NCT05254327

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

A Randomized Phase 2 Trial of the Efficacy and Safety of Short and Long Course Radiation Therapy With and Without BMX-001 as Part of Total Neoadjuvant Therapy in Patients With Newly Diagnosed Locally Advanced Rectal Adenocarcinoma

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 118 (estimated)
• Sponsor: University of Nebraska · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

In this trial, we will enroll patients in two cohorts: Cohort 1: A 6 patient safety lead-in for patients receiving capecitabine prior to beginning a randomized trial for the Long Course Chemo-radiation (LCCRT) cohort. There will be trial stopping rules to review safety and PK data prior to initiating the randomized trial of patients receiving LCCRT. Cohort 2: The Randomized Short Course Radiation (SCRT) cohort. Cohort 1 and 2 will begin enrolling concurrently. Cohort 1 will have a safety lead-in of patients receiving LCCRT (with capecitabine). Cohort 2 will begin with the randomized study of patients receiving SCRT, while the LCCRT cohort is going through the safety lead-in portion. There are stopping rules for the LCCRT cohort 1 pending PK analysis as well as an AE review from the treatment period plus 1 week post BMX-001 treatment for the 6 patients prior to moving on with the randomized portion of the study for the LCCRT cohort. All patients in the safety lead-in portion of the trial will receive BMX-001 (A loading dose of 28 mg/subject followed by maintenance doses of 14 mg/subject twice a week) with either SCRT or LCCRT. Patients will receive 3 cycles of FOLFOX or 2 cycles of CAPOX and then be assigned to SCRT or LCCRT prior to randomization. Patients will be randomized (stratified by gender) to BMX-001 or no BMX-001. The randomized trial in each cohort will involve SCRT or LCCRT with or without BMX-001. After conclusion of radiation, patients will then receive 6 more cycles of FOLFOX or 4 more cycles of CAPOX. Skin, GI, and GU symptoms will be measured on the day of screening, before and after RT.

Conditions

• Rectal Cancer

Interventions

Type	Name	Description
DRUG	BMX-001	Loading dose of 28 mg per subject, followed by maintenance doses of 14 mg per subject twice per week.

Timeline

• Start date: 2022-08-15
• Primary completion: 2027-05-01
• Completion: 2029-06-01
• First posted: 2022-02-24
• Last updated: 2025-08-07

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study