Sarcoma clinical trials
50 Sarcoma trials (Medical Oncology and Radiation Oncology) summarized for fellows and board review, newest first within each disease. Each summary covers design, results, toxicity and what changed in practice.
Jump to: Sarcoma · Soft tissue sarcoma · Benign disease RT · Pediatric — Ewing · Pediatric — RMS · Sarcoma - osteosarcoma · GIST - metastatic · Sarcoma - TGCT · Sarcoma - desmoid · Sarcoma - Ewing
Sarcoma (20)
- Temozolomide in Advanced Leiomyosarcoma, Immunotherapy Sensitization Anticancer Drugs 2026TEM-based regimens effective in advanced LMS. Post-TEM biopsies revealed dynamic changes in mismatch repair gene expression, tertiary lymphoid structures, and...
- LMS04 (LMS-04) NEJM 2024Doxorubicin + trabectedin followed by trabectedin maintenance significantly improved both PFS (12 vs 6 months, HR 0.37) and OS (33 vs 24 months, HR 0.65) compared...
- SPEARHEAD-1 Lancet 2024Afami-cel achieved a 37% ORR in heavily pre-treated advanced synovial sarcoma and MRCL with durable responses at 32.6-mo follow-up and a manageable safety profile...
- SU2C-SARC032 Lancet 2024Perioperative pembrolizumab added to preoperative RT significantly improved disease-free survival in UPS and dedifferentiated/pleomorphic liposarcoma of the...
- AcSé Pembrolizumab (Sarcoma) Lancet Oncol 2023Pembrolizumab monotherapy showed meaningful activity only in chordoma (ORR 15%) among 10 histotypes; ASPS activity (20%) limited by small cohort. Most sarcoma...
- AMPECT JCO 2021Nab-sirolimus achieved 39% ORR with 77% DCR and durable responses in malignant PEComa (mPFS 8.7 mo). Highest ORR (50%) seen in TSC1/TSC2-mutated patients, consistent...
- ANNOUNCE JAMA 2020Olaratumab added to doxorubicin did not improve OS compared with doxorubicin + placebo in advanced STS - neither in the unselected population nor in the LMS subgroup....
- STRASS (EORTC-62092) Lancet Oncol 2020Preoperative radiotherapy before surgery for retroperitoneal sarcoma did not improve abdominal recurrence-free survival compared with surgery alone and was associated...
- Tazemetostat (Epithelioid Sarcoma) Lancet Oncol 2020Tazemetostat achieved 15% ORR with 67% DCR in heavily pretreated INI1-negative epithelioid sarcoma, with durable responses and median OS over 12 months. The oral...
- GeDDiS trial, Doxorubicin vs gem/docetaxel in soft-tissue sarcoma 2017Single-agent doxorubicin and gemcitabine/docetaxel produced similar progression-free and overall survival in first-line soft-tissue sarcoma; doxorubicin should remain...
- ISG-STS 1001 Lancet Oncol 2017Histotype-tailored neoadjuvant chemotherapy did not improve DFS compared with standard epirubicin + ifosfamide and was associated with inferior DFS outcomes, leading...
- SARC021 (TH CR-406) Lancet Oncol 2017Adding evofosfamide to doxorubicin did not improve OS or PFS versus doxorubicin alone in advanced STS, despite higher response rates and toxicity. Hypoxia-activation...
- Eribulin vs dacarbazine liposarcoma 2016Eribulin significantly improved overall survival versus dacarbazine in advanced liposarcoma/leiomyosarcoma after anthracycline failure, with the benefit driven...
- Trabectedin vs Dacarbazine in liposarcoma 2016Trabectedin significantly improved progression-free survival and disease control over dacarbazine in advanced liposarcoma or leiomyosarcoma after anthracycline...
- EORTC 62012 Lancet Oncol 2014Intensified doxorubicin + ifosfamide significantly improved ORR and PFS compared with doxorubicin alone but did not improve overall survival (P=.076). This landmark...
- EORTC 62931 Lancet Oncol 2012Adjuvant chemotherapy with doxorubicin and ifosfamide showed no benefit in relapse-free survival or overall survival after resection of high-risk soft-tissue sarcoma....
