Anaplastic Thyroid CancerPhase 1NCT04420754
Study of AIC100 CAR T Cells in Relapsed/Refractory Thyroid Cancer
A Phase 1 study testing AIC100 CAR T Cells in people with Anaplastic Thyroid Cancer and Relapsed/Refractory Poorly Differentiated Thyroid Cancer, run by AffyImmune Therapeutics, Inc. at 4 sites in United States.
Trial summary
The purpose of this study is to assess the safety and tolerability and determine the recommended Phase 2 dose of AIC100 Chimeric Antigen Receptor (CAR) T cells in patients with relapsed/refractory poorly differentiated thyroid cancer and anaplastic thyroid cancer, including newly diagnosed. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Anaplastic Thyroid Cancer, Relapsed/Refractory Poorly Differentiated Thyroid Cancer
- What is being tested?
- AIC100 CAR T Cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 4 sitesUnited States
- Study dates
- 28 Sep 2020to 4 Aug 2030
- Phase
- Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Willing and able to participate in the study and provide written informed consent
- Be ≥ 18 years of age on the day of signing the Informed Consent Form
- Patients must have thyroid cancer that meets one of the following diagnoses, and, prior to lymphodepleting chemotherapy (LDC), have an identified available fresh or archival biopsy sample:
- Anaplastic Thyroid Cancer BRAF wild-type at any stage, including newly diagnosed
- Anaplastic Thyroid Cancer BRAF mutant after failure of or inability to tolerate BRAF- specific therapy
- Poorly Differentiated Thyroid Cancer that has failed any of the following treatments: surgery radioactive iodine, chemotherapy, radiation therapy, and/or targeted therapies
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- Measurable disease by Computed Tomography (CT) or Positron Emission Tomography (PET) PET/CT per RECIST v1.1
- For ATC patients who do not have measurable disease at Screening, they are required to have measurable disease at Baseline Day -7 to proceed in the study.
- Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
- Life expectancy greater than 8 weeks
- Overall adequate hepatic, renal, bone marrow, cardiac, and coagulation function, defined as the following:
- Estimated creatinine clearance ≥ 50 mL/minute
- Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST): normal or Grade 1. Note: Lymphodepleting Chemotherapy (LDC) agents can cause fluctuations in hepatic enzymes.
- Serum total bilirubin: normal or Grade 1. Note: LDC agents can cause fluctuations in hepatic enzymes.
- Serum albumin: normal or Grade 1. (human albumin supplementation is not allowed within 2 weeks prior to Screening assessment)
- Hemodynamically stable and left ventricular ejection fraction ≥ 45%
- Hematological parameters
- Absolute neutrophil count > 1000/μL without myeloid growth factor support for ≥ 2 weeks
- Absolute lymphocyte count ≥ 100/μL at screening and at apheresis
- Platelet count ≥ 50 × 1000/μL without platelet transfusion for ≥ 2 weeks
- Hemoglobin concentration > 7 g/dL without red blood cell transfusion for ≥ 2 weeks
- Has met the minimum washout time for previous cancer treatments before undergoing apheresis or LDC, and in the Investigator's judgement, the patient is able to safely undergo the procedure
- (incorporated into inclusion criteria #7)
- Females of reproductive potential (defined as all females physiologically capable of becoming pregnant) must agree to use 1 highly effective method of contraception and 1 additional effective method from at least 30 days before enrollment/apheresis and for at least 1 year after the infusion of AIC100 CAR T Cells.
