Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ended
Participants planned
200
Countries
1
Sites
9
Advanced or Metastatic Cancer Merkel Cell Carcinoma (MCC) Cutaneous Squamous Cell Carcinoma (CSCC) Basal Cell Carcinoma (BCC) Triple Negative Breast Cancer (TNBC) Non-Small Cell Lung Cancer (NSCLC)
To determine confirmed objective response rate (ORR) with vidutolimod in combination with cemiplimab per RECIST 1.1 by investigator
Key facts
- Sponsor
- Regeneron Pharmaceuticals Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Neoplasms [C04]
- Trial duration
- completed 31 Oct 2024
- Decision date (initial)
- 2024-01-30
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2023-507344-36-01
- ClinicalTrials.gov
- NCT04916002
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Safety
To determine confirmed objective response rate (ORR) with vidutolimod in combination with cemiplimab per RECIST 1.1 by investigator
Secondary objectives 2
- To evaluate the safety and tolerability of vidutolimod administered by intratumoral (IT) injection in combination with cemiplimab
- To evaluate the efficacy of vidutolimod in combination with cemiplimab
Conditions and MedDRA coding
Advanced or Metastatic Cancer Merkel Cell Carcinoma (MCC) Cutaneous Squamous Cell Carcinoma (CSCC) Basal Cell Carcinoma (BCC) Triple Negative Breast Cancer (TNBC) Non-Small Cell Lung Cancer (NSCLC)
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.0 | LLT | 10048683 | Advanced cancer | 10029104 |
Study design 8 periods
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Experimental: Vidutolimod and cemiplimab for cutaneous squamous cell carcinoma (CSCC) (A1) Participants who have not received prior systemic therapy for CSCC will receive vidutolimod intratumoral(ly) (IT) and cemiplimab intravenous (IV) according to the treatment schedule until a reason for treatment discontinuation is reached.
|
Not Applicable | None | Experimental: Vidutolimod and cemiplimab for CSCC (A1): Drug: vidutolimod Participants will receive vidutolimod 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule. Other Name: CMP-001 Drug: cemiplimab Participants will receive cemiplimab 350 mg IV over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter. Other Names: Libtayo REGN2810 |
|
| 2 | Experimental: Vidutolimod and cemiplimab for CSCC (A2) Participants who have progressed while receiving a PD-1-blocking antibody or are within 12 weeks of discontinuation of treatment for CSCC will receive vidutolimod IT and cemiplimab IV according to the treatment schedule until a reason for treatment discontinuation is reached.
|
Not Applicable | None | Experimental: Vidutolimod and cemiplimab for CSCC (A2): Drug: vidutolimod Participants will receive vidutolimod 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule. Other Name: CMP-001 Drug: cemiplimab Participants will receive cemiplimab 350 mg IV over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter. Other Names: Libtayo REGN2810 |
|
| 3 | Experimental: Vidutolimod and cemiplimab for Merkel cell carcinoma (MCC) (B1) Participants who have not received prior systemic therapy for MCC will receive vidutolimod IT and cemiplimab IV according to the treatment schedule until a reason for treatment discontinuation is reached.
|
Not Applicable | None | Experimental: Vidutolimod and cemiplimab for Merkel cell carcinoma MCC (B1): Drug: vidutolimod Participants will receive vidutolimod 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule. Other Name: CMP-001 Drug: cemiplimab Participants will receive cemiplimab 350 mg IV over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter. Other Names: Libtayo REGN2810 |
|
| 4 | Experimental: Vidutolimod and cemiplimab for for Merkel cell carcinoma (MCC) (B2) Participants who have progressed while receiving a PD-1-blocking antibody or are within 12 weeks of discontinuation of treatment for MCC will receive vidutolimod IT and cemiplimab IV according to the treatment schedule until a reason for treatment discontinuation is reached.
|
Not Applicable | None | Experimental: Vidutolimod and cemiplimab for for Merkel cell carcinoma (MCC) (B2): Drug: vidutolimod Participants will receive vidutolimod 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule. Other Name: CMP-001 Drug: cemiplimab Participants will receive cemiplimab 350 mg IV over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter. Other Names: Libtayo REGN2810 |
|
| 5 | Experimental: Vidutolimod and cemiplimab for triple negative breast cancer (TNBC) (C1) Participants who have not received prior therapy with immune checkpoint inhibitors (iCPIs) for TNBC will receive vidutolimod IT and cemiplimab IV according to the treatment schedule until a reason for treatment discontinuation is reached.
