A Phase 1/2 interventional study of BMX-001 in Anal Cancer, Squamous Cell Carcinoma and Radiation Exposure, sponsored by University of Nebraska. Active, not recruiting at 1 site in United States. Open to participants aged 19 Years and older. Per ClinicalTrials.gov, last updated 2026-05-18.
Sponsored by University of Nebraska · Phase 1/2, Interventional, and Treatment
Over 80% of anal cancers are squamous cell carcinoma (SCC). Current standard treatment for locally advanced squamous cell carcinoma of the anal canal is a combination of radiation therapy (RT) and concurrent chemotherapy. This allows for organ preservation in approximately 75% of patients. The use of concurrent radiation and chemotherapy with infusional 5-fluorouracil (5-FU) and mitomycin results in locoregional relapse rates of 20-32 and 5-year overall survival rates of 58-78%. However, while mitomycin significantly increases the rate of grade 4 toxicities, it improves local outcomes and has been considered a necessary agent in the care of anal cancer. Oxidative stress induced by radiotherapy and chemotherapy tends to protect tumor cells and promote normal tissue damage. A recently developed compound, BMX-001 (MnTnBuOE-2- PyP5+), is among the most highly potent metalloporphyrin compounds which reduce oxidative stress, thereby protecting normal tissues and augmenting tumor killing.
In this Phase 1/2 study, the investigators will conduct a safety and efficacy study of the combination of BMX-001 with standard radiation therapy and concurrent (5FU)/mitomycin in newly diagnosed Anal Squamous Cell Carcinoma (ASCC) patients. The primary Phase 1 objective is to determine the maximum tolerated dose (MTD) of BMX-001 in ASCC patients receiving RT and concurrent 5FU/mitomycin chemotherapy. Three participants will be treated at Dose Level 1 and three at Dose Level 2, then three at Dose Level 3. Dose Limiting Toxicities (DLT) experienced by any participant will be used to determine the MTD. The Phase II objective is to examine the impact of BMX-001 on the overall acute ≥ grade 3 toxicity rate of the normal tissue including rectum, bladder, and skin in combination with RT and concurrent 5FU/mitomycin in treatment of newly diagnosed ASCC patients. These will be determined by participant reports, biological materials (blood, tissue, urine) sampling and imaging. Participant health-related quality of life will be assessed by two questionnaires.
Over 80% of anal cancers are squamous cell carcinoma (SCC). Current standard treatment for locally advanced squamous cell carcinoma of the anal canal is a combination of radiation therapy (RT) and concurrent chemotherapy. This allows for organ preservation in approximately 75% of patients. The use of concurrent radiation and chemotherapy with infusional 5-fluorouracil (5-FU) and mitomycin results in locoregional relapse rates of 20-32 and 5-year overall survival rates of 58-78%. However, while mitomycin significantly increases the rate of grade 4 toxicities, it improves local outcomes and has been considered a necessary agent in the care of anal cancer. Oxidative stress induced by radiotherapy and chemotherapy tends to protect tumor cells and promote normal tissue damage. A recently developed compound, BMX-001 (MnTnBuOE-2- PyP5+), is among the most highly potent metalloporphyrin compounds which reduce oxidative stress, thereby protecting normal tissues and augmenting tumor killing.
In this Phase 1/2 study, the investigators will conduct a safety and efficacy study of the combination of BMX-001 with standard radiation therapy (RT) and concurrent 5-fluorouracil (5FU)/mitomycin in newly diagnosed anal squamous cell carcinoma (ASCC) participant. A recently completed Phase 1 study using BMX-001 with concurrent chemotherapy and radiation therapy in patient with newly diagnosed high-grade gliomas (BMX-HGG-001) demonstrated no adverse effects in subcutaneous dosing up to 28 mg/subject load with half the loading dose (14 mg/subject) given biweekly for 8 weeks in 12 participants. One of three participants dosed with 42 mg/subject load with half the loading dose given biweekly for 8 weeks experienced dose-limiting toxicity (DLT) of grade 3 tachycardia and grade 3 hypotension at loading dose. These resolved with treatment within 24 hours and the participant returned to the study with no recurrence for the maintenance does. The sponsor determined that the Recommended Phase 2 Dose (RP2D) of BMX-001 is 28 mg/subject load followed by 14 mg/subject twice a week for up to eight weeks as the maximum dose that was tolerated with no adverse effects. The most common related toxicity seen in this study grade 1 injection site reaction. There is no apparent toxicity to end organ tissues or bone marrow.
