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Vinblastine/Methotrexate For Severe Progressive Plexiform Neurofibromas: A Phase II Study
| City: |
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Philadelphia |
| State: |
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Pennsylvania |
| Zip Code: |
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19104 |
| Conditions: |
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Neurofibromatosis Type 1 - Precancerous Condition |
| Purpose: |
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RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing
so they stop growing or die. Combining methotrexate with vinblastine may be effective
treatment for neurofibromatosis type 1 associated with progressive plexiform neurofibromas.
PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating
patients who have neurofibromatosis type 1 associated with progressive plexiform
neurofibromas.
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| Study Summary: |
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OBJECTIVES:
- Determine the effect of chronic vinblastine and methotrexate on time to disease
progression in children or young adults with progressive plexiform neurofibroma
associated with neurofibromatosis type 1.
- Determine the objective response rate in patients treated with this regimen.
- Determine the toxic effects of this regimen in these patients.
- Determine the quality of life of patients treated with this regimen.
OUTLINE: Patients are stratified according to tumor status (severely debilitating and/or
life-threatening vs cosmetically disfiguring).
Patients receive methotrexate and vinblastine IV weekly for 26 weeks and then every 2 weeks
for 26 weeks in the absence of disease progression or unacceptable toxicity.
Quality of life is assessed at baseline and then every 3 months during study participation.
Patients are followed every 3 months until disease progression.
PROJECTED ACCRUAL: A total of 35 patients will be accrued for this study within
approximately 3 years.
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| Criteria: |
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DISEASE CHARACTERISTICS:
- Diagnosis of progressive, debilitating, severely disfiguring, or life-threatening
plexiform neurofibroma (PN) that is surgically unresectable (or surgery refused by
patient) and for which there is no other standard medical management
- Histologic confirmation of tumor not required in the presence of consistent
clinical and radiographic findings
- Tumor must be biopsied if any clinical observation or scan suggests
possible malignant transformation
- Measurable disease
- PN lesion that can be measured in at least 2 dimensions by direct physical
examination (clinical measurement and serial photography) or MRI
- Recurrent or progressive disease as documented by an increase in size or the presence
of new lesions on MRI
- Appearance of new tumors or a measurable increase in the sum of the product of
the two longest perpendicular diameters of the index lesion(s) over a time
period of no more than 12 months prior to study entry
- Must meet at least one other diagnostic criteria for neurofibromatosis type 1 (NF1):
- Six or more cafe-au-lait spots at least 0.5 cm in prepubertal patients or at
least 1.5 cm in postpubertal patients
- Freckling in the axilla or groin
- Optic glioma
- Two or more Lisch nodules
- Distinctive bony lesion (dysplasia of the sphenoid bone or dysplasia or thinning
of long bone cortex)
- First-degree relative with NF1
- Prior therapy for NF1 or PN is not required
PATIENT CHARACTERISTICS:
Age:
- 25 and under
Performance status:
- Lansky 60-100% OR
- Karnofsky 60-100%
Life expectancy:
- At least 12 months
Hematopoietic:
- CBC normal
- Absolute neutrophil count greater than 1,000/mm^3
- Platelet count greater than 100,000/mm^3
Hepatic:
- Bilirubin no greater than 1.5 times normal
- ALT/AST no greater than 1.5 times normal
Renal:
- BUN no greater than 1.5 times normal
- Creatinine no greater than 1.5 times normal
Other:
- Not pregnant or nursing
- Negative pregnancy test
PRIOR CONCURRENT THERAPY:
Biologic therapy:
- At least 1 week since prior filgrastim (G-CSF)
- No concurrent immunotherapy
Chemotherapy:
- At least 4 weeks since prior chemotherapy
- No other concurrent chemotherapy
Endocrine therapy:
- No concurrent hormonal therapy directed at the tumor
Radiotherapy:
- At least 6 weeks since prior radiotherapy
- No concurrent radiotherapy
Surgery:
- See Disease Characteristics
Other:
- Recovered from any prior therapy
- At least 30 days since prior investigational agents
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| NCT ID: |
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NCT00030264 |
| Primary Contact: |
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Study Chair
Jean B. Belasco, MD
Children's Hospital of Philadelphia
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| Backup Contact: |
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N/A |
| Location Contact: |
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Philadelphia, Pennsylvania 19104
United States
Jean B. Belasco, MD
Phone: 215-590-3129
Site Status: Recruiting |
| Data Source: |
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ClinicalTrials.gov |
| Date Processed: |
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May 25, 2013 |
| Modifications to this listing: |
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