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Thyroid CancerPhase 2NCT00936858

RAD001 for Patients With Radioiodine Refractory Thyroid Cancer

A Phase 2 study testing RAD001 in people with Thyroid Cancer, run by Dana-Farber Cancer Institute at 4 sites 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

Since thyroid cancer becomes refractory to radioactive iodine, treatment options are very limited. Tyrosine kinase inhibitors such as sorafenib have recently shown promise. This trial seeks to expand treatment options for this disease with a new, oral drug called RAD001. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Thyroid Cancer
What is being tested?
RAD001
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
4 sitesUnited States
Study dates
Jul 2009to 27 Feb 2020
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

  • Histologically confirmed locally advanced or metastatic thyroid cancer, excluding thyroid lymphomas not amenable to or refractory to surgical resection, external beam radiotherapy, radioiodine or other local therapies.
  • Prior therapy with chemotherapy and targeted therapies except for mTor inhibitors is allowed.
  • Medullary thyroid cancer with documented evidence of disease progression by modified RECIST within 6 months before study day 1 or symptomatic disease at the time of screening in the absence of documented disease progression.
  • Differentiated thyroid cancer with documented evidence of disease progression by modified RECIST within 6 months before study day 1.
  • Anaplastic thyroid cancer with disease progression with documented disease progression by modified RECIST within 6 months of study day 1.
  • Patients must have at least one measurable site of disease according to RECIST criteria that has not previously irradiated. If the patient has has previous radiation to the marker lesion(s), there must be evidence of progression since radiation.
Show all 10Show fewer
  • 18 years of age or older
  • WHO performance status 2 or less
  • Adequate bone marrow, liver, and renal function
  • Fasting serum cholesterol 300mg/dL or less OR 7.75 mmol/L or less AND fasting triglycerides 2.5x ULN or less

Who cannot join

  • Patients receiving anticancer therapies within last 2 weeks or who have received radiation therapy within 3 weeks of study day 1
  • Prior therapy with mTOR inhibitors
  • Patients who have had a major surgery or significant traumatic injury within 4 weeks of start of study drug, patients who have not recovered from the side effects of any major surgery or patients that may require major surgery during the course of the study
  • Prior treatment with any investigational drug within the preceding 3 weeks
  • Patients receiving chronic, systemic treatment with corticosteroids or another immunosuppressive agent. Topical or inhaled corticosteroids are allowed.
  • Patients should not receive immunization with attenuated live vaccines within 2 weeks of study entry or during study period
Show all 16Show fewer
  • Uncontrolled brain or leptomeningeal metastases, including patients who continue to require glucocorticoids for brain or leptomeningeal metastases
  • Other malignancies within the past 3 years except for adequately treated carcinoma of the cervix or basal or squamous cell carcinomas of the skin
  • Patients who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study
  • A known history of HIV seropositivity
  • Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of RAD001
  • Patients with an active, bleeding diathesis
  • Female patients who are pregnant or breast feeding, or adults of reproductive potential who are not using effective birth control methods
  • Patients who have received prior treatment wih an mTOR inhibitor
  • Patients with a known hypersensitivity to RAD001 or other rapamycins or to its excipients
  • History of noncompliance to medical regimens

Trial locations and contacts

4 sites in 1 country.

United States4 sites
  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02115

  • MD Anderson Cancer Center

    Houston, Texas, 77030

  • Mt. Sinai Medical Center

    New York, New York, 10029

Other trials in United States

About this study

Official title: A Phase II Trial Using RAD001 for Patients With Radioiodine Refractory Thyroid Cancer

Since thyroid cancer becomes refractory to radioactive iodine, treatment options are very limited. Tyrosine kinase inhibitors such as sorafenib have recently shown promise. This trial seeks to expand treatment options for this disease with a new, oral drug called RAD001. It is an inhibitor of the mTOR pathway and has shown activity in neuroendocrine cancers of the gastrointestinal tract and has been approved for the treatment of metastatic renal cell cancer.

