Trial of the Efficacy and Safety of Short and Long Course Radiation Therapy With/Without BMX-001

NCT05254327 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 0012-22-FBBMX-RAC-001
• Study Type: interventional
• Target: 118
• Locations: 1 country
• Sites: 3

Brief Summary

In this Phase 2 study, we will conduct an efficacy and safety study of the combination of investigational drug BMX-001, with short-course radiotherapy (SCRT) or long-course chemoradiotherapy (LCCRT) as part of total neoadjuvant therapy in newly diagnosed rectal adenocarcinoma (RAC) patients.

Conditions

Rectal Cancer

Keywords

Radiation

Outcome Measures

Primary Outcomes (4)

• Efficacy of BMX-001 as measured by Grade 3 and above associated with gastrointestinal Toxicities

  Grade 3 and above radiation-associated gastrointestinal, genitourinary, skin and hematologic toxicity (SCRT or LCCRT) including frequency, severity and duration of rectal bleeding, rectal pain, diarrhea, dysuria, hematuria, urinary frequency, radiation dermatitis and thrombocytopenia

  Three weeks (During and 2 weeks after RT)

• Efficacy of BMX-001 as measured by Grade 3 and above associated with genitourinary Toxicities

  Grade 3 and above radiation-associated gastrointestinal, genitourinary, skin and hematologic toxicity (SCRT or LCCRT) including frequency, severity and duration of rectal bleeding, rectal pain, diarrhea, dysuria, hematuria, urinary frequency, radiation dermatitis and thrombocytopenia

  Three weeks (During and 2 weeks after RT)

• Efficacy of BMX-001 as measured by Grade 3 and above associated with skin Toxicities

  Grade 3 and above radiation-associated gastrointestinal, genitourinary, skin and hematologic toxicity (SCRT or LCCRT) including frequency, severity and duration of rectal bleeding, rectal pain, diarrhea, dysuria, hematuria, urinary frequency, radiation dermatitis and thrombocytopenia

  Three weeks (During and 2 weeks after RT)

• Efficacy of BMX-001 as measured by Grade 3 and above associated with hematologic Toxicities

  Grade 3 and above radiation-associated gastrointestinal, genitourinary, skin and hematologic toxicity (SCRT or LCCRT) including frequency, severity and duration of rectal bleeding, rectal pain, diarrhea, dysuria, hematuria, urinary frequency, radiation dermatitis and thrombocytopenia

  Three weeks (During and 2 weeks after RT)

Study Arms (4)

Arm A

EXPERIMENTAL

Short Chemo-Radiation Therapy (SCRT) + BMX-001

Drug: BMX-001

Arm B

NO INTERVENTION

Short Chemo-Radiation Therapy (SCRT)

Arm C

EXPERIMENTAL

Long Course Chemo-Radiation Therapy (LCCRT) + BMX-001

Drug: BMX-001

Arm D

NO INTERVENTION

Long Course Chemo-Radiation Therapy (LCCRT)

Interventions

BMX-001 DRUG

Loading dose of 28 mg per subject, followed by maintenance doses of 14 mg per subject twice per week.

Arm A; Arm C

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with pathologically confirmed locally advanced rectal adenocarcinoma who will be receiving total neoadjuvant therapy regimen with curative intent.
• AJCC stage II to III rectal adenocarcinoma that will require total neoadjuvant therapy.
• Adult, age \> or equal to 18 years (for Nebraska, age of consent is ≥19 years old)
• ECOG Performance Status 0-2
• Hemoglobin ≥ 9.0 g/dl, ANC ≥ 1,500 /dl, platelets ≥ 100,000 /dl (The use of transfusion or other intervention to achieve Hgb \> 9.0 g/dl is acceptable)
• Serum SGOT and bilirubin ≤ 1.5 times upper limit of normal
• Adequate renal function defined as follows:
• )Serum creatinine \< 1.5 mg/dl within 2 weeks prior to enrollment or 2)Creatinine clearance (CC) ≥ 50 ml/min within 2 weeks prior to enrollment determined by 24-hour collection or estimated by Cockcroft-Gault formula: CCr male = \[(140 - age) x (wt in kg)\]/\[(Serum Cr mg/dl) x (72)\], CCr female = 0.85 x (CrCl male) 8. Signed, written informed consent prior to completing any study specific procedures 9. Negative pregnancy test for women of child-bearing potential at the time of screening 10. Women of childbearing potential and male participants must agree to use two forms of a medically effective means of birth control throughout their participation in the treatment phase of the study and until 12 months following the last study treatment 11. Chest/Abdominal/Pelvic (CAP) CT/ pelvic MRI done within 8 weeks prior to randomization.

You may not qualify if:

• Breast-feeding or pregnant
• Active infection requiring IV antibiotics 7 days before enrollment
• Prior, unrelated malignancy requiring current active treatment with the exception of 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 (Gleason 6 or less) prostate cancer
• Prior history of rectal adenocarcinoma (RAC)
• Prior history of pelvic radiotherapy for any other type of malignancy
• Known hypersensitivity or contraindication to any agent in FOLFOX or CAPOX regimen.
• Because corticosteroids are anti-inflammatory and could interrupt oxidative stress, patients will be excluded unless they are on stable or decreasing corticosteroids dose at the time of randomization.
• Inadequately controlled hypertension (defined as 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 (i.e. active) cardiovascular disease or cerebrovascular disease, for example 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 upon physical/neurological examination of central nervous system disease (e.g. seizures) unrelated to cancer unless adequately controlled by medication or potentially interfering with protocol treatment
• Significant vascular disease (e.g., aortic aneurysm requiring surgical repair or recent arterial thrombosis) within 6 months prior to start of study treatment
• A 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.
• A history of additional risk factors for Torsades de Pointes (TdP) (e.g., congestive heart failure, hypokalemia, known family history of Long QT Syndrome).

Sponsors & Collaborators

• University of Nebraska: lead
• BioMimetix JV, LLC: collaborator

Study Sites (3)

Markey Cancer Center

Lexington, Kentucky, 40536, United States

RECRUITING

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

RECRUITING

UT Health San Antonio MD Anderson Cancer Center

San Antonio, Texas, 78229, United States

RECRUITING

MeSH Terms

Conditions

Rectal Neoplasms

Condition Hierarchy (Ancestors)

Colorectal Neoplasms; Intestinal Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Digestive System Diseases; Gastrointestinal Diseases; Intestinal Diseases; Rectal Diseases

Study Officials

• Chi Lin, MD, PhD

  University of Nebraska

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jessi E Delaney, RN, BSN

CONTACT

402-599-8711; jessdelaney@unmc.edu

Samuel P Anderson, BS

CONTACT

402-559-1250; samuanderson@unmc.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Masking Details: Lab performing PK sample analysis will be blinded to the subjects and relationship of specimens to treatment with BMX-001
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 14, 2022
• First Posted: February 24, 2022
• Study Start: August 15, 2022
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): June 1, 2029
• Last Updated: August 7, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)