|
Not Applicable | None | Experimental: Vidutolimod and cemiplimab for triple negative breast cancer (TNBC) (C1): Participants who have not received prior therapy with immune checkpoint inhibitors (iCPIs) for TNBC will receive vidutolimod IT and cemiplimab IV according to the treatment schedule until a reason for treatment discontinuation is reached. Drug: vidutolimod Participants will receive vidutolimod 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule. Other Name: CMP-001 Drug: cemiplimab Participants will receive cemiplimab 350 mg IV over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter. Other Names: Libtayo REGN2810 |
|
| 6 | Experimental: Vidutolimod and cemiplimab for TNBC (C2) Participants who have progressed while receiving a PD-1-blocking antibody or are within 12 weeks of discontinuation of treatment for TNBC will receive vidutolimod IT and cemiplimab IV according to the treatment schedule until a reason for treatment discontinuation is reached
|
Not Applicable | None | Experimental: Vidutolimod and cemiplimab for TNBC (C2): Drug: vidutolimod Participants will receive vidutolimod 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule. Other Name: CMP-001 Drug: cemiplimab Participants will receive cemiplimab 350 mg IV over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter. Other Names: Libtayo REGN2810 |
|
| 7 | Experimental: Vidutolimod and cemiplimab for basal cell carcinoma (BCC) (D) Participants who have not received prior hedgehog pathway inhibitor therapy, nor prior therapy with immune checkpoint inhibitors (iCPIs), for metastatic or locally and/or regionally advanced unresectable BCC will receive vidutolimod IT and cemiplimab IV according to the treatment schedule until a reason for treatment discontinuation is reached.
|
Not Applicable | None | Experimental: Vidutolimod and cemiplimab for basal cell carcinoma (BCC) (D): Drug: vidutolimod Participants will receive vidutolimod 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule. Other Name: CMP-001 Drug: cemiplimab Participants will receive cemiplimab 350 mg IV over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter. Other Names: Libtayo REGN2810 |
|
| 8 | Experimental: Vidutolimod and cemiplimab for non-small cell lung cancer (NSCLC) Not conducted in EU Participants with advanced NSCLC (metastatic or locally advanced who are not candidates for definitive chemoradiation, nor candidates for surgical resection), whose tumors have high PD-L1 expression (TPS ≥50%, as determined by a CAP/CLIA or equivalently licensed lab), have not received prior anti-PD-1/programmed cell death ligand 1 (PD-L1) therapy, and are amenable to IT therapy for advanced NSCLC. Patients with EGFR, ALK or ROS1 aberrations, are not eligible.
|
Not Applicable | None | Experimental: Vidutolimod and cemiplimab for non-small cell lung cancer (NSCLC) Not conducted in EU: Drug: vidutolimod Participants will receive vidutolimod 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule. Other Name: CMP-001 Drug: cemiplimab Participants will receive cemiplimab 350 mg IV over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter. Other Names: Libtayo REGN2810 |
Regulatory references
| EU CT number | Title | Sponsor |
|---|---|---|
| 2022-501683-18-00 | A Multicenter, Open-label, Phase 2 study of Intratumoral Vidutolimod (CMP-001) in Combination with Intravenous Cemiplimab in Subjects with Selected Types of Advanced or Metastatic Cancer | Regeneron Pharmaceuticals Inc. |
| 2023-507344-36-00 | A Multicenter, Open-label, Phase 2 study of Intratumoral Vidutolimod (CMP-001) in Combination with Intravenous Cemiplimab in Subjects with Selected Types of Advanced or Metastatic Cancer | Regeneron Pharmaceuticals Inc. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 5
- Histopathologically-confirmed diagnosis of cancer, as defined by the protocol
- Measurable disease, as defined by RECIST v1.1 and as defined in the protocol
- Adequate organ function based on most recent laboratory values within 3 weeks before first dose of study treatment on Week 1 Day 1 (W1D1), as defined in the protocol
- Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 1 at Screening
- Other protocol defined inclusion criteria apply
Exclusion criteria 11
- Received radiation therapy (or other non-systemic therapy) within 2 weeks before first dose of study treatment on W1D1. Participants should have recovered (i.e. Grade ≤ 1 or at baseline) from radiation-related toxicities.
- Untreated, symptomatic, or enlarging central nervous system metastases or carcinomatous meningitis (including leptomeningeal metastases from solid tumors).
- Received systemic pharmacologic doses of corticosteroids > 10 mg/day prednisone within 30 days before first dose of study treatment on W1D1, as defined in the protocol.
- History of immune-mediated AE leading to permanent discontinuation due to prior PD-1-blocking antibody.
- Not fully recovered from AEs due to prior treatment (to Grade 1 or less, per Common Terminology Criteria for Adverse Events (CTCAE), with the exception of persistent vitiligo, alopecia, hypothyroidism, diabetes mellitus, and adrenal and/or pituitary insufficiency.
- Active pneumonitis or history of noninfectious pneumonitis that required steroids
- Severe uncontrolled medical disease within 12 months of screening, including but not limited to poorly controlled hypertension, unstable angina, myocardial infarction, congestive heart failure (New York Heart Association Class II or greater), pericarditis, cerebrovascular accident, or implanted or continuous use of a pacemaker or defibrillator, or emphysema with FEV1 ≤ 50% predicted.
- Known history of immunodeficiency.
- Known additional malignancy that is progressing or required active treatment within the past 3 years, as defined in the protocol.
- Active autoimmune disease that required systemic treatment in past 2 years; replacement therapy is not considered a form of systemic treatment.
