NCT00113360

Phase II Study of RAD001 Plus Octreotide Depot in Patients With Metastatic or Unresectable Low Grade Neuroendocrine Carcinoma (Carcinoid, Islet Cell)

Completed Phase 2 Results posted Last updated 24 April 2025

What this trial tests

Phase 2 trial testing RAD001 in Neuroendocrine Carcinoma in 67 participants. Completed in 1 July 2009.

Timeline

1 January 2005

Primary endpoint
1 July 2009

1 July 2009

Quick facts

Lead sponsor	M.D. Anderson Cancer Center
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	67
Start date	1 January 2005
Primary completion	1 July 2009
Estimated completion	1 July 2009
Sites	1 location across United States

Drugs / interventions tested

• RAD001 — full drug profile →
• Octreotide Depot — full drug profile →

Conditions studied

• Neuroendocrine Carcinoma — all drugs for Neuroendocrine Carcinoma →
• Islet Cell Carcinoma — all drugs for Islet Cell Carcinoma →

Sponsor

M.D. Anderson Cancer Center — full company profile →

Who can join

18 and older, any sex, with Neuroendocrine Carcinoma or Islet Cell Carcinoma. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Progression Free Survival (PFS)
  Time frame: PFS assessed every 12 weeks (at the end of every 3 cycles) or more frequently if clinically indicated
  PFS is measured from date of trial entry until documented progression of disease or death from any cause. PFS is measured by computed tomography (CT) scans or magnetic resonance imaging (MRI) performed at baseline and after every three cycles.

Sponsor's own description

Objectives:

Primary endpoint:

-Assess the clinical activity of RAD 001 plus depot octreotide as defined by progression free survival (PFS) duration defined by RECIST criteria in treated and untreated patients with metastatic, unresectable low grade neuroendocrine carcinoma.

Secondary endpoints:

* Assess the progression free survival duration of patients with metastatic, unresectable low grade neuroendocrine carcinoma treated with RAD 001 plus depot octreotide.
* Assess the safety of RAD 001 plus depot octreotide in patients with metastatic, unresectable low grade neuroendocrine carcinoma.
* To determine the expression/phosphorylation status of the components of the mTOR signaling pathway in the primary tumors, in order to determine whether these markers can be used as predictors of sensitivity to the combination of RAD001 and octreotide.
* To determine the effect of the combination of RAD001 and octreotide on the expression and phosphorylation of mTOR's targets in the accessible tumor tissue, in order to identify potential pharmacodynamics markers of response to this drug combination.
* To observe the effects of treatment with RAD001 on plasma angiogenic biomarkers.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. PIK3CA/PTEN mutations and Akt activation as markers of sensitivity to allosteric mTOR inhibitors.
   Meric-Bernstam F, Akcakanat A, Chen H, Do KA, et al · · 2012 · cited 175× · PMID 22422409 · DOI 10.1158/1078-0432.ccr-11-2123
2. Genomic Determinants of PI3K Pathway Inhibitor Response in Cancer.
   Weigelt B, Downward J. · · 2012 · cited 72× · PMID 22970424 · DOI 10.3389/fonc.2012.00109
3. PI3K-AKT-mTOR-signaling and beyond: the complex network in gastroenteropancreatic neuroendocrine neoplasms.
   Briest F, Grabowski P. · · 2014 · cited 68× · PMID 24578720 · DOI 10.7150/thno.7851
4. Endocrine tumours in neurofibromatosis type 1, tuberous sclerosis and related syndromes.
   Lodish MB, Stratakis CA. · · 2010 · cited 54× · PMID 20833335 · DOI 10.1016/j.beem.2010.02.002