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Concurrent Chemoradiation + 5-FU + Mitomycin-C in Anal Carcinoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01858025
Recruitment Status : Active, not recruiting
First Posted : May 20, 2013
Last Update Posted : April 2, 2019
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Jennifer Wo, Massachusetts General Hospital

Brief Summary:

This research study is a Pilot Study. Pilot studies are conducted to see if it is practical to do this type of research ona larger scale in the future. The pilot part of the study is to assess the possibility of using pencil beam proton radiation to treat your type of cancer. Proton radiation is used for many other types of malignancies, but its use for the treatment of anal cancer has been limited. The treatment is still being studied as research doctors are trying to find out more about its use in the treatment of anal cancer. Proton beam radiation therapy is an FDA approved radiation delivery system.

You are being asked to participate in this study because you have cancer in the anal canal. Conventional radiation therapy with photons in combination with 5-FU and mitomycin-C is used as standard treatment for many patients with anal cancer.

In this research study we are looking at another type of radiation called proton radiation, which is known to spare surrounding tissue and organs from radiation. Proton radiation delivers radiation to the area requiring radiation but delivers no dose beyond the region requiring treatment. There are several techniques that can be used to deliver proton radiation therapy. One of the newer techniques, called pencil beam scanning, allows for more accurate delivery of radiation to your tumor and further reduces the amount of normal tissue exposed to radiation. Most proton patients are treated with a number of beams that study doctors conform to the shape of your tumor. Pencil beam scanning delivers radiation with a single, narrow proton beam that is swept over the area of your tumor. This may reduce side effects that patients would normally experience with conventional radiation therapy or other means of delivering proton radiation therapy, and also minimizes treatment time.

In this research study, we are evaluating the effectiveness of using pencil beam proton radiation delivered to reduce side effects associated with radiation treatment.

Condition or disease Intervention/treatment Phase
Carcinoma of the Anal Canal Radiation: Pencil Beam scanning Radiation Drug: 5-fluorouracil Drug: Mitomycin-C Early Phase 1

Detailed Description:

If you agree to participate in this study, you will be asked to undergo some screening tests or procedures to confirm that you are eligible. Many of these tests and procedures are likely to be part of regular cancer care and may be done even if it turns out that you do not take part in the research study. If you have had some of these tests or procedures recently, they may or may not have to be repeated. The screening will include the following: a medical history, physical examination, biopsy of your tumor, anal and groin exam, archival tumor tissue, CT or PET-CT, MRI, blood tests and a serum pregnancy test. If these tests show that you are eligible to participate in the research study, you will begin the study treatment. If you do not meet the eligibility criteria, you will not be able to participate in this research study.

Proton Radiation will be delivered daily for 5-6 weeks, depending on the dose prescribed by your physician. Treatment is delivered (Monday-Friday) for 5 days (no weekends or holidays). Each treatment will require that you lie on a table for 25-30 minutes.

You will receive radiation therapy as an outpatient at the Massachusetts General Hospital.

You will start receiving chemotherapy within 5 days of starting radiation treatment. The day you start chemotherapy will be considered Day 1.

You will receive Mitomycin-C on Days 1 and 29 of chemotherapy. Mitomycin-C will be delivered via IV infusion over a period of 10-30 minutes.

You will receive 5-fluorouracil on Day 1 and 29 of chemotherapy. You will be fitted with an ambulatory infusion pump that will administer 5-FU via IV infusion continuously over a period of 96 hours. The infusion pump will be about the size of a paperback book that can fit into a front-pack or "fanny-pack" around your waist. This pump will be connected to your body. The nurses in the infusion room will start the pump and disconnect it after 4 days.

At your 6 and 12 week follow up visits, a physical examination and the tests and procedures you receive will tell your study doctor whether your disease has gotten better, worse or stayed the same.

If your cancer has gotten better, you will continue to be monitored at your follow up visits.

If your cancer is suspected to have gotten worse, you will need to have a biopsy to confirm. If the biopsy is negative, you will need to come back for re-evaluation at 16 weeks (about 4 months) after completing radiation therapy.

If the biopsy confirms that your cancer has gotten worse, your doctor will recommend that you have surgery for the removal of your tumor.

If your cancer has stayed the same, your doctor will recommend that you have additional procedures done to develop a plan for treatment.