Read the detailed description
  • RAD001 will be taken once a day in the morning starting on Day 1 and continue until the participant is no longer participating in the study treatment.
  • A history and physical exam will be performed the first day of the study and then once a month. Blood tests including coagulation studies, and thyroid studies will be performed monthly. A urine sample will need to be provided on the first day of treatment and then every 2 months. Imaging consisting of a CT or MRI of the neck, chest and abdomen will be done every 8 weeks after starting RAD001.
  • Participants will remain on this research study for up to 24 months. However, if the participants doctor feels that they are benefiting from the study drug and they do not have severe side effects, they may be given the option to continue taking RAD001.
Conditions
Thyroid Cancer
Being tested
RAD001
Keywords
radioiodine refractory thyroid cancer, RAD001
Medical subject headings
Thyroid Neoplasms

What participants receive

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

  • RAD001Drug

    Also called Afinitor (everolimus)

Study groups in NCT00936858
Study groupRoleWhat they receive
Arm AExperimentalRAD001 will be administered orally as once daily dose of 10 mg (one 10mg tablet or two 5mg tablets) continuously from study day 1 until progression of disease or unacceptable toxicity.
Receives: Drug: RAD001

What is being measured

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

  1. Median Progression Free Survival

    Progression free survival (PFS) is defined as the time from start of treatment to disease progression or death from any cause as estimated by Kaplan Meier methods. Progression is measured using RECIST 1.1 criteria, defined as at least a 20% increase in size in target lesion and/or unequivocal progression of non-target lesions and/or appearance of new lesions. Patients who have not progressed and are alive are censored at the date the patient is known to be progression-free.

    Measured: Every 2 months for first 24 months, then every 3 months from >24 to 60 months; up to 48 months.

Secondary outcomes (3)
  1. Objective Response Rate

    The objective response rate is the proportion of participants achieving complete response (CR) or partial response (PR) on treatment based on Response Evaluation Criteria In Solid Tumors Criteria (RECIST 1.1) criteria.

    PR or better is achieved if the following are true:

    Target Lesions:

    -At least a 30% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm.

    Non-target Lesions:

    No progression. No appearance new lesions or unequivocal progression of existing non-target lesions. Unequivocal progression should not normally trump target lesion status. It must be representative of overall disease status change, not a single lesion increase.

    Bone Lesions:

    ->50% increase in lesions.

    -No new lesions.

    Measured: Every 2 months for first 24 months, then every 3 months from >24 to 60 months; up to 48 months.

  2. Median Overall Survival

    Overall Survival (OS) is defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive.

    Measured: Every 2 months for first 24 months, then every 3 months from >24 to 60 months; up to 5 years post study registration.

  3. Mean Change in Quality of Life [Medullary Thyroid Cancer Population Only]

    The M. D. Anderson Symptom Inventory (MDASI) questionnaire was used to assess quality of life. Questions 1 to 19 were scored. Each of the 19 questions have a response range of 0 - 10, where 0 represents "not present" and 10 represents "as bad as you can imagine". The 19 responses are averaged together to create a mean score, with a lower score indicating a better quality of life. All questionnaire mean scores at each timepoint are averaged together to give a mean score at that timepoint (baseline and week 8) The mean change in quality of life is calculated by subtracting the baseline mean score from the week 8 mean score.

    Measured: Measured at baseline and then again at cycle 8 (8 months).

Timeline

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

  1. First posted10 Jul 2009 (estimated)
  2. Study startJul 2009
  3. Primary completion27 Feb 2020
  4. Study completion27 Feb 2020

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
50 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00936858
Sponsor's protocol ID
09-049
Status last verified
Jul 2026
First submitted
9 Jul 2009
Last updated
12 Aug 2026
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing RAD001 in people with Thyroid Cancer, run by Dana-Farber Cancer Institute 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 10 inclusion and 16 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 12 Aug 2026. 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 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 28 Sep 2021). 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?

Median Progression Free Survival (time frame: Every 2 months for first 24 months, then every 3 months from >24 to 60 months; up to 48 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 12 Aug 2026.

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