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Phase II Study of RAD001monotherapy in Patients With Unresectable Adenoid Cystic Carcinoma

Phase 2
18 Years
Open (Enrolling)
Adenoid Cystic Carcinoma

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Trial Information

Phase II Study of RAD001monotherapy in Patients With Unresectable Adenoid Cystic Carcinoma

Although the histologic appearance of adenoid cystic carcinoma is low grade, management of
this malignancy is a distinct therapeutic challenge because of its insidious local growth
pattern, propensity for perineural involvement, tendency for distant metastasis, and
pronounced ability to recur over a prolonged period.

In prospectively performed clinical trials, objective responses to any cytotoxic agent or
regimen are infrequent, whereas stabilization of disease was observed more commonly.

In adenoid cystic carcinoma, the study focusing on PI3-K/AKT/mTOR pathway is rare.

According to Younes MN et al's study, adenoid cystic carcinoma cell lines have increased
pAkt activity when EGF-stimulation is added. And when treated with EGFR/VEGFR TK dual
inhibitor, the phosphorylated form of Akt decreased despite of total level of Akt is
remained unchanged.

When the investigators consider that the increased pAkt activity is one of possible
predictor to mTOR inhibitor, the mTOR inhibitor might have an activity in adenoid cystic

Although mTOR is clearly an attractive therapeutic target in tumor, no clinical study on
mTOR inhibition by RAD001 has been systematically conducted in adenoid cystic carcinoma.

In phase I study of RAD001, 2 patients with adenoid cystic carcinoma show some response to
RAD001 (unpublished data).

So the investigators design this phase II study of RAD001 in adenoid cystic carcinoma to
evaluate the efficacy of RAD001 in this orphan disease.

Inclusion Criteria:

- 1. Histologically or cytologically confirmed adenoid cystic carcinoma

- 2. Local, locally-advanced or metastatic disease documented as having shown
progression on a scan (CT, MRI, MIBI scan) taken 2 to 12 months prior to baseline
compared to a previous scan taken at any time in the past. Progression must be
documented according to RECIST criteria.

- 3. Disease that is not amenable to surgery, radiation or combined modality therapy
with curative intent and who is previously treated with chemotherapy or local
treatment (e,g transarterial chemoembolization)

- 4. Presence of at least one measurable target lesion for further evaluation according
to RECIST criteria

- 5. 18 years or older

- 6. ECOG performance status 0, 1

- 7. Previous treatment with chemotherapy, loco-regional therapy (e.g
chemoembolization) 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

- 8. Adequate organ function

- 9. A patient with the willingness to comply with the study protocol during the study
period and capable of complying with it

- 10. A patient who signed the informed consent prior to the participation of the study
and who understands that he/she has a right to withdrawal from participation in the
study at any time without any disadvantages.

Exclusion Criteria:

- 1. A patient with no measurable disease

- 2. Prior chemotherapy, radiation therapy or surgery within 4 weeks prior to study
entry except palliative radiotherapy to non-target lesions (within 2 weeks prior to
study entry)

- 3. A patient with previous active or passive immunotherapy

- 4. A patient with intestinal obstruction or impending obstruction, recent active
upper GI bleeding

- 5. A pregnant or lactating patient

- 6. A patient of childbearing potential without being tested for pregnancy at baseline
or with being tested for positive. (A postmenopausal woman with the amenorrhea period
of at least 12 months or longer is considered to have non-childbearing potential)

- 7. A man or woman of childbearing potential who has no willingness to use a
contraceptive measure during the study

- 8. A patient with history of another malignant disease within past 5 years, except
curatively treated basal cell carcinoma of skin and cervical carcinoma in situ.

- 9. A patient with history of uncontrolled seizures, central nervous system disorder
or psychiatric disorders that are considered clinically significant by the
investigator that would prohibit the understanding of informed consent or that may be
considered to interfere with the compliance of the administration of the study

- 10. A patient with clinically significant heart disease (e.g. congestive heart
failure, symptomatic coronary artery diseases, cardiac arrhythmia, etc) or myocardial
infarction within past 12 months.

- 11. Ongoing cardiac arrhythmia of grade ≥2, atrial fibrillation of any grade, or QTc
interval>450msec for males or >470msec for female.

- 12. A patient with interstitial pneumonia or diffuse symptomatic fibrosis of the

- 13. A patient with peripheral neuropathy of grade 1 by NCI CTC, caused by other
factors (e.g. alcohol, diabetes, etc). If the absence of deep tendon reflexes is the
only neurologic disorder, this condition does not apply to the exclusion criteria.

- 14. A patient with organ transplantation requiring immunosuppressive therapy

Type of Study:


Study Design:

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

Outcome Measure:

progression free survival rate at 4 months

Outcome Description:

proportion of patients who are alive and progression-free at the time of 4 months of treatment among all patients

Outcome Time Frame:

10 months

Safety Issue:


Principal Investigator

Yung-Jue Bang, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Seoul National University Hospital


Korean Food and Drug Administration:Korea, Republic of

Study ID:




Start Date:

July 2008

Completion Date:

December 2011

Related Keywords:

  • Adenoid Cystic Carcinoma
  • Adenoid cystic carcinoma
  • RAD001
  • Carcinoma
  • Carcinoma, Adenoid Cystic