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NCT03519412: ARETHUSA

Pembrolizumab in MMR-Proficient Metastatic Colorectal Cancer Pharmacologically Primed to Trigger Hypermutation Status

Completed Phase 2 Results posted Last updated 3 March 2026
What this trial tests

Phase 2 trial testing temozolomide (induction), in Colorectal Neoplasms in 107 participants. Completed in 8 January 2025.

Timeline
23 January 2019
Primary endpoint
8 January 2025
8 January 2025

Quick facts

Lead sponsorIFOM ETS - The AIRC Institute of Molecular Oncology
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationnon randomized
Designparallel
Maskingnone
Primary purposetreatment
Enrollment107
Start date23 January 2019
Primary completion8 January 2025
Estimated completion8 January 2025
Sites4 locations across Italy

Drugs / interventions tested

Conditions studied

Sponsor

IFOM ETS - The AIRC Institute of Molecular Oncology

Who can join

18 and older, any sex, with Colorectal Neoplasms or Microsatellite Instability. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Overall Response Rate (ORR) Primary · Tumor assessments every 8-9 weeks from date of enrollment (in Trial Phase of the study) until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 36 months.

Overall response rate (ORR) to pembrolizumab according to RECIST v1.1. An objective response is defined as either a CR or PR assessed by RECIST v1.1 at the discretion of the local investigator.

GroupValue95% CI
TMZ-primed MMR-proficient (MMRp)00 – 0
Overall Response Rate (ORR) Secondary · Tumor assessments every 8-9 weeks from date of enrollment (in Trial Phase of the study) until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 36 months.

Overall response rate (ORR) to pembrolizumab according to RECIST v1.1. An objective response is defined as either a CR or PR assessed by RECIST v1.1 criteria at the discretion of the local investigator.

GroupValue95% CI
MMR-deficient (MMRd)54.332.7 – 84.8
Progression Free Survival (PFS) Secondary · From the date of treatment initiation with pembrolizumab to the date of first documented progression or date of death from any cause, whichever came first, up to maximum 72 months.

Progression Free Survival (PFS) in pembrolizumab treated patients. PFS was estimated using the Kaplan-Meier method. Patient without disease progression were censored at the last follow-up visit available.

GroupValue95% CI
TMZ-primed MMR-proficient (MMRp)3.32.2 – 6.4
MMR-deficient (MMRd)49.73.5 – NA
Overall Survival (OS) Secondary · Every 8-12 weeks from date of treatment initiation with pembrolizumab to the date of death due to any cause (up to maximum 72 months). A participant who has not died will be censored at last known date alive.

Overall Survival (OS) in pembrolizumab treated patients. OS was estimated using the Kaplan-Meier method. Patient without disease progression were censored at the last survival follow-up available.

GroupValue95% CI
TMZ-primed MMR-proficient (MMRp)5.62.9 – NA
MMR-deficient (MMRd)NA15.4 – NA
Safety and Tolerability Secondary · From the time the first dose of study medication (either pembrolizumab or temozolomide) is administered until 30 days following discontinuation of study treatment.

Safety and Tolerability according to CTCAE version 4.03

All treatment-related Adverse Event (AE), any grade
GroupValue95% CI
MMR-proficient (MMRp)42
MMR-deficient (MMRd)/TMZ-primed MMR-proficient (MMRp)15
All treatment-related Adverse Event (AE), grade ≥ 3
GroupValue95% CI
MMR-proficient (MMRp)8
MMR-deficient (MMRd)/TMZ-primed MMR-proficient (MMRp)5

Adverse events — posted to ClinicalTrials.gov

Time frame: All-Cause Mortality monitored/assessed up to 72 months. Adverse Events monitored/assessed from the time the first dose of study medication (either pembrolizumab or temozolomide) is administered until 30 days following discontinuation of study treatment.. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

MMR-proficient (MMRp)
Serious: 14/66 (21%)
Deaths: 6/66
MMR-deficient (MMRd)/TMZ-primed TMB-high MMR-proficient (MMRp)
Serious: 20/57 (35%)
Deaths: 13/57

Serious adverse events (25 terms)

