Active — Not RecruitingPhase 1/2

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

United States24 participantsStarted 2017-11-28

Plain-language summary

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.

Who can participate

Age range19 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * 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 * Karnofsky Performance Status (KPS) ≥ 60% * 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 Exclusion Criteria: * 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 hist…

What they're measuring

Maximum Tolerated Dose of BMX-001

Timeframe: Within first year of the study

Count of Adverse Events, Serious Adverse Events and Dose Limiting Toxicities

Timeframe: 10 months post-radiation therapy

Trial details

NCT IDNCT03386500

SponsorUniversity of Nebraska

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-07