Real-world Efficacy and Safety of Neoadjuvant Dostarlimab in Patients With dMMR/MSI-H Locally Advanced Rectal Cancer

NCT07269249 | Recruiting

• 1 other identifier: RW-NEDOS
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 2

Brief Summary

This is an observational, retrospective-prospective, multicentre trial enrolling all patients included in the AIFA monitoring registry of Dostarlimab for the indication in rectal cancer. The aims of the study are to describe the clinical outcomes and safety of patients with dMMR/MSI-H locally advanced rectal cancer (LARC) receiving neoadjuvant dostarlimab in the real-world setting.

Conditions

Locally Advanced Rectal Cancer (LARC)

Keywords

dostarlimab; mismatch repair-deficient LARC; real-world; retrospective-prospective study; neoadjuvant setting

Outcome Measures

Primary Outcomes (1)

• Objective response rate (ORR)

  Defined as the percentage of trial participants having a CR or PR, as per RECIST v1.1.

  after 3 , 6 and 9 cycles of dostarlimab. Each cycle is 21 days. Up to 6 months

Secondary Outcomes (10)

• duration of clinical complete response

  up to to 12 months

• near-complete clinical response rate

  at 6 and 12 months up to 12 months

• duration of near-complete clinical response

  up to 12 months

• rate of surgery (total mesorectal excision or local excision)

  at 6 months (at the end of neoadjuvant dostarlimab)

• pathological complete response rate

  after surgery (at 6 months)

Study Arms (1)

Stage II-III rectal cancer (LARC)

patients with dMMR/MSI LARC treated with neoadjuvant dostarlimab

Drug: Dostarlimab

Interventions

Dostarlimab DRUG

Dostarlimab 500 mg iv every 3 weeks for 9 cycles

Stage II-III rectal cancer (LARC)

Eligibility Criteria

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

Study Population

Patients (any sex/gender) with dMMR/MSI-H locally advanced rectal cancer treated with dostarlimab are eligible for this study.

You may qualify if:

• Signed informed consent form
• Age \> 18 years
• Histologically confirmed stage II-III rectal cancer
• dMMR/MSI status assessed locally by Immunohistochemistry, next-generation sequencing or PCR
• For the retrospective part of the study:
• At least one dose of neoadjuvant dostarlimab from November 2023 (i.e. within the period of AIFA dostarlimab monitoring registry activation for the indication in rectal cancer, which is reimbursed according to the Italian law 648/1996, GU n.252 as of 27/10/2023)
• Eligible deceased or unreachable patients will also be included to avoid selection biases, in respect of the article 110 bis, paragraph 4 of the Italian Privacy Code (a Data Protection Impact Assessment will be produced and published on the Sponsor website before study initiation, and patients explicitly unwilling before death will be not included).
• For the prospective part of the study:

You may not qualify if:

• \- Distant metastasis
• Major cognitive dysfunction or psychiatric disorders
• Any previous systemic or local treatment for rectal cancer
• Dostarlimab received within an interventional clinical trial

Sponsors & Collaborators

• National Cancer Institute, Naples: lead

Study Sites (2)

ASL Napoli 1, Presidio Ospedaliero Ospedale del mare, Oncologia medica

Naples, 80131, Italy

NOT YET RECRUITING

IRCCS Fondazione G. Pascale, Unità Operativa Complessa Oncologia Addominale

Naples, Italy

RECRUITING

MeSH Terms

Interventions

dostarlimab

Study Officials

• Maria Carmela Piccirillo, MD

  National Cancer Institute, IRCCS Fondazione G. Pascale

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Maria Carmela Piccirillo, MD

CONTACT

+39 08117770280; m.piccirillo@istitutotumori.na.it

Piera Gargiulo, MD

CONTACT

+39 081 1777 0279; piera.gargiulo@istitutotumori.na.it

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 25, 2025
• First Posted: December 8, 2025
• Study Start: September 1, 2025
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: December 8, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share

Locations

IT (2)