Phase 2 study of the mTOR inhibitor RAD001 (everolimus) in combination with bevacizumab (avastin) in...
-
Upload
britton-harper -
Category
Documents
-
view
214 -
download
1
Transcript of Phase 2 study of the mTOR inhibitor RAD001 (everolimus) in combination with bevacizumab (avastin) in...
Phase 2 study of the mTOR inhibitor RAD001 (everolimus) in combination with bevacizumab
(avastin) in patients with sporadic and neurofibromatosis type 1 (NF1) related
refractory malignant peripheral nerve sheath tumors
Principal Investigator:
Brigitte Widemann, MDNational Cancer Institute, National Institutes of Health
SARC016
SARC016
SARC Sponsor and coordinating center
DoD Grant supporting clinical trial
Novartis Providing everolimus
Genetech Providing bevacizumab
SARC016
Background NF1 activation results in aberrant activation
mTOR Sirolimus halts tumor growth and prolongs
survival in a genetically engineered NF1 mouse model of MPNST
VEGF expression is increased in MPNST and correlates with poor outcome
Patients with NF1 related or sporadic refractory MPNST
N=25 patients
28 day cycles of everolimus (daily dosing) + bevacizumab
(day 1 and 14)2 stage design:• If > 1 of 15 pt respond
accrual will continue to a total of 25 patients
Ongoing tumor assessment imaging every 2 cycles until disease
progression or unacceptable toxicity for a maximum of 2 years
SARC016
Primary objectives: • Clinical benefit rate (CR, PR, SD at ≥ 4
months, WHO)
• Toxicity and safety of RAD001 and bevacizumab
SARC016 Participation Sites open to enrollment
National Cancer Institute Pennsylvania Oncology Lurie Children’s Hospital of
Chicago University of Michigan Washington University, St.
Louis
Children’s National Medical Center
Johns Hopkins University of Iowa Cincinnati Children’s Hospital Massachusetts General
Hospital and Dana Farber Sites pending University of Alabama at Birmingham Children’s Hospital of Philadelphia
Study Status
Current Enrollment n= 9
On study treatment n= 2
Off study n= 7