- Females of reproductive potential must have a negative serum beta-human chorionic gonadotropin pregnancy test result at Screening
Who cannot join
- Women who are pregnant or breastfeeding
- Clinically significant, active, uncontrolled, systemic infection; the following are not exclusionary:
- Patients with Human immunodeficiency virus (HIV) must have been on effective antiretroviral therapy for ≥ 4 weeks prior to enrollment; must have an HIV viral load < 400 copies/µL; no acquired immunodeficiency syndrome related opportunistic infections in the previous 12 months; and a CD4+ cell count ≥ 350 cells/µL
- Patients with chronic hepatitis B virus (HBV) infection must be on antiviral therapy and have an HBV viral load below the limits of detection
- Patients with chronic hepatitis C virus (HCV) infection must have completed therapy and have an HCV viral load below the limits of detection
- Prior treatment with investigational gene therapy or CAR T cell therapy
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- Presence of active and clinically relevant central nervous system disorder such as epilepsy, stroke, or symptomatic or uncontrolled brain metastases
- Evidence of another malignancy within 2 years prior to Screening (except in-situ non melanoma skin cancers, localized controlled prostate cancer, adequately treated Stage 1 uterine cancer that has a low risk of recurrence, or any other malignancies with similar outcome)
- (incorporated into exclusion criteria #2)
- Active autoimmune disease (including but not limited to systemic lupus erythematosus, Sjögren's Syndrome, rheumatoid arthritis (RA), psoriasis, multiple sclerosis, inflammatory bowel disease) requiring immunosuppressive therapy within 4 weeks prior to eligibility confirmation
- Patients with severe chronic diseases of the kidney, liver, heart, lung; or any other serious illness that, in the opinion of the Investigator, may affect the patient's treatment, follow up, or assessments, including but not limited to uncontrolled clinically significant neurological or psychiatric disorders or metabolic diseases
- Patients who need long-term use of systemic corticosteroids > 10 mg/day prednisone or equivalent
- Allergy to the chemotherapy drugs given during lymphodepletion or known hypersensitivity to any component of AIC100 CAR T Cells, including excipients
- Receipt of a COVID-19 vaccine within 4 weeks before Screening
- Concurrent participation in another interventional clinical study during participation in this study
- Prior treatment with any gene therapy or genetically modified cell therapy, including CAR T cells
- Prior treatment with ICAM-1 directed antibody, bispecific T cell engager, or antibody drug conjugate, unless there is confirmed ICAM-1 expression (by immunohistochemistry) after progression or relapse following most recent ICAM-1 directed treatment.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Northwestern Memorial Hospital
Chicago, Illinois, 60611
City of Hope National Medical Center, City of Hope Medical Center
Duarte, California, 91010
MD Anderson Cancer Center
Houston, Texas, 77030
Weill Cornell Medical College
New York, New York, 10065
About this study
Official title: A Multicenter Phase I Study of AIC100 CAR T Cells in Relapsed and/or Refractory Advanced Thyroid Cancer or Anaplastic Thyroid Cancer
The purpose of this study is to assess the safety and tolerability and determine the recommended Phase 2 dose of AIC100 Chimeric Antigen Receptor (CAR) T cells in patients with relapsed/refractory poorly differentiated thyroid cancer and anaplastic thyroid cancer, including newly diagnosed.
Read the detailed description
The primary objective of this study is to assess the safety and tolerability of AIC100 CAR T Cells and determine the recommended Phase 2 dose of AIC100 in patients with relapsed/refractory poorly differentiated thyroid cancer and in patients with anaplastic thyroid cancer that are BRAF wild-type, including newly diagnosed, or BRAF mutant anaplastic thyroid cancer after failure of BRAF mutant specific therapy.
Upon enrollment, patients will undergo apheresis for collection of autologous lymphocytes. The autologous T cells will be transfected and expanded in vitro to generate the AIC100 CAR T Cell product. After lymphodepleting chemotherapy, AIC100 CAR T Cells will be infused.
The study drug product, AIC100, consists of autologous CAR T cells targeting intercellular adhesion molecule-1 (ICAM-1) on thyroid cancer. In addition, AIC100 cells express the somatostatin receptor subtype 2 (SSTR2), which should enable imaging of AIC100 CAR T Cells.
- Being tested
- AIC100 CAR T Cells
- Keywords
- AIC100, CAR T Cell, Anaplastic Thyroid Cancer, Poorly Differentiated Thyroid Cancer
- Medical subject headings
- Thyroid Carcinoma, Anaplastic, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AIC100 CAR T CellsBiological
Also called AIC100
Autologous CAR T cells directed against ICAM-1
| Study group | Role | What they receive |
|---|---|---|
| Cohort -1 | Experimental | AIC100 CAR T Cell Dose Level -1 (Flat Dose): 1 x 10e6 CAR T cells Receives: Biological: AIC100 CAR T Cells |
| Cohort 1 | Experimental | AIC100 CAR T Cell Dose Level 1 (Flat Dose): 1 x 10e7 CAR T cells Receives: Biological: AIC100 CAR T Cells |
| Cohort 2 | Experimental | AIC100 CAR T Cell Dose Level 2 (Flat Dose): 1 x 10e8 CAR T cells Receives: Biological: AIC100 CAR T Cells |
| Cohort 3 | Experimental | AIC100 CAR T Cell Dose Level 3 (Flat Dose): 5 x 10e8 CAR T cells Receives: Biological: AIC100 CAR T Cells |
| Cohort 2.5 | Experimental | AIC100 CAR T Cell Dose Level 2.5 (Flat Dose): 2.5 x 10e8 CAR T cells. The interim step-down dose of Cohort 2.5 may be evaluated, if needed, based on ongoing safety and efficacy data. Receives: Biological: AIC100 CAR T Cells |
| Cohort 4 | Experimental | AIC100 CAR T Cell Dose Level 4 (Flat Dose): 7.5 x 10e8 CAR T cells. The proposed escalation dose of Cohort 4 may be evaluated, if needed, based on ongoing safety and efficacy data. Receives: Biological: AIC100 CAR T Cells |
| Cohort 5 | Experimental | AIC100 CAR T Cell Dose Level 5 (Flat Dose): 1 x 10e9 CAR T cells. The proposed escalation dose of Cohort 5 may be evaluated, if needed, based on ongoing safety and efficacy data. Receives: Biological: AIC100 CAR T Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of overall Grade >=3 Adverse Events (AE) and Serious Adverse Events (SAE)
The number of Grade 3, 4 and 5 AEs and SAEs that occur throughout the study.
