NCT03516708

Brief Summary

The purpose of this research study is to evaluate epacadostat when given with routine radiation therapy and chemotherapy (capecitabine and oxaliplatin) to treat rectal cancer before routine surgery is performed to remove the tumor.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
49

participants targeted

Target at P50-P75 for phase_1

Timeline
52mo left

Started Jan 2020

Longer than P75 for phase_1

Geographic Reach
1 country

4 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress60%
Jan 2020Aug 2030

First Submitted

Initial submission to the registry

April 24, 2018

Completed
10 days until next milestone

First Posted

Study publicly available on registry

May 4, 2018

Completed
1.7 years until next milestone

Study Start

First participant enrolled

January 6, 2020

Completed
7.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 15, 2027

Expected
3.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

August 12, 2030

Last Updated

February 11, 2026

Status Verified

February 1, 2026

Enrollment Period

7.5 years

First QC Date

April 24, 2018

Last Update Submit

February 8, 2026

Conditions

Keywords

ImmunotherapyKynurenineColonIndoleamine 2,3 dioxygenaseIDO1

Outcome Measures

Primary Outcomes (2)

  • Phase I only: Recommended phase II dose (RP2D) of epacadostat with standard of care radiation and chemotherapy in preoperative treatment of locally advanced rectal cancer

    The recommended phase 2 dose (RP2D) is defined as the dose level immediately below the maximally administered dose at which 0 or 1 of a cohort of 3 to 6 patients experienced a dose-limiting toxicity (DLT) during first 2 cycles.

    Completion of the first 2 cycles of treatment for all patients (estimated to be 86 months)

  • Phase II Treatment Cohort only: Neoadjuvant Rectal (NAR) Score

    Neoadjuvant Rectal Score (NAR Score) is calculated by following formula: NAR = (\[5pN \- 3(cT - pT) + 12\]2)/9.61 (with higher scores representing poorer prognosis), where cT=clinical tumor stage, pT=pathologic tumor stage, and pN=pathologic nodal stage.

    At the time of surgery (approximately week 28)

Secondary Outcomes (5)

  • Phase I and Phase II Treatment Cohort only: Safety and toxicity profile of the combination as measured by adverse events experienced

    Through 4 weeks after completion of treatment (approximately 28 weeks)

  • Phase I only: Neoadjuvant Rectal (NAR) Score

    At the time of surgery (approximately week 28)

  • Phase I and Phase II Treatment Cohort only: Pathological complete response rate (pCR)

    At the time of surgery (approximately week 25)

  • Phase I and Phase II Treatment Cohort only: Progression-free survival (PFS)

    Through completion of follow-up (estimated to be 3 years and 32 weeks)

  • Phase I and Phase II Treatment Cohort only: Complete clinical response rate (cCR)

    At the time of preoperative assessment (approximately week 28)

Study Arms (3)

Dose Escalation Cohort (Phase I): Epacadostat + SCRT + Chemotherapy + Surgery

EXPERIMENTAL

* Epacadostat at the designated dose level starting on the 1st day of radiation therapy and continuing throughout chemotherapy until the day of surgery. * Standard of care preoperative therapy will consist of a total of approximately 20-24 weeks of short-course pelvic radiation and chemotherapy: * Cycle 0 Days 1-7(Week 1): Short-course pelvic radiation therapy (SCRT), 5 fractions over 1 week * Cycle 0 Days 8-21 or 8-28 (Weeks 2-4): Treatment break for 2 to 3 weeks; for patients enrolled at Washington University and Dana Farber only, tumor biopsy will be obtained between the end of RT and prior to chemotherapy (target Days 14-28) * Cycles 1-6: (6) 21-day cycles of CAPOX for a total of 18 weeks. CAPOX is typically capecitabine at 1000 mg/m\^2 PO BID (days 1-14 of each cycle) and oxaliplatin 130 mg/m\^2 IV Q3W. * Surgery will follow approximately 4 to 6 weeks after completion of CAPOX

Drug: EpacadostatRadiation: Short-course radiation therapyDrug: CAPOX chemotherapy

