Trial Outcomes & Findings for Topical MTS-01 for Dermatitis During Radiation and Chemotherapy for Anal Cancer (NCT NCT01324141)

NCT ID: NCT01324141

Last Updated: 2021-11-23

Results Overview

Adverse events were assessed by the Common Terminology Criteria for Adverse Events (CTCAE) v4.0. Grade 1 is mild, Grade 2 is moderate, Grade 3 is severe, Grade 4 is life threatening, and Grade 5 is death related to adverse event.

• Recruitment status: TERMINATED
• Study phase: PHASE1
• Target enrollment: 6 participants
• Primary outcome timeframe: Date treatment consent signed to date off study, approximately, 36 months and 10 days.
• Results posted on: 2021-11-23

Participant Flow

Participant milestones

Measure	1/Chemo + Radiation Chemo + Radiation Tempol: Tempol gel will be applied to the bilateral groins and the gluteal cleft, avoiding a 3 cm radius from the anal verge, immediately prior to each fraction of radiation therapy (RT). 5-Fluorouracil: 5-FU will be delivered as 1000mg/m(2)/day as 96 hour continuous infusion beginning on day 1 and 29. Mitomycin-C: Mitomycin-C (MMC) will be delivered at a dose of 10mg/m(2) on days 1 and 29 Radiation Therapy: Radiation therapy (RT) will be delivered to a total dose of 50-54 Gray (Gy) based on tumor characteristics.
Overall Study STARTED	6
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	1/Chemo + Radiation Chemo + Radiation Tempol: Tempol gel will be applied to the bilateral groins and the gluteal cleft, avoiding a 3 cm radius from the anal verge, immediately prior to each fraction of radiation therapy (RT). 5-Fluorouracil: 5-FU will be delivered as 1000mg/m(2)/day as 96 hour continuous infusion beginning on day 1 and 29. Mitomycin-C: Mitomycin-C (MMC) will be delivered at a dose of 10mg/m(2) on days 1 and 29 Radiation Therapy: Radiation therapy (RT) will be delivered to a total dose of 50-54 Gray (Gy) based on tumor characteristics.
Overall Study Declined to participate before treatment started	1
Overall Study Disease progression on study	1

Baseline Characteristics

ECOG data was not captured for 1/6 participants that withdrew before treatment.

Baseline characteristics by cohort

Measure	1/Chemo + Radiation n=6 Participants Chemo + Radiation Tempol: Tempol gel will be applied to the bilateral groins and the gluteal cleft, avoiding a 3 cm radius from the anal verge, immediately prior to each fraction of radiation therapy (RT). 5-Fluorouracil: 5-FU will be delivered as 1000mg/m(2)/day as 96 hour continuous infusion beginning on day 1 and 29. Mitomycin-C: Mitomycin-C (MMC) will be delivered at a dose of 10mg/m(2) on days 1 and 29 Radiation Therapy: Radiation therapy (RT) will be delivered to a total dose of 50-54 Gray (Gy) based on tumor characteristics.
Age, Categorical <=18 years	0 Participants n=6 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=6 Participants
Age, Categorical >=65 years	0 Participants n=6 Participants
Age, Continuous	55.85 years STANDARD_DEVIATION 5.16 • n=6 Participants
Sex: Female, Male Female	4 Participants n=6 Participants
Sex: Female, Male Male	2 Participants n=6 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=6 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=6 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=6 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=6 Participants
Race (NIH/OMB) Asian	0 Participants n=6 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=6 Participants
Race (NIH/OMB) Black or African American	1 Participants n=6 Participants
Race (NIH/OMB) White	5 Participants n=6 Participants
Race (NIH/OMB) More than one race	0 Participants n=6 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=6 Participants
Region of Enrollment United States	6 participants n=6 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status ECOG 0	3 Participants n=5 Participants • ECOG data was not captured for 1/6 participants that withdrew before treatment.
Eastern Cooperative Oncology Group (ECOG) Performance Status ECOG 1	2 Participants n=5 Participants • ECOG data was not captured for 1/6 participants that withdrew before treatment.
Eastern Cooperative Oncology Group (ECOG) Performance Status ECOG 2	0 Participants n=5 Participants • ECOG data was not captured for 1/6 participants that withdrew before treatment.
Eastern Cooperative Oncology Group (ECOG) Performance Status ECOG3	0 Participants n=5 Participants • ECOG data was not captured for 1/6 participants that withdrew before treatment.
Eastern Cooperative Oncology Group (ECOG) Performance Status ECOG4	0 Participants n=5 Participants • ECOG data was not captured for 1/6 participants that withdrew before treatment.
Eastern Cooperative Oncology Group (ECOG) Performance Status ECOG 5	0 Participants n=5 Participants • ECOG data was not captured for 1/6 participants that withdrew before treatment.
Human Immunodeficiency Virus (HIV) Status HIV Positive	1 Participants n=5 Participants • HIV status was not captured for 1/6 participants that withdrew before treatment.
Human Immunodeficiency Virus (HIV) Status HIV Negative	4 Participants n=5 Participants • HIV status was not captured for 1/6 participants that withdrew before treatment.
Human Papilloma Status (Anal Swab) HPV Positive	0 Participants n=5 Participants • HPV status was not captured for 1/6 participants that withdrew before treatment.
Human Papilloma Status (Anal Swab) HPV Negative	5 Participants n=5 Participants • HPV status was not captured for 1/6 participants that withdrew before treatment.
T Stage T1	0 Participants n=5 Participants • T Stage was not captured for 1/6 participants that withdrew before treatment.
T Stage T2	2 Participants n=5 Participants • T Stage was not captured for 1/6 participants that withdrew before treatment.
T Stage T3	3 Participants n=5 Participants • T Stage was not captured for 1/6 participants that withdrew before treatment.
T Stage T4	0 Participants n=5 Participants • T Stage was not captured for 1/6 participants that withdrew before treatment.
N Stage N0	4 Participants n=5 Participants • N Stage was not captured for 1/6 participants that withdrew before treatment.
N Stage N1	0 Participants n=5 Participants • N Stage was not captured for 1/6 participants that withdrew before treatment.
N Stage N2	0 Participants n=5 Participants • N Stage was not captured for 1/6 participants that withdrew before treatment.
N Stage N3	1 Participants n=5 Participants • N Stage was not captured for 1/6 participants that withdrew before treatment.
Stage I	0 Participants n=5 Participants • Stage was not captured for 1/6 participants that withdrew before treatment.
Stage II	4 Participants n=5 Participants • Stage was not captured for 1/6 participants that withdrew before treatment.
Stage IIIA	0 Participants n=5 Participants • Stage was not captured for 1/6 participants that withdrew before treatment.
Stage IIIB	1 Participants n=5 Participants • Stage was not captured for 1/6 participants that withdrew before treatment.
Stage IV	0 Participants n=5 Participants • Stage was not captured for 1/6 participants that withdrew before treatment.

