Trials · Topics · Skin Cancer

Skin Cancer clinical trials

78 Skin Cancer 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: Melanoma - metastatic · Melanoma - stage III · Melanoma - BRAF+ · Melanoma · Merkel · BCC / SCC · Benign disease · Merkel Cell Carcinoma · Basal Cell Carcinoma - advanced · CTCL / MF · Cutaneous SCC · PCBCL · DFSP

Melanoma - metastatic (17)

• Tebentafusp 5-year OS (IMCgp100-202) Ann Oncol 2026
  With a minimum of 5 years of follow-up, tebentafusp continued to deliver a clinically meaningful overall survival benefit (5-yr OS 16% vs 8%; HR 0.67) in first-line...
• RELATIVITY-047 JCO 2025
  Relatlimab plus nivolumab significantly improved PFS vs nivolumab alone in untreated advanced melanoma (HR 0.75, P=.006). Three-year follow-up confirms sustained...
• KEYNOTE-006 Ann Oncol 2024
  Pembrolizumab significantly improved OS (HR 0.71) and PFS (HR 0.64) vs ipilimumab in untreated advanced melanoma. 10-yr data confirm durable benefit: 34% alive at 10...
• C-144-01 (Lifileucel) J Immunother Cancer 2022
  Lifileucel produced a clinically meaningful ORR of 31.4% with durable responses (mDOR not reached, 41.7% lasting ≥18 mo) in heavily pretreated advanced melanoma...
• Tebentafusp in Metastatic Uveal Melanoma 2022
  Tebentafusp significantly prolonged overall survival versus investigator's choice in previously untreated HLA-A*02:01-positive metastatic uveal melanoma, despite low...
• CheckMate 066 trial JCO 2020
  First-line nivolumab provided durable, sustained OS and PFS benefit over dacarbazine in BRAF wild-type advanced melanoma, with roughly half of deaths and the majority...
• IMMUNED trial Lancet 2020
  In resected stage IV melanoma with no evidence of disease, adjuvant nivolumab alone or with ipilimumab significantly improved RFS over placebo, establishing...
• CheckMate 067 trial NEJM 2019
  Combination nivolumab plus ipilimumab provided superior OS and PFS versus either monotherapy, with durable 5-year survival, at the cost of substantially higher grade...
• CheckMate 511 trial JCO 2019
  NIVO3+IPI1 significantly reduced grade 3-5 toxicity versus NIVO1+IPI3 in unresectable stage III/IV melanoma, with no apparent loss of efficacy on descriptive comparison.
• KEYNOTE-029 trial, pembro vs low ipi Lancet 2017
  Standard-dose pembrolizumab with reduced-dose ipilimumab showed a manageable safety profile and robust antitumor activity in treatment-naive advanced melanoma...
• CheckMate 037 trial Lancet Onc 2015
  Nivolumab produced markedly higher and more durable response rates than chemotherapy in ipilimumab-refractory melanoma with a substantially better safety profile...
• CheckMate 069 trial, ipi/nivo vs ipi NEJM 2015
  Combination nivolumab plus ipilimumab markedly increased objective response and prolonged progression-free survival versus ipilimumab alone in treatment-naive...
• OPTiM JCO 2015
  T-VEC significantly improved durable response rate (16.3% vs 2.1%, P<.0001) and ORR (26.4% vs 5.7%) vs GM-CSF in unresectable melanoma with injectable lesions. OS...
• CA184-024 NEJM 2011
  Ipilimumab 10 mg/kg + dacarbazine significantly improved OS vs dacarbazine alone in untreated advanced melanoma (HR 0.72, P<.001), with durable long-term survival in...
• SEER-metastasectomy analysis JSO 2011
  Metastasectomy was associated with longer survival across M1a and M1b subgroups, supporting consideration of surgical resection in selected stage IV melanoma patients...
• MDX010-20 NEJM 2010
  Ipilimumab 3 mg/kg significantly improved OS vs gp100 vaccine alone in previously treated advanced melanoma (HR 0.68, P<.001) - the first treatment to improve OS in...
• HD-IL-2 meta-analysis JCO 1999
  High-dose IL-2 produced durable complete responses in a minority of metastatic melanoma patients (median OS 11.4mo), establishing the first immunotherapy capable of...

