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Hepatocellular CarcinomaPhase 1/2DUET-1NCT05120271

BOXR1030 T Cells in Subjects With Advanced GPC3-Positive Solid Tumors

A Phase 1/2 study testing CAR-GPC3 T Cells in people with Hepatocellular Carcinoma and Squamous Cell Carcinoma of the Lung, run by Sotio Biotech Inc. at 8 sites in 2 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

This is a first-in-human (FIH), Phase 1/2, open-label, multicenter study to assess safety and determine the recommended Phase 2 dose (RP2D) of BOXR1030 administration after lymphodepleting chemotherapy (LD chemotherapy) in subjects with glypican-3 positive (GPC3+) advanced solid tumors. Read more about this study

Who is it for?
Ages 18 to 80 years
What is being tested?
CAR-GPC3 T Cells
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
7 participants
Where
8 sitesin 2 countries
Study dates
26 Oct 2022to Dec 2042
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

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 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Aged 18 to 80 years at time of enrollment
  • Body weight >/= 50kg
  • Able to provide a recent tumor specimen taken within 6 months prior to signing consent and after the initiation of the subject's most recent systemic anti-cancer therapy, for GPC3 expression assessment by immunohistochemistry (IHC). Previously collected tumor tissue older than 6 months at time of GPC3 IHC testing or collected prior to initiation of current or last systemic therapy may be permitted for GPC3 prescreening. If prescreening sample is found to be GPC3+, a new tumor biopsy will be needed to confirm tumor remains GPC3+ in order to proceed.
  • Histologically confirmed advanced unresectable or metastatic hepatocellular carcinoma (HCC), squamous cell carcinoma (SCC) of the lung, myxoid/round cell liposarcoma (MRCLS), or Merkel cell carcinoma (MCC) with GPC3 overexpression by IHC. Subjects must consent to IHC testing in a separate informed consent. Note: Tumor samples will be sent to a central laboratory for GPC3 expression analysis.
  • Documentation of disease progression or refractory disease or intolerance to prior lines of standard-of-care therapies. Patients with tumors with genetic alterations and mutations (e.g., breast cancer gene, epidermal growth factor receptor mutations, and anaplastic lymphoma kinase translocation) who have approved targeted therapies available for their cancer will need to have been treated with such approved therapies or refused such approved targeted therapy for their cancer prior to enrolling in this study.
  • Life expectancy >16 weeks
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  • Have adequate organ function (renal/hepatic/pulmonary)
  • Left ventricular ejection fraction ≥50% by multiple-gated acquisition scan or echocardiogram
  • Eastern Cooperative Group performance status of 0 to 1
  • For subjects with HCC:
  • Child-Pugh Score of A
  • No fibrolamellar carcinoma or mixed hepatocellular cholangiocarcinoma histology
  • No grade 2 or grade 3 ascites based on the European Association for the Study of Liver guidelines.
  • A minimum of 2 sites of disease, including at least 1 site that is measurable by RECIST 1.1 criteria to ensure sufficient disease for response assessment. At least 1 of the other lesions must be considered adequate for Protocol-required tumor biopsy.
  • Adequate wash-out of prior systemic therapy for underlying malignancy, relative to leukapheresis:
  • Last dose of any antineoplastic treatment must be at least 2 weeks before leukapheresis.
  • Last dose of any investigational agent must be at least 3 half-lives of the treatment, or 28 days, before leukapheresis (whichever is shorter).
  • Adequate wash-out of prior systemic therapy for underlying malignancy, relative to LD chemotherapy:
  • Subjects may receive an additional dose of their last received form of therapy at the same dose and dosing schedule following leukapheresis as bridging therapy; however, the required wash-out period prior to start of LD chemotherapy must be adhered to:
  • The last dose of systemic nitrosourea or systemic mitomycin-C must be at least 6 weeks before the first dose of LD chemotherapy in this study.
  • For all other systemic cytotoxic chemotherapy, the wash-out must be the duration of a full cycle of that therapy. For example, if the prior therapy is given in 3-week cycles, there must be at least 3 weeks between the last dose of that therapy and the first dose of LD chemotherapy in this study. If the prior therapy is administered more frequently than every 2 weeks, a minimum 2-week wash-out is required.
  • For biologic therapy (e.g., antibodies) the wash-out must be either the duration of the biologic agent dosing interval, or 3 weeks, whichever is shorter.
  • For small molecule therapies, the wash-out must be 5 half-lives of the drug.
  • For any investigational agent, the wash-out must be 3 half-lives of the investigational agent, or 4 weeks, whichever is shorter.
  • Note: Local radiation of lesions is allowed if indicated for palliation requiring 1 week wash-out prior to start of BOXR1030 dosing if < 2 weeks of radiotherapy for non-central nervous system (CNS) disease; locally treated lesions will be considered non-target lesions. Hormone ablation is also allowed as clinically indicated.

