Hepatocellular CarcinomaPhase 1/2NCT02587689
Phase I/II Study of Anti-Mucin1 (MUC1) CAR T Cells for Patients With MUC1+ Advanced Refractory Solid Tumor
A Phase 1/2 study testing anti-MUC1 CAR T Cells in people with Hepatocellular Carcinoma and Non-small Cell Lung Cancer, run by PersonGen BioTherapeutics (Suzhou) Co., Ltd. at 1 site in China.
Trial summary
The purpose of this study is to determine whether autologous T cells bearing chimeric antigen receptor that can specifically recognize (Mucin 1) MUC1 is safe and effective for patients with relapsed or refractory solid tumor. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Hepatocellular Carcinoma, Non-small Cell Lung Cancer, Pancreatic Carcinoma
- What is being tested?
- anti-MUC1 CAR T Cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- Oct 2015to Oct 2018
- 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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female subjects with MUC1+ malignancies in patients with no available curative treatment options who have limited prognosis (several months to < 2 year survival) with currently available therapies will be enrolled:
- Eligible diseases: MUC1+ hepatocellular carcinoma, non-small cell lung cancer, pancreatic carcinoma and triple-negative basal-like breast carcinoma.
- Hepatocellular carcinoma (HCC)
- Clinical diagnosis of HCC was confirmed by histopathological examination of surgical samples in all patients;
- Non-small cell lung cancer
- Refractory or recurrent histologically or cytologically confirmed; unresectable; non-squamous NSCLC must have been tested for epidermal growth factor receptor (EGFR) mutation and anaplastic lymphoma kinase (ALK) translocation and if positive should have received appropriate tyrosine kinase inhibitor therapy prior to enrollment;
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- Pancreatic carcinoma
- Patients with histologic verification of carcinoma of the pancreas (T1-3, N0-1) who have undergone surgical resection within the past 4 - 12 weeks. Patients with R1 resections are excluded;
- Triple-negative basal-like breast carcinoma
- Patients with basal-like breast carcinoma must have confirmed triple negative (estrogen receptor negative [ER-]/ progesterone receptor (PR) negative [PR-]/ human epidermal growth factor receptor-2 (HER2) negative [HER2-]) .
- MUC1 is expressed in malignancy tissues by immuno-histochemical (IHC).
- Eastern cooperative oncology group (ECOG) performance status of 0-1 or karnofsky performance status (KPS) score is higher than 60.
- Patients 18 years of age or older, and must have a life expectancy > 12 weeks.
- Adequate venous access for apheresis or venous sampling, and no other contraindications for leukapheresis.
- Females of child-bearing potential must have a negative pregnancy test and all subjects must agree to use an effective method of contraception for up to two weeks after the last infusion of CAR T cells.
- Adequate bone marrow, liver and renal function as assessed by the following laboratory requirements: White blood cell count (WBC) ≥ 2500c/ml, Platelets ≥ 50×10^9/L, Hb ≥ 9.0g/dL, lymphocyte (LY) ≥ 0.7×10^9/L, LY% ≥ 15%, Alb ≥ 2.8g/dL, serum lipase and amylase < 1.5×upper limit of normal, serum creatinine ≤ 2.5mg/dL, aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 5×upper limit of normal, serum total bilirubin ≤ 2.0mg/dL. These tests must be conducted within 7 days prior to registration.
- Ability to give informed consent.
Who cannot join
- The transduction efficiency of the T cells is less than 10% or the amplification of the T cells via artificial antigen presenting cell (aAPC) stimulation is less than 5 times.
- Patients with symptomatic central nervous system (CNS) involvement.
- Pregnant or nursing women may not participate.
- Known HIV, hepatitis B virus (HBV) or hepatitis C virus (HCV) infection.
- Serious illness or medical condition which would not permit the patient to be managed according to the protocol, including active uncontrolled infection, major cardiovascular, coagulation disorders, respiratory or immune system, myocardial infarction, cardiac arrhythmias, obstructive/restrictive pulmonary disease, or psychiatric or emotional disorders.
- History of severe immediate hypersensitivity to any of the agents including cyclophosphamide, fludarabine, or aldesleukin.
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- Concurrent use of systemic steroids. Recent or current use of inhaled steroids is not exclusionary.
- Previously treatment with any gene therapy products.
- The existence of unstable or active ulcers or gastrointestinal bleeding.
- Patients with portal vein vascular invasion or extrahepatic, are excluded from this study.
- Patients with a history of organ transplantation or are waiting for organ transplantation.
- Patients need anticoagulant therapy (such as warfarin or heparin).
- Patients need long-term antiplatelet therapy (aspirin at a dose > 300mg/d; clopidogrel at a dose > 75mg/d).
- Patients treated by radiotherapy within 4 weeks prior the first apheresis.
- Patients using fludarabine or cladribine chemotherapy within two years.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
PersonGen Biomedicine (Suzhou) Co., Ltd.
Suzhou, Jiangsu, 215123
Lin Yang, Ph.D., 86-512-65922190, lin.yang@persongen.com
Yangyi Bao, MD
About this study
Official title: Phase I/II Study of Anti-MUC1 CAR T Cells for Patients With MUC1+ Advanced Refractory Solid Tumor
The purpose of this study is to determine whether autologous T cells bearing chimeric antigen receptor that can specifically recognize (Mucin 1) MUC1 is safe and effective for patients with relapsed or refractory solid tumor.