PRIMARY outcome

Timeframe: Date treatment consent signed to date off study, approximately, 36 months and 10 days.

Population: 1/6 participants enrolled withdrew before treatment.

Adverse events were assessed by the Common Terminology Criteria for Adverse Events (CTCAE) v4.0. Grade 1 is mild, Grade 2 is moderate, Grade 3 is severe, Grade 4 is life threatening, and Grade 5 is death related to adverse event.

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 n=5 Participants Grade 2 is moderate adverse events.	MTS-01 Grade 3 n=5 Participants Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 n=5 Participants Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 n=5 Participants Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Radiation dermatitis	0 Adverse events	0 Adverse events	0 Adverse events	2 Adverse events	3 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Nausea	0 Adverse events	0 Adverse events	0 Adverse events	3 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Hypocalcemia	0 Adverse events	0 Adverse events	0 Adverse events	1 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Pain	0 Adverse events	0 Adverse events	0 Adverse events	2 Adverse events	2 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Leukopenia	0 Adverse events	0 Adverse events	0 Adverse events	0 Adverse events	5 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Febrile neutropenia	0 Adverse events	0 Adverse events	0 Adverse events	0 Adverse events	1 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Thrombocytopenia	0 Adverse events	0 Adverse events	0 Adverse events	2 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Abdominal pain	0 Adverse events	0 Adverse events	0 Adverse events	1 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Diarrhea	1 Adverse events	1 Adverse events	0 Adverse events	1 Adverse events	2 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Gastroesophageal reflux	0 Adverse events	0 Adverse events	0 Adverse events	1 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Mucositis	0 Adverse events	0 Adverse events	0 Adverse events	1 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Urinary tract pain	0 Adverse events	0 Adverse events	0 Adverse events	2 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Bladder spasm	0 Adverse events	0 Adverse events	0 Adverse events	1 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Urinary incontinence	0 Adverse events	0 Adverse events	0 Adverse events	1 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Fatigue	1 Adverse events	0 Adverse events	1 Adverse events	1 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Hypoglycemia	1 Adverse events	0 Adverse events	0 Adverse events	0 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Transaminitis	0 Adverse events	0 Adverse events	0 Adverse events	1 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Hypoalbuminemia	0 Adverse events	0 Adverse events	0 Adverse events	1 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Myalgia	0 Adverse events	0 Adverse events	0 Adverse events	1 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Headache	0 Adverse events	0 Adverse events	0 Adverse events	0 Adverse events	1 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Syncope	0 Adverse events	0 Adverse events	0 Adverse events	0 Adverse events	1 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Lymphopenia	0 Adverse events	0 Adverse events	0 Adverse events	0 Adverse events	5 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Infection	0 Adverse events	0 Adverse events	0 Adverse events	1 Adverse events	1 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Insomnia	0 Adverse events	0 Adverse events	0 Adverse events	1 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Neutropenia	0 Adverse events	0 Adverse events	0 Adverse events	2 Adverse events	3 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Anemia	0 Adverse events	0 Adverse events	0 Adverse events	3 Adverse events	0 Adverse events
Number of Grade 1 and Higher Adverse Events Possibly, Probably, or Definitely Related to Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) Cluster of Differentiation (CD4) count decrease	0 Adverse events	0 Adverse events	1 Adverse events	0 Adverse events	0 Adverse events

PRIMARY outcome

Timeframe: Date treatment consent signed to date off study, approximately, 36 months and 10 days.

