NCT03954067

Brief Summary

The purpose of this study is to assess the safety and tolerability of ASP9801 and to determine the recommended phase 2 dose (RP2D). The study will also evaluate antitumor activity, objective response rate, pharmacokinetics and virus shedding of ASP9801 as a single agent, as well as in combination with pembrolizumab, an anti-programmed cell death protein 1 (PD-1) checkpoint inhibitor.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
72

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Sep 2019

Longer than P75 for phase_1

Geographic Reach
1 country

17 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

May 14, 2019

Completed
3 days until next milestone

First Posted

Study publicly available on registry

May 17, 2019

Completed
4 months until next milestone

Study Start

First participant enrolled

September 3, 2019

Completed
4.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 19, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 19, 2024

Completed
Last Updated

April 22, 2025

Status Verified

April 1, 2025

Enrollment Period

4.6 years

First QC Date

May 14, 2019

Last Update Submit

April 17, 2025

Conditions

Keywords

immunotherapyvaccinia virusvisceral lesionscutaneous lesionsintratumoral injectionASP9801subcutaneous lesionsoncolytic virus

Outcome Measures

Primary Outcomes (5)

  • Dose Limiting Toxicities (DLT) - dose escalation part

    Incidence of dose limiting toxicities

    Up to 28 days

  • Safety and tolerability assessed by Adverse Events (AEs)

    An AE is any untoward medical occurrence in a subject administered an investigational product (IP), and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IP whether or not considered related to the IP.

    Up to 12 months

  • Number of participants with laboratory value abnormalities and/or adverse events

    Number of participants with potentially clinically significant laboratory values.

    Up to 12 months

  • Number of participants with vital sign abnormalities and /or adverse events

    Number of participants with potentially clinically significant vital sign values.

    Up to 12 months

  • Safety assessed by 12- lead electrocardiograms (ECGs) adverse events

    12-lead ECGs will be read and assessed locally. Any clinically significant adverse changes on the ECG will be reported as Adverse Events.

    Up to 12 months

Secondary Outcomes (8)

  • Percent change from baseline in antitumor activity of ASP9801

    Up to 12 months

  • Objective Response Rate per imRECIST

    Up to 12 months

  • ASP9801 viral DNA in blood

    Up to 12 months

  • Viral shedding of ASP9801 in saliva

    Up to 12 months

  • Viral shedding of ASP9801 in urine

    Up to 12 months

  • +3 more secondary outcomes

Study Arms (8)

Dose Escalation - cutaneous or subcutaneous lesions

EXPERIMENTAL

Participants will receive ASP9801 (1x10\^7 pfu/mL) on days 1 and 15 of 28 day cycles to determine the recommended phase 2 dose. After cycle 2, participants who have not met any discontinuation criteria and receiving clinical benefit may be treated on continuous cycles until treatment discontinuation criteria are met.

Biological: ASP9801

Dose Escalation - visceral lesions

EXPERIMENTAL

Participants will receive ASP9801 (1x10\^8 pfu/mL) on days 1 and 15 of 28 day cycles to determine the recommended phase 2 dose. After cycle 2, participants who have not met any discontinuation criteria and receiving clinical benefit may be treated on continuous cycles until treatment discontinuation criteria are met.

Biological: ASP9801

Dose Expansion (Monotherapy) - cutaneous or subcutaneous lesions

EXPERIMENTAL

Participants will receive ASP9801 (1x10\^8 pfu/mL) on days 1 and 15 of 28 day cycles at the dose recommended by the dose escalation phase. After cycle 2, participants who have not met any discontinuation criteria and receiving clinical benefit may be treated on continuous cycles until treatment discontinuation criteria are met.

Biological: ASP9801

Dose Expansion (Monotherapy) - visceral lesions

EXPERIMENTAL

Participants will receive ASP9801 (5x10\^8 pfu/mL) on days 1 and 15 of 28 day cycles at the dose recommended by the dose escalation phase. After cycle 2, participants who have not met any discontinuation criteria and receiving clinical benefit may be treated on continuous cycles until treatment discontinuation criteria are met.

Biological: ASP9801

Dose Expansion (Monotherapy Induction) - cutaneous or subcutaneous lesions

EXPERIMENTAL

Participants will receive ASP9801 (5x10\^8 pfu/mL) on days 1, 8, 15 and 22 of the first 28 day cycle. Participants will receive ASP9801 on days 1 and 15 on the second 28 day cycle at the dose recommended by the dose escalation phase. After cycle 2, participants who have not met any discontinuation criteria and receiving clinical benefit may be treated on continuous cycles until treatment discontinuation criteria are met.

