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Unresectable MelanomaPhase 1NCT04625205

A Clinical Study of the Safety and Activity of the Investigational Cell Therapy NEO-PTC-01 in Patients With Advanced Melanoma

A Phase 1 study testing NEO-PTC-01, IL-2 and PD-1 Inhibitors in people with Unresectable Melanoma and Metastatic Melanoma, run by BioNTech US Inc. at 2 sites in 2 countries.

Terminated

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

Official record
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Trial summary

This study will investigate the safety and activity of NEO-PTC-01 in patients with unresectable or metastatic melanoma. NEO-PTC-01 is an autologous personalized T cell (PTC) product for adoptive cell therapy that is manufactured ex vivo and targets neoantigens displayed on the patient's tumor and the tumor… Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Unresectable Melanoma, Metastatic Melanoma
What is being tested?
NEO-PTC-01, IL-2 and PD-1 Inhibitors
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
22 participants
Where
2 sitesin 2 countries
Study dates
1 Dec 2020to 26 Mar 2025
Phase
Phase 1
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 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Adult (age 18 to 75) men and women willing and able to give written informed consent.
  • Histologically confirmed unresectable or metastatic melanoma.
  • Part 1:
  • Have previously received a PD-1/PD-L1 inhibitor (either as single agent or in combination) and a cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4) inhibitor-containing regimen (single agent or combination) prior to NEO-PTC-01, with disease progression following these therapies or otherwise lack of clinical benefit as determined by the study investigator.
  • Part 2:
  • Have received/are currently receiving a PD-1/PD-L1 inhibitor (as a single agent or in combination with CTLA-4) for at least 3 months.
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  • Have documented SD by RECIST v1.1 or clinically asymptomatic progressive disease on the most recent imaging assessment, which must have occurred within 3 months of enrollment.
  • In the opinion of the investigator, are medically eligible and able to continue with PD-1/PD-L1 inhibitor therapy.
  • In the opinion of the investigator, would benefit from the addition of a T-cell based therapy.
  • For known serine-threonine kinase (BRAF) mutant patients: Patients must have also received targeted therapy (B-Raf inhibitor or B-Raf/mitogen-activated protein kinase enzyme [MEK] combination therapy) prior to NEO-PTC-01, unless deemed not appropriate to receive these treatments by the investigator.
  • Have at least one site of measurable disease by RECIST v1.1.
  • At least one site of disease must be accessible to biopsy for tumor tissue for sequence and immunological analysis. The biopsy site may be the same as the measurable site so long as it remains measurable. Surgical resection of the measurable site may not be performed if that site is the only measurable lesion. An archival biopsy may be used in place if the biopsy was taken within 6 months of informed consent.
  • Have Eastern Cooperative Oncology Group Performance Status of 0 or 1.
  • Recovered from all toxicities associated with prior treatment to acceptable baseline status (for laboratory toxicities see below limits for inclusion) or a National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0, Grade of 0 or 1, except for toxicities not considered by the treating physician to be a safety risk (e.g., alopecia).
  • Screening laboratory values must meet the following criteria and should be obtained prior to any Production phase assessments:
  • White blood cell count ≥ 3 × 10^3/μL.
  • Absolute neutrophil count ≥ 1.5 × 10^3/μL.
  • Platelet count ≥ 100 × 10^3/μL.
  • Hemoglobin > 9 g/dL or 6 mmol/L.
  • Serum creatinine ≤ 1.5 × upper limit of normal (ULN) or creatinine clearance ≥ 50 mL/min by Cockcroft-Gault.
  • Aspartate aminotransferase and alanine aminotransferase ≤ 3 × ULN.
  • Total bilirubin ≤ 1.5 × ULN (except in patients with Gilbert Syndrome, who can have total bilirubin < 3.0 mg/dL).
  • International Normalized Ratio, Prothrombin Time (PT), or Activated Partial Thromboplastin Time (aPTT) ≤ 1.5 × ULN unless the patient is receiving anticoagulant therapy, as long as PT or aPTT is within therapeutic range of intended use of anticoagulants.

