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Midostaurin (PKC412) for Locally Advanced Rectal Cancer

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ClinicalTrials.gov Identifier: NCT01282502
Recruitment Status : Active, not recruiting
First Posted : January 25, 2011
Last Update Posted : December 21, 2017
Information provided by (Responsible Party):

January 21, 2011
January 25, 2011
December 21, 2017
August 2011
March 2015   (Final data collection date for primary outcome measure)
To determine Maximum Tolerated Dose (MTD) of midostaurin in combination with standard 5-FU chemoradiation [ Time Frame: 1.5 years ]
To determine MTD of midostaurin in combination with standard 5-FU chemoradiation [ Time Frame: 1.5 years ]
Complete list of historical versions of study NCT01282502 on ClinicalTrials.gov Archive Site
  • 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 ]
  • To determine surgical complication rate in patients who received preoperative radiation therapy [ Time Frame: 1.5 years ]
  • 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 ]
  • To evaluate proteomic markers of response and resistance to midostaurin-based chemoradiation [ Time Frame: 1.5 years ]
Same as current
Not Provided
Not Provided
Midostaurin (PKC412) for Locally Advanced Rectal Cancer
Phase I Study of Chemoradiation With 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.

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.

Phase 1
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Adenocarcinoma of the Rectum
Drug: Midostaurin
50 mg BID for 8 cycles
Experimental: Midostaurin with chemoradiation
Intervention: Drug: Midostaurin
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Active, not recruiting
March 2022
March 2015   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
Contact information is only displayed when the study is recruiting subjects
United States
Not Provided
Not Provided
Theodore Sunki Hong, Massachusetts General Hospital
Massachusetts General Hospital
Not Provided
Principal Investigator: Theodore S Hong, MD Massachusetts General Hospital
Massachusetts General Hospital
December 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP