Phase 2
N=31
BEZ235 Phase II Trial in Patients With Advanced Pancreatic Neuroendocrine Tumors (pNET) After Failure of mTOR Inhibitor Therapy.
Pancreatic Neuroendocrine Tumors (pNET)
Bottom Line
View on ClinicalTrials.gov: NCT01658436 ↗Enrolled (actual)
31
Serious AEs
41.9%
Results posted
May 2016
Primary outcome: Primary: Stage 1 - Progression Free Survival (PFS) Rate Analysis at 16 Weeks as Per Local Radiology Review — 51.6 Percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- BEZ235 (Stage 1) (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Jul 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Stage 1 - Progression Free Survival (PFS) Rate Analysis at 16 Weeks as Per Local Radiology Review |
51.6 | — |
| SECONDARY Stage 1- Overall Response Rate (ORR) |
0.0; 0.0 | — |
| SECONDARY Stage 1 - Disease Control Rate |
71.0 | — |
Summary
This is a Phase II study in 2 stages, evaluating BEZ235 plus best supportive care (BSC) versus placebo plus BSC in patients with advanced pancreatic neuroendocrine tumors (pNET) after failure of mTOR inhibitor therapy.
Study design: This was a Phase II, two-stage, multicenter study, where Stage 1 was a single arm, open label design and Stage 2 was planned to be a randomized, double-blind study.
However, at the end of Stage 1, the futility was met and hence the Stage 2 was not initiated.
Eligibility Criteria
Inclusion Criteria
- Unresectable or metastatic, histologically confirmed low or intermediate grade pancreatic neuroendocrine tumor with radiological evidence of disease progression since last treatment
- Refractory disease to treatment with mTOR inhibitor
- Measurable disease per RECIST Version 1.1 using Computed Tomography (CT) or Magnetic Resonance Imaging (MRI)
- Prior or concurrent therapy with SSA is permitted; a stable dose at least 2 months prior to study start and must continue on the stable dose while receiving study treatment; SSA is not considered as a systemic treatment.
- WHO PS ≤ 1
- Adequate bone marrow function or organ function
Exclusion Criteria
- Previous treatment with any PI3K or AKT inhibitor
- Discontinuation prior mTOR inhibitor therapy due to toxicity
- Poorly differentiated neuroendocrine carcinoma, high-grade neuroendocrine carcinoma, adenocarcinoid, goblet cell carcinoid and small cell carcinoma
- Radiotherapy, or major surgery within 4 weeks prior to study treatment start
- Hepatic artery embolization or cryoablation/ radiofrequency ablation of hepatic metastasis within 2 months of study treatment start.
- More than 3 prior systemic treatment regimens (including cytotoxic chemotherapy, targeted therapy, immunotherapy)
Data sourced from ClinicalTrials.gov (NCT01658436). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.