Can reprogrammed immune cells take on a rare thyroid cancer?

NCT ID NCT04877613

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 22, 2026 · Last updated Jul 23, 2026 · Updated 1 time

Summary

This early-phase trial is testing whether a single infusion of specially engineered immune cells (called CAR T cells) can safely target and attack medullary thyroid cancer that has spread or come back. The cells are designed to recognize a protein called GFRα4 found on the cancer cells. Participants receive chemotherapy first to make room for the new cells, then the infusion. The main goal is to check safety, but researchers will also watch for any signs that the tumors shrink or stop growing.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
a single dose of engineered immune cells (CAR T cells) targeting GFRα4, given after chemotherapy to prepare the body
What this could lead to
If successful, this approach could offer a new treatment option for people with advanced medullary thyroid cancer that has stopped responding to standard therapies.
What could go wrong
This is a very early phase 1 trial with only 9 participants, focused mainly on safety. CAR T-cell therapy can cause serious side effects, and it is unknown whether it will shrink tumors or improve survival.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

9 people

The number who actually took part.

Started

Aug 2021

Expected to finish

Jun 2039

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Signed, written informed consent 2. Male or female age ≥ 18 years 3. Histologically or cytologically confirmed diagnosis of medullary thyroid cancer (MTC). 4. Incurable recurrent/metastatic disease that is progressive after at least 1 prior tyrosine kinase inhibitor (TKI) containing regimen, or the patient was intolerant of or declined such therapy. 5. Adequate organ function defined as: 1. Serum creatinine ≤ 2.5 mg/dl or estimated creatinine clearance ≥ 30 ml/min and not on dialysis. 2. AST ≤ 5x upper limit of normal range and total bilirubin ≤ 2.0 mg/dl; except for patients in whom hyperbilirubinemia is attributed to Gilbert's syndrome. 3. Left Ventricular Ejection Fraction (LVEF) ≥ 45% confirmed by ECHO/MUGA 4. Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and pulse oxygen greater than 92% on room air. 6. ECOG Performance Status that is either 0 or 1. 7. Toxicities from prior therapies must have recovered to grade ≤ 2 according to the CTCAE 5.0 criteria or to the patient's prior baseline. 8. Patients must have evaluable disease as defined by RECIST 1.1. 9. Subjects of reproductive potential must agree to use acceptable birth control methods. Exclusion Criteria: 1. Evidence of active hepatitis B or hepatitis C infection. The following would not qualify as an active infection, thus would not exclude the subject from participating 1. Positive HBV serology with undetectable viral load and ongoing antiviral prophylaxis for potential HBV reactivation. 2. Positive HCV serology with quantitative PCR for plasma HCV RNA below the lower limit of detection, with or without concurrent antiviral HCV treatment. 2. Any other active, uncontrolled infection. 3. Any prior history of moderate to severe (Grade 2 or higher) pneumonitis. 4. Subjects with chronic kidney disease with Grade 2 or higher renal impairment (eGFR or CrCl 59-30 ml/min/1.73 m2). 5. Class III/IV cardiovascular disability according to the New York Heart Association Classification. 6. Clinically apparent arrhythmia or arrhythmias that are not stable on medical management within two weeks of physician-investigator confirmation of eligibility. 7. Planned concurrent treatment with systemic high dose corticosteroids. Patients may be on a stable low dose of steroids (≤10mg equivalent of prednisone). Use of inhaled steroids is allowable. Corticosteroid treatment as anti-emetic prophylaxis on the day of lymphodepleting chemotherapy administration is allowed per institutional practice. 8. Any moderate to severe skin rash or allergies requiring systemic treatment. 9. Receipt of immune checkpoint inhibitors within 2 months prior to physician-investigator confirmation of eligibility - Retired with Protocol Version 3. 10. Pregnant or nursing (lactating) women. 11. Active autoimmune disease requiring systemic immunosuppressive treatment equivalent to ≥ 10mg daily of prednisone. Patients with autoimmune neurological diseases (such as MS or Parkinson's) will be excluded. 12. Have any history of prior or active central nervous system (CNS) involvement (e.g., leptomeningeal disease, parenchymal masses) with MTC. Screening for this (e.g., with lumbar puncture and/or brain MRI) is not required unless suspicious symptoms and/or radiographic findings are present. Subjects with calvarial metastatic disease that extends intracranially and involves the dura will be excluded, even if CSF is negative for MTC. 13. Known seizure disorder or history of prior seizures requiring medication. 14. History of allergy or hypersensitivity to study product excipients (human serum albumin, DMSO, and Dextran 40).

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Metastatic medullary thyroid cancer are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.