Skip to main content
OpenTrials
Completed

NCT Number: NCT01282502

Midostaurin (PKC412) for Locally Advanced Rectal Cancer

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Adenocarcinoma of the Rectum Colorectal Neoplasms Digestive System Diseases Digestive System Neoplasms Gastrointestinal Diseases Gastrointestinal Neoplasms Intestinal Diseases Intestinal Neoplasms Neoplasms Neoplasms by Site Rectal Diseases Rectal Neoplasms

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Dana Farber Cancer Institute, Boston, Massachusetts, United States

Loading trial locations.

About this study

Midostaurin capsules will be taken by mouth for 8 weeks. For the first 2 weeks midostaurin will be taken alone (no chemoradiation). After 2 weeks standard chemoradiation will be added to the midostaurin regimen. Subjects receive midostaurin and chemoradiation for an additional 6 weeks. Physical exams will be done weekly. Blood samples will be taken and an optional tumor biopsy will be performed in week 2.

4-5 weeks after completing chemoradiation and midostaurin subjects will undergo surgery as standard of care. Tumor tissue from the surgery will be used for research purposes. A Ct scan of chest, abdomen, and pelvis will be performed.

After completion of surgery, subjects will have an end of study visit with physical exam, blood tests. CT scans of chest, abdomen, and pelvis will be performed yearly for 5 years.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • 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

Treatment and study plan

Midostaurin

Drug

50 mg BID for 8 cycles

Primary outcomes

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

    Time frame: 1.5 years

Secondary outcomes

  1. 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)

    Time frame: 1.5 years

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

    Time frame: 1.5 years

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

    Time frame: 1.5 year

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

    Time frame: 1.5 years

Sponsors and collaborators

Lead sponsor

Massachusetts General Hospital

Other

Registry information

Official study title

Phase I Study of Chemoradiation With Midostaurin (PKC412) For Locally Advanced Rectal Cancer

Important dates

Study start
2011
Primary completion
2015
Study completion
2018
First posted
Jan 25, 2011
Registry last updated
Dec 12, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.