NCT04472429

Brief Summary

This study is a Phase 3 global, multicenter, placebo-controlled double-blind randomized study that will enroll participants with inoperable locally recurrent or metastatic SCAC not previously treated with systemic chemotherapy.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
308

participants targeted

Target at P50-P75 for phase_3

Timeline
Completed

Started Jan 2021

Longer than P75 for phase_3

Geographic Reach
13 countries

84 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 13, 2020

Completed
2 days until next milestone

First Posted

Study publicly available on registry

July 15, 2020

Completed
6 months until next milestone

Study Start

First participant enrolled

January 12, 2021

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 15, 2024

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

May 23, 2025

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 26, 2025

Completed
Last Updated

November 3, 2025

Status Verified

October 1, 2025

Enrollment Period

3.3 years

First QC Date

July 13, 2020

Results QC Date

April 8, 2025

Last Update Submit

October 16, 2025

Conditions

Keywords

Squamous cell carcinomacarboplatinpaclitaxelPD-1 InhibitorAnal CancerSCAC

Outcome Measures

Primary Outcomes (1)

  • Progression-free Survival (PFS)

    PFS was defined as the time from the date of randomization to the date of the first documented disease progression (PD), according to Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) by blinded independent central review committee (BICR), or death due to any cause, whichever occurred first. PD: progression of a target or non-target lesion or presence of a new lesion.

    up to 33.9 months

Secondary Outcomes (12)

  • Overall Survival

    up to 40.4 months

  • Objective Response Rate (ORR)

    up to 445 days

  • Duration of Response (DOR)

    up to 32.1 months

  • Disease Control Rate (DCR)

    up to 445 days

  • Number of Participants With Any Treatment-emergent Adverse Event (TEAE ) During the Randomized Period

    up to 535 days

  • +7 more secondary outcomes

Study Arms (2)

Group A : carboplatin+paclitaxel+placebo

PLACEBO COMPARATOR

Participants will receive carboplatin on Day 1,paclitaxel on Day1,8, 15, and placebo on Day 1 of each 28 day cycle

Drug: carboplatinDrug: paclitaxel

Group B : carboplatin+paclitaxel+retifanlimab

EXPERIMENTAL

Participants will receive carboplatin on Day 1,paclitaxel on Day1,8, 15, and retifanlimab on Day 1 of each 28 day cycle

Drug: carboplatinDrug: paclitaxelDrug: retifanlimab

Interventions

carboplatin will be administered intravenous on Day 1 of each 28 day cycle

Group A : carboplatin+paclitaxel+placeboGroup B : carboplatin+paclitaxel+retifanlimab

paclitaxel will be administered intravenous on Days 1,8, and 15 of each 28 day cycle

Group A : carboplatin+paclitaxel+placeboGroup B : carboplatin+paclitaxel+retifanlimab

retifanlimab will be administered intravenous on Day 1 of each 28 day cycle

Also known as: INCMGA00012
Group B : carboplatin+paclitaxel+retifanlimab

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Able to comprehend and willing to sign a written ICF for the study.
  • Are 18 years of age or older (or as applicable per local country requirements).
  • Histologically or cytologically verified, inoperable locally recurrent or metastatic SCAC.
  • No prior systemic therapy other than the following: a. Chemotherapy administered concomitantly with radiotherapy as a radiosensitizing agent is permitted.
  • b. Prior neoadjuvant or adjuvant therapy if completed ≥ 6 months before study entry.
  • Has measurable disease per RECIST v1.1 as determined by local site investigator/radiology assessment. Tumor lesions situated in a previously irradiated area, or in an area subjected to other loco-regional therapy, are usually not considered measurable unless there has been demonstrated progression in the lesion.
  • Able and willing to provide adequate tissue sample and whole blood sample with central testing result prior to randomization. Biopsy for archival samples should have occurred within 9 months prior to randomization.
  • ECOG performance status 0 to 1.
  • If HIV-positive, then must be stable as defined by: a. CD4+ count ≥ 200/μL, b. Undetectable viral load per standard of care assay, c. Receiving antiretroviral therapy (ART/HAART) for at least 4 weeks prior to study enrollment, and have not experienced any HIV-related opportunistic infection for at least 4 weeks prior to study enrollment.
  • Willingness to avoid pregnancy or fathering children