Phase II Treatment Cohort: Epacadostat + SCRT + Chemotherapy + Surgery

EXPERIMENTAL

* Epacadostat 400 mg BID 1st day of radiation therapy and continuing for \~28 days, until biopsy (with the exception of patients enrolling to dose expansion and starting on epacadostat with neoadjuvant chemotherapy prior to approval of post-sIRB A2) * Preoperative therapy approximately 20-24 weeks of chemoradiation: * Week 1: SCRT, typically 5 Gy x 5 fractions over 1 week, with epacadostat 400 mg BID starting on D1 of SCRT * Weeks 2-4: Epacadostat monotherapy 400 mg BID \& continued for a minimum of 21 days, until the day prior to chemotherapy * For patients enrolled at Washington University and Dana Farber Cancer Institute ONLY, tumor biopsy between the end of RT and prior to initiation of chemotherapy. Tumor biopsy target of between Days 15-28 * Weeks 3-6: 4-5 weeks after completion of SCRT \& after completion of approximately 21-35 days of epacadostat, SOC neoadjuvant chemotherapy of CAPOX or FOLFOX will be initiated * Surgery will occur approximately 4-6 weeks after chemotherapy

Drug: EpacadostatRadiation: Short-course radiation therapyDrug: CAPOX chemotherapyDrug: FOLFOX chemotherapy

Phase II Biomarker Cohort: SCRT + Chemotherapy + Surgery

ACTIVE COMPARATOR

Washington University and Dana Farber only: Patients enrolled to this cohort will not receive epacadostat. Patients will undergo standard of care preoperative therapy consisting of approximately 20 to 24 weeks of chemoradiation. All treatment will be administered in this cohort as per institutional standard. The expected schedule for these patients will consist of 1 week of short-course pelvic radiation therapy, followed by a treatment break, followed by neoadjuvant chemotherapy. Approximately 4 to 6 weeks after completion of neoadjuvant chemotherapy, patients may undergo surgery. Tumor biopsy may occur at screening and after completion of RT, prior to starting neoadjuvant chemotherapy.

Radiation: Short-course radiation therapyDrug: CAPOX chemotherapyDrug: FOLFOX chemotherapy

Interventions

Drug provided.

Also known as: INCB024360
Dose Escalation Cohort (Phase I): Epacadostat + SCRT + Chemotherapy + SurgeryPhase II Treatment Cohort: Epacadostat + SCRT + Chemotherapy + Surgery

Short-course pelvic radiation therapy, 5 Gy x 5 fractions over 1 week

Also known as: SCRT
Dose Escalation Cohort (Phase I): Epacadostat + SCRT + Chemotherapy + SurgeryPhase II Biomarker Cohort: SCRT + Chemotherapy + SurgeryPhase II Treatment Cohort: Epacadostat + SCRT + Chemotherapy + Surgery

Standard of care

Dose Escalation Cohort (Phase I): Epacadostat + SCRT + Chemotherapy + SurgeryPhase II Biomarker Cohort: SCRT + Chemotherapy + SurgeryPhase II Treatment Cohort: Epacadostat + SCRT + Chemotherapy + Surgery

Standard of care

Phase II Biomarker Cohort: SCRT + Chemotherapy + SurgeryPhase II Treatment Cohort: Epacadostat + SCRT + Chemotherapy + Surgery

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Newly diagnosed pathologically-confirmed locally advanced rectal cancer (defined by 8th edition AJCC stage 2 or 3, or stage 1 not eligible for sphincter-sparing surgery) with plans to proceed with total neoadjuvant short course radiation as part of their neoadjuvant therapy as confirmed by treating physician
  • At least 18 years of age.
  • ECOG performance status 0, 1, or 2
  • Adequate bone marrow and organ function as defined below:
  • Absolute neutrophil count ≥ 1.5 K/cumm
  • Platelets ≥ 100 K/cumm
  • Hemoglobin \> 9 g/dL
  • Total bilirubin ≤ 1.5 x IULN
  • AST(SGOT)/ALT(SGPT) ≤ 2.5 x IULN
  • Serum creatinine \< 1.5 x IULN OR measured or calculated creatinine clearance ≥ 50 mL/min/1.73 m2
  • Applicable to subjects enrolled at Washington University and Dana Farber Cancer Institute only: Willing to undergo study-related biopsies subject to accessibility of tumor, appropriateness of biopsy (not contraindicated), and continued subject consent. If biopsy is not safe and feasible per treating physician, then patient may still enroll with permission of sponsor-investigator.
  • Women of childbearing potential and men must agree to contraceptive methods as described in protocol prior to study entry, for the duration of study participation, and for 120 days after the last dose of study treatment. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately.
  • Able to understand and willing to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable).