- PALETTE Lancet 2012Pazopanib significantly improved PFS (HR 0.31, P<.0001) compared with placebo in pre-treated advanced STS, with a 3-month absolute PFS gain, but did not significantly...
- Paclitaxel vs peg-Doxorubicin in in Kaposi sarcoma 2010Paclitaxel and pegylated liposomal doxorubicin produced similar response, survival, and significant improvements in pain and swelling in advanced symptomatic...
- RTOG 9514 J Clin Oncol 2006The neoadjuvant MAID + interdigitated RT regimen was deliverable in a multi-institutional setting with efficacy consistent with prior single-institution experience....
- Peg-doxorubicin vs ABV in Kaposi sarcoma 1998Pegylated liposomal doxorubicin produced higher response rates with comparable or lower toxicity and better treatment completion than ABV combination chemotherapy for...
Soft tissue sarcoma (8)
- Brightline-1 trial Clin Cancer Res 2026Brigimadlin showed antitumor activity with a higher response rate but did not improve PFS over doxorubicin in first-line MDM2-amplified DDLPS; the doxorubicin control...
- Expert Consensus: Pre-Op RT for Retroperitoneal Sarcoma (Baldini) Int J Radiat Oncol Biol Phys 2015International consensus supports pre-op RT for resectable RPS, using IMRT to achieve 50.4 Gy with strict bowel constraints. This provides better bowel displacement...
- IMRT vs Conventional EBRT for STS (Folkert MSKCC) JCO 2014IMRT is associated with significantly reduced local recurrence compared to conventional EBRT for primary extremity STS, even after adjusting for the higher proportion...
- Review: RT for Extremity Soft Tissue Sarcoma (Haas) Int J Radiat Oncol Biol Phys 2012RT is standard of care for most extremity STS treated with limb-sparing surgery. Pre-op RT is increasingly preferred for functional advantages and lower total dose...
- RTOG GTV/CTV Contouring Consensus for STS (Wang) Int J Radiat Oncol Biol Phys 2011A standardized RTOG atlas for GTV/CTV delineation in preoperative RT for extremity STS was developed, with 3 cm longitudinal and 1.5 cm radial CTV margins from GTV as...
- Pre-Op vs Post-Op RT in Extremity STS (O'Sullivan) Lancet 2002Preoperative RT results in significantly more wound complications than postoperative RT. However, late toxicity is greater with post-op RT (larger field, higher...
- Adjuvant RT in Extremity STS (Yang NCI) JCO 1998Adjuvant EBRT significantly improves local control in both high- and low-grade extremity STS after limb-sparing surgery, but without OS benefit. Establishes RT as...
- Limb-Sparing Surgery vs Amputation in STS (Rosenberg) Ann Surg 1982Limb-sparing surgery plus adjuvant RT achieves equivalent overall survival and disease-free survival to amputation in high-grade extremity STS, establishing limb...
Benign disease RT (6)
- Dupuytren's RT RCT Int J Radiat Oncol Biol Phys 2001RT significantly reduces disease progression in early-stage Dupuytren's compared to observation. At 13 months, 81% of RT patients had stable disease vs only 23% in...
- HO RT Prophylaxis Meta-Analysis IJROBP 2004Single-fraction RT (7-8 Gy) and NSAIDs (indomethacin) are equally effective in preventing HO after THA. Both significantly reduce severe HO (Brooker III-IV) compared...
- Dupuytren's RT Long-Term Strahlenther Onkol 2001RT provides durable long-term disease stabilization for early-stage Dupuytren's contracture with minimal late toxicity at 10 years. Optimal results in Tubiana stage N...
- HO RT Prophylaxis Multicenter IJROBP 2001Single-fraction 7 Gy is equivalent to 12 Gy/4 fractions for HO prophylaxis after THA. Single-fraction is preferred for patient convenience and equivalent efficacy. RT...
- Peyronie's Disease RT IJROBP 2006RT at 36 Gy for Peyronie's disease effectively relieves penile pain and stabilizes plaque size in the majority of patients with tolerable acute toxicity. RT does not...