ReactionSystemMMR-proficient (MMRp)MMR-deficient (MMRd)/TMZ-p…
Intestinal obstructionGastrointestinal disorders
Alanine aminotransferase increasedInvestigations
ThrombocytopeniaBlood and lymphatic system disorders
Acute kidney injuryRenal and urinary disorders
Blood bilirubin increasedInvestigations
Hepatic failureHepatobiliary disorders
Spinal cord compressionNervous system disorders
PancytopeniaBlood and lymphatic system disorders
General physical health deteriorationGeneral disorders
Disease progressionGeneral disorders
Sinus tachycardiaCardiac disorders
DysarthriaNervous system disorders
JaundiceHepatobiliary disorders
Cardiac failureCardiac disorders
InfectionInfections and infestations
Back painMusculoskeletal and connective tissue disorders
Device breakageProduct Issues
Pathological fractureMusculoskeletal and connective tissue disorders
Device related infectionInfections and infestations
Urinary tract obstructionRenal and urinary disorders
HepatotoxicityHepatobiliary disorders
Intra-abdominal haemorrhageGastrointestinal disorders
SeizureNervous system disorders
Pleural effusionRespiratory, thoracic and mediastinal disorders
PyrexiaGeneral disorders
Other adverse events (5 terms — click to expand)

ReactionSystemMMR-proficient (MMRp)MMR-deficient (MMRd)/TMZ-p…
NauseaGastrointestinal disorders
AnaemiaBlood and lymphatic system disorders
ThrombocytopeniaBlood and lymphatic system disorders
NeutropeniaBlood and lymphatic system disorders
AstheniaGeneral disorders

Most-reported serious reactions: Intestinal obstruction, Alanine aminotransferase increased, Thrombocytopenia, Acute kidney injury, Blood bilirubin increased, Hepatic failure, Spinal cord compression, Pancytopenia.

Data from ClinicalTrials.gov NCT03519412 adverse events section.

Sponsor's own description

In this study, MMRd metastatic colorectal cancer (mCRC) patients who failed standard therapies will undergo treatment with pembrolizumab, while RAS-extended mutated MMR-proficient mCRC patients will be tested for o6-methylguanine-DNA-methyltransferase (MGMT) expression (IHC) and then for MGMT promoter methylation. MGMT IHC-negative, promoter methylation positive patients will be treated with temozolomide (TMZ). Patients progressing under temozolomide will be tested for tumor mutational burden (TMB) and proceed to pembrolizumab if TMB is \> 20 mutations/Mb. The primary study hypothesis is that tumors with acquired resistance to temozolomide become hypermutated and are sensitive to pembrolizumab.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Development of tumor mutation burden as an immunotherapy biomarker: utility for the oncology clinic.
    Chan TA, Yarchoan M, Jaffee E, Swanton C, et al · · 2019 · cited 2009× · PMID 30395155 · DOI 10.1093/annonc/mdy495
  2. Immunotherapy efficacy on mismatch repair-deficient colorectal cancer: From bench to bedside.
    Lizardo DY, Kuang C, Hao S, Yu J, et al · · 2020 · cited 160× · PMID 33035640 · DOI 10.1016/j.bbcan.2020.188447
  3. Review of PD-1/PD-L1 Inhibitors in Metastatic dMMR/MSI-H Colorectal Cancer.
    Oliveira AF, Bretes L, Furtado I. · · 2019 · cited 152× · PMID 31139574 · DOI 10.3389/fonc.2019.00396
  4. Temozolomide Treatment Alters Mismatch Repair and Boosts Mutational Burden in Tumor and Blood of Colorectal Cancer Patients.
    Crisafulli G, Sartore-Bianchi A, Lazzari L, Pietrantonio F, et al · · 2022 · cited 103× · PMID 35522273 · DOI 10.1158/2159-8290.cd-21-1434
  5. Liquid biopsies to monitor and direct cancer treatment in colorectal cancer.
    Mauri G, Vitiello PP, Sogari A, Crisafulli G, et al · · 2022 · cited 79× · PMID 35264786 · DOI 10.1038/s41416-022-01769-8
  6. Burden of tumor mutations, neoepitopes, and other variants are weak predictors of cancer immunotherapy response and overall survival.
    Wood MA, Weeder BR, David JK, Nellore A, et al · · 2020 · cited 74× · PMID 32228719 · DOI 10.1186/s13073-020-00729-2
  7. Strategies to tackle RAS-mutated metastatic colorectal cancer.
    Patelli G, Tosi F, Amatu A, Mauri G, et al · · 2021 · cited 65× · PMID 34044286 · DOI 10.1016/j.esmoop.2021.100156
  8. Molecular mechanisms targeting drug-resistance and metastasis in colorectal cancer: Updates and beyond.
    Al Bitar S, El-Sabban M, Doughan S, Abou-Kheir W. · · 2023 · cited 53× · PMID 36998426 · DOI 10.3748/wjg.v29.i9.1395

Verify or expand the search:

Other recruiting trials for Colorectal Neoplasms

Currently open trials in the same condition.

Other IFOM ETS - The AIRC Institute of Molecular Oncology trials

Trials by the same sponsor.

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