Who cannot join

  • Prior treatment with adoptive cell therapy (e.g., CAR T-cell therapy, natural killer cell therapy, engineered T-cell receptor therapy).
  • History of allogenic hematopoietic stem cell transplant.
  • Known untreated CNS tumors or brain metastasis. Subjects are eligible if CNS metastases are asymptomatic, have been treated with radiotherapy for at least 1 month prior to informed consent, are off corticosteroids and have neurologically returned to baseline (residual signs or symptoms related to the CNS treatment are permitted). Imaging obtained for the purpose of CNS metastases management performed during screening must document radiographic stability of CNS lesions for at least 1 month prior to leukapheresis and be performed after completion of any CNS directed therapy. If brain scans are performed, magnetic resonance scans are preferred; however, computed tomography scans are acceptable if magnetic resonance imaging is medically contraindicated. CNS evaluation for subjects with no suspicion of brain tumors in their history is not required for the study. Subjects with known leptomeningeal metastases are excluded.
  • Subjects who have not recovered to < 1 or baseline from all AEs due to previous therapies (subjects with ≤ grade 2 peripheral neuropathy that has been stable for at least 4 weeks or < grade 2 endocrine-related AEs that has been stable for at least 4 weeks on replacement therapy).
  • Planned use of any antineoplastic treatment or investigational agent from the time of the first dose of LD chemotherapy through the end of study participation, except for allowed local radiation of lesions for palliation (to be considered non-target lesions after treatment) and hormone ablation.
  • Uncontrolled or life-threatening symptomatic concomitant disease including clinically significant gastrointestinal bleeding or pulmonary hemorrhage within 4 weeks before screening, known symptomatic human immunodeficiency virus (HIV) positive with an acquired immunodeficiency syndrome-defining opportunistic infection within the past 12 months prior to screening, or a current CD4 count <350 cells/µL, symptomatic active hepatitis B or C checked at screening, or active tuberculosis therapy.
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  • Subjects with HIV are eligible if:
  • They have received antiretroviral therapy (ART) as clinically indicated for at least 12 months prior to starting LD therapy and have an HIV viral load less than 40 copies/mL prior to start of LD therapy.
  • They continue on ART as clinically indicated while enrolled on study.
  • CD4 counts> 350 cells/µL and CD4 counts and viral load are monitored per standard of care by a local health care provider.
  • They are fully vaccinated against SARS-CoV-2.
  • Has received prior radiotherapy within 2 weeks of the start of BOXR1030. Subjects must have recovered from all radiation-related toxicities, not require corticosteroids, and not have had severe radiation pneumonitis.
  • Potentially life-threatening second malignancy requiring systemic treatment within the last 3 years (i.e., subjects with a history of prior malignancy are eligible if treatment was completed at least 3 years before entering the Treatment Period and the subject has no evidence of disease) or which would impede evaluation of treatment response.
  • Clinically significant (i.e., active) cardiovascular disease: cerebral vascular accident/stroke (<6 months prior to enrollment), myocardial infarction (<6 months prior to enrollment), unstable angina, congestive heart failure (New York Heart Association Classification Class II), or the presence of any condition that can increase proarrhythmic risk (e.g., hypokalemia, bradycardia, heart block) including any new, unstable, or serious cardiac arrhythmia requiring medication, or other baseline arrhythmia that might interfere with interpretation of electrocardiograms on study (e.g., bundle branch block).
  • Has an active infection excluding controlled HIV.
  • Has an active autoimmune disease requiring systemic (immunosuppressive) therapy.
  • NOTE: Additional inclusion/exclusion criteria may apply. The above information is not intended to be an exhaustive list of considerations for potential participation in a clinical trial.

Trial locations and contacts

8 sites in 2 countries.