Who cannot join

  • Age greater than 75 years or less than 18 years.
  • Prior cell therapy (including tumor-infiltrating lymphocyte, chimeric antigen receptor T cell, and T-cell receptor-based products) within 12 months prior to signing of informed consent form.
  • Have an active or history of autoimmune disease (known or suspected). Exceptions are permitted for vitiligo, type I diabetes mellitus, residual hypothyroidism due to autoimmune condition requiring only hormone replacement, psoriasis not requiring systemic treatment, or conditions not expected to recur in the absence of an external trigger.
  • Have known active central nervous system metastases and/or carcinomatous meningitis. Patients with previously treated brain metastases may participate provided they are stable (without evidence of progression by imaging [using the identical imaging modality for each assessment, either MRI or CT scan] for at least 4 weeks prior to enrollment and any neurologic symptoms have returned to baseline), have no evidence of new or enlarging brain metastases, and are not using steroids for at least 7 days prior to enrollment. This exception does not include carcinomatous meningitis, which is excluded regardless of clinical and/or radiographic stability.
  • Active systemic infections requiring intravenous antimicrobial therapy, coagulation disorders or other active major medical illnesses of the cardiovascular, respiratory or immune system, as evidenced by a positive stress thallium or comparable test, myocardial infarction, clinically significant cardiac arrhythmias such as uncontrolled atrial fibrillation, ventricular tachycardia, or second- or third-degree heart block, and obstructive or restrictive pulmonary disease.
  • Active major medical illnesses of the immune system including conditions requiring systemic treatment with either corticosteroids (> 10 mg daily prednisone equivalents) or other immunosuppressive medications within 14 days prior to NEO-PTC-01 infusion. Inhaled or topical steroids and adrenal replacement doses (≤ 10 mg daily prednisone equivalents) are permitted in the absence of active autoimmune disease.
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  • Known human immunodeficiency virus infection, active chronic hepatitis B or C, and/or life-threatening illnesses unrelated to cancer that could, in the investigator's opinion, interfere with participation in this study.
  • Have any underlying medical condition, psychiatric condition, or social situation that, in the investigator's opinion, would interfere with participation in the study.
  • Have a planned major surgery that is expected to interfere with study participation or confound the ability to analyze study data.
  • Are pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the study, starting with the screening visit through 150 days after the end of the trial visit. Nursing women are excluded from this study because there is an unknown but potential risk of AEs in nursing infants secondary to treatment of the mother with treatments to be administered in this study.
  • Have a history of another invasive malignancy aside from melanoma, except for the following circumstances:
  • Patient has been disease-free for at least 2 years and is deemed by the investigator to be at low risk for recurrence of that malignancy.
  • Patient was not treated with systemic chemotherapy for carcinoma in situ of the breast, oral cavity, or cervix, basal cell, or squamous cell carcinoma of the skin.

Trial locations and contacts

2 sites in 2 countries.

2 sites in 2 countries

Belgium1 site

  • Universitair Ziekenhuis Brussel

    Brussels, 1090

Other trials in Belgium

Netherlands1 site

  • Netherlands Cancer Institute - Antoni van Leeuwenhoek

    Amsterdam, 1066 CX

Other trials in Netherlands

About this study

Official title: An Open-label, Phase I Study of NEO-PTC-01 in Patients With Advanced or Metastatic Melanoma

This study will investigate the safety and activity of NEO-PTC-01 in patients with unresectable or metastatic melanoma. NEO-PTC-01 is an autologous personalized T cell (PTC) product for adoptive cell therapy that is manufactured ex vivo and targets neoantigens displayed on the patient's tumor and the tumor microenvironment.

The study will be conducted in two parts, Part 1 (Dose Finding) and Part 2 (Dose Expansion).

Read the detailed description

Part 1 will test two doses of NEO-PTC-01 and will be structured according to a 3+3 dose escalation design. After the highest tolerated NEO-PTC-01 dose is identified, 2 additional evaluations in Part 1 are planned to investigate the combination of NEO-PTC-01 with either interleukin (IL) 2-directed or programmed cell death protein 1 (PD-1) inhibitor therapy, respectively.

Part 2 will test the dose deemed to be safe in the dose-finding part of the study to further define the safety of NEO-PTC-01 in patients currently receiving PD-1/ programmed death ligand 1 (PD-L1) inhibitors (as single agent or in combination with cytotoxic T-lymphocyte-associated antigen-4 [CTLA4] inhibitors) as first line therapy for metastatic melanoma.

Patients who were treated on previous protocol versions and left the study due to disease progression or who have completed the 52-week follow-up period, may re-enter the study for an extended 5-year follow up in which additional assessments will be conducted.

Being tested
NEO-PTC-01, IL-2, PD-1 Inhibitors
Keywords
Melanoma, Solid Tumor
Medical subject headings
Melanoma

What participants receive

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

  • NEO-PTC-01Biological

    Administered via intravenous infusion.

  • IL-2Drug

    Administered via intravenous infusion.

  • PD-1 InhibitorsDrug

    Administered via intravenous infusion.

Study groups in NCT04625205
Study groupRoleWhat they receive
Part 1 dose finding phase: NEO-PTC-01 Dose 1ExperimentalMonotherapy - Dose 1
Receives: Biological: NEO-PTC-01
Part 1 dose finding phase: NEO-PTC-01 Dose 2ExperimentalMonotherapy - Dose 2
Receives: Biological: NEO-PTC-01
Part 1 dose finding phase: NEO-PTC-01 plus IL-2ExperimentalNEO-PTC-01 in combination with a fixed dose of IL-2 (cohort will only be open in countries where IL-2 is approved)
Receives: Biological: NEO-PTC-01; Drug: IL-2
Part 1 dose finding phase: NEO-PTC-01 plus PD-1 inhibitorExperimentalThe PD-1 inhibitor therapy will be introduced, beginning 1 to 2 weeks post NEO-PTC-01, to patients who failed PD-1/PD-L1 inhibitor therapy prior to enrollment to the NTC-001 study
Receives: Biological: NEO-PTC-01; Drug: PD-1 Inhibitors
Part 2 dose expansion phase: NEO-PTC-01ExperimentalPatients currently receiving PD-1/PD-L1 inhibitors (as single agent or in combination with CTLA4 inhibitors) as therapy for metastatic melanoma
Receives: Biological: NEO-PTC-01

