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Mono Centre, Open Label Proof of Concept Study SOM230 in Progressive Medullary Thyroid Cancer Patients and the Combination With RAD001 Upon Progression

Phase 2
18 Years
80 Years
Open (Enrolling)
Medullary Thyroid Cancer

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Trial Information

Mono Centre, Open Label Proof of Concept Study SOM230 in Progressive Medullary Thyroid Cancer Patients and the Combination With RAD001 Upon Progression

Medullary thyroid cancer (MTC) is a neuroendocrine tumor originating from thyroid C cells.
Neuroendocrine tumors have been demonstrated to express somatostatin receptors as well as
mTOR pathway. The somatostatin analogues now available (octreotide and lanreotide) act
preferentially through the somatostatin receptor subtype 2 (sst2). In MTC, these compounds
have been reported to exert anti-secretive effects on calcitonin but no anti-proliferative
effects.SOM230 (pasireotide) is a new somatostatin analogue showing a peculiar binding
profile with high affinity for sst1, sst2, sst3, sst5. Preliminary data show SOM230 to be
effective in a phase II study on patients with metastatic carcinoid. RAD001 (everolimus) is
a novel agent that interacts with mTOR. It was demonstrated to inhibit tumor growth in
neuroendocrine tumor cell lines. Some clinical trials have explored the efficacy of a
combined therapy with RAD001 plus octreotide in patients with digestive neuroendocrine
tumors, highlighting encouraging results in term of tumor control.In particular, octreotide
and RAD001 seem to show a synergistic activity in inhibiting neuroendocrine tumor

Inclusion Criteria:

- Patients with progressive metastatic or postoperative persistent medullary thyroid
cancer who have histopathologically confirmed disease and measurable tumor lesions.
(Postoperative persistent after surgical removal is characterized by increased levels
of calcitonin with or without radiological detectable tumour relapse or metastases.)

- Patients with evidence of biochemical progression of disease, as expressed by
progressive increase of serum calcitonin levels, assessed once a month for at least
three months before study entry, according to RECIST definitions (elevation of the
markers for at least 25 %).

- Disease that is not amenable to surgery, radiation, or combined modality therapy with
curative intent.

- Adequate organ function - Karnofsky-Index performance status >60%

- Life expectancy > 6 months

- Age > 18 years

- Women of childbearing potential must have a negative serum pregnancy test within 14
days of randomization and a urine pregnancy test 48 hours prior to the administration
of the first study treatment.

- Patients with a known history of impaired fasting glucose or diabetes mellitus (DM)
may be included, however blood glucose and antidiabetic treatment must be monitored
closely throughout the trial and adjusted as necessary.

- Signed and dated informed consent document indicating that the patient has been
informed of all pertinent aspects of the trial prior to enrolment.

Exclusion Criteria:

- Unstable systemic diseases including uncontrolled hypertension, active uncontrolled
infections, psychiatric condition or laboratory abnormality that may increase the
risk associated with study participation or study drug administration, or may
interfere with the interpretation of study results, and in the judgment of the
investigator would make the patient inappropriate for entry into this study.

- Known hypersensitivity to somatostatin analogues.

- Pregnant or breast-feeding patients

- Sign of recurrence of prior or concomitant malignancies (within the last 3 years or
requiring active treatment) other than MTC; with the exception of previous basal cell
skin cancer, previous cervical carcinoma in situ

- Prior therapy with mTOR inhibitors (e.g. sirolimus, temsirolimus, everolimus)

- Participation in a clinical trial to test an investigational drug within 4 weeks
prior to visit 1.

- Any of severe and/or uncontrolled medical conditions:

- Patients who have congestive heart failure (NYHA Class III or IV), unstable angina,
sustained ventricular tachycardia, ventricular fibrillation, clinically significant
bradycardia, advanced heart block or a history of acute myocardial infarction within
the six months preceding enrollment,

- QT related exclusion criteria

- Previous treatments with chemotherapy, loco regional therapy (eg, chemoembolization)
or interferon are permitted providing that toxicity has resolved to < Grade 1 at
study entry and that last treatment was at least 4 weeks prior to baseline

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:

Efficacy of SOM230 in patients with progressive metastatic or postoperative persistent medullary thyroid cancer

Outcome Description:

Efficacy is defined as progression-free survival (PFS), according to RECIST criteria, in patients treated with SOM230.

Outcome Time Frame:

From date of start therapy until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 6 months.

Safety Issue:


Principal Investigator

Annamaria Colao

Investigator Role:

Principal Investigator

Investigator Affiliation:

"Federico II" University of Naples, Italy


Italy: Ethics Committee

Study ID:




Start Date:

February 2012

Completion Date:

Related Keywords:

  • Medullary Thyroid Cancer
  • Progressive metastatic
  • postoperative persistent
  • Thyroid Neoplasms
  • Thyroid Diseases