8 sites in 2 countries

United States5 sites

  • Baylor Scott and White Research Institute

    Dallas, Texas, 75246

  • University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030-4008

  • Froedtert and Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • Hoag Hospital Newport Beach

    Newport Beach, California, 92663

  • Fred Hutchinson Cancer Center - Seattle Cancer Care Alliance (SCCA) Location

    Seattle, Washington, 98109

Other trials in United States

United Kingdom3 sites

  • Beatson Institute for Cancer Research Wolfson Wohl Cancer Research Centre

    Glasgow, G61 1BD

  • University College London Hospitals NHS Foundation Trust

    London, NW1 2PG

  • The Royal Marsden NHS Foundation Trust

    London, SW3 6JJ

Other trials in United Kingdom

About this study

Official title: A First-in-Human, Phase 1/2, Dose Escalation Study of BOXR1030 T Cells in Subjects With Advanced GPC3-Positive Solid Tumors

This is a first-in-human (FIH), Phase 1/2, open-label, multicenter study to assess safety and determine the recommended Phase 2 dose (RP2D) of BOXR1030 administration after lymphodepleting chemotherapy (LD chemotherapy) in subjects with glypican-3 positive (GPC3+) advanced solid tumors.

Read the detailed description

This is a FIH, Phase 1/2, open-label, multicenter study to assess safety and determine the maximum tolerated dose (MTD) and/or RP2D of BOXR1030 administration after LD chemotherapy in subjects with GPC3+ advanced solid tumors. After signing informed consent and completing all screening assessments, eligible subjects will be enrolled and undergo leukapheresis to obtain T cells for BOXR1030 manufacturing. Subjects will receive a 3-day LD chemotherapy regimen with fludarabine and cyclophosphamide, administered according to institutional standard practice for these drugs, including inpatient administration as appropriate. Subjects must be hospitalized for BOXR1030 administration and will remain hospitalized for 10 days after the infusion. For 28 days after BOXR1030 administration, all subjects must stay within a distance that requires no more than 2 hours of travel to the study site.

During the Post-treatment Evaluation Period (within 6 months after BOXR1030 administration), study visits will occur daily for the first week, twice in the second week, and then once weekly at Weeks 3, 4, 6, 9, 12, 15, 18, and 24. Safety (targeted physical examination, adverse event [AE] assessment, and clinical laboratory tests) will be evaluated and samples will be collected for endpoint analyses. For 28 days after BOXR1030 administration, subjects will be required to monitor their temperature and complete neurological evaluation via the immune effector cell-associated encephalopathy assessment tool every day (to be administered by site staff during clinical visits and by a caregiver at home on non-clinic days). At regular intervals, antitumor activity will be assessed per RECIST 1.1 and iRECIST criteria.

After 6 months of follow-up from BOXR1030 administration, subjects will enter the Long-term Follow-up Period for a total duration of 15 years after BOXR1030 dosing. Study visits are scheduled at Months 7, 9, 11, 13, 15, 18, 21, and 24, every 6 months thereafter until Year 5, and then annually through Year 15. Long-term follow-up assessments will focus on long-term safety and disease status. Subjects whose disease does not progress before Week 24 will enter the Long-term Follow-up at Month 7. Survival status will be checked at these visits.

Being tested
CAR-GPC3 T Cells
Keywords
HCC, SCC, MCC, MRCLS
Medical subject headings
Carcinoma, Hepatocellular, Carcinoma, Merkel Cell, Liposarcoma, Myxoid

What participants receive

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

  • CAR-GPC3 T CellsBiological

    Also called BOXR1030

    Five dose levels each with LD chemotherapy

Study groups in NCT05120271
Study groupRoleWhat they receive
GPC3+ solid tumorsExperimentalOne time intravenous administration of BOXR1030 after completion of cyclophosphamide and fludarabine LD chemotherapy
Receives: Biological: CAR-GPC3 T Cells

What is being measured

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

  1. Dose limiting toxicity

    Defined using National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0 or American Society for Transplantation and Cellular Therapy (ASTCT) criteria for cytokine release syndrome or immune effector cell-associated neurotoxicity syndrome.

    Measured: From the time of BOXR1030 administration (Study Day 1) through 28 days after BOXR1030 administration (Study Day 28/Week 4)

  2. MTD

    Defined as the dose that maximizes the probability of targeted toxicity among doses that satisfy the escalation with overdose control criterion.

    Measured: From the time of BOXR1030 administration (Study Day 1) through 28 days after BOXR1030 administration (Study Day 28/Week 4)

  3. RP2D

    The RP2D may be the same as the MTD, a previously tested dose, or an intermediate/alternative dose below the MTD that is yet unexplored. Alternatively, the RP2D may be selected on the basis of observed safety and activity in dose escalation before the MTD is reached.

