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Anaplastic Thyroid CancerPhase 2NCT02688608

Pembrolizumab in Anaplastic/Undifferentiated Thyroid Cancer

A Phase 2 study testing Pembrolizumab in people with Anaplastic Thyroid Cancer, run by Saad A Khan at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This study is being done because there are currently no approved and no commonly working targeted therapies in anaplastic thyroid cancer (ATC). This is an area of urgent need for patients, not just for approved treatments but also rationally-designed clinical trials designed specifically for ATC. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Anaplastic Thyroid Cancer
What is being tested?
Pembrolizumab
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
1 siteUnited States
Study dates
Oct 2016to Oct 2021
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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

  • Be willing and able to provide written informed consent for the trial.
  • Histologically or cytologically confirmed diagnosis of anaplastic thyroid cancer or undifferentiated thyroid cancer. A diagnosis of possible ATC/UTC will be allowed if the clinical presentation is consistent with anaplastic or undifferentiated thyroid cancer.
  • Be ≥ 18 years of age on day of signing informed consent.
  • Have measurable disease based on RECIST 1.1.
  • Be willing to provide tissue from a newly obtained core or excisional biopsy of a tumor lesion. Newly-obtained is defined as a specimen obtained up to 6 weeks (42 days) prior to initiation of treatment on Day 1. Subjects for whom newly-obtained samples cannot be provided (e.g. inaccessible or subject safety concern) may submit an archived sample.
  • Have a performance status of 0-1 on the Eastern Cooperative Oncology Group (ECOG) Performance Scale.
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  • Demonstrate adequate organ function as defined in the protocol. ,
  • Female subject of childbearing potential should have a negative urine or serum pregnancy within 72 hours prior to receiving the first dose of study medication. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required.
  • Female subjects of childbearing potential should be willing to use 2 methods of birth control or be surgically sterile, or abstain from heterosexual activity for the course of the study through 120 days after the last dose of study medication (Reference Section 5.7.2). Subjects of childbearing potential are those who have not been surgically sterilized or have not been free from menses for > 1 year.
  • Male subjects should agree to use an adequate method of contraception starting with the first dose of study therapy through 120 days after the last dose of study therapy.

Who cannot join

  • Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the first dose of treatment.
  • Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of trial treatment.
  • Has a known history of active TB (Bacillus Tuberculosis).
  • Hypersensitivity to pembrolizumab or any of its excipients.
  • Has had a prior anti-cancer monoclonal antibody (mAb) within 4 weeks prior to study Day 1 or who has not recovered (i.e., ≤ Grade 1 or at baseline) from adverse events due to agents administered more than 4 weeks earlier.
  • Has had prior chemotherapy, targeted small molecule therapy, or radiation therapy within 2 weeks prior to study Day 1 or who has not recovered (i.e., ≤ Grade 1 or at baseline) from adverse events due to a previously administered agent.
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  • Note: Subjects with ≤ Grade 2 neuropathy are an exception to this criterion and may qualify for the study.
  • Note: If subject received major surgery, they must have recovered adequately from the toxicity and/or complications from the intervention prior to starting therapy.
  • Has a known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer.
  • Has known active central nervous system (CNS) metastases and/or carcinomatous meningitis. Subjects with previously treated brain metastases may participate provided they are stable (without evidence of progression by imaging for at least four weeks prior to the first dose of trial treatment and any neurologic symptoms have returned to baseline), have no evidence of new or enlarging brain metastases, and are not using steroids for at least 7 days prior to trial treatment. This exception does not include carcinomatous meningitis which is excluded regardless of clinical stability.
  • Has active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.
  • Has known history of, or any evidence of active, non-infectious pneumonitis.
  • Has an active infection requiring systemic therapy.
  • Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject's participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating investigator.
  • Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
  • Is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 120 days after the last dose of trial treatment.
  • Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent.
  • Has a known history of Human Immunodeficiency Virus (HIV) (HIV 1/2 antibodies).
  • Has known active Hepatitis B (e.g., HBsAg reactive) or Hepatitis C (e.g., HCV RNA [qualitative] is detected).
  • Has received a live vaccine within 30 days of planned start of study therapy. Note: Seasonal influenza vaccines for injection are generally inactivated flu vaccines and are allowed; however intranasal influenza vaccines (e.g., Flu-Mist®) are live attenuated vaccines, and are not allowed.

Trial locations and contacts

1 site in 1 country.

