NCT01282502

Brief Summary

This study combines midostaurin (PKC412) with radiation and a standard chemotherapy drug call 5-Fluorouracil (5-FU) for subjects with advanced rectal cancer. Midostaurin is a type of kinase inhibitor which works by blocking proteins associated with cancer cell growth. Previous studies also suggest that midostaurin may help increase the effectiveness of radiation therapy. In this research we are looking for the highest dose of midostaurin that can be given safely in combination with standard chemoradiation.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
19

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Aug 2011

Longer than P75 for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

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

First Submitted

Initial submission to the registry

January 21, 2011

Completed
4 days until next milestone

First Posted

Study publicly available on registry

January 25, 2011

Completed
6 months until next milestone

Study Start

First participant enrolled

August 1, 2011

Completed
3.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2015

Completed
3.8 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2018

Completed
Last Updated

December 12, 2018

Status Verified

December 1, 2018

Enrollment Period

3.6 years

First QC Date

January 21, 2011

Last Update Submit

December 11, 2018

Conditions

Keywords

rectal cancerchemotherapychemoradiation

Outcome Measures

Primary Outcomes (1)

  • To determine Maximum Tolerated Dose (MTD) of midostaurin in combination with standard 5-FU chemoradiation

    1.5 years

Secondary Outcomes (4)

  • To determine the rate of Dworak Tumor Regression Grade 3/4 for locally advanced rectal cancer treated with study combination at the MTD, stratified by KRAS status (mutant vs. wild type)

    1.5 years

  • To determine surgical complication rate in patients who received preoperative radiation therapy

    1.5 years

  • Perform an exploratory analysis of the impact of selected mutations in APC, PTEN, BRAF, NRAS, and PIK3CA, among other genes

    1.5 year

  • To evaluate proteomic markers of response and resistance to midostaurin-based chemoradiation

    1.5 years

Study Arms (1)

Midostaurin with chemoradiation

EXPERIMENTAL
Drug: Midostaurin

Interventions

50 mg BID for 8 cycles

Midostaurin with chemoradiation

Eligibility Criteria

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

You may qualify if:

  • Adenocarcinoma of the rectum
  • T3/4 or N+ disease
  • Life expectancy \> 3 months
  • Normal organ and marrow function

You may not qualify if:

  • Metastatic disease
  • Pregnant or breastfeeding
  • Prior radiotherapy
  • Receiving other investigational agents
  • History of inflammatory bowel disease
  • Active scleroderma or CREST syndrome
  • Uncontrolled intercurrent illness
  • History of a different malignancy unless disease free for at least 5 years
  • HIV or active viral hepatitis
  • Impaired cardiac function

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

MeSH Terms

Conditions

Rectal Neoplasms

Interventions

midostaurin

Condition Hierarchy (Ancestors)

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

Study Officials

  • Theodore S Hong, MD

    Massachusetts General Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Radiation Oncologist

Study Record Dates

First Submitted

January 21, 2011

First Posted

January 25, 2011

Study Start

August 1, 2011

Primary Completion

March 1, 2015

Study Completion

December 1, 2018

Last Updated

December 12, 2018

Record last verified: 2018-12

Locations