Safety Study of BMX-001 (Radio-protector) in Patients With Newly Diagnosed Anal Cancer

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Anal Cancer, Squamous Cell Carcinoma, Radiation Exposure

In brief

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.

Key facts

Study ID: NCT03386500
Run by: University of Nebraska
People needed: 24
Starts: 2017-11-28
Expected to finish: 2029-12-01
Last updated by the study team: 2026-05-18

Who can join

Age: 19 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Pathologically confirmed locally advanced anal squamous cell carcinoma (including oligometastatic disease) with concurrent chemoradiation with standard Fluorouracil (5FU)/mitomycin regimen with curative intent
Any cancer stage requiring a dose of 59.4 cGy
19 years of age or older
ECOG Performance Status (ECOG) ≥ 2
Hemoglobin ≥ 9.0 g/dl (a transfusion or other intervention to achieve Hgb > 9.0 g/dl is acceptable)
Absolute neutrophil count (ANC) ≥ 1,500 /dl
Platelets ≥ 100,000 /dl
Serum creatinine ≤ 1.5 mg/dl, serum SGOT and bilirubin ≤ 1.5 times upper limit of normal
Negative pregnancy test for women of child-bearing potential within 48 hours prior to first dose of BMX-001
Use of a medically effective means of birth control until 12 months following the last study treatment for women of childbearing potential and male participants
Positron Emission Tomography (PET)/ Computed Tomography (CT)/pelvic magnetic resonance imaging (MRI) done within 8 weeks of trial initiation

You may not qualify if…

Breast-feeding
Active infection requiring IV antibiotics within 7 days before enrollment
Prior, unrelated malignancy requiring current active treatment, exception: cervical carcinoma in situ, basal cell or carcinoma of the skin, invasive cancers with a 5-year disease-free interval, resected cancer of the bladder, or low-grade prostate cancer (Gleason 6 or less)
Prior history of Acantholytic squamous cell carcinoma (ASCC)
Prior history of pelvic radiotherapy for any other type of malignancy
Known hypersensitivity to Fluorouracil (5FU) and/or mitomycin
Current corticosteroid use unless dose is stable or decreasing at study enrollment (anti-inflammatory properties could interrupt oxidative stress)
Inadequately controlled hypertension (systolic blood pressure >150 mmHg and/or diastolic blood pressure > 100 mmHg)
Active or history of postural hypotension and autonomic dysfunction within the past year
Known hypersensitivity to BMX-001
Clinically significant (active) cardiovascular disease or cerebrovascular disease, (e.g., cerebrovascular accidents ≤ 6 months prior to study enrollment, myocardial infarction ≤ 6 months prior to study enrollment, unstable angina, New York Heart Association (NYHA) Grade II or greater congestive heart failure (CHF), or serious cardiac arrhythmia uncontrolled by medication or potentially interfering with protocol treatment
History or evidence from physical/neurological examination of central nervous system disease (e.g. seizures) unrelated to cancer, potentially interfering with protocol treatment unless adequately controlled by medication
Significant vascular disease (e.g., aortic aneurysm requiring surgical repair or recent arterial thrombosis, including structural heart disease) within 6 months prior to start of study treatment
Marked baseline prolongation of QT/QTc interval [e.g., repeated demonstration of a QTc interval >450 milliseconds (ms), CTCAE grade 1, using the specific/usual choice by clinical center for correction factor
History of additional risk factors for Torsades de Pointes (TdP) (e.g., congestive heart failure, hypokalemia, known family history of Long QT Syndrome)
Documented recent electrolyte disturbances (hypokalemia, hypomagnesemia, or hypocalcemia) that were clinically significant and not corrected. No additional laboratory testing is required solely for screening.
Medical necessity for anti-arrhythmics with significant risk of QTc prolongation such as class I and class III anti-arrhythmics. These include but are not limited to amiodarone, quinidine, dofetilide, sotalol, flecainide, and lidocaine

Where it is running

University of Nebraska Medical Center — Omaha, Nebraska, United States

Full record on ClinicalTrials.gov

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