- NOTE: Other protocol defined Inclusion/Exclusion Criteria apply
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- ORR, defined as the proportion of subjects with a confirmed objective response of complete response (CR) or partial response (PR) based on Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) by investigator assessment
Secondary endpoints 5
- Adverse events (AEs), serious adverse events (SAEs), and AEs leading to discontinuation or death, and severity of AEs as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events Version 5.0 (CTCAE v5.0)
- Duration of response (DOR), defined as the time from date of first documented response (CR or PR) to date of documented progressive disease (PD), based on RECIST v1.1, per investigator
- Progression free survival (PFS), defined as the time from date of first dose of study treatment to date of documented PD based on RECIST v1.1 or death, whichever occurs first
- Response in injected and noninjected target lesions per RECIST v1.1
- Overall survival (OS), defined as the time from date of first dose of study treatment to date of death
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 3
LIBTAYO 350 mg concentrate for solution for infusion.
PRD7514335 · Product
- Active substance
- Cemiplimab
- Substance synonyms
- REGN2810
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS
- Max daily dose
- 350 mg milligram(s)
- Max total dose
- 350 mg milligram(s)
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Authorised
- ATC code
- L01XC33 — -
- Marketing authorisation
- EU/1/19/1376/001
- MA holder
- REGENERON IRELAND D.A.C.
- MA country
- Liechtenstein
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- Difference in pack, label and QP release sites. Material for clinical use may be assigned a longer shelf life compared to the MA
PRD10054531 · Product
- Active substance
- Vidutolimod
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRATUMORAL USE
- Max daily dose
- 10 mg milligram(s)
- Max total dose
- 10 mg milligram(s)
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- REGENERON PHARMACEUTICALS, INC.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10054530 · Product
- Active substance
- Vidutolimod
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 10 mg/g milligram(s)/gram
- Max total dose
- 10 mg milligram(s)
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- REGENERON PHARMACEUTICALS, INC.
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Regeneron Pharmaceuticals Inc.
- Sponsor organisation
- Regeneron Pharmaceuticals Inc.
- Address
- 777 Old Saw Mill River Road
- City
- Tarrytown
- Postcode
- 10591-6717
- Country
- United States
Scientific contact point
- Organisation
- Regeneron Pharmaceuticals Inc.
- Contact name
- Medical Affairs
Public contact point
- Organisation
- Regeneron Pharmaceuticals Inc.
- Contact name
- Medical Affairs
Third parties 7
| Organisation | City, country | Duties |
|---|---|---|
| Parexel International (IRL) Limited ORG-100022780
|
Dublin 2, Ireland | Other, Code 5, Data management |
| WCG Clinical Inc. ORG-100040730
|
Puyallup, United States | Other |
| Q2 Solutions LLC ORG-100017000
|
Ithaca, United States | Other |
| 4g Clinical LLC ORG-100042775
|
Wellesley, United States | Other |
| Icon Laboratories Inc. ORG-100037135
|
Farmingdale, United States | Other |
| Fisher Clinical Services UK Limited ORG-100012049
|
Horsham, United Kingdom | Other |
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | Other, Code 5 |
Locations
1 EU/EEA country · 9 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ended | 26 | 9 |
| Rest of world
Australia, United States
|
— | 174 | — |
Investigational sites
Centre Hospitalier Universitaire De Bordeaux
Oncology, 1 Rue Jean Burguet, 33000, Bordeaux
Hospital Hotel Dieu
Oncology, 1 Place Alexis Ricordeau, 44000, Nantes
Hopital Saint Louis
Oncology, 1 Avenue Claude Vellefaux, 75010, Paris
Centre Hospitalier Universitaire De Dijon
Oncology, 14 Rue Paul Gaffarel, 21000, Dijon
Centre Hospitalier Universitaire Grenoble Alpes
Oncology, Boulevard De La Chantourne, Cs 10217, Grenoble Cedex 9
Centre Hospitalier Universitaire De Lille
Oncology, Rue Michel Polonowski, 59000, Lille
Institut Gustave Roussy
Oncology, 114 Rue Edouard Vaillant, 94800, Villejuif
Assistance Publique Hopitaux De Marseille
Oncology, 144 Rue Saint Pierre, 13005, Marseille
Centre Leon Berard
Oncology, 28 Rue Laennec, 69008, Lyon
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Summary of results Art. 37(4) CTR
| Title | Submission date | Status | Type |
|---|---|---|---|
| CMP-001-009 SUM-104374
|
2025-10-30T19:09:20 | Submitted | Summary of Results |
Layperson summary Annex V
| Title | Submission date | Status | Type |
|---|---|---|---|
| REGN_CMP-001-009 PLS | 2025-10-30T19:10:01 | Submitted | Laypersons Summary of Results |
Documents 2 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Laypersons summary of results (for publication) | REGN_CMP-001-009 PLS | 1 |
| Summary of results (for publication) | CMP-001-009 CTIS Results Submission | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-12-01 | France | Acceptable with conditions 2024-01-25
|
2024-01-30 |