HepatoblastomaPhase 1/2ARYA-2NCT04634357
ET140203 T Cells in Pediatric Subjects With Hepatoblastoma, HCN-NOS, or Hepatocellular Carcinoma
A Phase 1/2 study testing ET140203 T Cells in people with Hepatoblastoma and Hepatocellular Carcinoma (HCC), run by Eureka Therapeutics Inc. at 4 sites in United States.
Trial summary
Open-label, dose escalation, multi-center, Phase I/II clinical trial to assess the safety/tolerability and determine the recommended Phase II Dose (RP2D) of ET140203 T-cells in pediatric subjects who are AFP-positive/HLA-A2-positive and have relapsed/refractory HB, HCN-NOS, or HCC. Read more about this study
- Who is it for?
- Ages 1 year to 21 years
- Conditions the study is for
- Hepatoblastoma, Hepatocellular Carcinoma (HCC), Liver Neoplasms
- What is being tested?
- ET140203 T Cells
- Type of treatment
- Vaccines
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 4 sitesUnited States
- Study dates
- 19 Jul 2022to 31 Jan 2028
- Phase
- Phase 1/2
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 1 year to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Histologically confirmed HB, HCN-NOS, or HCC with serum AFP >100ng/mL at the time of screening and following the most recent line of therapy.
- Disease recurrence after remission following initial standard-of care (SOC) treatment (i.e., relapse) or failure of response to SOC treatment (i.e., refractory).
- Age ≥ 1 year and ≤ 21 years.
- Molecular Human Leukocyte Antigen (HLA) class I allele typing that confirms subject carries at least one HLA-A2 allele.
- Life expectancy of > 4 months per the Investigator's opinion.
- Lansky or Karnofsky Performance Scale ≥ 70.
Show all 9Show fewer
- For enrollment to the dose-finding cohort, subjects must have at least one (1) lesion ≥ 5 mm in diameter or two (2) or more lesions ≥ 3 mm in diameter. For the dose-expansion cohort, subjects must have measurable disease by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1.
- Child-Pugh score of A6 or better.
- Adequate organ function.
Who cannot join
- Recurrent HB who are candidates for complete surgical resection (e.g., isolated pulmonary relapse amendable to pulmonary metastasectomy).
- Pre-existing illness including heart failure, uncontrolled pulmonary disease not cancer-related, or psychiatric illness/social situation that would limit compliance with study requirements.
- Active, uncontrolled systemic bacterial, fungal, or viral infection. Subjects with Human Immunodeficiency Virus (HIV), hepatitis B, or hepatitis C are eligible provided their infection is being treated and the viral load is controlled.
- Any known active malignancy (other than HB, HCN-NOS, or HCC).
- Pregnant or lactating women.
- Received the following within one (1) week of leukapheresis or within two (2) weeks of conditioning chemotherapy: cytotoxic chemotherapy, radiation, other anti-cancer therapies (including immunotherapeutic agents), or immunosuppressive therapy. Systemic corticosteroids at doses greater than 5 mg/day of prednisone or equivalent doses of other corticosteroids within two (2) weeks prior to leukapheresis or conditioning chemotherapy or receipt of a T-cell engager (TCE) within two (2) months prior to leukapheresis or at any time as bridging therapy between leukapheresis and conditioning chemotherapy is exclusionary. (Note: Topical and inhaled corticosteroids in standard doses and physiological replacement doses of corticosteroids for adrenal insufficiency are allowed).
Show all 12Show fewer
- Concurrently receiving other investigational agents, biological, chemical, or radiation therapies, while participating in the study.
- Contraindication for receipt of conditioning chemotherapeutic agents including Fludarabine and Cyclophosphamide.
- Active autoimmune disease requiring systemic immunosuppressive therapy.
- Compromised circulation in the main portal vein, hepatic vein, or vena cava due to partial or complete obstruction which, in the opinion of the Investigator, would make the subject unsuitable for the study.
- History of organ transplant.
- HB, HCN-NOS, or HCC involving greater than 50% of the liver (volumetric).