Biological: ASP9801

Dose Expansion (Combination Therapy) - cutaneous or subcutaneous lesions

EXPERIMENTAL

Participants will receive ASP9801 (1x10\^8 pfu/mL) on days 1 and 15 of 28 day cycles at the dose recommended by the dose escalation phase. Participants will also receive pembrolizumab starting on day 1 and once every 6 weeks. After cycle 2, participants who have not met any discontinuation criteria and receiving clinical benefit may be treated on continuous cycles until treatment discontinuation criteria are met.

Combination Product: Pembrolizumab

Dose Expansion (Combination Induction Therapy) - cutaneous or subcutaneous lesions

EXPERIMENTAL

Participants will receive ASP9801 (5x10\^8 pfu/mL) on days 1, 8, 15 and 22 of first 28 day cycle at the dose recommended by the dose escalation phase. Participants will also receive pembrolizumab starting on day 1 and once every 6 weeks. Participants will receive ASP9801 on days 1 and 15 on the second 28 day cycle at the dose recommended by the dose escalation phase. After cycle 2, participants who have not met any discontinuation criteria and receiving clinical benefit may be treated on continuous cycles until treatment discontinuation criteria are met.

Combination Product: Pembrolizumab

Dose Expansion (Combination Therapy) - visceral lesions

EXPERIMENTAL

Participants will receive ASP9801 (5x10\^8 pfu/mL) on days 1 and 15 of 28 day cycles at the dose recommended by the dose escalation phase. Participants will also receive pembrolizumab starting on day 1 and once every 6 weeks. After cycle 2, participants who have not met any discontinuation criteria and receiving clinical benefit may be treated on continuous cycles until treatment discontinuation criteria are met.

Combination Product: Pembrolizumab

Interventions

ASP9801BIOLOGICAL

Administered by intratumoral injection

Dose Escalation - cutaneous or subcutaneous lesionsDose Escalation - visceral lesionsDose Expansion (Monotherapy Induction) - cutaneous or subcutaneous lesionsDose Expansion (Monotherapy) - cutaneous or subcutaneous lesionsDose Expansion (Monotherapy) - visceral lesions
PembrolizumabCOMBINATION_PRODUCT

Administered by Intravenous Infusion

Also known as: KEYTRUDA®
Dose Expansion (Combination Induction Therapy) - cutaneous or subcutaneous lesionsDose Expansion (Combination Therapy) - cutaneous or subcutaneous lesionsDose Expansion (Combination Therapy) - visceral lesions

Eligibility Criteria

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

You may qualify if:

  • Subject must have histologically- or cytologically-confirmed diagnosis of advanced or metastatic solid tumor(s).
  • Subject has measurable disease as determined by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. At least 1 lesion must be suitable for intratumoral (IT) injection. Lesions for injection must be ≥ 10 mm and ≤ 60 mm in longest diameter.
  • Subject has had disease progression after, been intolerant to, or has refused all available therapies that are known to confer clinical benefit. Note: There is no limit to the number of prior treatment regimens.
  • Subject has a predicted life expectancy ≥ 12 weeks.
  • Subject has at least 2 sites of disease suitable for biopsy and is willing and able to undergo required tumor biopsies according to the treating institution's guidelines at screening and during study treatment.
  • Subject has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
  • A female subject is eligible to participate if she is not pregnant as documented by negative pregnancy test within 72 hours prior to treatment and at least 1 of the following conditions applies:
  • Not a woman of childbearing potential (WOCBP) OR
  • WOCBP who agrees to follow the contraceptive guidance throughout the treatment period and for at least 180 days after the final study investigational product (IP) administration.
  • Female subject must agree not to breastfeed starting at screening, and throughout the study period and 180 days after the final study IP administration.
  • Female subject must not donate ova starting at screening, and throughout the study period and for 180 days after the final study IP administration.
  • Male subject must agree to remain abstinent or use a condom throughout the study period and for 180 days after the final study IP administration.
  • Male subject with female partner(s) of childbearing potential must agree to use contraception during the treatment period and for at least 180 days after the final study IP administration.
  • Male subject must not donate sperm during the treatment period and for at least 180 days after the final study IP administration.
  • Subject must be willing and able to comply with the study requirements including prohibited concomitant medication restrictions.
  • +2 more criteria

You may not qualify if:

