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

Phase 2
18 Years
Open (Enrolling)
Thyroid Cancer

Thank you

Trial Information

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

- 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.

Inclusion Criteria:

- 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

- 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

- 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

Exclusion Criteria:

- 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

- 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

- 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

- History of noncompliance to medical regimens

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Progression free survival

Outcome Time Frame:

2 years

Safety Issue:


Principal Investigator

Jochen Lorch, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Dana-Farber Cancer Institute


United States: Food and Drug Administration

Study ID:




Start Date:

July 2009

Completion Date:

Related Keywords:

  • Thyroid Cancer
  • radioiodine refractory thyroid cancer
  • RAD001
  • Thyroid Neoplasms
  • Thyroid Diseases



MD Anderson Cancer CenterHouston, Texas  77030-4096
Dana-Farber Cancer InstituteBoston, Massachusetts  02115
Massachusetts General HospitalBoston, Massachusetts  02114-2617
Mt. Sinai Medical CenterNew York, New York  10029