Population: 1/6 participants enrolled withdrew before treatment.

Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE)	5 Participants	—	—	—	—

SECONDARY outcome

Timeframe: baseline, weeks 1-6, follow up at 1 week, follow up at 2 weeks, and follow up at 4 weeks

Population: 1/6 participants enrolled withdrew before treatment.

RTOG grade 1-5 were used to assess skin toxicity in the groin (right and left inguinal area) and gluteal cleft, as well as two control sites. Grade 0 is no skin toxicity, Grade 1 is follicular, faint or dull erythema, Grade 3 is tender or bright erythema, Grade 3 is confluent, moist desquamation, Grade 4 is ulceration, hemorrhage, or necrosis, and Grade 5 is death due to radiation toxicity.

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 n=5 Participants Grade 2 is moderate adverse events.	MTS-01 Grade 3 n=5 Participants Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 n=5 Participants Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 n=5 Participants Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Mean Grade of Toxicity at Each Timepoint and Location for All Participants Who Underwent Treatment Week 5	1 Grade Standard Deviation 0.7	0.8 Grade Standard Deviation 0.5	0.8 Grade Standard Deviation 0.5	0.2 Grade Standard Deviation 0.5	0 Grade Standard Deviation 0
Mean Grade of Toxicity at Each Timepoint and Location for All Participants Who Underwent Treatment Week 6	1.2 Grade Standard Deviation 1.1	1.2 Grade Standard Deviation 0.5	1.2 Grade Standard Deviation 0.5	0.4 Grade Standard Deviation 0.5	0 Grade Standard Deviation 0
Mean Grade of Toxicity at Each Timepoint and Location for All Participants Who Underwent Treatment Baseline	0 Grade Standard Deviation 0	0 Grade Standard Deviation 0	0 Grade Standard Deviation 0	0 Grade Standard Deviation 0	0 Grade Standard Deviation 0
Mean Grade of Toxicity at Each Timepoint and Location for All Participants Who Underwent Treatment Week 1	0 Grade Standard Deviation 0	0 Grade Standard Deviation 0	0 Grade Standard Deviation 0	0 Grade Standard Deviation 0	0 Grade Standard Deviation 0
Mean Grade of Toxicity at Each Timepoint and Location for All Participants Who Underwent Treatment Week 2	0 Grade Standard Deviation 0	0 Grade Standard Deviation 0	0 Grade Standard Deviation 0	0 Grade Standard Deviation 0	0 Grade Standard Deviation 0
Mean Grade of Toxicity at Each Timepoint and Location for All Participants Who Underwent Treatment Week 3	0.4 Grade Standard Deviation 0.5	0.2 Grade Standard Deviation 0.5	0.2 Grade Standard Deviation 0.5	0.2 Grade Standard Deviation 0.5	0 Grade Standard Deviation 0
Mean Grade of Toxicity at Each Timepoint and Location for All Participants Who Underwent Treatment Week 4	0.6 Grade Standard Deviation 0.5	0.6 Grade Standard Deviation 0.5	0.6 Grade Standard Deviation 0.5	0.2 Grade Standard Deviation 0.5	0 Grade Standard Deviation 0
Mean Grade of Toxicity at Each Timepoint and Location for All Participants Who Underwent Treatment Follow up at 1 week	1.8 Grade Standard Deviation 0.8	1 Grade Standard Deviation 0	1 Grade Standard Deviation 0	0.6 Grade Standard Deviation 0.5	0 Grade Standard Deviation 0
Mean Grade of Toxicity at Each Timepoint and Location for All Participants Who Underwent Treatment Follow up at 2 weeks	1.2 Grade Standard Deviation 0.5	1 Grade Standard Deviation 0	1 Grade Standard Deviation 0	0.8 Grade Standard Deviation 0.5	0 Grade Standard Deviation 0
Mean Grade of Toxicity at Each Timepoint and Location for All Participants Who Underwent Treatment Follow up at 4 weeks	0.4 Grade Standard Deviation 0.5	0.2 Grade Standard Deviation 0.5	0.2 Grade Standard Deviation 0.5	0.6 Grade Standard Deviation 0	0 Grade Standard Deviation 0

SECONDARY outcome

Timeframe: From beginning until completion of radiation treatment up to 46 days

Population: 1/6 participants enrolled withdrew before treatment.

Number of participants that required a treatment break relative to hematologic (e.g. thrombocytopenia, leukopenia) toxicity (non-dermatologic).