Measured: Up to 1 year post-infusion
Incidence of anticipated AIC100 CAR T Cell related AEs, SAEs and adverse events of special interest (AESI) (infusion-related reactions, CRS, ICANS, HLH/MAS, TLS, new malignancies, AEs leading to death and DLT AEs)
The number of CAR T related adverse events that occur throughout the study, including AESIs, infusion-related reactions, cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS), tumor lysis syndrome (TLS) and new malignancies.
Measured: Up to 15 years post-infusion
Determine recommended phase 2 dose
The recommended phase 2 dose will be determined through the dose escalation process
Measured: Up to 1 year post infusion
Secondary outcomes (1)
Assessment of presence and frequency of AIC100 CAR T cells in peripheral blood and tumor samples (when available)
Patients will be monitored for expansion and persistence of AIC100 CAR transgenes after infusion by vector copy number (VCN) analysis
Measured: Up to 15 years post-infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2020
- Study start28 Sep 2020
- Primary completion4 Aug 2026 (estimated)
- Study completion4 Aug 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anaplastic Thyroid CancerStudies 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AffyImmune Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sonal Gupta, MD PhD, Study director, AffyImmune Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04420754
- Sponsor's protocol ID
- 19-12021154
- Status last verified
- Jan 2025
- First submitted
- 3 Jun 2020
- Last updated
- 30 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Phase I Study of AIC100 CAR T Cells in Relapsed and/or Refractory Advanced Thyroid Cancer or Anaplastic Thyroid Cancer
- ClinicalTrials.gov ID
- NCT04420754
- Acronym / sponsor ID
- 19-12021154
- Keywords
- AIC100, CAR T Cell, Anaplastic Thyroid Cancer, Poorly Differentiated Thyroid Cancer
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- AffyImmune Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 28 Sep 2020 (actual)
- Primary completion
- 4 Aug 2026 (estimated)
- Study completion
- 4 Aug 2030 (estimated)
- First posted
- 9 Jun 2020 (actual)
- 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 assess the safety and tolerability and determine the recommended Phase 2 dose of AIC100 Chimeric Antigen Receptor (CAR) T cells in patients with relapsed/refractory poorly differentiated thyroid cancer and anaplastic thyroid cancer, including newly diagnosed.
Detailed description
The primary objective of this study is to assess the safety and tolerability of AIC100 CAR T Cells and determine the recommended Phase 2 dose of AIC100 in patients with relapsed/refractory poorly differentiated thyroid cancer and in patients with anaplastic thyroid cancer that are BRAF wild-type, including newly diagnosed, or BRAF mutant anaplastic thyroid cancer after failure of BRAF mutant specific therapy.
Upon enrollment, patients will undergo apheresis for collection of autologous lymphocytes. The autologous T cells will be transfected and expanded in vitro to generate the AIC100 CAR T Cell product. After lymphodepleting chemotherapy, AIC100 CAR T Cells will be infused.
