Squamous Cell Carcinoma of Anal CanalPhase 2NCT03597295
A Study of INCMGA00012 in Squamous Carcinoma of the Anal Canal Following Platinum-Based Chemotherapy (POD1UM-202)
A Phase 2 study testing Retifanlimab in people with Squamous Cell Carcinoma of Anal Canal, run by Incyte Corporation at 46 sites in 9 countries.
Trial summary
The purpose of this study is to assess the efficacy of INCMGA00012 in participants with locally advanced or metastatic squamous carcinoma of the anal canal (SCAC) who have progressed after platinum-based chemotherapy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Squamous Cell Carcinoma of Anal Canal
- What is being tested?
- Retifanlimab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 94 participants
- Where
- 46 sitesin 9 countries
- Study dates
- 8 Oct 2018to 10 Nov 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Ability to comprehend and willingness to sign a written informed consent form.
- Confirmed diagnosis of locally advanced or metastatic SCAC.
- Must have received (or been intolerant to or ineligible for) at least 1 prior line of platinum-based chemotherapy and received no more than 2 prior systemic treatments.
- Must have measurable disease by RECIST v1.1.
- Eastern Cooperative Oncology Group performance status of 0 to 1.
- If HIV-positive, then all of the following criteria must also be met: CD4+ count ≥ 300/μL, undetectable viral load, and receiving highly active antiretroviral therapy.
Who cannot join
- Receipt of anticancer therapy or participation in another interventional clinical study within 21 days before the first administration of study drug; 6 weeks for mitomycin C.
- Radiotherapy within 14 days of first dose of study treatment with the following caveats: 28 days for pelvic radiotherapy, 6 months for thoracic region radiotherapy that is > 30 Gy.
- Prior treatment with programmed cell death protein 1 (PD-1) or programmed cell death ligand protein 1 (PD-L1)-directed therapy.
- Active autoimmune disease requiring systemic immunosuppression.
- Known central nervous system (CNS) metastases and/or carcinomatous meningitis.
- Known active hepatitis infection.
- Active infections requiring systemic therapy.
- Is pregnant or breastfeeding or is expecting to conceive or father children within the projected duration of the study, from screening through 6 months after the last dose of study drug.
Trial locations and contacts
46 sites in 9 countries.
46 sites in 9 countries
Italy10 sites
Azienda Ospedaliero Universitaria Ospedali Riuniti
Ancona, 60126
Ospedale A. Perrino - Brindisi
Brindisi, 72100
A.O.U. Policlinico V. Emanuele G. Rodolico
Catania, 95123
Ospedale Degli Infermi - Faenza
Faenza, 48018
Irccs Azienda Ospedaliera Universitaria San Martino
Genova, 16132
Niguarda Cancer Center
Milan, 20162
Aou Modena - Policlinico
Modena, 41124
Clinica La Maddalena
Palermo, 90146
Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore
Rome, 00168
IRCCS Casa Sollievo Della Sofferenza
San Giovanni Rotondo, 71013
United Kingdom10 sites
Royal Sussex County Hospital
Brighton, BN2 5BE
Royal Marsden Hospital
Chelsea, SW3 6JJ
Castle Hill Hospital
Cottingham, HU16 5JQ
St. James U Hospital
Leeds, L59 7TF
St. James Univ Hospital
Leeds, L59 7TF
Royal Free London Nhs Foundation Trust
London, NW3 2QG
The Royal Marsden Nhs Foundation Trust - Chelsea
London, SW3 6JI
The Christie NHS Foundation Trust
Manchester, M20 4BV
Royal Marsden Hospital
Sutton, SM2 5PT
Royal Cornwall Hospital, Sunrise Centre
Truro, TR1 3LQ
United States7 sites
Texas Oncology-Baylor Charles A. Sammons
Dallas, Texas, 75246
City of Hope National Medical Center
Duarte, California, 91010
Texas Oncology-McKinney
McKinney, Texas, 75071
Maryland Oncology Hematology P.A.