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Number of Participants That Required a Treatment Break Relative to Hematologic Toxicity (Non-dermatologic)	0 Participants	—	—	—	—

SECONDARY outcome

Timeframe: Completion of study, approximately 14 months after start of treatment

Population: 1/6 participants enrolled withdrew before treatment.

Opiates are strong drugs prescribed by prescription used for maximum pain relief.

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Number of Participants Treated With Topical MTS-01 and 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT) That Required Opiates	5 Participants	—	—	—	—

SECONDARY outcome

Timeframe: 12 months

Population: 1/6 participants enrolled withdrew before treatment.

PFS is defined as the duration of time from start of treatment to time of progression or death, whichever occurs first. Progression was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST0 version 1.1. Progression is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study. And the appearance of one or more new lesions.

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Number of Participants With 12-month Progression-free Survival Treated With 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT)	4 Participants	—	—	—	—

SECONDARY outcome

Timeframe: Completion of study, approximately 14 months

Population: The participants were screened for HIV before enrollment. Only 1 participant was HIV positive. However, the participant with HIV came off study before post treatment was collected so change cannot be assessed. As pre-specified in the protocol, participants are taken off study for disease progression.

Change in the levels of number of Human Immunodeficiency Virus (HIV) in the circulation pre and post treatment

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Pre-treatment and post treatment tissue (CD4), and pre-treatment and post treatment circulation (CD8)

Population: 3/6 participants were analyzed because 1 participant was enrolled but withdrew before treatment, samples were collected at the time of optional biopsy which was conducted in 3 patients.

Ratio of the number Cluster of Differentiation 4 (CD4): Cluster of Differentiation 8 (CD8) cells in the circulation and tissue pre and post treatment. The number of cells of CD4 are divided by the number of cells of CD8.

Outcome measures

Measure	MTS-01 Grade 1 n=3 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Ratio of the Number of Cluster of Differentiation 4 (CD4): Cluster of Differentiation 8 (CD8) Cells in the Circulation and Tissue Pre and Post Treatment CD4:CD8 Pre-treatment tissue	5.2 Ratio of CD4:CD8 cells Interval 3.6 to 7.0	—	—	—	—
Ratio of the Number of Cluster of Differentiation 4 (CD4): Cluster of Differentiation 8 (CD8) Cells in the Circulation and Tissue Pre and Post Treatment CD4:CD8 post treatment tissue	1.89 Ratio of CD4:CD8 cells Interval 0.5 to 3.2	—	—	—	—
Ratio of the Number of Cluster of Differentiation 4 (CD4): Cluster of Differentiation 8 (CD8) Cells in the Circulation and Tissue Pre and Post Treatment CD4:CD8 pre-treatment circulation	2.95 Ratio of CD4:CD8 cells Interval 2.4 to 3.3	—	—	—	—
Ratio of the Number of Cluster of Differentiation 4 (CD4): Cluster of Differentiation 8 (CD8) Cells in the Circulation and Tissue Pre and Post Treatment CD4:CD8 post treatment circulation	1.56 Ratio of CD4:CD8 cells Interval 0.9 to 2.1	—	—	—	—

SECONDARY outcome

Timeframe: 12 months

Population: 1/6 participants enrolled withdrew before treatment.

DFS is defined as the duration of time from start of treatment to time of progression. Progression was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST0 version 1.1. Progression is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study. And the appearance of one or more new lesions.

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Number of Participants With 12 Month Disease Free Survival (DFS) Treated With 5-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT)	4 Participants	—	—	—	—

SECONDARY outcome

Timeframe: start of treatment to time of death, approximately 14 months

Population: This outcome measure could not be completed.

OS is defined as the duration of time from start of treatment to time of death.

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Overall Survival	NA months The overall survival was to be reported as a median. One participant withdrew. Of the 5 remaining participants, none died while on study, one of these patients was removed from study for progression and was censored at that time.	—	—	—	—

SECONDARY outcome

Timeframe: Baseline and Course 1 Day 28

Population: 1/6 participants enrolled withdrew before treatment.

Peripheral blood mononuclear cells (Vascular Endothelial Growth Factor (VEGF), Tumor Necrosis Factor alpha (TNF-alpha), Interleukin-7 (IL-7), and Transforming Growth Factor beta-1 (TGF-beta1) were sampled from peripheral blood from rectal associated lymphoid tissue to evaluate immune cell subsets at baseline and after treatment with MTS-0 and 15-Fluoruracil (5-FU)/Mitomycin C (MMC)/Intensity-modulated Radiotherapy (IMRT). It is unknown if a lower or higher numbers has prognostic significance.