United States1 site
  • UT Southwestern Medical Center

    Dallas, Texas, 75390

Other trials in United States

About this study

Official title: Phase II Trial of Pembrolizumab in Metastatic or Locally Advanced Anaplastic/ Undifferentiated Thyroid Cancer

This study is being done because there are currently no approved and no commonly working targeted therapies in anaplastic thyroid cancer (ATC). This is an area of urgent need for patients, not just for approved treatments but also rationally-designed clinical trials designed specifically for ATC. Patients diagnosed with anaplastic thyroid cancer have a very high likelihood of dying because of their disease. As such there is a clear need for improving therapy for ATC.

Read the detailed description

The goal of this multi-center, open-label trial is to measure the impact of treating metastatic anaplastic thyroid cancer patients with immune checkpoint therapy. This trial will potentially lead to the development of new therapy for anaplastic thyroid cancer. The drug to be administered, pembrolizumab is FDA approved with known side effects and is active in many tumor types.

Programmed death 1 or (PD-1) PD-1/PD-L1 expression will be measured and reported for patients undergoing therapy with pembrolizumab. This will help determine if there PD-1 or PD-L1 are predictive biomarkers for anti-PD-1 therapy. It will also add to the data regarding their frequency in aggressive thyroid cancer. Where available, genomic profiling data will be analyzed to determine if there is a correlation between response and mutational status.

Being tested
Pembrolizumab
Medical subject headings
Thyroid Carcinoma, Anaplastic

What participants receive

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

  • PembrolizumabDrug

    Also called Keytruda-Merck

    200 mg IV once every 3 weeks

Study groups in NCT02688608
Study groupRoleWhat they receive
PembrolizumabExperimental200 milligrams of Pembrolizumab will be given intravenously every 3 weeks.
Receives: Drug: Pembrolizumab

What is being measured

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

  1. Overall Response (OR)

    Overall response (OR) represents those participants that collectively achieve either complete response (CR) or partial response (PR) within 18 months, as defined below per the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.

    • Complete Response (CR) = Disappearance of all target lesions
    • Partial Response (PR) = ≥ 30% decrease in the sum of the longest diameter of target lesions
    • Overall Response (OR) = CR + PR
    • Progressive disease (PD) = 20% increase in the sum of the longest diameter of target lesions, and/or the appearance of one or more new lesion(s)
    • Stable disease (SD) = Small changes that do not meet any of the above criteria The outcome is reported as the cumulative number of participants receiving at least one dose of study treatment, that achieve either a CR or PR with 6 months, a number without dispersion.

    Measured: up to 18 months.

Secondary outcomes (3)
  1. Progression-free Survival (PFS)

    Progression-free survival (PFS) means to remain alive without disease progression. Progressive disease is defined as a 20% increase in the sum of the longest diameter of target lesions, and/or the appearance of one or more new lesion(s). The outcome is reported as the number of participants who received at least one dose of study treatment and remained alive without progression at 2 years after the beginning of treatment, a number without dispersion.

    Measured: 2 years

  2. Adverse Events Associated With Pembrolizumab

    Safety of the study treatment pembrolizumab is based on the number of adverse effects caused by pembrolizumab, referred to as related adverse events or toxicities. Reported adverse events (related) will be serious as defined by the Code of Federal Regulations at 21CFR§312.32, or non-serious. The outcome is reported as the number of non-serious and serious adverse events that occurred with the nominal study period (35 cycles or 2 years) that were reported as possibly, probably, or definitely related to pembrolizumab, a number without dispersion.

    Measured: 2 years

  3. Overall Survival (OS)

    Overall survival (OS) means to remain alive without consideration of treatment response status.

    The outcome is reported as the number of participants who received at least one dose of study treatment and were known to remain alive at 2 years after the beginning of treatment, a number without dispersion. Subjects who become lost-to-follow-up before the 2-year assessment are not included.

    Measured: 2 years

Timeline

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

  1. First posted23 Feb 2016 (estimated)
  2. Study startOct 2016
  3. Primary completionOct 2020
  4. Study completionOct 2021

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02688608
Sponsor's protocol ID
STU 012016-019
Other IDs
STU 012016-019
Status last verified
Feb 2022
First submitted
20 Jan 2016
Last updated
10 Feb 2022
Data monitoring committee
Yes
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pembrolizumab in people with Anaplastic Thyroid Cancer, run by Saad A Khan at 1 site 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 10 inclusion and 20 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: finished, as of the sponsor's last update on 10 Feb 2022. 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 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 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?

Yes. Results are posted in the registry (first posted 10 Feb 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Overall Response (OR) (time frame: up to 18 months.). 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 10 Feb 2022.

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