Melanoma - stage III (15)

• Daromun (L19IL2 + L19TNF) Neoadjuvant Ann Oncol 2025
  Neoadjuvant daromun significantly improves RFS vs upfront surgery in stage III melanoma. Offers new option for locoregional recurrence after prior surgery and...
• NADINA NEJM 2024
  Neoadjuvant ipilimumab + nivolumab followed by response-adapted adjuvant therapy dramatically improved EFS vs adjuvant nivolumab alone (12-month EFS 83.7% vs 57.2%...
• CheckMate 76K Nat Med 2023
  Adjuvant nivolumab significantly reduced recurrence risk vs placebo in resected stage IIB/IIC melanoma (HR 0.58, P=.0004), confirming adjuvant PD-1 inhibition as...
• SWOG S1801 NEJM 2023
  Neoadjuvant pembrolizumab followed by surgery and adjuvant pembrolizumab significantly improved 2-year EFS vs adjuvant pembrolizumab alone (72% vs 49%, HR 0.58...
• KEYNOTE-716 Lancet 2022
  Adjuvant pembrolizumab significantly reduced recurrence risk vs placebo in resected stage IIB/IIC melanoma (HR 0.65, P=.0132), establishing adjuvant PD-1 inhibition...
• CheckMate 238 trial Lancet Oncol 2020
  Adjuvant nivolumab provided sustained RFS benefit over high-dose ipilimumab with a more favorable safety profile; the OS difference was not statistically significant...
• COMBI-AD trial NEJM 2020
  Twelve months of adjuvant dabrafenib plus trametinib produced durable, sustained improvements in RFS and DMFS over placebo in resected BRAF-mutant stage III melanoma.
• E1609 trial JCO 2020
  Adjuvant ipilimumab 3mg/kg significantly improved OS vs high-dose interferon with less toxicity than the 10mg/kg dose. However, ipilimumab is no longer a preferred...
• KEYNOTE-054 Trial/EORTC-1325 NEJM 2020
  Adjuvant pembrolizumab significantly prolonged RFS across the overall and PD-L1-positive populations and irrespective of BRAF status; OS impact was not yet...
• OpACIN-neo Lancet Oncol 2019
  Ipilimumab 3 mg/kg + nivolumab 1 mg/kg × 2 cycles (Arm B) achieved 57% pCR with acceptable toxicity (Grade ≥3 AEs 20%) and was selected as the recommended neoadjuvant...
• MSLT-II trial NEJM 2017
  CLND improved regional disease control and provided prognostic staging information but did not improve melanoma-specific survival in patients with a positive SLN, at...
• DeCOG-SLT Lancet Oncol 2016
  CLND after positive SLNB did not improve DMFS, DFS, or OS vs observation with ultrasound surveillance in melanoma with occult sentinel-node metastases, while...
• EORTC 18071 NEJM 2016
  Adjuvant ipilimumab 10 mg/kg significantly improved RFS and OS vs placebo in resected stage III melanoma (OS HR 0.72, P=.001), the first adjuvant trial to demonstrate...
• MSLT-I NEJM 2014
  SLNB improved DFS and provided accurate pathologic staging in intermediate-thickness melanoma but did not significantly improve MSS in the ITT population. Among...
• E-1684 trial JCO 1996
  High-dose interferon improved both RFS and OS over observation, becoming the first adjuvant therapy to alter the natural history of high-risk resected melanoma...