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Peripheral Blood Mononuclear Cells (Vascular Endothelial Growth Factor (VEGF), Tumor Necrosis Factor Alpha (TNF-alpha), Interleukin-7 (IL-7), and Transforming Growth Factor Beta-1 (TGF-beta1) Cell Counts at Baseline and Course 1 Day 28 VEGF at Course 1 Day 28	127.26 pg/ml Standard Deviation 63.6	—	—	—	—
Peripheral Blood Mononuclear Cells (Vascular Endothelial Growth Factor (VEGF), Tumor Necrosis Factor Alpha (TNF-alpha), Interleukin-7 (IL-7), and Transforming Growth Factor Beta-1 (TGF-beta1) Cell Counts at Baseline and Course 1 Day 28 VEGF at baseline	151.24 pg/ml Standard Deviation 45.0	—	—	—	—
Peripheral Blood Mononuclear Cells (Vascular Endothelial Growth Factor (VEGF), Tumor Necrosis Factor Alpha (TNF-alpha), Interleukin-7 (IL-7), and Transforming Growth Factor Beta-1 (TGF-beta1) Cell Counts at Baseline and Course 1 Day 28 TNF-alpha at baseline	3.36 pg/ml Standard Deviation 1.2	—	—	—	—
Peripheral Blood Mononuclear Cells (Vascular Endothelial Growth Factor (VEGF), Tumor Necrosis Factor Alpha (TNF-alpha), Interleukin-7 (IL-7), and Transforming Growth Factor Beta-1 (TGF-beta1) Cell Counts at Baseline and Course 1 Day 28 TNF-alpha at Course 1 Day 28	3.21 pg/ml Standard Deviation 2.5	—	—	—	—
Peripheral Blood Mononuclear Cells (Vascular Endothelial Growth Factor (VEGF), Tumor Necrosis Factor Alpha (TNF-alpha), Interleukin-7 (IL-7), and Transforming Growth Factor Beta-1 (TGF-beta1) Cell Counts at Baseline and Course 1 Day 28 IL-7 at baseline	11.32 pg/ml Standard Deviation 6.2	—	—	—	—
Peripheral Blood Mononuclear Cells (Vascular Endothelial Growth Factor (VEGF), Tumor Necrosis Factor Alpha (TNF-alpha), Interleukin-7 (IL-7), and Transforming Growth Factor Beta-1 (TGF-beta1) Cell Counts at Baseline and Course 1 Day 28 IL-7 at Course 1 Day 28	15.62 pg/ml Standard Deviation 10.3	—	—	—	—
Peripheral Blood Mononuclear Cells (Vascular Endothelial Growth Factor (VEGF), Tumor Necrosis Factor Alpha (TNF-alpha), Interleukin-7 (IL-7), and Transforming Growth Factor Beta-1 (TGF-beta1) Cell Counts at Baseline and Course 1 Day 28 TGF-beta 1 at baseline	14435 pg/ml Standard Deviation 3711.7	—	—	—	—
Peripheral Blood Mononuclear Cells (Vascular Endothelial Growth Factor (VEGF), Tumor Necrosis Factor Alpha (TNF-alpha), Interleukin-7 (IL-7), and Transforming Growth Factor Beta-1 (TGF-beta1) Cell Counts at Baseline and Course 1 Day 28 TGF-beta 1 at Course 1 Day 28	7946 pg/ml Standard Deviation 2729.3	—	—	—	—

SECONDARY outcome

Timeframe: Pretreatment

Population: 1/6 participants enrolled withdrew before treatment.

HPV is a sexually transmitted infection that can cause warts and cervical cancer. HPV test detects deoxyribonucleic acid (DNA) or ribonucleic acid (RNA) of HPV in a cell sample (i.e. cervical).

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Number of Participants With Tumor Tissue Negative for Human Papilloma Virus (HPV)	5 Participants	—	—	—	—

SECONDARY outcome

Timeframe: Baseline, Week 3, Week 6, 1 month follow up, and 3 months follow up

Population: 1/6 participants enrolled withdrew before treatment.

The Brief Pain Inventory Scale is a questionnaire that asks the participant to rate their pain on a scale of 0 (no pain) to 10 (worst pain).