Rockville, Maryland, 20850
UC Davis Comprehensive Cancer Center
Sacramento, California, 95817
Ridley-Tree Cancer Center
Santa Barbara, California, 95817
Renovatio Clinical
Spring, Texas, 77380
France7 sites
Centre Hospitalier Universitaire de Besancon
Besançon, 2500
CHU Hopital De La Timone
Marseille, 13385
Icm Montpellier
Montpellier, 34298
Hopital Universitaire Pitie-Salpetriere
Paris, 75013
Chu de Rennes - Hôpital Pontchaillou
Rennes, 35033
Centre de Lutte Contre Le Cancer - Institut de Cancerologie de L'Ouest - Rene Gauducheau
Saint-Herblain, 44805
CHU Toulouse Hopital Rangueil
Toulouse, 31059
Denmark3 sites
Aarhus Universitets Hospital
Aarhus, 8000
Herlev Og Gentofte Hospital
Herlev, 2730
Vejle Hospital
Vejle, 7100
Spain3 sites
Hospital General Universitari Vall D Hebron
Barcelona, 08035
Hospital Clinic I Provincial
Barcelona, 08036
Hospital Universitario 12 de Octubre
Madrid, 28041
Germany2 sites
University Medical Centre Hamburg-Eppendorf, Centre of Oncology
Hamburg, 20246
Asklepios Klinik Altona
Hamburg, 22763
About this study
Official title: A Phase 2 Study of INCMGA00012 in Participants With Squamous Carcinoma of the Anal Canal Who Have Progressed Following Platinum-Based Chemotherapy (POD1UM-202)
The purpose of this study is to assess the efficacy of INCMGA00012 in participants with locally advanced or metastatic squamous carcinoma of the anal canal (SCAC) who have progressed after platinum-based chemotherapy.
- Conditions
- Squamous Cell Carcinoma of Anal Canal
- Being tested
- Retifanlimab
- Keywords
- Squamous carcinoma of the anal canal, anti-PD-1 antibody, IgG4 monoclonal antibody, INCMGA00012
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RetifanlimabDrug
Also called MGA012, INCMGA00012
Retifanlimab 500 milligrams (mg) intravenously every 4 weeks (Q4W).
| Study group | Role | What they receive |
|---|---|---|
| Retifanlimab 500 mg | Experimental | Retifanlimab 500 milligrams (mg) intravenously every 4 weeks (Q4W). Receives: Drug: Retifanlimab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate (ORR)
ORR was defined as the percentage of participants with a best overall response of complete response (CR) or partial response (PR), per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 (v1.1), as assessed by independent central radiographic (ICR) review, at any post-Baseline visit until new anti-cancer therapy or first Progressive Disease. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to <10 millimeters (mm). PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions.
Measured: Cycle 1 Day 1, and every 4 weeks throughout the study, up to approximately 24 months
Secondary outcomes (9)
Duration of Response (DOR)
DOR was defined as the time from an initial objective response (CR or PR) per RECIST v1.1 until the first observation of documented disease progression (PD), as determined by ICR, or death due to any cause. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to <10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. PD: progression of a target or non-target lesion or presence of a new lesion.
Measured: up to 18.2 months
Disease Control Rate (DCR)
DCR was defined as the percentage of participants with a confirmed overall response of CR, PR, or stable disease (SD), per RECIST v1.1, at any post-baseline visit until the first progressive disease (PD) or new anti-cancer therapy. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to <10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. PD: progression of a target or non-target lesion or presence of a new lesion. SD: no change in target lesions to qualify for CR, PR, or PD.
Measured: Cycle 1 Day 1, and every 4 weeks throughout the study, up to approximately 24 months
Progression-free Survival (PFS)
According to RECIST 1.1, PFS was defined as the length of time from the initial infusion of study drug until the earliest date of disease progression, as determined by ICR, or death due to any cause, if occurring sooner than progression.