You may not qualify if:

  • Has received prior PD-(L)1 directed therapy
  • Has received prior radiotherapy with or without radiosensitizing chemotherapy within 28 days of Cycle 1 Day 1 except for palliative radiation (30 Gy or less) which is restricted for 14 days of Cycle 1 Day 1 (note: all toxicities associated should have resolved to Grade ≤ 1).
  • Participants with laboratory outside of the protocol defined ranges.
  • History of second malignancy within 3 years (with exceptions).
  • Clinically significant pulmonary, cardiac, gastrointestinal or autoimmune disorders.
  • Active bacterial, fungal, or viral infections, including hepatitis A, B, and C and IV antibiotic use within 7 days of Cycle 1 Day 1.
  • Receipt of a live vaccine within 28 days of planned start of study therapy.
  • History of organ transplant, including allogeneic stem cell transplantation.
  • Known active CNS metastases and/or carcinomatous meningitis.
  • Known hypersensitivity to platinum, paclitaxel, another monoclonal antibody, or any of the excipients that cannot be controlled with standard measures (eg, antihistamines, corticosteroids).
  • Participant is pregnant or breastfeeding.
  • Current use of protocol defined prohibited medication.
  • Has pre-existing peripheral neuropathy that is ≥ Grade 2 by CTCAE v5.
  • Inability or unlikely, in the opinion of the investigator, to comply with the Protocol requirements

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (84)

The University of Arizona Cancer Center

Tucson, Arizona, 85724, United States

Location

City of Hope National Medical Center

Duarte, California, 91010, United States

Location

UC Davis Comprehensive Cancer Center

Sacramento, California, 95817, United States

Location

Sansum Clinic

Santa Barbara, California, 93105, United States

Location

Rocky Mountain Cancer Center

Denver, Colorado, 80218, United States

Location

Ochsner Clinic

New Orleans, Louisiana, 70121, United States

Location

Maryland Oncology Hematology, P.A.

Columbia, Maryland, 21044, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Texas Oncology