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Brief Pain Inventory Score Average pain at 3 months follow up	3.0 Scores on a scale Interval 0.0 to 6.0	—	—	—	—
Brief Pain Inventory Score Pain at time of survey at baseline	1.6 Scores on a scale Interval 0.0 to 6.0	—	—	—	—
Brief Pain Inventory Score Pain at time of survey at treatment week 3	1.3 Scores on a scale Interval 0.0 to 5.0	—	—	—	—
Brief Pain Inventory Score Pain at time of survey at treatment week 6	5.4 Scores on a scale Interval 2.0 to 9.0	—	—	—	—
Brief Pain Inventory Score Worst pain at baseline	4.8 Scores on a scale Interval 0.0 to 10.0	—	—	—	—
Brief Pain Inventory Score Worst pain at treatment week 3	3.5 Scores on a scale Interval 0.0 to 8.0	—	—	—	—
Brief Pain Inventory Score Worst pain at treatment week 6	7.2 Scores on a scale Interval 4.0 to 10.0	—	—	—	—
Brief Pain Inventory Score Worst pain at 1 month follow up	3.1 Scores on a scale Interval 0.0 to 9.0	—	—	—	—
Brief Pain Inventory Score Worst pain at 3 months follow up	2.1 Scores on a scale Interval 0.0 to 5.0	—	—	—	—
Brief Pain Inventory Score Least pain at baseline	1.4 Scores on a scale Interval 0.0 to 4.0	—	—	—	—
Brief Pain Inventory Score Least pain at treatment week 3	1.5 Scores on a scale Interval 0.0 to 4.0	—	—	—	—
Brief Pain Inventory Score least pain at treatment week 6	3.6 Scores on a scale Interval 1.0 to 5.0	—	—	—	—
Brief Pain Inventory Score Least pain at 1 month follow up	1.2 Scores on a scale Interval 0.0 to 3.0	—	—	—	—
Brief Pain Inventory Score Least pain at 3 months follow up	2.8 Scores on a scale Interval 0.0 to 5.0	—	—	—	—
Brief Pain Inventory Score Average pain at baseline	1.8 Scores on a scale Interval 0.0 to 4.0	—	—	—	—
Brief Pain Inventory Score Average pain at treatment week 3	1.9 Scores on a scale Interval 0.0 to 3.5	—	—	—	—
Brief Pain Inventory Score Average pain at treatment week 6	5.0 Scores on a scale Interval 2.0 to 7.0	—	—	—	—
Brief Pain Inventory Score Average pain at 1 month follow up	2.0 Scores on a scale Interval 0.0 to 6.0	—	—	—	—
Brief Pain Inventory Score Pain at time of survey a 1 month follow up	2.2 Scores on a scale Interval 0.0 to 6.0	—	—	—	—
Brief Pain Inventory Score Pain at time of survey at 3 months follow up	2.2 Scores on a scale Interval 0.0 to 5.0	—	—	—	—

SECONDARY outcome

Timeframe: Baseline, Week 3, Week 6, 1 month follow up, and 3 months follow up

Population: 1/6 participants enrolled withdrew before treatment.

The Brief Pain Inventory Scale questionnaire were used to assess pain interference. Participants rated pain interference on a scale of 0 (does not interfere) to 10 (completely interferes).

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Pain Interference Pain interference at treatment week 3	3.00 Scores on a scale Interval 0.44 to 8.0	—	—	—	—
Pain Interference Pain interference at treatment week 6	6.70 Scores on a scale Interval 2.89 to 9.44	—	—	—	—
Pain Interference Pain interference at 1 month follow up	3.17 Scores on a scale Interval 0.0 to 8.33	—	—	—	—
Pain Interference Pain interference at baseline	1.73 Scores on a scale Interval 0.33 to 3.89	—	—	—	—
Pain Interference Pain interference at 3 months follow up	1.87 Scores on a scale Interval 0.0 to 4.56	—	—	—	—

SECONDARY outcome

Timeframe: Baseline, Week 3, Week 6, 1 month follow up, and 3 months follow up

Population: 1/6 participants enrolled withdrew before treatment.

The Brief Pain Inventory Scale questionnaire were used to assess pain relief after medication. Participants rated pain relief on a scale of 0% (no relief) to 100% (complete relief) and a mean and full range were reported.

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Mean Percentage Pain Relief After Medication Baseline	90.0 percentage pain relief Interval 60.0 to 100.0	—	—	—	—
Mean Percentage Pain Relief After Medication Treatment week 3	93.8 percentage pain relief Interval 85.0 to 100.0	—	—	—	—
Mean Percentage Pain Relief After Medication Treatment week 6	44.0 percentage pain relief Interval 10.0 to 90.0	—	—	—	—
Mean Percentage Pain Relief After Medication 1 month follow up	89.0 percentage pain relief Interval 75.0 to 100.0	—	—	—	—
Mean Percentage Pain Relief After Medication 3 months follow up	83.0 percentage pain relief Interval 50.0 to 100.0	—	—	—	—

SECONDARY outcome

Timeframe: 12 months of follow up, approximately 14 months

Population: This outcome measure was not measurable.

Duration of overall response is measured from the time measurement criteria are met for Complete Response until the first date that recurrent or progressive disease is objectively document. Complete Response was measured by the Response Evaluation Criteria in Solid Tumors (RECIST0 version 1.1. Complete Response is disappearance of all target lesions.

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Duration of Overall Response (DOR)	NA months 1/6 participants enrolled withdrew before treatment. 1 participant never reached complete response. DOR was censored for all responders due to completion of follow up \& median censoring time is 329 days. Median cannot be calculated as the median was not reached. A median cannot be reported unless 50% of participants have reached the endpoint. 50% did not reach the endpoint and came off study for complete follow-up.	—	—	—	—

SECONDARY outcome

Timeframe: Baseline and 1 year

Population: 1/6 participants enrolled withdrew before treatment. 3 participants underwent optional biopsy.

Percentage of total viable cells that were Cluster of differentiation 3+ (CD3+) cells in biopsied tissue.

