Carcinoma of the Anal CanalEarly Phase 1NCT01858025
Concurrent Chemoradiation + 5-FU + Mitomycin-C in Anal Carcinoma
A Early Phase 1 study testing Pencil Beam scanning Radiation, 5-fluorouracil and Mitomycin-C in people with Carcinoma of the Anal Canal, run by Massachusetts General Hospital at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Carcinoma of the Anal Canal
- What is being tested?
- Pencil Beam scanning Radiation, 5-fluorouracil and Mitomycin-C
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 2 sitesUnited States
- Study dates
- Jul 2013to Nov 2021
- Phase
- Early Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Histologically or cytologically confirmed invasive primary squamous, basaloid or cloacogenic carcinoma of the anal canal
- Life expectancy of at least 3 months
Who cannot join
- 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
Show all 10Show fewer
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Roberts Proton Therapy Center of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
About this study
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
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.
Read more
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.
Read the 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.
- Conditions
- Carcinoma of the Anal Canal
- Being tested
- Pencil Beam scanning Radiation, 5-fluorouracil, Mitomycin-C
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pencil Beam scanning RadiationRadiation
5-fluorouracilDrug
Also called 5-FU
Mitomycin-CDrug
| Study group | Role | What they receive |
|---|---|---|
| Pencil Beam Scanning Radiation | Experimental | 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 Receives: Radiation: Pencil Beam scanning Radiation; Drug: 5-fluorouracil; Drug: Mitomycin-C |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Grade 3+ Dermatologic Toxicity
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)
Measured: 2 years
Secondary outcomes (5)
Clinical Complete Response on Digital Rectal Exam
Overall rate of clinical complete response using digital rectal examination performed every 4 to 6 weeks until complete clinical response (cCR) up to 6 months. Patients were followed with digital rectal examination every 3 months until year 2, then every 6 months until year 5, and annual scans for 3 years thereafter.
Measured: Median follow up of 27 months
Local Failure
Estimate the rate of local failure; local failure will be measured from study entry to date of first local or regional failure
Measured: 2 years
Colostomy-Free Survival
Estimate the rate of colostomy-free survival; colostomy-free survival will be measured from study entry.
Measured: 2 years
Progression Free Survival
Estimate the rate of progression free survival; progression free survival will be measured from study entry to date of first local, regional, distant or second primary failure or date of death.
Measured: 2 years
Overall Survival
Estimate the rate of overall survival; overall survival will be measured from study entry to 2 years
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2013 (estimated)
- Study startJul 2013
- Primary completionJul 2017
- Study completionNov 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Carcinoma of the Anal CanalStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Principal investigator, Jennifer Wo, Massachusetts General Hospital
- Investigators
- Jennifer Y. Wo, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01858025
- Sponsor's protocol ID
- 13-075
- Status last verified
- Sep 2026
- First submitted
- 16 May 2013
- Last updated
- 6 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Wo JY, Plastaras JP, Metz JM, Jiang W, Yeap BY, Drapek LC, Adams J, Baglini C, Ryan DP, Murphy JE, Parikh AR, Allen JN, Clark JW, Blaszkowsky LS, DeLaney TF, Ben-Josef E, Hong TS. Pencil Beam Scanning Proton Beam Chemoradiation Therapy With 5-Fluorouracil and Mitomycin-C for Definitive Treatment of Carcinoma of the Anal Canal: A Multi-institutional Pilot Feasibility Study. Int J Radiat Oncol Biol Phys. 2019 Sep 1;105(1):90-95. doi: 10.1016/j.ijrobp.2019.04.040. Epub 2019 May 22.
PubMed 31128146
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01858025
- Acronym / sponsor ID
- 13-075
- Conditions
- Carcinoma of the Anal Canal
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jul 2013 (actual)
- Primary completion
- Jul 2017 (actual)
- Study completion
- Nov 2021 (actual)
- First posted
- 20 May 2013 (estimated)
- Results
- Posted 6 Oct 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pencil Beam Scanning Radiation | Experimental | Radiation: Pencil Beam scanning Radiation, Drug: 5-fluorouracil, Drug: Mitomycin-CPencil 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Histologically or cytologically confirmed invasive primary squamous, basaloid or cloacogenic carcinoma of the anal canal
- Life expectancy of at least 3 months
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Pencil Beam scanning Radiation | Radiation | Pencil Beam Scanning Radiation |
| 5-fluorouracilAlso: 5-FU | Drug | Pencil Beam Scanning Radiation |
| Mitomycin-C | Drug | Pencil Beam Scanning Radiation |
Primary outcome
Number of Participants With Grade 3+ Dermatologic Toxicity
Time frame: 2 years
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)
5 secondary outcomes
Clinical Complete Response on Digital Rectal Exam
Time frame: Median follow up of 27 months
Local Failure
Time frame: 2 years
Colostomy-Free Survival
Time frame: 2 years
Progression Free Survival
Time frame: 2 years
Overall Survival
Time frame: 2 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 31128146Wo JY, Plastaras JP, Metz JM, Jiang W, Yeap BY, Drapek LC, Adams J, Baglini C, Ryan DP, Murphy JE, Parikh AR, Allen JN, Clark JW, Blaszkowsky LS, DeLaney TF, Ben-Josef E, Hong TS. Pencil Beam Scanning Proton Beam Chemoradiation Therapy With 5-Fluorouracil and Mitomycin-C for Definitive Treatment of Carcinoma of the Anal Canal: A Multi-institutional Pilot Feasibility Study. Int J Radiat Oncol Biol Phys. 2019 Sep 1;105(1):90-95. doi: 10.1016/j.ijrobp.2019.04.040. Epub 2019 May 22.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jennifer Y. Wo, MD principal investigator
Massachusetts General Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Pencil Beam scanning Radiation, 5-fluorouracil and Mitomycin-C in people with Carcinoma of the Anal Canal, run by Massachusetts General Hospital at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Oct 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Grade 3+ Dermatologic Toxicity (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01858025, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Oct 2026.