Phase 2
N=48
Combination Chemotherapy in Treating Patients With Stage III or Stage IV Malignant Peripheral Nerve Sheath Tumors
Neurofibromatosis Type 1 · Sarcoma
Bottom Line
View on ClinicalTrials.gov: NCT00304083 ↗Enrolled (actual)
48
Serious AEs
27.1%
Results posted
May 2018
Primary outcome: Primary: Number of Participants With Response Rate (Complete Response and Partial Response) — 5; 4; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- filgrastim (Biological); doxorubicin hydrochloride (Drug); etoposide (Drug); ifosfamide (Drug); conventional surgery (Procedure); radiation therapy (Radiation)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Sarcoma Alliance for Research through Collaboration
- Primary completion
- Jun 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Response Rate (Complete Response and Partial Response) |
5; 4; 0; 0; 20; 4 | — |
| SECONDARY Response of Plexiform Neurofibroma to Neoadjuvant Chemotherapy Using Volumetric MRI Analysis |
— | — |
| SECONDARY Utility of Fludeoxyglucose F18 Positron Emission Tomography (18FDG-PET) and Automated MRI Volumetric Tumor Analysis to Assess Response to Treatment |
— | — |
| SECONDARY Response Evaluation Using WHO, RECIST, 18 FDG-PET and Volumetric MRI With Percent Necrosis in Tumor Specimens |
29; 4 | — |
| SECONDARY Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response |
15; 7; 1; 0; 0; 2 | — |
| SECONDARY Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue |
3; 1; 5; 2; 2; 0 | — |
| SECONDARY Identify the Number of Participants With a Serum Biomarker to Predict the Presence of MPNST Versus Benign Plexiform Neurofibroma |
3; 2 | — |
| SECONDARY Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs. |
10; 17; 19; 26; 12; 12 | — |
Summary
RATIONALE: Drugs used in chemotherapy, such as doxorubicin, ifosfamide, and etoposide, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Radiation therapy uses high-energy x-rays to kill tumor cells. Giving combination chemotherapy with or without radiation therapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving combination chemotherapy after surgery may kill any tumor cells that remain after surgery.
PURPOSE: This phase II trial is studying how well combination chemotherapy works in treating patients with stage III or stage IV malignant peripheral nerve sheath tumors.
Eligibility Criteria
DISEASE CHARACTERISTICS:
- Newly diagnosed sporadic or neurofibromatosis type 1 (NF1)-associated high-grade malignant peripheral nerve sheath tumors (MPNSTs)
- Stage III or stage IV (metastatic) disease
- Measurable disease, defined as at least 1 tumor that is measurable in 2 dimensions on CT scan or MRI
PATIENT CHARACTERISTICS:
- Ejection fraction normal by echocardiogram or MUGA
- Serum creatinine normal for age OR creatinine clearance > 60 mL/min
- SGPT < 5 times upper limit of normal (ULN)
- Bilirubin < 2.5 times ULN
- Absolute neutrophil count ≥ 1,500/mm^3*
- Hemoglobin ≥ 9.0 g/dL*
- Platelet count ≥ 100,000/mm^3*
- ECOG performance status 0-2
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception during and for 6 months after completion of study treatment NOTE: * Unsupported
PRIOR CONCURRENT THERAPY:
- No prior chemotherapy for MPNST
- Prior surgical resection of MPNST allowed provided residual or recurrent measurable disease is present
- Recovered from toxic effects of all prior therapy
- At least 3 weeks since prior chemotherapy or biologic therapy for treatment of a plexiform neurofibroma, optical pathway tumor, or other NF1-associated tumor (in patients with NF1)
- At least 6 weeks since prior radiotherapy for treatment of a plexiform neurofibroma, optical pathway tumor, or other NF1-associated tumor (in patients with NF1)
- At least 4 weeks since prior radiotherapy to the area involved by MPNST
- No other concurrent growth factors (e.g., sargramostim [GM-CSF] or interleukin-11)
- Concurrent epoetin alfa allowed
Data sourced from ClinicalTrials.gov (NCT00304083). 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.