    Measured: From the time of BOXR1030 administration (Study Day 1) through 28 days after BOXR1030 administration (Study Day 28/Week 4)

  4. Treatment-emergent AEs (TEAEs)

    Type, frequency, and severity of TEAEs; clinically significant abnormal safety laboratory findings; and vital signs. TEAEs and laboratory findings according to NCI CTCAE version 5.0 and ASTCT criteria.

    Measured: From the time of BOXR1030 administration (Study Day 1) through Week 24

Secondary outcomes (10)

  1. Overall response rate

    Defined according to RECIST 1.1 criteria.

    Measured: From the time of BOXR1030 administration (Study Day 1) until disease progression/recurrence or start of new anti-cancer therapy, whichever came first, assessed up to approximately 15 years

  2. Best overall response

    Defined as the best response recorded from the date of treatment to disease progression or death.

    Measured: From the time of BOXR1030 administration (Study Day 1) until disease progression or death, whichever came first, assessed up to approximately 15 years

  3. Duration of response

    Defined as the time from the first achieved response (complete or partial) to the first date of radiological progression.

    Measured: From the date of response for patients with response (complete or partial) until disease progression or death, whichever came first, assessed up to approximately 15 years

  4. Progression-free survival

    Defined as the time from the date of treatment to the first date of radiological progression or death.

    Measured: From the time of BOXR1030 administration (Study Day 1) until disease progression or death, whichever came first, assessed up to approximately 15 years

  5. Clinical benefit rate

    Defined as the total number (or percentage) of patients who achieved a complete response, partial response, or had stable disease for 6 months or longer.

    Measured: From the time of BOXR1030 administration (Study Day 1) until disease progression or death, whichever came first, assessed up to approximately 15 years

  6. Time to response

    Defined as the time from the date of treatment to the first objective tumor response.

    Measured: From the time of BOXR1030 administration (Study Day 1) until response, assessed up to approximately 15 years

  7. Time to progression

    Defined as the time from the date of treatment to disease progression.

    Measured: From the time of BOXR1030 administration (Study Day 1) until disease progression, assessed up to approximately 15 years

  8. BOXR1030 T-cell levels in blood

    BOXR1030 T-cell levels in blood will be analyzed.

    Measured: From the time of BOXR1030 administration (Study Day 1) until the end of the study, assessed up to approximately 15 years

  9. BOXR1030 T-cell characterization in blood

    DNA samples will be analyzed to detect BOXR1030 transgene levels (GPC3 chimeric antigen receptor [CAR]). Peripheral blood mononuclear cells will be used to evaluate immune cell surface markers along with GPC3 CAR for characterization of BOXR1030 T cells in blood.

    Measured: From the time of BOXR1030 administration (Study Day 1) until the end of the study, assessed up to approximately 15 years

  10. Levels of inflammatory markers

    Inflammatory markers will include, e.g., ferritin, C-reactive protein, erythrocyte sedimentation rate, and triglycerides.

    Measured: From the time of BOXR1030 administration (Study Day 1) until the end of the study, assessed up to approximately 15 years

Timeline

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

  1. First posted15 Nov 2021
  2. Study start26 Oct 2022
  3. Primary completion24 Oct 2025
  4. Study completionDec 2042 (estimated)

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
7 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05120271
Sponsor's protocol ID
B1030-101
Acronym
DUET-1
Status last verified
Dec 2025
First submitted
2 Nov 2021
Last updated
5 Dec 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Hickman TL, Choi E, Whiteman KR, Muralidharan S, Pai T, Johnson T, Parikh A, Friedman T, Gilbert M, Shen B, Barron L, McGinness KE, Ettenberg SA, Motz GT, Weiss GJ, Jensen-Smith A. BOXR1030, an anti-GPC3 CAR with exogenous GOT2 expression, shows enhanced T cell metabolism and improved anti-cell line derived tumor xenograft activity. PLoS One. 2022 May 4;17(5):e0266980. doi: 10.1371/journal.pone.0266980. eCollection 2022.

    PubMed 35507536

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing CAR-GPC3 T Cells in people with Hepatocellular Carcinoma and Squamous Cell Carcinoma of the Lung, run by Sotio Biotech Inc. at 8 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 25 inclusion and 17 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: under way, not looking for participants, as of the sponsor's last update on 5 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in United States, United Kingdom. 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?

This study is still under way. Results are not available in the source record yet.

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?

Dose limiting toxicity (time frame: From the time of BOXR1030 administration (Study Day 1) through 28 days after BOXR1030 administration (Study Day 28/Week 4)). 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 NCT05120271, 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 5 Dec 2025.

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