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
Dana-Farber/Boston Children's Cancer and Blood Disorders Center
Boston, Massachusetts, 02215
Jill MacDonald, 617-632-4930, Jill_Macdonald@DFCI.Harvard.edu
Allison O'Neill, MD
- Recruiting
Children's Hospital Los Angeles
Los Angeles, California, 90027
Silpa Sharma, MPH, CCRC, 323-361-6475, sisharma@chla.usc.edu
Rachana Shah, MD, MS
- Recruiting
Children's Hospital Orange County
Orange, California, 92868
Dorian Chan, 714-509-7868, dchan@choc.org
Rishikesh Chavan, MD
- Recruiting
UCSF Benioff Children's Hospitals
San Francisco, California, 94158
Brian Luong, CCRP, Brian.Luong@ucsf.edu
Arun Rangaswami, MD
About this study
Official title: An Open-Label, Dose Escalation, Phase I/II Clinical Trial of ET140203 T Cells in Pediatric Subjects With Relapsed/Refractory Hepatoblastoma (HB), Hepatocellular Neoplasm-Not Otherwise Specified (HCN-NOS), or Hepatocellular Carcinoma (HCC)
Open-label, dose escalation, multi-center, Phase I/II clinical trial to assess the safety/tolerability and determine the recommended Phase II Dose (RP2D) of ET140203 T-cells in pediatric subjects who are AFP-positive/HLA-A2-positive and have relapsed/refractory HB, HCN-NOS, or HCC.
Read the detailed description
This is an open-label, dose escalation, multi-center, Phase I/II clinical trial to assess the safety of intravenous (IV) administration of ET140203 T cells and determine the recommended Phase II dose (RP2D) of ET140203 T-cell therapy in pediatric subjects (age ≥ 1 year and ≤ 21 years) who are AFP-positive/HLA-A2-positive and have relapsed/refractory HB, HCN-NOS, or HCC.
A traditional dose escalation model (3+3 design) will be used to determine the RP2D, and once determined, the expansion phase will commence. A statistically relevant number of subjects will be treated at the RP2D in the expansion phase to adequately assess the therapeutic benefits and risks of ET140203 T-cell therapy. Tumor response assessments will be performed prior to 1st infusion (baseline) and at Months 1, 3, 6, 9, 12, 18, and 24. At each tumor response assessment visit, radiographic imaging will be performed and used for response evaluation. Serum AFP levels will also be measured at each tumor response assessment visit.
The active assessment period of the study will continue for 2 years. Subjects will be followed for assessment of treatment safety and overall survival during long-term follow-up (LTFU; Year 2-15).
- Conditions
- Hepatoblastoma, Hepatocellular Carcinoma (HCC), Liver Neoplasms, Metastatic Liver Cancer, Liver Cancer, HEMNOS
- Being tested
- ET140203 T Cells
- Keywords
- Relapsed/Refractory Hepatoblastoma (HB), Pediatric, Hepatocellular Neoplasm-Not Otherwise Specified (HCN-NOS), Hepatocellular Carcinoma (HCC), Liver Cancer, T-cell therapy, Metastatic Liver Cancer, Liver neoplasms, HEMNOS
- Medical subject headings
- Hepatoblastoma, Carcinoma, Hepatocellular, Liver Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ET140203 T CellsDrug
Biological/Vaccine: ET140203 autologous T-cell product
Autologous T cells transduced with lentivirus encoding an ET140203 expression construct
| Study group | Role | What they receive |
|---|---|---|
| ET140203 T Cells | Experimental | ET140203 Autologous T Cells Receives: Drug: ET140203 T Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence rates of adverse events (AEs) after infusion of ET140203 T cells
Safety of ET140203 T cells as assessed by the number of adverse events (AEs) after infusion
Measured: 28 days
Severity rates of adverse events (AEs) after infusion of ET140203 T cells
Safety of ET140203 T cells as assessed by the severity of adverse events (AEs) after infusion.
Measured: 28 days
Incidence rates of dose limiting toxicities (DLTs) after infusion of ET140203 T cells
Tolerability of ET140203 T cells after infusions assessed by committee review of dose limiting toxicities (DLTs)
Measured: 28 days
The recommended phase 2 dose (RP2D) regimen of ET140203 T cell therapy primarily based on DLT
The RP2D will be determined by the study Dose Escalation Committee (DEC) and primarily based on DLTs.
Measured: Up to 2 years
Secondary outcomes (2)
Assess the efficacy of ET140203 T cells in pediatric subjects with relapsed/refractory HB, HCN-NOS, or HCC
Response rate will be assessed by radiographic scans and assessed according to RECIST criteria.
Measured: Up to 2 years
Determine the pharmacokinetics of ET140203 T cells after infusion.
Assess the expansion and persistence of ET140203 T cells circulating in blood over time.
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.
- First posted18 Nov 2020
- Study start19 Jul 2022
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Jan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HepatoblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eureka Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pei Wang, PhD, Study director, Eureka Therapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04634357
- Sponsor's protocol ID
- ETUS20AFPAR123
- Acronym
- ARYA-2
- Status last verified
- Jul 2026
- First submitted
- 4 Nov 2020
- Last updated
- 6 Jul 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No