AzTrials
AzTrials

Metastatic CancerPhase 1/2NCT04725331

A Clinical Trial Assessing BT-001 Alone and in Combination With Pembrolizumab in Metastatic or Advanced Solid Tumors

A Phase 1/2 study testing BT-001 and Pembrolizumab [KEYTRUDA®] in people with Metastatic Cancer and Soft Tissue Sarcoma, run by Transgene at 5 sites in 2 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
Search all trials

Trial summary

This is a Phase I/IIa, multicenter, open-label, consecutive cohorts, dose-escalation study of BT-001 with repeated IT administrations alone and in combination with IV infusions of pembrolizumab. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
BT-001 and Pembrolizumab [KEYTRUDA®]
Could I receive a placebo?
No, everyone receives an active treatment
How many people
31 participants
Where
5 sitesin 2 countries
Study dates
25 Feb 2021to 22 Oct 2025
Phase
Phase 1/2
Are there results?
None posted in the registry

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

  • Have at least 1 injectable measurable cutaneous, subcutaneous or nodal lesion (direct injection or through the use of ultrasound guidance) not exceeding 50mm in longest diameter and whenever possible 1 distant non-injected measurable lesion.
  • Provision of a fresh tumor sample of the lesion that will be injected first and, whenever possible, from another lesion that is planned to be injected, at baseline and be willing to supply new tumor samples from a biopsy during treatment.
  • Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 or 1.
  • Have adequate hematological, hepatic and renal functions.
  • Have histologically confirmed, advanced/metastatic sarcoma (soft tissue and bone), Merkel cell carcinoma, melanoma, triple negative breast cancer or non-small cell lung cancer, with cutaneous or, palpable subcutaneous lesions or easily injectable lymph nodes.
  • Have failed and/or are intolerant to standard therapeutic options.

Who cannot join

  • Have had major surgery within 4 weeks of first study drug administration.
  • Have received prior treatment with a vaccinia oncolytic virus.
  • Have received prior systemic anti-cancer therapy including investigational agents within 4 weeks prior to the start of treatment.
  • Have received prior radiotherapy within 2 weeks of start of study treatment or have had a history of radiation pneumonitis
  • Has a diagnosis of immunodeficiency or is receiving chronic systemic steroid therapy (in dosing exceeding 10 mg daily of prednisone equivalent) or any other form of immunosuppressive therapy within 28 days prior the first dose of study drugs
  • Have a known additional malignancy that is progressing or has required active treatment within the past 3 years.
Show all 22Show fewer
  • Have an active autoimmune disease that has required systemic treatment in past 2 years (i.e., with use of disease modifying agents, corticosteroids or immunosuppressive drugs).
  • Have a history of (non-infectious) pneumonitis / interstitial lung disease that required steroids or has current pneumonitis / interstitial lung disease
  • Have an active infection requiring systemic therapy
  • Have a known history of HIV infection
  • Is taking an anticoagulant medication that cannot be interrupted prior to IT injections
  • Have had an allogenic tissue/solid organ transplant or allogenic stem cell or bone marrow transplantation
  • History of severe exfoliative skin conditions (e.g., eczema or atopic dermatitis) requiring systemic therapy for more than 4 weeks within 2 years prior to BT-001 initiation.
  • Have received prior therapy with an anti-PD-1, anti-PD-L1, or anti PD L2 agent or with an agent directed to another stimulatory or co-inhibitory T-cell receptor (e.g., CTLA-4, OX 40, CD137), and was discontinued from that treatment due to a Grade 3 or higher immune-related Adverse Event (irAE).
  • Have known active CNS metastases and/or carcinomatous meningitis.
  • Have a known history of Hepatitis B (defined as HBsAg reactive) or known active Hepatitis C virus (defined as HCV RNA [qualitative] is detected) infection.n.
  • Woman of childbearing potential who has a positive serum pregnancy test (within 72 hours) prior to the start of treatment.
  • Have received or receiving any live or live-attenuated vaccine within 30 days prior to the first dose of study intervention..
  • History of myocarditis or congestive heart failure, unstable angina, uncontrolled infection, or myocardial infarction 6 months prior to clinical trial entry.
  • Interstitial lung disease that is symptomatic and may interfere with the detection or management of suspected drug-related pulmonary toxicity
  • Is currently participating in or has participated in a study of an investigational agent or has used an investigational device within 4 weeks prior to the first dose of study treatment
  • Have severe hypersensitivity to the active substance or, to any of the excipients of (≥Grade 3) to pembrolizumab. and/or any of its excipients

Trial locations and contacts

5 sites in 2 countries.