  • Subject has ongoing toxicity ≥ National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI CTCAE) grade 2 attributable to prior antineoplastic therapies considered clinically significant.
  • Subject who has had major surgery ≤ 4 weeks of screening. Subjects must have recovered from prior procedures and/or any complications from surgery prior to starting study treatment.
  • Subject is concurrently participating in another interventional study or has received an investigational product ≤ 30 days or 5 half-lives whichever is shorter, prior to first IP administration.
  • Subject with symptomatic or untreated central nervous system (CNS) metastases or leptomeningeal disease. Subjects with treated symptomatic brain metastases should be neurologically stable (without evidence of progression by imaging for at least 4 weeks prior to screening and any neurologic symptoms have returned to baseline) and off steroids for at least 2 weeks prior to first IP administration. Subjects with carcinomatous meningitis are excluded regardless of clinical stability.
  • Subject with active or prior autoimmune or inflammatory disorders requiring systemic therapy within past 2 years (including inflammatory skin conditions or severe eczema, inflammatory bowel disease (e.g., colitis or Crohn's disease), diverticulitis (with the exception of diverticulosis), celiac disease, systemic lupus erythematosus, Sarcoidosis syndrome, Wegener syndrome (granulomatosis with polyangiitis), Graves' disease, rheumatoid arthritis, hypophysitis, uveitis, etc.
  • The following are exceptions to this criterion:
  • Subject with vitiligo or alopecia
  • Subject with hypothyroidism (e.g., following Hashimoto syndrome) stable on hormone replacement
  • Any chronic skin condition that does not require systemic therapy
  • Subject with another malignancy that currently requires treatment.
  • Subject with only tumors encasing major vascular structures such as the carotid artery, tumors adjacent to vital neurovascular structures or tumors in locations that are at high risk for adverse events (AEs) or otherwise not considered appropriate for IT injection. Subjects with such tumors that have other injectable tumors would be eligible.
  • Subject with inadequate organ and marrow functions meeting any of the below criteria:
  • Leukocytes \< 3000/μL
  • Absolute neutrophil count \< 1500/μL
  • Platelets \< 100,000/μL
  • +24 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (17)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

University of Arizona - Arizona Cancer Center

Tucson, Arizona, 85719, United States

Location

The Angeles Clinic and Research Institute

Los Angeles, California, 90025, United States

Location

The University of Chicago Medical Center

Chicago, Illinois, 60637, United States

Location

University of Kentucky, Markey Cancer Center

Lexington, Kentucky, 40536, United States

Location

Norton Cancer Institute

Louisville, Kentucky, 40241, United States

Location

University of Maryland Medical Center

Baltimore, Maryland, 21201, United States

Location

Henry Ford Hospital

Detroit, Michigan, 48202, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Nebraska Methodist Hospital

Omaha, Nebraska, 68130, United States

Location

Roswell Park Cancer Institute - Medical Oncology

Buffalo, New York, 14263, United States

Location

Fox Chase Cancer Center

Philadelphia, Pennsylvania, 19111, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15260, United States

Location

Mary Crowley Cancer Research Center

Dallas, Texas, 75251, United States

Location

The University of Texas - MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

UVA Cancer Center

Charlottesville, Virginia, 22903, United States

Location

VCU Massey Cancer Center

Richmond, Virginia, 23298, United States

Location

Related Links

MeSH Terms

Conditions

Neoplasm MetastasisVaccinia

Interventions

pembrolizumab

Condition Hierarchy (Ancestors)

Neoplastic ProcessesNeoplasmsPathologic ProcessesPathological Conditions, Signs and SymptomsPoxviridae InfectionsDNA Virus InfectionsVirus DiseasesInfections

Study Officials

  • Medical Director

    Astellas Pharma Global Development, Inc.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 14, 2019

First Posted

May 17, 2019

Study Start

September 3, 2019

Primary Completion

April 19, 2024

Study Completion

April 19, 2024

Last Updated

April 22, 2025

Record last verified: 2025-04

Data Sharing

IPD Sharing
Will share

Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as products terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.

Shared Documents
STUDY PROTOCOL, SAP, CSR
Time Frame
Access to participant level data is offered to researchers after publication of the primary manuscript (if applicable) and is available as long as Astellas has legal authority to provide the data.
Access Criteria
Researchers must submit a proposal to conduct a scientifically relevant analysis of the study data. The research proposal is reviewed by an Independent Research Panel. If the proposal is approved, access to the study data is provided in a secure data sharing environment after receipt of a signed Data Sharing Agreement.
More information

Locations