- Conditions
- Hepatocellular Carcinoma, Non-small Cell Lung Cancer, Pancreatic Carcinoma, Triple-Negative Invasive Breast Carcinoma
- Being tested
- anti-MUC1 CAR T Cells
- Medical subject headings
- Carcinoma, Hepatocellular, Carcinoma, Non-Small-Cell Lung, Pancreatic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-MUC1 CAR T CellsBiological
Also called anti-MUC1-CAR transduced autologous T cells
| Study group | Role | What they receive |
|---|---|---|
| anti-MUC1 CAR T Cells | Experimental | The subject's T cells will be modified in one or two different ways that will allow the cells to identify and kill the MUC1+ tumor cells. Receives: Biological: anti-MUC1 CAR T Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase I: Adverse events attributed to the administration of the anti-MUC1 CAR T cells
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2015 (estimated)
- Study startOct 2015
- Primary completionOct 2017 (estimated)
- Study completionOct 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hepatocellular CarcinomaStudies of the same condition, open and under way.
- More trials run by PersonGen BioTherapeutics (Suzhou) Co., Ltd.The sponsor's other registered studies.
- 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
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- PersonGen BioTherapeutics (Suzhou) Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The First People's Hospital of Hefei, Hefei Binhu Hospital
- Responsible party
- Sponsor
- Investigators
- Lin Yang, Ph.D., Principal investigator, PersonGen BioTherapeutics (Suzhou) Co., Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02587689
- Sponsor's protocol ID
- PG-021-001
- Status last verified
- Dec 2016
- First submitted
- 25 Oct 2015
- Last updated
- 5 Dec 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 5 Dec 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Study of Anti-MUC1 CAR T Cells for Patients With MUC1+ Advanced Refractory Solid Tumor
- ClinicalTrials.gov ID
- NCT02587689
- Acronym / sponsor ID
- PG-021-001
- Conditions
- Hepatocellular Carcinoma, Non-small Cell Lung Cancer, Pancreatic Carcinoma, Triple-Negative Invasive Breast Carcinoma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- PersonGen BioTherapeutics (Suzhou) Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- The First People's Hospital of Hefei, Hefei Binhu Hospital
- Study start
- Oct 2015
- Primary completion
- Oct 2017 (estimated)
- Study completion
- Oct 2018 (estimated)
- First posted
- 27 Oct 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine whether autologous T cells bearing chimeric antigen receptor that can specifically recognize (Mucin 1) MUC1 is safe and effective for patients with relapsed or refractory solid tumor.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| anti-MUC1 CAR T Cells | Experimental | Biological: anti-MUC1 CAR T CellsThe subject's T cells will be modified in one or two different ways that will allow the cells to identify and kill the MUC1+ tumor cells. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Male and female subjects with MUC1+ malignancies in patients with no available curative treatment options who have limited prognosis (several months to < 2 year survival) with currently available therapies will be enrolled:
- Eligible diseases: MUC1+ hepatocellular carcinoma, non-small cell lung cancer, pancreatic carcinoma and triple-negative basal-like breast carcinoma.
- Clinical diagnosis of HCC was confirmed by histopathological examination of surgical samples in all patients;
- Refractory or recurrent histologically or cytologically confirmed; unresectable; non-squamous NSCLC must have been tested for epidermal growth factor receptor (EGFR) mutation and anaplastic lymphoma kinase (ALK) translocation and if positive should have received appropriate tyrosine kinase inhibitor therapy prior to enrollment;
- Patients with histologic verification of carcinoma of the pancreas (T1-3, N0-1) who have undergone surgical resection within the past 4 - 12 weeks. Patients with R1 resections are excluded;
Key exclusion criteria (15 listed)
- The transduction efficiency of the T cells is less than 10% or the amplification of the T cells via artificial antigen presenting cell (aAPC) stimulation is less than 5 times.
- Patients with symptomatic central nervous system (CNS) involvement.
- Pregnant or nursing women may not participate.
- Known HIV, hepatitis B virus (HBV) or hepatitis C virus (HCV) infection.
- Serious illness or medical condition which would not permit the patient to be managed according to the protocol, including active uncontrolled infection, major cardiovascular, coagulation disorders, respiratory or immune system, myocardial infarction, cardiac arrhythmias, obstructive/restrictive pulmonary disease, or psychiatric or emotional disorders.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| anti-MUC1 CAR T CellsAlso: anti-MUC1-CAR transduced autologous T cells | Biological | anti-MUC1 CAR T Cells |
Primary outcome
Phase I: Adverse events attributed to the administration of the anti-MUC1 CAR T cells
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Dec 2016.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lin Yang, Ph.D. principal investigator
PersonGen BioTherapeutics (Suzhou) Co., Ltd.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing anti-MUC1 CAR T Cells in people with Hepatocellular Carcinoma and Non-small Cell Lung Cancer, run by PersonGen BioTherapeutics (Suzhou) Co., Ltd. at 1 site in China.
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 17 inclusion and 15 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: status not confirmed, as of the sponsor's last update on 5 Dec 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Lin Yang, Ph.D. (86-512-65922190, lin.yang@persongen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase I: Adverse events attributed to the administration of the anti-MUC1 CAR T cells (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 2016.