5 sites in 2 countries

France4 sites

  • Institut Bergonié

    Bordeaux, 33000

  • Centre Léon Bérard

    Lyon, 69008

  • Hôpital Saint-Louis AP-HP

    Paris, 75010

  • Institut Gustave Roussy

    Villejuif, 94800

Other trials in France

Belgium1 site

  • Clinique Universitaire Saint-Luc

    Brussels, 1200

Other trials in Belgium

About this study

Official title: A Phase I/IIa Study of Intra-tumoral BT-001 (TG6030) Administered Alone and in Combination With Pembrolizumab in Patients With Cutaneous or, Subcutaneous Lesions or Easily Injectable Lymph Nodes of Metastatic/Advanced Solid Tumors.

This is a Phase I/IIa, multicenter, open-label, consecutive cohorts, dose-escalation study of BT-001 with repeated IT administrations alone and in combination with IV infusions of pembrolizumab.

Read the detailed description

This study will include 3 parts:

  • Phase I, Part A: Repeated intra-tumoral (IT) administrations of BT-001 as a single agent, in patients with metastatic/advanced solid tumors; dose-escalation will be employed.
  • Phase I, Part B: Repeated IT administrations of BT-001 in combination with intravenous (IV) infusions of pembrolizumab in patients with metastatic/advanced soft tissue sarcoma (STS), Merkel cell carcinoma (MCC), melanoma, triple negative breast cancer (TNBC) or non-small cell lung cancer (NSCLC)..
  • Phase IIa: Repeated IT administrations of BT-001 in combination with IV infusions of pembrolizumab in several cohorts of patients with defined metastatic or advanced solid tumor conditions.
Being tested
BT-001, Pembrolizumab [KEYTRUDA®]
Keywords
Superficial tumors, Metastatic cancer, Intratumoral, Safety, Oncolytic virus, Vaccinia virus, Solid tumors, Melanoma, Merkel, Sarcoma, TNBC, NSCLC, Pembrolizumab, BT-001, TG6030, Phase 1
Medical subject headings
Neoplasm Metastasis, Sarcoma, Carcinoma, Merkel Cell, Melanoma, Triple Negative Breast Neoplasms, Carcinoma, Non-Small-Cell Lung, Vaccinia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BT-001Biological

    Oncolytic Vaccinia virus containing genes encoding the 4-E03 human recombinant anti-hCTLA4 antibody and human GM-CSF administered at different dose [Phase I, Part A]; one dose lower and at Recommended Dose for Part B [Phase I, Part B] by intra-tumoral (IT) route.

  • Pembrolizumab [KEYTRUDA®]Drug

    Also called KEYTRUDA®

    Programmed death receptor (PD-1) blocking antibody administered at 200mg by intravenous (IV) infusions every 3 weeks.

Study groups in NCT04725331
Study groupRoleWhat they receive
Phase I, Part A - Dose escalation and safety of BT-001 aloneExperimentalDose escalation with repeated administrations of BT-001 directly into tumor as a single agent, in patients with metastatic or advanced solid tumors.
Receives: Biological: BT-001
Phase I, Part B - Safety of BT-001 in combination with pembrolizumabExperimentalRepeated administrations of BT-001 directly into tumor in combination with infusions of pembrolizumab in patients with metastatic or advanced soft tissue sarcoma (STS), Merkel cell carcinoma (MCC), melanoma, triple negative breast cancer (TNBC) or non-small cell lung cancer (NSCLC)..
Receives: Biological: BT-001; Drug: Pembrolizumab [KEYTRUDA®]
Phase IIa - Expansion cohorts of BT-001 in combination with pembrolizumabExperimentalRepeated administrations of BT-001 directly into tumor in combination with infusions of pembrolizumab in several cohorts of patients with defined metastatic or advanced solid tumor conditions: soft tissue sarcoma, Merkel cell carcinoma, melanoma, triple negative breast cancer, non-small cell lung cancer.
Receives: Biological: BT-001; Drug: Pembrolizumab [KEYTRUDA®]

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Phase I: Safety and tolerability (Adverse Event reported per NCI-CTCAE v5.0)

    Incidence of Adverse Event reported per NCI-CTCAE v5.0, Dose limiting toxicity and Serious Adverse Events.