Melanoma - BRAF+ (9)

• COLUMBUS-AD trial (EORTC-2139-MG) Eur J Cancer 2026
  COLUMBUS-AD demonstrated a consistent and manageable safety profile and numerically encouraging 12-month RFS (86% vs 70%) and DMFS (92% vs 82%) for one year of...
• DREAMseq JCO 2023
  IO-first sequencing (ipi+nivo then BRAF/MEK on progression) was significantly superior to targeted-first in BRAF V600-mutant advanced melanoma, with 2-yr OS 71.8% vs...
• COLUMBUS JCO 2022
  Encorafenib plus binimetinib significantly improved PFS (HR 0.54) and OS (HR 0.61) vs vemurafenib monotherapy in BRAF V600-mutant advanced melanoma, with...
• IMspire 150 trial Lancet 2020
  Adding atezolizumab to vemurafenib plus cobimetinib significantly prolonged PFS in BRAF V600-mutant melanoma without a significant increase in grade ≥3 toxicity...
• COMBI-d trial J Ann Onc 2017
  Dabrafenib plus trametinib improved long-term PFS and OS over dabrafenib alone, confirming that combined BRAF and MEK inhibition is superior to BRAF inhibitor...
• COMBI-v trial NEJM 2015
  Dabrafenib plus trametinib significantly improved OS, PFS, and ORR over vemurafenib monotherapy, establishing combined BRAF/MEK inhibition as superior to single-agent...
• coBRIM trial Lancet Oncol 2016
  Adding cobimetinib to vemurafenib significantly improved PFS and OS in BRAF V600-mutant melanoma, supporting combined BRAF/MEK inhibition as superior to BRAF monotherapy.
• BREAK-3 trial NEJM 2011
  Vemurafenib significantly improved both OS and PFS over dacarbazine in BRAF V600E-mutant metastatic melanoma, validating BRAF inhibition as a transformative targeted...
• BRIM-3 trial Lancet 2012
  Dabrafenib significantly improved PFS over dacarbazine in BRAF V600E-mutant metastatic melanoma, establishing it as an effective BRAF-targeted monotherapy.

Melanoma (7)

• RT + Ipilimumab in Metastatic Melanoma (Hiniker Stanford) Int J Radiat Oncol Biol Phys 2016
  RT combined with ipilimumab is safe and feasible in metastatic melanoma, with 50% clinical benefit rate and 27% complete systemic response - results substantially...
• ANZNTG 01.02/TROG 02.01: Adjuvant RT vs Observation in Melanoma (Henderson) Lancet Oncol 2015
  Adjuvant RT to the lymph node field after CLND significantly reduces lymph node field relapse in high-risk melanoma (HR 0.56, P=.023). However, no improvement in OS...
• RT for Desmoplastic Melanoma (Guadagnolo MDACC) Cancer 2014
  Adjuvant RT significantly reduces local recurrence in desmoplastic melanoma compared to surgery alone (~7-10% vs ~20-26%). Given DM's neurotropic nature and high LR...
• RT for Axillary Melanoma Metastases (Beadle MDACC) Int J Radiat Oncol Biol Phys 2009
  Adjuvant RT (30 Gy/5 fx) to the axillary basin after CLND achieves excellent regional control (~5-10% relapse) in high-risk Stage III melanoma with acceptable...
• Hypofractionation for Malignant Melanoma (Chang MDACC) Int J Radiat Oncol Biol Phys 2006
  Melanoma has a low alpha/beta ratio (~0.6 Gy) based on clinical data, supporting hypofractionated RT as biologically equivalent or superior to conventional...
• MDACC Adjuvant RT for Melanoma LN Metastases (Ballo) Cancer 2003
  Adjuvant hypofractionated RT (30 Gy in 5 fractions) achieves ~90% regional lymph node control in high-risk Stage III melanoma with acceptable late toxicity. The 6...
• RTOG 83-05: Hypofractionation vs Conventional RT for Melanoma (Sause) Int J Radiat Oncol Biol Phys 1991
  Both hypofractionated and conventional RT schedules achieve similar complete response rates (~24-35%) for melanoma, with no statistically significant difference. This...