- Keilholz Dupuytren's RT IJROBP 1996Low-dose RT (30 Gy in 2 courses) effectively stabilizes early Dupuytren's contracture in the majority of patients, preventing progression to more advanced contracture...
Pediatric: Ewing (4)
- AEWS0031 JCO 2012Interval-compressed VDC/IE (q2 weeks) significantly improved both EFS and OS in localized Ewing sarcoma with manageable toxicity using G-CSF support. Interval...
- Disseminated Ewing Sarcoma WLI Cancer 2010Whole lung irradiation improves outcomes in patients with lung-only metastatic Ewing sarcoma, particularly when combined with HDCT consolidation. Bone/BM metastatic...
- INT-0154 JCO 2009Dose intensification of VDC/IE beyond standard doses does not improve EFS or OS in non-metastatic Ewing sarcoma and significantly increases toxicity. Standard-dose...
- INT-0091/IESS-III NEJM 2003Adding ifosfamide and etoposide (IE) to VAC significantly improved EFS and OS in localized Ewing sarcoma/PNET. The four-drug VACIE regimen (or alternating VDC/IE)...
Pediatric: RMS (3)
- ARST0431 JCO 2016Dose-compressed VDC/IE with RT and irinotecan maintenance improved EFS in high-risk RMS compared to historical controls, with 38% 3-year EFS - a meaningful...
- ARST0331 JCO 2014Adding vincristine/irinotecan (VI) to VAC did not improve FFS or OS in intermediate-risk rhabdomyosarcoma. VAC plus local RT at 45-50.4 Gy remains the standard of...
- D9803 JCO 2009Adding topotecan to VAC did not improve outcomes in intermediate-risk rhabdomyosarcoma and increased hematologic toxicity. VAC plus local RT remains the standard...
Sarcoma - osteosarcoma (3)
- OLIE (ITCC-082) JAMA Oncol 2024Adding lenvatinib to IE reduced progression risk ~46% (HR 0.54, 1-sided P=.04) in relapsed/refractory osteosarcoma but did not meet the prespecified primary threshold...
- EURAMOS-1 (Poor Response / MAPIE) Lancet Oncol 2016MAPIE did not improve EFS or OS vs MAP in poor-responding high-grade osteosarcoma (HR 0.98, P=.72) with significantly increased toxicity. Poor histologic response...
- EURAMOS-1 (Good Response) JCO 2015Pegylated interferon alfa-2b maintenance added to MAP did not improve EFS or OS in good histologic responders. IFN maintenance cannot be recommended; MAP remains...
GIST - metastatic (2)
- VOYAGER JCO 2021Avapritinib did not meet superior PFS vs regorafenib in molecularly unselected ≥3rd-line GIST (HR 1.25, P=.055); mPFS shorter despite higher ORR. Benefit concentrated...
- B2222 NEJM 2002Imatinib produced durable objective responses in 53.7% of advanced Kit-positive GIST - unprecedented in a historically chemotherapy-resistant disease - with no...
Sarcoma - TGCT (2)
- MOTION Lancet 2024Vimseltinib achieved 40% ORR vs 0% placebo in advanced TGCT with significant pain/function improvement. The intermittent 5-on/2-off schedule was designed to reduce...
- ENLIVEN Lancet 2019Pexidartinib achieved 39% ORR (vs 0% placebo, P<.0001) with durable responses and improved function in TGCT, establishing the first FDA-approved systemic therapy and...
Sarcoma - desmoid (1)
- DeFi NEJM 2023Nirogacestat significantly improved PFS (HR 0.29, P<.001), ORR (41% vs 8%), pain, and quality of life versus placebo in progressing desmoid tumours - the first...
Sarcoma - Ewing (1)
- Euro-E.W.I.N.G.99 / Ewing-2008 (HDCT) JCO 2018High-dose BuMel+ASCT showed numerically improved EFS vs standard VAI consolidation in high-risk localized Ewing sarcoma, particularly in very high-risk exploratory...