You may not qualify if:

  • Received prior anti-cancer therapy for rectal cancer.
  • Prior treatment with agents targeting IDO pathway (including indoximod)
  • Previous radiotherapy in the pelvic region or previous rectal surgery (e.g. TEM) or any investigational treatment for rectal cancer within the past month.
  • Known or suspected presence of another malignancy that could be mistaken for the malignancy under study during disease assessments.
  • Currently receiving any other investigational agents.
  • Extensive growth into cranial part of the sacrum (above S3) or the lumbosacral nerve roots indicating that surgery will never be possible even if substantial tumor downsizing is seen.
  • Presence of metastatic disease or recurrent rectal tumor.
  • Diagnosis of Familial Adenomatosis Polyposis coli (FAP), Hereditary Non-Polyposis Colorectal Cancer (HNPCC), active Crohn's disease, or active ulcerative colitis.
  • A history of allergic reactions attributed to compounds of similar chemical or biologic composition to epacadostat, pembrolizumab, 5-FU, oxaliplatin, or other agents used in the study.
  • Has an active infection requiring systemic therapy.
  • Warfarin (Coumadin): patients currently on warfarin are excluded. Patients who go off warfarin and have INR within normal limits have no washout period.
  • Any history of serotonin syndrome (SS) after receiving serotonergic drugs. This syndrome has been most closely associated with the use of MAOIs, meriperidine, linezolid, or methylene blue; all of these agents are prohibited during the study
  • Uncontrolled intercurrent illness including, but not limited to active tuberculosis infection, pneumonitis requiring treatment, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia.
  • Has an active or inactive autoimmune disease or syndrome (i.e. rheumatoid arthritis, moderate or severe psoriasis, multiple sclerosis, inflammatory bowel disease) that has required systemic treatment in the past 2 years or is receiving systemic therapy for an autoimmune or inflammatory disease (i.e. with use of modifying agents, corticosteroids, or immunosuppressive drugs). Exceptions include subjects with vitiligo or resolved childhood asthma/atopy, hypothyroidism stable on hormone replacement, controlled asthma, Type I diabetes, Graves' disease, or Hashimoto's disease.
  • An abnormal screening ECG that, in the investigator's opinion, is clinically meaningful.
  • +5 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

University of California Irvine - Chao Family Comprehensive Cancer Center

Orange, California, 92868, United States

WITHDRAWN

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States

RECRUITING

Henry Ford Cancer Institute

Detroit, Michigan, 48202, United States

WITHDRAWN

Washington University School of Medicine

St Louis, Missouri, 63110, United States

RECRUITING

Related Links

MeSH Terms

Conditions

Rectal Neoplasms

Interventions

epacadostat

Condition Hierarchy (Ancestors)

Colorectal NeoplasmsIntestinal NeoplasmsGastrointestinal NeoplasmsDigestive System NeoplasmsNeoplasms by SiteNeoplasmsDigestive System DiseasesGastrointestinal DiseasesIntestinal DiseasesRectal Diseases

Study Officials

  • Moh'd Khusman, M.D.

    Washington University School of Medicine

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Moh'd Khushman, M.D.

CONTACT

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 24, 2018

First Posted

May 4, 2018

Study Start

January 6, 2020

Primary Completion (Estimated)

July 15, 2027

Study Completion (Estimated)

August 12, 2030

Last Updated

February 11, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will not share

Locations