What is being measured

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

  1. Rate of adverse events (AEs), including serious adverse events (SAEs) and AEs leading to treatment discontinuation

    Rate of AEs, including SAEs and AEs leading to treatment discontinuation and those AEs and SAEs detected during symptom-directed physical examinations (changes in safety laboratory evaluations, physical examination findings, and vital signs).

    Measured: Day 1 up 5 years

Secondary outcomes (7)

  1. Progression-free survival, defined as the time from the date of first dosing of NEO-PTC-01 to the date of first documented progressive disease (PD) or death, whichever comes first

    Clinical activity endpoints, based on investigator assessment of serial radiographic evaluations [Computed Tomography (CT) or Magnetic Resonance Imaging (MRI)] to determine response to treatment and progression of disease based on Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1.

    Measured: Day 1 up 5 years

  2. Objective response rate, defined as the proportion of patients who achieve complete response (CR) or partial response (PR) based on RECIST v1.1

    Clinical activity endpoints, based on investigator assessment of serial radiographic evaluations (CT or MRI) to determine response to treatment and progression of disease based on RECIST version 1.1.

    Measured: Day 1 up 5 years

  3. Duration of response, defined as the date of the first documentation of a confirmed response to the date of the first documented PD

    Clinical activity endpoints, based on investigator assessment of serial radiographic evaluations (CT or MRI) to determine response to treatment and progression of disease based on RECIST version 1.1.

    Measured: Day 1 up 5 years

  4. Clinical benefit rate, defined as the proportion of patients who achieve CR, PR, or stable disease (SD) based on RECIST v1.1

    Clinical activity endpoints, based on investigator assessment of serial radiographic evaluations (CT or MRI) to determine response to treatment and progression of disease based on RECIST version 1.1.

    Measured: Day 1 up 5 years

  5. Best overall response, defined as the best response according to RECIST 1.1

    Clinical activity endpoints, based on investigator assessment of serial radiographic evaluations (CT or MRI) to determine response to treatment and progression of disease based on RECIST version 1.1

    Measured: Day 1 up 5 years

  6. Time to first subsequent therapy, defined as the time from end of treatment to the start date of first subsequent therapy

    Clinical activity endpoints, based on investigator assessment of serial radiographic evaluations (CT or MRI) to determine response to treatment and progression of disease based on RECIST version 1.1.

    Measured: Day 1 up 5 years

  7. Overall survival, defined as the time from the date of first dosing of NEO-PTC-01 to the date of death due to any cause

    Measured: Day 1 up 5 years

Timeline

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

  1. First posted12 Nov 2020
  2. Study start1 Dec 2020
  3. Primary completion26 Mar 2025
  4. Study completion26 Mar 2025

Why it stopped: Sponsor decision

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
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
22 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04625205
Sponsor's protocol ID
NTC-001
Other IDs
2019-003908-13, 2023-508524-35-00, 2023, 2019
Status last verified
Apr 2025
First submitted
6 Nov 2020
Last updated
6 Apr 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Borgers JSW, Lenkala D, Kohler V, Jackson EK, Linssen MD, Hymson S, McCarthy B, O'Reilly Cosgrove E, Balogh KN, Esaulova E, Starr K, Ware Y, Klobuch S, Sciuto T, Chen X, Mahimkar G, Sheen JHF, Ramesh S, Wilgenhof S, van Thienen JV, Scheiner KC, Jedema I, Rooney M, Dong JZ, Srouji JR, Juneja VR, Arieta CM, Nuijen B, Gottstein C, Finney OC, Manson K, Nijenhuis CM, Gaynor RB, DeMario M, Haanen JB, van Buuren MM. Personalized, autologous neoantigen-specific T cell therapy in metastatic melanoma: a phase 1 trial. Nat Med. 2025 Mar;31(3):881-893. doi: 10.1038/s41591-024-03418-4. Epub 2025 Jan 3.

    PubMed 39753970

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing NEO-PTC-01, IL-2 and PD-1 Inhibitors in people with Unresectable Melanoma and Metastatic Melanoma, run by BioNTech US Inc. at 2 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 23 inclusion and 13 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 6 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in Belgium, Netherlands. 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 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.

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). 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?

Rate of adverse events (AEs), including serious adverse events (SAEs) and AEs leading to treatment discontinuation (time frame: Day 1 up 5 years). 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 NCT04625205, 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 6 Apr 2025.

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