIVEN investigators. Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial. Lancet. 2019 Aug 10;394(10197):478-487. Epub 2019 Jun 19. link to original article link to PMC article PubMed NCT02371369
2. MOTION: Gelderblom H, Bhadri V, Stacchiotti S, Bauer S, Wagner AJ, van de Sande M, Bernthal NM, López Pousa A, Razak AA, Italiano A, Ahmed M, Le Cesne A, Tinoco G, Boye K, Martín-Broto J, Palmerini E, Tafuto S, Pratap S, Powers BC, Reichardt P, Casado Herráez A, Rutkowski P, Tait C, Zarins F, Harrow B, Sharma MG, Ruiz-Soto R, Sherman ML, Blay JY, Tap WD; MOTION investigators. Vimseltinib versus placebo for tenosynovial giant cell tumour (MOTION): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2024 Jun 22;403(10445):2709-2719. Epub 2024 Jun 3. link to original article PubMed NCT05059262