For this study, after completion of Phase 1 and establishment of a RP2D for the treatment regimen in participants undergoing radiation therapy and chemotherapy, the protocol will proceed to Dose Level 3, after completion of Dose Level 1 and Dose Level 2. Up to 20 participants will be enrolled with a safety lead-in of 6 participants to confirm safety in subjects in this cohort receiving radiation therapy and 5FU/mitomycin. To evaluate the pharmacokinetics (PK) of BMX-001 in combination with current chemoradiation, blood samples will be drawn for analysis in three to six participants in enrolled in the safety lead-in. Next, the first dose of BMX-001 will be administered subcutaneously from 4 days up to 1 hour prior to the start of radiation treatment. Blood will be drawn for PK on the following days: Day 1 (before and after loading dose), Day 8, Day 22 and Day 36. Measures will be obtained at approximately the following times: -1 to 0 hour (before loading dose), 30 minutes after the drug is given, 4 hours post-dose, and 24 hours post-dose. Samples will be analyzed for BMX-001 using validated analytical methods. Skin, gastrointestinal (GI), and genitourinary (GU) symptoms will be measured on the day of screening, weekly during RT, 1 months, 4 months and 10 months after the completion of RT. Perianal skin will be assessed weekly during RT, 1 month, 4 months, and 10 months after completion of RT. Further follow up will be per standard of care.
297 studies on the registry are indexed under Anus Neoplasms; 74 are open to participants now.
This study's enrollment of 24 is below the median of 66 across 211 interventional studies indexed under Anus Neoplasms.
Browse Anus Neoplasms studies →University of Nebraska is the lead sponsor of 496 studies on the registry; 67 are open to participants now.
Of its 83 completed or terminated interventional studies of FDA-regulated products, 53 (64%) have results posted.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CTCAE grade
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
One arm includes all enrolled patients.
Drug: BMX-001
BMX-001 at escalating doses in combination with standard RT, 33 fractions to 59.4 Gy, plus Mitomycin at the standard dosing of 10 mg/m2 administered IV bolus at day 1 and day 29 as well as 5FU 1g/m2/d day 1-4 and 29-32.
Also known as: MnTnBuOE-2-PyP5+, manganese butoxyethyl pyridyl porphyrin
Maximum Tolerated Dose of BMX-001
Maximum tolerated dose (MTD) of BMX-001 will be determined by the highest dose level at which at least 1 out of 6 participants experienced a Dose-Limiting Toxicity (DLT).
Time frame: Within first year of the study
Count of Adverse Events, Serious Adverse Events and Dose Limiting Toxicities
Acute grade 3 of normal tissue for adverse events (AE), serious adverse events (SAE) and dose limiting toxicities (DLT) will be recorded per Common Terminology Criteria for Adverse Events (CTCAE) 4.0
Time frame: 10 months post-radiation therapy
Impact of Study Treatment on Acute Rectal Bleeding
Acute grade 3 of normal tissue for rectal bleeding will be recorded per Common Terminology Criteria for Adverse Events (CTCAE) 4.0.
Time frame: 10 months post-radiation therapy
Impact of Study Treatment on Acute Rectal Pain
Acute grade 3 of normal tissue for rectal pain will be recorded per Common Terminology Criteria for Adverse Events (CTCAE) 4.0.
Time frame: 10 months post-radiation therapy
Impact of Study Treatment on Bowel Movements
Acute grade 3 of normal tissue for diarrhea will be recorded per Common Terminology Criteria for Adverse Events (CTCAE) 4.0.
