This site became the new on June 19th. Learn more.
Show more Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more... Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more... Menu
Give us feedback

Midostaurin (PKC412) for Locally Advanced Rectal Cancer

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Theodore Sunki Hong, Massachusetts General Hospital Identifier:
First received: January 21, 2011
Last updated: June 20, 2017
Last verified: June 2017
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.

Condition Intervention Phase
Adenocarcinoma of the Rectum Drug: Midostaurin Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Study of Chemoradiation With Midostaurin (PKC412) For Locally Advanced Rectal Cancer

Resource links provided by NLM:

Further study details as provided by Theodore Sunki Hong, Massachusetts General Hospital:

Primary Outcome Measures:
  • To determine Maximum Tolerated Dose (MTD) of midostaurin in combination with standard 5-FU chemoradiation [ Time Frame: 1.5 years ]

Secondary Outcome Measures:
  • 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 ]

Enrollment: 19
Study Start Date: August 2011
Estimated Study Completion Date: March 2022
Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Midostaurin with chemoradiation Drug: Midostaurin
50 mg BID for 8 cycles

Detailed Description:

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.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01282502

United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Sponsors and Collaborators
Massachusetts General Hospital
Principal Investigator: Theodore S Hong, MD Massachusetts General Hospital
  More Information

Responsible Party: Theodore Sunki Hong, Radiation Oncologist, Massachusetts General Hospital Identifier: NCT01282502     History of Changes
Other Study ID Numbers: 10-457
Study First Received: January 21, 2011
Last Updated: June 20, 2017

Keywords provided by Theodore Sunki Hong, Massachusetts General Hospital:
rectal cancer

Additional relevant MeSH terms:
Rectal Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Colorectal Neoplasms
Intestinal Neoplasms
Gastrointestinal Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Gastrointestinal Diseases
Intestinal Diseases
Rectal Diseases
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action processed this record on September 21, 2017