Merkel (6)

• NCDB: Adjuvant RT and Chemo in Merkel Cell Carcinoma (Bhatia) J Natl Cancer Inst 2016
  Adjuvant RT is associated with significantly improved OS in Stage I and II MCC, but not Stage III. Adjuvant chemotherapy provides no OS benefit in any stage. This is...
• MDACC Head & Neck Merkel Cell Carcinoma (Bishop) Head Neck 2015
  Adjuvant RT achieves excellent local and regional control in H&N MCC. Comprehensive nodal irradiation (parotid + cervical levels) is recommended for H&N MCC given the...
• RCT: Adjuvant RT vs Observation in Stage I MCC (Jouary) Ann Oncol 2012
  Adjuvant prophylactic RT to the primary site and regional nodes eliminates locoregional relapse in Stage I MCC (0% vs 16.7%, P=.007). This is the only RCT in MCC...
• Sentinel Node Positivity Predictors in MCC (Schwartz) JCO 2011
  All clinicopathologic features of MCC tested were associated with increased likelihood of SLN positivity, and no low-risk subgroup had <15-20% SLN positivity. This...
• RT Dose and Volume Effects on Relapse in MCC (Foote) Int J Radiat Oncol Biol Phys 2010
  RT doses ≥50 Gy and comprehensive nodal irradiation achieve better regional control in MCC than lower doses or limited fields. Supports use of 50-56 Gy for...
• Chemoradiation for High-Risk Merkel Cell Carcinoma (Poulsen) JCO 2003
  Concurrent CRT achieves 75% 3-year locoregional control and 71% OS in high-risk MCC, with significant but manageable acute toxicity. Provides proof-of-concept that...

BCC / SCC (5)

• ASTRO Guidelines: RT for BCC and SCC (Likhacheva) Pract Radiat Oncol 2020
  RT is effective definitive or adjuvant treatment for BCC and SCC with local control 90-95% for well-selected lesions, providing equivalent local control to surgery...
• BCC Treatment Network Meta-Analysis (Drucker) Ann Intern Med 2018
  EBRT, excision, Mohs surgery, and curettage+diathermy all achieve similarly low BCC recurrence rates (~3.5-7%) for low-risk lesions. Topical therapies (imiquimod...
• TROG 05.01: Post-Op CRT vs RT for High-Risk cSCC H&N (Porceddu) JCO 2018
  Adding concurrent carboplatin to postoperative RT did not significantly improve locoregional relapse-free survival in high-risk cSCC of the H&N, though a...
• CTV Margins for Non-Melanoma Skin Cancer (Khan) Radiother Oncol 2012
  CTV margins of 10-14 mm (depending on histology and size) are required to cover 95% of microscopic disease extension beyond gross BCC/SCC. Larger tumors and SCC...
• RT for Epithelial Skin Cancer: Dose-Fractionation (Locke) Int J Radiat Oncol Biol Phys 2001
  RT achieves excellent local control for BCC and SCC (~90-95%) using conventional fractionation (2 Gy/fx to 60-66 Gy). Hypofractionation is acceptable for...

Benign disease (4)

• Keloid RT Meta-Analysis Ann Plast Surg 2017
  Post-excision RT reduces keloid recurrence to ~23% overall. Single-fraction and multi-fraction regimens have similar efficacy. Immediate RT (within 24 hours) is...
• Keloid RT BED Meta-Analysis IJROBP 2009
  A BED of ~30-40 Gy (using α/β 10) is the threshold for effective keloid prevention after surgery. Single-fraction doses of 10-15 Gy or 15 Gy/3 fractions are practical...
• Definitive RT for Unresectable Keloids Clin Oncol 2004
  Primary RT without surgery achieves meaningful volume reduction and symptom relief in the majority of large/unresectable keloids. It is a valid approach for patients...
• Post-Excision Keloid RT IJROBP 2004
  Post-excision RT within 24-48 hours at 15-20 Gy effectively reduces keloid recurrence. Site-specific recurrence rates are substantially higher for sternal and...

Merkel Cell Carcinoma (4)

• STAMP Trial (Pembrolizumab) in Merkel Cell Carcinoma Cancers 2025
  STAMP represents significant advancement in adjuvant/early-line MCC therapy; pending results expected to inform standard-of-care in stage I-III disease.
• CheckMate 358 (MCC) JCO 2020
  Neoadjuvant nivolumab followed by surgery achieved a 47% pCR rate in resectable MCC - a remarkably high pathologic response. pCR strongly predicted improved...
• JAVELIN Merkel 200 Lancet Oncol 2016
  Avelumab achieved a 31.8% ORR with durable responses (82% ongoing) in chemotherapy-refractory metastatic MCC with a favorable safety profile, establishing anti-PD-L1...
• KEYNOTE-017 NEJM 2016
  First-line pembrolizumab achieved 56% ORR with durable responses (mDOR not reached) in advanced MCC, outperforming historical chemotherapy (ORR ~50%, median duration...

Basal Cell Carcinoma - advanced (3)

• EMPOWER-BCC-1 Lancet Oncol 2021
  Cemiplimab achieved 31% ORR including 6% CR in laBCC after HHI failure - a population with no prior standard second-line option. Established cemiplimab as the first...
• BOLT Lancet Oncol 2015
  Sonidegib at both doses achieved comparable ORRs (~36-43% in laBCC), but the 200 mg dose demonstrated a markedly superior benefit-risk profile with fewer grade 3-4...
• ERIVANCE BCC NEJM 2012
  Vismodegib produced clinically meaningful ORRs in metastatic BCC (30%) and locally advanced BCC (43%), with 21% CR in laBCC - the first targeted therapy for advanced...

CTCL / MF (3)

• Low-Dose TSEBT for Erythrodermic MF/Sézary (Morris) Int J Radiat Oncol Biol Phys 2017
  Low-dose TSEBT (12 Gy) achieves high response rates in erythrodermic MF/SS with substantially reduced toxicity compared to conventional 30 Gy. When combined with ECP...
• Stanford TSEBT for Mycosis Fungoides (Navi) Arch Dermatol 2011
  TSEBT ≥30 Gy achieves universal response (100% ORR) with 60% CR in T2/T3 MF. T2 disease responds significantly better than T3 (CR 75% vs 47%). Adjuvant HN2 after...
• NCI Mycosis Fungoides RCT (Kaye) NEJM 1989
  Despite a higher complete response rate with aggressive combined-modality therapy, there was no improvement in overall survival compared to sequential topical...

Cutaneous SCC (2)

• KEYNOTE-629 JCO 2020
  Pembrolizumab achieved 34% ORR with mOS 23.8 months in recurrent/metastatic cSCC and a favorable toxicity profile. Provided the confirmatory single-arm dataset...
• EMPOWER-CSCC-1 NEJM 2018
  Cemiplimab achieved ~47% ORR with durable responses in metastatic CSCC and ~44% ORR in locally advanced CSCC - the first effective systemic therapy for this disease....

PCBCL (2)

• MDACC Low-Dose RT for PCBCL (Akhtari) Leuk Lymphoma 2015
  Low-dose RT (as low as 4 Gy) achieves 100% complete response for PCBCL non-leg type with no in-field failures. No dose-response advantage was identified for doses...
• Yale Primary Cutaneous B-Cell Lymphoma (Smith) JCO 2004
  RT is highly effective for indolent PCBCL (PCFCL and PCMZL), with excellent local control. PCDLBCL-LT has inferior outcomes and requires systemic therapy....

DFSP (1)

• Imatinib DFSP JCO 2010
  Imatinib achieved 46% ORR and median TTP 1.7 years in t(17;22)-positive advanced DFSP with favorable tolerability. Responses in both classic and fibrosarcomatous...