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Vinblastine/Methotrexate For Severe Progressive Plexiform Neurofibromas: A Phase II Study

Phase 2
25 Years
Open (Enrolling)
Neurofibromatosis Type 1, Precancerous Condition

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Trial Information

Vinblastine/Methotrexate For Severe Progressive Plexiform Neurofibromas: A Phase II Study


- 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.

Inclusion Criteria


- 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



- 25 and under

Performance status:

- Lansky 60-100% OR

- Karnofsky 60-100%

Life expectancy:

- At least 12 months


- CBC normal

- Absolute neutrophil count greater than 1,000/mm^3

- Platelet count greater than 100,000/mm^3


- Bilirubin no greater than 1.5 times normal

- ALT/AST no greater than 1.5 times normal


- BUN no greater than 1.5 times normal

- Creatinine no greater than 1.5 times normal


- Not pregnant or nursing

- Negative pregnancy test


Biologic therapy:

- At least 1 week since prior filgrastim (G-CSF)

- No concurrent immunotherapy


- At least 4 weeks since prior chemotherapy

- No other concurrent chemotherapy

Endocrine therapy:

- No concurrent hormonal therapy directed at the tumor


- At least 6 weeks since prior radiotherapy

- No concurrent radiotherapy


- See Disease Characteristics


- Recovered from any prior therapy

- At least 30 days since prior investigational agents

Type of Study:


Study Design:

Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Time to disease progression after 6 months

Safety Issue:


Principal Investigator

Jean B. Belasco, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Children's Hospital of Philadelphia



Study ID:




Start Date:

February 2001

Completion Date:

Related Keywords:

  • Neurofibromatosis Type 1
  • Precancerous Condition
  • plexiform neurofibroma
  • neurofibromatosis type 1
  • Neurofibroma
  • Neurofibromatoses
  • Neurofibromatosis 1
  • Osteitis Fibrosa Cystica
  • Precancerous Conditions
  • Neurofibroma, Plexiform



Children's Hospital of Philadelphia Philadelphia, Pennsylvania  19104