Austin, Texas, 78745, United States

Location

Baylor Scott and White Research Institute

Dallas, Texas, 75246, United States

Location

Renovatio Clinical Consultants Llc

The Woodlands, Texas, 77380, United States

Location

Texas Oncology-Wichita Falls Texoma Cancer Center

Wichita Falls, Texas, 76310, United States

Location

Virginia Cancer Specialists, Pc

Arlington, Virginia, 22205, United States

Location

Blue Ridge Cancer Care

Roanoke, Virginia, 24014, United States

Location

Princess Alexandra Hospital Australia

Woolloongabba, Queensland, 04102, Australia

Location

Flinders Medical Centre

Bedford Park, South Australia, 5042, Australia

Location

Monash Medical Centre Clayton

Clayton, Victoria, 03168, Australia

Location

Zna Middelheim

Antwerp, 02020, Belgium

Location

Ulb Hospital Erasme

Brussels, 01070, Belgium

Location

Herlev Og Gentofte Hospital

Herlev, 02730, Denmark

Location

Vejle Hospital

Vejle, 07100, Denmark

Location

Institut de Cancerologie de L Ouest - Site Paul Papin

Angers, 49055, France

Location

Chu Besancon Hospital Jean Minjoz

Besançon, 25030, France

Location

Centre Hospitalier Universitaire de Bordeaux

Bordeaux, 33075, France

Location

Centre Leon Berard

Lyon, 69008, France

Location

Chu Hopital de La Timone

Marseille, 13385, France

Location

Institut Du Cancer de Montpellier

Montpellier, 34298, France

Location

Centre Antoine Laccassagne

Nice, 06200, France

Location

Hospital Universitaire Pitie-Salpetriere

Paris, 75651, France

Location

Hospital de La Miletrie

Poitiers, 86021, France

Location

Chu de Rennes - Hospital Pontchaillou

Rennes, 35033, France

Location

Hopital Charles Nicolle Chu Rouen Hospital de Bois-Guillaume

Rouen, 76031, France

Location

Centre de Lutte Contre Le Cancer - Institut de Cancerologie de L'Ouest - Rene Gauducheau

Saint-Herblain, 44800, France

Location

Institut de Cancerologie de Strasbourg

Strasbourg, 67200, France

Location

Chu Toulouse Hopital Rangueil

Toulouse, 31059, France

Location

Institut Gustave Roussy

Villejuif, 94805, France

Location

Universitatsklinikum Bonn Aoer

Bonn, 53127, Germany

Location

University Clinic Carl Gustav Carus Technical University Dresden

Dresden, 01307, Germany

Location

Asklepios Klinik Altona

Hamburg, 22763, Germany

Location

Fondazione Irccs Ca Granda Ospedale Maggiore

Milan, 20122, Italy

Location

Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda

Milan, 20162, Italy

Location

Comitato Etico Fondazione Irccs Istituto Nazionale Dei Tumori Milano

Milan, 20133, Italy

Location

European Institute of Oncology

Milan, 20141, Italy

Location

Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda

Milan, 20162, Italy

Location

University Di Cagliari-Presidio Policlinico Monserrato

Monserrato, 09042, Italy

Location

Azienda Ospedaliera Universitaria University Degli Studi Della Campania Luigi Vanvitelli

Napoli, 80131, Italy

Location

Iov - Istituto Oncologico Veneto Irccs

Padua, 35128, Italy

Location

Azienda Ospedaliero Universitaria Pisana

Pisa, 56126, Italy

Location

Ospedale Degli Infermi

Rimini, 47923, Italy

Location

I.R.C.C.S. Casa Sollievo Della Sofferenza

San Giovanni Rotondo, 71013, Italy

Location

Azienda Ospedaliero Universitaria Ospedali Riuniti

Torrette, 60020, Italy

Location

National Cancer Center Hospital

Chūōku, 104-0045, Japan

Location

Kyushu University Hospital

Fukuoka, 812-8582, Japan

Location

Saitama Medical University International Medical Center

Hidaka-shi, 350-1298, Japan

Location

Aichi Cancer Center Hospital

Nagoya, 464-8681, Japan

Location

Osaka International Cancer Institute

Osaka, 541-8567, Japan

Location

Tohoku University Hospital

Sendai, 980-8574, Japan

Location

Center Hospital of the National Center For Global Health and Medicine

Shinjuku-ku, 162-8655, Japan

Location

Haukeland University Hospital

Bergen, 05021, Norway

Location

Oslo Universitetssykehus

Oslo, 00450, Norway

Location

Panoncology Trials Pan American Center For Oncology Trials, Llc

San Juan, 00935, Puerto Rico

Location

Complejo Hospitalario Universitario A Coruna

A Coruña, 15006, Spain

Location

Hospital General Universitario Vall D Hebron

Barcelona, 08035, Spain

Location

Hospital Clinic de Barcelona

Barcelona, 08036, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Hospital Universitario de La Paz

