SARC006: Phase II Trial of Chemotherapy in Sporadic and

Neurofibromatosis Type 1 Associated High Grade Unresectable Malignant

Peripheral Nerve Sheath Tumors

Brigitte Widemann, NCI, POB

Trial Schema

• Eight chemotherapy courses total

MPNST

NF1

Sporadic

IE x2

IA x2

IA x 2

Response Evaluation

Local Control Chemotherapy

IE x2

PET3D MRI

SurgeryXRTMRI

PET3D MRI

Patient Characteristics

• Two patients enrolled:

– Pt. 1: 24 y/o male with NF1 and metastatic MPNST

• Toxicity: Tolerated chemotherapy well

• Response evaluation post cy 4: Stable (WHO), 37% 3D

• Complicated post surgery course, removed from protocol

– Pt. 2: 21 y/o male with sporadic metastatic MPNST

• Pt. presented with leukemoid reaction, and widespread metastases

• Toxicity: Tolerated cy 1 of IA chemotherapy well (stable WHO)

• Removed from protocol after cy. 1 for palliative radiation for pain control and due to delayed platelet recovery as a result of bone marrow involvement.

SARC006 Protocol Status

– Protocol (amendment #3) open for enrollment:

• National Cancer Institute, Pennsylvania Oncology

– NF1 Sites:

• Close to approval: Children’s Memorial Chicago, Children’s Hospital and Clinics of Minnesota, Children’s Hospital of Philadelphia

• In review/consideration: Cincinnati Children’s Hospital, Children’s Hospital of Pittsburgh, St. Louis Children’s Hospital, University of Alabama at Birmingham, Children’s National Medical Center

SARC006 Protocol Status– SARC Sites:

• Close to approval: University of Michigan, Texas Children’s Hospital, Dana Farber Cancer Institute

• In review/consideration: University of Minnesota, MD Anderson, Mayo Clinic, Arkansas Children’s, Vanderbilt University, Emory University, Cleveland Clinic, Carolinas, Cedars Sinai, Lee Moffitt Cancer Center, Fox Chase Cancer Center, City of Hope, University of Utah, Stanford Comprehensive Cancer Center, Huntsman Cancer Center

• Not participating: Washington Cancer Institute, MSKCC, Johns Hopkins, Seattle Cancer Care, UCLA, University of Florida, Sarcoma Oncology Center, Oregon Health and Science University

SARC006 Approval Process • Contract with SARC• Institutional protocol review• US Army IRB review:

– Informed consent form, assent (ages 13-17 years) form, and information page (ages 7-12 years old), site specific protocol appendices

– CV for all investigators– Facility safety plan– PI assurance plan– GCP training for PI

• US Army will perform pre-review of documents prior to local IRB submission

• All communication with US Army IRB through SARC