This is a multi-site, prospective, non-randomized phase 2 study evaluating neoadjuvant botensilimab in combination with balstilimab for patients with microsatellite stable (MSS) / mismatch repair proficient (MMRp) early rectal cancer staged T1-T2 N0 by MRI and considered candidates for surgical resection without standard neoadjuvant therapies. Participants will receive a single IV dose of botensilimab on Day 1 followed by balstilimab IV every 2 weeks for up to 6 months, with tumor response assessments during treatment and follow-up afterward.
This study is a multi-site, prospective, non-randomized, phase 2 trial. The protocol proposes to treat 16 participants with a single fixed dose of botensilimab 75 mg IV on the first day of treatment, followed by balstilimab 240 mg IV every 2 weeks for up to 6 months. The schema states that patients with stage I MSS/MMRp rectal cancer (T1-2 N0 by MRI) will undergo tumor response assessment during treatment for 6 months. After treatment, participants with complete or near complete response and pathologic complete response at local excision may undergo non-operative management with long-term surveillance, while those with residual disease or tumor progression will undergo management per standard of care. The study calendar includes treatment cycles, discontinuation, 30-day and 90-day safety follow-up, efficacy follow-up, and survival follow-up by phone every 12 weeks.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
16
one IV infusion on Cycle 1 Day 1, over about 30 minutes
IV infusion every 2 weeks on Day 1 of each treatment cycle, over about 30 minutes, for up to 6 months
Brigham and Women's Hospital
Boston, Massachusetts, United States
RECRUITINGDana-Farber Cancer Institute
Boston, Massachusetts, United States
RECRUITINGBeth Israel Deaconess Medical Center (BIDMC)
Boston, Massachusetts, United States
ACTIVE_NOT_RECRUITINGComplete response (cCR plus nCR with pCR at resection) within 6 months from initiation of therapy or nCR with pathologic complete response (pCR) with TES
Tumor response will be evaluated by a qualified colorectal surgeon using flexible sigmoidoscopy (or equivalent), with biopsy as clinically indicated, and rectal MRI using standard response criteria. Response categories are clinical complete response, near-complete clinical response, no response, or progression. Participants with near-complete clinical response must have pathologic complete response at TES/local excision to meet the endpoint.
Time frame: From baseline to 6 months from initiation of therapy
Incidence of treatment-emergent adverse events
Adverse events will be classified and graded according to NCI CTCAE version 6.0. Toxicity results will be presented descriptively using frequencies and percentages.
Time frame: From first treatment through survival follow-up, up to 5 years from initiation of therapy
Incidence of surgical complications
Surgical complications will be graded according to the Clavien-Dindo classification among participants who undergo local excision/TES or total mesorectal excision.
Time frame: From surgery through 90-day safety follow-up
Organ preservation rate
Organ preservation rate is defined as the proportion of participants who avoid total mesorectal excision. Local excision/TES without total mesorectal excision is considered organ preservation.
Time frame: From initiation of therapy through post-treatment surgical management decision, approximately 6 months
3-year locoregional recurrence rate
Locoregional recurrence rate is defined as the proportion of participants with local or regional recurrence as the initial site of failure within 3 years from initiation of therapy.
Time frame: From initiation of therapy to 3 years.
Disease-free survival
Disease-free survival is defined as survival without evidence of local recurrence, distant metastasis, or death from any cause within 3 years from initiation of therapy. Participants alive without disease progression are censored at the date of last disease evaluation.
Time frame: From initiation of therapy to 3 years.
Overall survival
Overall survival is defined as survival without death from any cause within 5 years from initiation of therapy.
Time frame: From initiation of therapy to 5 years.
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