Measured: up to 16.8 months
Overall Survival
Overall survival was defined as the time in months between the first dose date (Day 1) and the date of death due to any cause.
Measured: up to 28.2 months
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE could have been any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of the study drug. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of retifanlimab and within 90 days of the last administration of retifanlimab.
Measured: up to 913 days
Cmax of Retifanlimab
Cmax was defined as the maximum observed plasma concentration.
Measured: pre-infusion on Day 1 of Cycles 1, 2, 4, 6, and 7; 10 minutes and 4 hours post-infusion on Day 1 of Cycles 1 and 6
Tmax of Retifanlimab
tmax was defined as the time to the maximum concentration.
Measured: pre-infusion on Day 1 of Cycles 1, 2, 4, 6, and 7; 10 minutes and 4 hours post-infusion on Day 1 of Cycles 1 and 6
Cmin of Retifanlimab
Cmin was defined as the minimum observed plasma concentration over the dose interval.
Measured: pre-infusion on Day 1 of Cycles 1, 2, 4, 6, and 7; 10 minutes and 4 hours post-infusion on Day 1 of Cycles 1 and 6
AUC0-t of Retifanlimab
AUC0-t was defined as the area under the plasma concentration-time curve from time = 0 to the last measurable concentration at time = t.
Measured: pre-infusion on Day 1 of Cycles 1, 2, 4, 6, and 7; 10 minutes and 4 hours post-infusion on Day 1 of Cycles 1 and 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2018
- Study start8 Oct 2018
- Primary completion8 Jun 2020
- Study completion10 Nov 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Squamous Cell Carcinoma of Anal CanalStudies of the same condition, open and under way.
- More trials run by Incyte CorporationThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 94 participants (actual)
Sponsor and investigators
- Lead sponsor
- Incyte Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Incyte Medical Monitor, Study director, Incyte Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03597295
- Sponsor's protocol ID
- INCMGA 0012-202
- Other IDs
- 2018-002070-51, 2018
- Status last verified
- Aug 2025
- First submitted
- 16 Jul 2018
- Last updated
- 21 Aug 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Rao S, Anandappa G, Capdevila J, Dahan L, Evesque L, Kim S, Saunders MP, Gilbert DC, Jensen LH, Samalin E, Spindler KL, Tamberi S, Demols A, Guren MG, Arnold D, Fakih M, Kayyal T, Cornfeld M, Tian C, Catlett M, Smith M, Spano JP. A phase II study of retifanlimab (INCMGA00012) in patients with squamous carcinoma of the anal canal who have progressed following platinum-based chemotherapy (POD1UM-202). ESMO Open. 2022 Aug;7(4):100529. doi: 10.1016/j.esmoop.2022.100529. Epub 2022 Jul 8.
PubMed 35816951
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study of INCMGA00012 in Participants With Squamous Carcinoma of the Anal Canal Who Have Progressed Following Platinum-Based Chemotherapy (POD1UM-202)
- ClinicalTrials.gov ID
- NCT03597295
- Acronym / sponsor ID
- INCMGA 0012-202
- Conditions
- Squamous Cell Carcinoma of Anal Canal
- Keywords
- Squamous carcinoma of the anal canal, anti-PD-1 antibody, IgG4 monoclonal antibody, INCMGA00012
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 94 participants (actual)
- Lead sponsor
- Incyte CorporationIndustry (pharma, biotech, devices)
- Study start
- 8 Oct 2018 (actual)
- Primary completion
- 8 Jun 2020 (actual)
- Study completion
- 10 Nov 2021 (actual)
- First posted
- 24 Jul 2018 (actual)
- Results
- Posted 23 Aug 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the efficacy of INCMGA00012 in participants with locally advanced or metastatic squamous carcinoma of the anal canal (SCAC) who have progressed after platinum-based chemotherapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Retifanlimab 500 mg | Experimental | Drug: RetifanlimabRetifanlimab 500 milligrams (mg) intravenously every 4 weeks (Q4W). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Ability to comprehend and willingness to sign a written informed consent form.