The study drug product, AIC100, consists of autologous CAR T cells targeting intercellular adhesion molecule-1 (ICAM-1) on thyroid cancer. In addition, AIC100 cells express the somatostatin receptor subtype 2 (SSTR2), which should enable imaging of AIC100 CAR T Cells.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 7
| Arm | Type | Receives |
|---|---|---|
| Cohort -1 | Experimental | Biological: AIC100 CAR T CellsAIC100 CAR T Cell Dose Level -1 (Flat Dose): 1 x 10e6 CAR T cells |
| Cohort 1 | Experimental | Biological: AIC100 CAR T CellsAIC100 CAR T Cell Dose Level 1 (Flat Dose): 1 x 10e7 CAR T cells |
| Cohort 2 | Experimental | Biological: AIC100 CAR T CellsAIC100 CAR T Cell Dose Level 2 (Flat Dose): 1 x 10e8 CAR T cells |
| Cohort 3 | Experimental | Biological: AIC100 CAR T CellsAIC100 CAR T Cell Dose Level 3 (Flat Dose): 5 x 10e8 CAR T cells |
| Cohort 2.5 | Experimental | Biological: AIC100 CAR T CellsAIC100 CAR T Cell Dose Level 2.5 (Flat Dose): 2.5 x 10e8 CAR T cells. The interim step-down dose of Cohort 2.5 may be evaluated, if needed, based on ongoing safety and efficacy data. |
| Cohort 4 | Experimental | Biological: AIC100 CAR T CellsAIC100 CAR T Cell Dose Level 4 (Flat Dose): 7.5 x 10e8 CAR T cells. The proposed escalation dose of Cohort 4 may be evaluated, if needed, based on ongoing safety and efficacy data. |
| Cohort 5 | Experimental | Biological: AIC100 CAR T CellsAIC100 CAR T Cell Dose Level 5 (Flat Dose): 1 x 10e9 CAR T cells. The proposed escalation dose of Cohort 5 may be evaluated, if needed, based on ongoing safety and efficacy data. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (25 listed)
- Willing and able to participate in the study and provide written informed consent
- Be ≥ 18 years of age on the day of signing the Informed Consent Form
- Patients must have thyroid cancer that meets one of the following diagnoses, and, prior to lymphodepleting chemotherapy (LDC), have an identified available fresh or archival biopsy sample:
- Measurable disease by Computed Tomography (CT) or Positron Emission Tomography (PET) PET/CT per RECIST v1.1
- Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
Key exclusion criteria (17 listed)
- Women who are pregnant or breastfeeding
- Clinically significant, active, uncontrolled, systemic infection; the following are not exclusionary:
- Prior treatment with investigational gene therapy or CAR T cell therapy
- Presence of active and clinically relevant central nervous system disorder such as epilepsy, stroke, or symptomatic or uncontrolled brain metastases
- Evidence of another malignancy within 2 years prior to Screening (except in-situ non melanoma skin cancers, localized controlled prostate cancer, adequately treated Stage 1 uterine cancer that has a low risk of recurrence, or any other malignancies with similar outcome)
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 |
|---|---|---|
| AIC100 CAR T CellsAlso: AIC100Autologous CAR T cells directed against ICAM-1 | Biological | Cohort -1, Cohort 1, Cohort 2, Cohort 2.5, Cohort 3, Cohort 4, Cohort 5 |
Primary outcome
Incidence of overall Grade >=3 Adverse Events (AE) and Serious Adverse Events (SAE)
Time frame: Up to 1 year post-infusion
The number of Grade 3, 4 and 5 AEs and SAEs that occur throughout the study.
Incidence of anticipated AIC100 CAR T Cell related AEs, SAEs and adverse events of special interest (AESI) (infusion-related reactions, CRS, ICANS, HLH/MAS, TLS, new malignancies, AEs leading to death and DLT AEs)
Time frame: Up to 15 years post-infusion
The number of CAR T related adverse events that occur throughout the study, including AESIs, infusion-related reactions, cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS), tumor lysis syndrome (TLS) and new malignancies.
Determine recommended phase 2 dose
Time frame: Up to 1 year post infusion
The recommended phase 2 dose will be determined through the dose escalation process
1 secondary outcome
Assessment of presence and frequency of AIC100 CAR T cells in peripheral blood and tumor samples (when available)
Time frame: Up to 15 years post-infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2025.
- Individual participant dataSharing planned: No
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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.
Sonal Gupta, MD PhD study director
AffyImmune Therapeutics, Inc.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AIC100 CAR T Cells in people with Anaplastic Thyroid Cancer and Relapsed/Refractory Poorly Differentiated Thyroid Cancer, run by AffyImmune Therapeutics, Inc. at 4 sites in United States.
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 30 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. 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?
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?
Incidence of overall Grade >=3 Adverse Events (AE) and Serious Adverse Events (SAE) (time frame: Up to 1 year post-infusion). The study lists 3 primary outcomes in all. 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 30 Mar 2025.