Outcome measures

Measure	MTS-01 Grade 1 n=3 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Percentage of Total Viable Cells That Were Cluster of Differentiation 3+ (CD3+) Cells in Biopsied Tissue Baseline	7.93 percentage of cells Interval 5.69 to 9.1	—	—	—	—
Percentage of Total Viable Cells That Were Cluster of Differentiation 3+ (CD3+) Cells in Biopsied Tissue 1 year	4.40 percentage of cells Interval 2.4 to 5.7	—	—	—	—

SECONDARY outcome

Timeframe: Baseline and 1 year follow up

Population: 1/6 participants enrolled withdrew before treatment. 3 participants underwent optional biopsy.

Absolute number of Cluster of differentiation 3+ (CD3) cells per gram of biopsied intestinal tissue. The number of CD3 positive cells were analyzed with flow cytometry of cellular suspensions from biopsy tissue.

Outcome measures

Measure	MTS-01 Grade 1 n=3 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Absolute Number of Cluster of Differentiation 3+ (CD3) Cells Per Gram of Biopsied Intestinal Tissue Baseline	22.98 CD3+cells/gm tissue(x 10^5) Interval 12.1 to 29.3	—	—	—	—
Absolute Number of Cluster of Differentiation 3+ (CD3) Cells Per Gram of Biopsied Intestinal Tissue 1 year follow up	13.62 CD3+cells/gm tissue(x 10^5) Interval 5.94 to 21.6	—	—	—	—

SECONDARY outcome

Timeframe: Baseline and 1 year follow up

Population: 1/6 participants enrolled withdrew before treatment. 3 participants underwent optional biopsy.

Number of Cluster of differentiation 8+ (CD8) cells per gram of biopsied intestinal tissue. The number of CD8 positive cells were analyzed with flow cytometry of cellular suspensions from biopsy tissue.

Outcome measures

Measure	MTS-01 Grade 1 n=3 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Number of Cluster of Differentiation 8+ (CD8) Cells Per Gram of Biopsied Intestinal Tissue Baseline	4.8 CD8+cells/gm tissue(x 10^5) Interval 1.4 to 5.6	—	—	—	—
Number of Cluster of Differentiation 8+ (CD8) Cells Per Gram of Biopsied Intestinal Tissue 1 year follow up	4.0 CD8+cells/gm tissue(x 10^5) Interval 1.77 to 5.58	—	—	—	—

SECONDARY outcome

Timeframe: Baseline and 1 year follow up

Population: 1/6 participants enrolled withdrew before treatment.

Number of Cluster of Differentiation 4+ (CD4) cells per gram of biopsied intestinal tissue. The number of CD4 positive cells were analyzed with flow cytometry of cellular suspensions from biopsy tissue.

Outcome measures

Measure	MTS-01 Grade 1 n=5 Participants Grade 1 is mild adverse events.	MTS-01 Grade 2 Grade 2 is moderate adverse events.	MTS-01 Grade 3 Grade 3 is severe adverse events.	5FU/MMC/IMRT Grade 2 Grade 2 is moderate adverse events.	5-FU/MMC/IMRT Grade 3-4 Grade 3 is severe adverse events and Grade 4 is life threatening adverse events.
Number of Cluster of Differentiation 4+ (CD4) Cells Per Gram of Biopsied Intestinal Tissue Baseline	17.64 CD4+cells/gm tissue(x 10^5) Interval 9.54 to 23.2	—	—	—	—
Number of Cluster of Differentiation 4+ (CD4) Cells Per Gram of Biopsied Intestinal Tissue 1 year follow up	12.72 CD4+cells/gm tissue(x 10^5) Interval 3.5 to 14.5	—	—	—	—

SECONDARY outcome

Timeframe: Baseline and Course 1 Day 28

Population: This outcome measure could not be completed. 1/6 participants enrolled withdrew before treatment. No participants with HIV ribonucleic acid (RNA) underwent optional biopsy. HIV RNA is not analyzed unless a patient has HIV. Thus, data not collected.

Optional snag biopsies pre and post were collected from participants rectal mucosa to enumerate cell counts.

Outcome measures

Outcome data not reported

Adverse Events

1/Chemo + Radiation

Serious events: 3 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Measure	1/Chemo + Radiation n=5 participants at risk Chemo + Radiation Tempol: Tempol gel will be applied to the bilateral groins and the gluteal cleft, avoiding a 3 cm radius from the anal verge, immediately prior to each fraction of radiation therapy (RT). 5-Fluorouracil: 5-FU will be delivered as 1000mg/m(2)/day as 96 hour continuous infusion beginning on day 1 and 29. Mitomycin-C: Mitomycin-C (MMC) will be delivered at a dose of 10mg/m(2) on days 1 and 29 Radiation Therapy: Radiation therapy (RT) will be delivered to a total dose of 50-54 Gray (Gy) based on tumor characteristics.
Metabolism and nutrition disorders Dehydration	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Respiratory, thoracic and mediastinal disorders Dyspnea	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Investigations Febrile neutropenia	40.0% 2/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
General disorders Fever	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Vascular disorders Hypotension	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Investigations Neutrophil count decreased	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Infections and infestations Skin infection	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Vascular disorders Thromboembolic event	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.