- Confirmed diagnosis of locally advanced or metastatic SCAC.
- Must have received (or been intolerant to or ineligible for) at least 1 prior line of platinum-based chemotherapy and received no more than 2 prior systemic treatments.
- Must have measurable disease by RECIST v1.1.
- Eastern Cooperative Oncology Group performance status of 0 to 1.
Key exclusion criteria (8 listed)
- Receipt of anticancer therapy or participation in another interventional clinical study within 21 days before the first administration of study drug; 6 weeks for mitomycin C.
- Radiotherapy within 14 days of first dose of study treatment with the following caveats: 28 days for pelvic radiotherapy, 6 months for thoracic region radiotherapy that is > 30 Gy.
- Prior treatment with programmed cell death protein 1 (PD-1) or programmed cell death ligand protein 1 (PD-L1)-directed therapy.
- Active autoimmune disease requiring systemic immunosuppression.
- Known central nervous system (CNS) metastases and/or carcinomatous meningitis.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RetifanlimabAlso: MGA012, INCMGA00012Retifanlimab 500 milligrams (mg) intravenously every 4 weeks (Q4W). | Drug | Retifanlimab 500 mg |
Primary outcome
Objective Response Rate (ORR)
Time frame: Cycle 1 Day 1, and every 4 weeks throughout the study, up to approximately 24 months
ORR was defined as the percentage of participants with a best overall response of complete response (CR) or partial response (PR), per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 (v1.1), as assessed by independent central radiographic (ICR) review, at any post-Baseline visit until new anti-cancer therapy or first Progressive Disease. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to <10 millimeters (mm). PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions.
9 secondary outcomes
Duration of Response (DOR)
Time frame: up to 18.2 months
Disease Control Rate (DCR)
Time frame: Cycle 1 Day 1, and every 4 weeks throughout the study, up to approximately 24 months
Progression-free Survival (PFS)
Time frame: up to 16.8 months
Overall Survival
Time frame: up to 28.2 months
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: up to 913 days
Cmax of Retifanlimab
Time frame: pre-infusion on Day 1 of Cycles 1, 2, 4, 6, and 7; 10 minutes and 4 hours post-infusion on Day 1 of Cycles 1 and 6
Tmax of Retifanlimab
Time frame: pre-infusion on Day 1 of Cycles 1, 2, 4, 6, and 7; 10 minutes and 4 hours post-infusion on Day 1 of Cycles 1 and 6
Cmin of Retifanlimab
Time frame: pre-infusion on Day 1 of Cycles 1, 2, 4, 6, and 7; 10 minutes and 4 hours post-infusion on Day 1 of Cycles 1 and 6
AUC0-t of Retifanlimab
Time frame: pre-infusion on Day 1 of Cycles 1, 2, 4, 6, and 7; 10 minutes and 4 hours post-infusion on Day 1 of Cycles 1 and 6
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Aug 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 35816951Rao S, Anandappa G, Capdevila J, Dahan L, Evesque L, Kim S, Saunders MP, Gilbert DC, Jensen LH, Samalin E, Spindler KL, Tamberi S, Demols A, Guren MG, Arnold D, Fakih M, Kayyal T, Cornfeld M, Tian C, Catlett M, Smith M, Spano JP. A phase II study of retifanlimab (INCMGA00012) in patients with squamous carcinoma of the anal canal who have progressed following platinum-based chemotherapy (POD1UM-202). ESMO Open. 2022 Aug;7(4):100529. doi: 10.1016/j.esmoop.2022.100529. Epub 2022 Jul 8.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Incyte Medical Monitor study director
Incyte Corporation
46 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Retifanlimab in people with Squamous Cell Carcinoma of Anal Canal, run by Incyte Corporation at 46 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 46 sites in Italy, United Kingdom, United States, France, Denmark and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Aug 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (ORR) (time frame: Cycle 1 Day 1, and every 4 weeks throughout the study, up to approximately 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03597295, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Aug 2025.