Madrid, 28046, Spain

Location

Son Espases University Hospital

Palma de Mallorca, 07120, Spain

Location

Hospital Universitario Virgen Del Rocio

Seville, 41013, Spain

Location

Hospital Universitario Miguel Servet

Zaragoza, 50009, Spain

Location

Sahlgrenska University Hospital

Gothenburg, 413 45, Sweden

Location

Skaenes Universitetssjukhus Lund

Lund, 22185, Sweden

Location

Stockholm South General Hospital Sodersjukhuset

Stockholm, 118 83, Sweden

Location

Royal Sussex County Hospital

Brighton, BN2 5BE, United Kingdom

Location

Addenbrooke'S Hospital

Cambridge, CB2 0QQ, United Kingdom

Location

Royal Surrey County Hospital

Guildford, GU2 7XX, United Kingdom

Location

Castle Hill Hospital

Hull, HU16 5JQ, United Kingdom

Location

Leeds Teaching Hospital

Leeds, LS 9 7TF, United Kingdom

Location

Royal Free London Nhs Foundation Trust

London, NW3 2QG, United Kingdom

Location

The Royal Marsden Nhs Foundation Trust - Chelsea

London, SW3 6JJ, United Kingdom

Location

The Christie Nhs Foundation Trust Uk

Manchester, M20 4BV, United Kingdom

Location

Churchill Hospital

Oxford, OX3 7LE, United Kingdom

Location

Royal Preston Hospital

Preston, PR2 9HT, United Kingdom

Location

The Royal Marsden Nhs Foundation Trust - Sutton

Sutton, SM2 5PT, United Kingdom

Location

Royal Cornwall Hospital Truro Sunrise Centre

Truro, TR1 3LQ, United Kingdom

Location

Related Publications (2)

  • Rao S, Samalin-Scalzi E, Evesque L, Ben Abdelghani M, Morano F, Roy A, Dahan L, Tamberi S, Dhadda AS, Saunders MP, Casanova N, Guimbaud R, Lievre A, Maurel J, Fakih M, Tian C, Harrison J, Jones MM, Cornfeld M, Spano JP, Rochefort P; POD1UM-303/InterAACT-2 study investigators. Retifanlimab with carboplatin and paclitaxel for locally recurrent or metastatic squamous cell carcinoma of the anal canal (POD1UM-303/InterAACT-2): a global, phase 3 randomised controlled trial. Lancet. 2025 Jun 14;405(10495):2144-2152. doi: 10.1016/S0140-6736(25)00631-2.

  • Rao S, Jones M, Bowman J, Tian C, Spano JP. POD1UM-303/InterAACT 2: A phase III, global, randomized, double-blind study of retifanlimab or placebo plus carboplatin-paclitaxel in patients with locally advanced or metastatic squamous cell anal carcinoma. Front Oncol. 2022 Aug 24;12:935383. doi: 10.3389/fonc.2022.935383. eCollection 2022.

MeSH Terms

Conditions

Carcinoma, Squamous CellAnus Neoplasms

Interventions

CarboplatinPaclitaxel

Condition Hierarchy (Ancestors)

CarcinomaNeoplasms, Glandular and EpithelialNeoplasms by Histologic TypeNeoplasmsNeoplasms, Squamous CellRectal NeoplasmsColorectal NeoplasmsIntestinal NeoplasmsGastrointestinal NeoplasmsDigestive System NeoplasmsNeoplasms by SiteDigestive System DiseasesGastrointestinal DiseasesIntestinal DiseasesAnus DiseasesRectal Diseases

Intervention Hierarchy (Ancestors)

Coordination ComplexesOrganic ChemicalsTaxoidsCyclodecanesCycloparaffinsHydrocarbons, AlicyclicHydrocarbons, CyclicHydrocarbonsDiterpenesTerpenes

Results Point of Contact

Title
Study Director
Organization
Incyte Corporation

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Masking Details
Double Blind
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR
Expanded Access
Yes

Study Record Dates

First Submitted

July 13, 2020

First Posted

July 15, 2020

Study Start

January 12, 2021

Primary Completion

April 15, 2024

Study Completion

September 26, 2025

Last Updated

November 3, 2025

Results First Posted

May 23, 2025

Record last verified: 2025-10

Data Sharing

IPD Sharing
Will share

Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency

Shared Documents
STUDY PROTOCOL, SAP
Time Frame
Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
Access Criteria
Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement.
More information

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