    Measured: Up to 5 years

  2. Phase I, Part A: Recommended dose for Part B (RDPB) definition

    RDPB based on the safety data collected during the dose escalation phase (Phase I, Part A).

    Measured: Week 10-12

  3. Phase IIa (except Soft Tissue Sarcoma cohort): Immune Overall Response Rate (iORR) by iRECIST

    Percentage of patients whose best overall response is either a Complete Response or a Partial Response according to immune Response Evaluation Criteria In Solid Tumors (iRECIST) criteria over the the total number of evaluable patients. for injected and non-injected lesion(s)

    Measured: Up to 2 years

  4. Phase IIa (Soft Tissue Sarcoma cohort): Immune Disease Control Rate (iDCR) at 6 months by iRECIST

    Percentage of patients whose best overall response is either a Complete Response, a Partial Response or Stable Disease according to immune Response Evaluation Criteria In Solid Tumors (iRECIST) criteria over the the total number of evaluable patients.

    Measured: Up to 6 months

Secondary outcomes (5)

  1. Phase IIa: Safety and tolerability (Adverse Event reported per NCI-CTCAE v5.0)

    Incidence of Adverse Event reported per NCI-CTCAE v5.0, Dose limiting toxicity and Serious Adverse Events.

    Measured: Up to 5 years

  2. Disease Control Rate (DCR) and immune DCR by RECIST version 1.1 and iRECIST

    Percentage of patients whose best overall response is either a Complete Response, a Partial Response or Stable Disease according to Response Evaluation Criteria In Solid Tumors version 1.1 (RECIST 1.1) criteria or immune RECIST over the total number of evaluable patients.

    Measured: 4 months or 6 months

  3. Progression Free Survival (PFS) and immune PFS duration by RECIST version 1.1 and iRECIST

    Time from the first BT-001 administration to the date of first documented tumor progression according to Response Evaluation Criteria In Solid Tumors version 1.1 (RECIST 1.1) criteria or immune RECIST or death due to any cause, whichever occurs first over evaluable patients

    Measured: Up to 2 years

  4. Duration of overall Response (DoR) and immune DOR by RECIST version 1.1 and iRECIST

    Time from the date of first documented response (CR or PR) to the date of first documented disease progression according to Response Evaluation Criteria In Solid Tumors version 1.1 (RECIST 1.1) criteria or immune RECIST or the date of death due to underlying cancer over the number of patients whose Best Overall Response is Complete Response or Partial Response.

    Measured: Up to 2 years

  5. Overall Survival (OS) duration

    Time from first BT-001 administration to the date of death due to any cause over evalauable patients.

    Measured: Up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Jan 2021
  2. Study start25 Feb 2021
  3. Primary completion22 Oct 2025
  4. Study completion22 Oct 2025

Why it stopped: Sponsor decision following completion of Phase I part not driven by safety reason

Resources

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 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
31 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04725331
Sponsor's protocol ID
BT-001.01
Other IDs
2020-000505-80, MK3475-E37, KEYNOTE-E37
Status last verified
Nov 2025
First submitted
25 Jan 2021
Last updated
21 Nov 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing BT-001 and Pembrolizumab [KEYTRUDA®] in people with Metastatic Cancer and Soft Tissue Sarcoma, run by Transgene at 5 sites in 2 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 22 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: stopped early, as of the sponsor's last update on 21 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in France, Belgium. 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

The study was stopped early (reason given: Sponsor decision following completion of Phase I part not driven by safety reason). Results are not available in the source record.

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?

Phase I: Safety and tolerability (Adverse Event reported per NCI-CTCAE v5.0) (time frame: Up to 5 years). The study lists 4 primary outcomes in all. 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 NCT04725331.

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 Nov 2025.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in