Other adverse events

Measure	1/Chemo + Radiation n=5 participants at risk Chemo + Radiation Tempol: Tempol gel will be applied to the bilateral groins and the gluteal cleft, avoiding a 3 cm radius from the anal verge, immediately prior to each fraction of radiation therapy (RT). 5-Fluorouracil: 5-FU will be delivered as 1000mg/m(2)/day as 96 hour continuous infusion beginning on day 1 and 29. Mitomycin-C: Mitomycin-C (MMC) will be delivered at a dose of 10mg/m(2) on days 1 and 29 Radiation Therapy: Radiation therapy (RT) will be delivered to a total dose of 50-54 Gray (Gy) based on tumor characteristics.
Gastrointestinal disorders Abdominal pain	40.0% 2/5 • Number of events 4 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Investigations Alanine aminotransferase increased	80.0% 4/5 • Number of events 7 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Investigations Alkaline phosphatase increased	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Skin and subcutaneous tissue disorders Alopecia	40.0% 2/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Anal hemorrhage	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Anal pain	20.0% 1/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Blood and lymphatic system disorders Anemia	80.0% 4/5 • Number of events 8 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Metabolism and nutrition disorders Anorexia	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Investigations Aspartate aminotransferase increased	60.0% 3/5 • Number of events 4 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Renal and urinary disorders Bladder spasm	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Eye disorders Blurred vision	40.0% 2/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Musculoskeletal and connective tissue disorders Bone pain	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Investigations CD4 lymphocytes decreased	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
General disorders Chills	40.0% 2/5 • Number of events 3 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Constipation	40.0% 2/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Injury, poisoning and procedural complications Dermatitis radiation	100.0% 5/5 • Number of events 19 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Diarrhea	80.0% 4/5 • Number of events 14 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Nervous system disorders Dizziness	40.0% 2/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Dry mouth	40.0% 2/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Dyspepsia	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Reproductive system and breast disorders Ejaculation disorder	20.0% 1/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Respiratory, thoracic and mediastinal disorders Epistaxis	20.0% 1/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
General disorders Fatigue	100.0% 5/5 • Number of events 8 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Fecal incontinence	20.0% 1/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
General disorders Fever	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Flatulence	60.0% 3/5 • Number of events 5 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Gastroesophageal reflux disease	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Gastrointestinal disorders - Other, Tongue deviation	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Nervous system disorders Headache	60.0% 3/5 • Number of events 8 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Hemorrhoidal hemorrhage	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Vascular disorders Hot flashes	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Metabolism and nutrition disorders Hyperglycemia	40.0% 2/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Metabolism and nutrition disorders Hypermagnesemia	40.0% 2/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Metabolism and nutrition disorders Hypoalbuminemia	100.0% 5/5 • Number of events 8 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Metabolism and nutrition disorders Hypocalcemia	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Metabolism and nutrition disorders Hypoglycemia	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Metabolism and nutrition disorders Hyponatremia	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Vascular disorders Hypotension	20.0% 1/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Psychiatric disorders Insomnia	60.0% 3/5 • Number of events 3 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Investigations Lymphocyte count decreased	100.0% 5/5 • Number of events 34 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Nervous system disorders Memory impairment	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Infections and infestations Mucosal infection	40.0% 2/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Mucositis oral	60.0% 3/5 • Number of events 4 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Musculoskeletal and connective tissue disorders Myalgia	20.0% 1/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Nausea	80.0% 4/5 • Number of events 8 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Investigations Neutrophil count decreased	100.0% 5/5 • Number of events 15 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Oral dysesthesia	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Skin and subcutaneous tissue disorders Pain of skin	20.0% 1/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Reproductive system and breast disorders Perineal pain	40.0% 2/5 • Number of events 4 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Eye disorders Photophobia	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Investigations Platelet count decreased	100.0% 5/5 • Number of events 10 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Skin and subcutaneous tissue disorders Pruritus	40.0% 2/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Rectal pain	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, Clamminess	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, carbunkle	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, sweating	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Infections and infestations Skin infection	20.0% 1/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Nervous system disorders Syncope	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Renal and urinary disorders Urinary frequency	60.0% 3/5 • Number of events 3 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Renal and urinary disorders Urinary incontinence	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Infections and infestations Urinary tract infection	20.0% 1/5 • Number of events 2 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Renal and urinary disorders Urinary tract pain	80.0% 4/5 • Number of events 7 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Renal and urinary disorders Urinary urgency	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Reproductive system and breast disorders Vaginal pain	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Gastrointestinal disorders Vomiting	40.0% 2/5 • Number of events 3 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.
Investigations White blood cell decreased	100.0% 5/5 • Number of events 22 • Date treatment consent signed to date off study, approximately, 36 months and 10 days. 1/6 participants enrolled withdrew before treatment.

Additional Information

Dr. Deborah E. Citrin

National Cancer Institute

Phone: 301-496-5457

Email: citrind@mail.nih.gov

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place