After you complete radiation therapy, you will be followed for up to 5 years.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Feasibility Study of Definitive Concurrent Chemoradiation With Pencil Beam Scanning Proton Beam in Combination With 5-Fluorouracil and Mitomycin-C for Carcinoma of the Anal Canal
Study Start Date : July 2013
Actual Primary Completion Date : July 2017
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Pencil Beam Scanning Radiation
Pencil Beam Radiation daily, Monday-Friday, for 5-6 weeks Mitomycin-C via IV on Days 1 and 29 5-Fluorouracil via infusion pump over 4 days, starting Day 1 and 29 of chemotherapy
Radiation: Pencil Beam scanning Radiation
Drug: 5-fluorouracil
Other Name: 5-FU

Drug: Mitomycin-C

Primary Outcome Measures :
  1. Feasibility of administration of pencil beam scanning proton beam radiation with chemotherapy for anal cancer [ Time Frame: 2 years ]
    The primary objective of this study is to determine feasibility of administration of pencil beam scanning proton beam radiotherapy in combination with concurrent 5-fluorouracil (5-FU) and mitomycin-C for carcinoma of the anal canal. Proton radiotherapy will be considered feasible if grade 3+ skin toxicity seen on this protocol is less than 48% (reported grade 3+ dermatologic toxicity from RTOG 98-11)

Secondary Outcome Measures :
  1. Assessment of Adverse Events [ Time Frame: 2 years ]
    To assess rates of grade 3+ acute toxicities in the first 90 days following the start of combined modality treatment. To evaluate adverse events associated with the treatment regimens after 90 days from the start of combined modality treatment.

  2. Quality of Life Outcomes [ Time Frame: 2 years ]
    To assess health related quality-of-life outcomes after proton radiotherapy for anal carcinoma using objective measurements and validated quality-of-life instruments. Health quality of life questionnaire will be completed before treatment, within 2 weeks of completion of radiotherapy and quarterly for 15 months.

  3. Clinical Complete Response [ Time Frame: 12 weeks ]
    Estimate the clinical complete response rate at 6 weeks and 12 weeks after completion of treatment.

  4. Radiotherapy treatment time [ Time Frame: 2 years ]
    Evaluate elapsed time form radiotherapy treatment start to radiotherapy treatment end

  5. Local-regional failure [ Time Frame: 2 years ]
    Estimate the rate of local-regional failure; local-regional failure will be measured from study entry to date of first local or regional failure

  6. Colostomy Failure and Colostomy-Free Survival [ Time Frame: 2 years ]
    Estimate the rate of colostomy failure; colostomy failure will be measured from study entry to date of colostomy failure. Estimate the rate of colostomy-free survival; colostomy-free survival will be measured from study entry.

  7. Disease-free Survival [ Time Frame: 2 years ]
    Estimate the rate of disease-free survival; disease-free survival will be measured from study entry to date of first local, regional, distant or second primary failure or date of death.

  8. Overall Survival [ Time Frame: 2 years ]
    Estimate the rate of overall survival; overall survival will be measured from study entry to date of death.

  9. Correlative Studies [ Time Frame: 2 years ]
    Evaluate prevalence and prognostic significance of Bcl-2, Bcl-X1, HPV

  10. To describe mood in the study population [ Time Frame: 2 years ]
    To describe QOL and mood in this study population to help us better identify the side effects and challenges faced by patients.

  11. To measure utilization of health services [ Time Frame: 2 years ]
    To measure utilization of health services (emergency room, hospital and intensive care unit) in the study population.

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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

Inclusion Criteria:

  • Histologically or cytologically confirmed invasive primary squamous, basaloid or cloacogenic carcinoma of the anal canal
  • Life expectancy of at least 3 months

Exclusion Criteria:

  • Prior abdominopelvic radiotherapy
  • Prior systemic therapy for anal cancer
  • Pregnant or breastfeeding
  • Receiving other investigational agents
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to mitomycin-C or 5-fluorouracil
  • Prior surgery for cancer of the anus that removed all macroscopic anal cancer
  • Uncontrolled intercurrent illness
  • AIDS based on current CDC definition
  • Other immunocompromised status
  • Individuals with a history of a different malignancy are ineligible except for the following circumstances: disease-free for at least 5 years or at low risk for recurrence or cervical cancer in situ, and basal cell or squamous cell carcinoma of the skin

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01858025

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United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, Pennsylvania
Roberts Proton Therapy Center of the University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Massachusetts General Hospital
National Cancer Institute (NCI)
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Principal Investigator: Jennifer Y. Wo, MD Massachusetts General Hospital

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Responsible Party: Jennifer Wo, Principal Investigator, Massachusetts General Hospital Identifier: NCT01858025     History of Changes
Other Study ID Numbers: 13-075
First Posted: May 20, 2013    Key Record Dates
Last Update Posted: April 2, 2019
Last Verified: April 2019

Additional relevant MeSH terms:
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Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Molecular Mechanisms of Pharmacological Action
Antimetabolites, Antineoplastic
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antibiotics, Antineoplastic
Alkylating Agents
Nucleic Acid Synthesis Inhibitors
Enzyme Inhibitors