Time frame: 10 months post-radiation therapy
Impact of Study Treatment on Acute Dysuria
Acute grade 3 of normal tissue for dysuria will be recorded per Common Terminology Criteria for Adverse Events (CTCAE) 4.0.
Time frame: 10 months post-radiation therapy
Impact of Study Treatment on Acute Hematuria
Acute grade 3 of normal tissue for hematuria will be recorded per Common Terminology Criteria for Adverse Events (CTCAE) 4.0.
Time frame: 10 months post-radiation therapy
Impact of Study Treatment on Acute Urinary Frequency
Acute grade 3 of normal tissue for urinary frequency will be recorded per Common Terminology Criteria for Adverse Events (CTCAE) 4.0.
Time frame: 10 months post-radiation therapy
Impact of Study Treatment on Acute Perianal Grade 3 Radiation Dermatitis
Acute grade 3 of normal tissue for acute perianal grade 3 radiation dermatitis will be recorded per Common Terminology Criteria for Adverse Events (CTCAE) 4.0.
Time frame: 10 months post-radiation therapy
Impact of Study Treatment on Late Rectal Bleeding
Acute grade 3 of normal tissue for late rectal bleeding per will be recorded per Common Terminology Criteria for Adverse Events (CTCAE) 4.0.
Time frame: 10 months post-radiation therapy
Impact of Study Treatment on Rectal Fibrosis
Acute grade 3 of normal tissue for rectal fibrosis will be evaluated by endoscopy.
Time frame: 10 months post-radiation therapy
Effect of Study Treatment on Local Control
The local recurrence rate will be assessed.
Time frame: 10 months post-radiation therapy
Effect of Study Treatment on Overall Survival
Overall survival (OS) rate will be determined.
Time frame: 10 months post-radiation therapy
Effect of Study Treatment on Locoreginal Progression-free Survival
Locoreginal progression-free survival rate survival (PFS) will determined.
Time frame: 10 months post-radiation therapy
Effect of Study Treatment on Urine Levels of 4-hydroxynonenal
4-hydroxynonenal (4-HNE) will be measured in urine.
Time frame: 4 months post-radiation therapy
Effect of Study Treatment on Plasma Levels of 4-hydroxynonenal
4-hydroxynonenal (4-HNE) will be measured in plasma.
Time frame: 4 months post-radiation therapy
Effect of Study Treatment on Serum Level of 8-OHdG
8-OHdG will be measured in serum.
Time frame: 4 months post-radiation therapy
Effect of Study Treatment on Blood Cells Level of 8-OHdG
8-OHdG will be measure in blood cells.
Time frame: 4 months post-radiation therapy
Effect of Study Treatment on Urine Level of 8-OHdG
8-OHdG will be measured in urine.
Time frame: 4 months post-radiation therapy
Effect of Study Treatment on Urine Levels of Malondialdehyde
Malondialdehyde (MDA) will be measured in urine.
Time frame: 4 months post-radiation therapy
Effect of Study Treatment on Plasma Levels of Malondialdehyde
Malondialdehyde (MDA) will be measured in plasma.
Time frame: 4 months post-radiation therapy
Single-dose and Repeated-dose Pharmacokinetic Profiles of BMX-001
Serum drug concentration of BMX-001 will be measured after single-dose and repeated-dose BMX-001 delivery.
Time frame: Up to 36 days of the chemoradiation phase
Effect of Study Treatment on Participant-reported Outcomes of Health-related Quality of Life
Participant-reported outcomes will be evaluated with the EORTC QLQ-CR29 questionnaire. This instrument consists of five functional scales, four subscales and 19 single items related to various colorectal cancer-related symptoms and functional problems. Scores can be linearly transformed to a range from 0 to 100, where higher scores generally indicate better functioning or a higher level of symptoms.
Time frame: 10 months post-radiation therapy
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
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Studies of the drug tested here: BMX-001