Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas

The recruitment status of this study is unknown because the information has not been verified recently.
Verified June 2009 by National Cancer Institute (NCI).
Recruitment status was  Recruiting
Information provided by:
National Cancer Institute (NCI) Identifier:
First received: February 14, 2002
Last updated: November 2, 2010
Last verified: June 2009

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.

Condition Intervention Phase
Neurofibromatosis Type 1
Precancerous Condition
Drug: methotrexate
Drug: vinblastine sulfate
Phase 2

Study Type: Interventional
Study Design: Masking: Open Label
Primary Purpose: Treatment
Official Title: Vinblastine/Methotrexate For Severe Progressive Plexiform Neurofibromas: A Phase II Study

Resource links provided by NLM:

Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:
  • Time to disease progression after 6 months [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Objective response rate [ Designated as safety issue: No ]
  • Toxicity [ Designated as safety issue: Yes ]
  • Quality of life parameters as measured by standard, validated, age-calibrated performance, pain, and mood scales [ Designated as safety issue: No ]
  • Perception of treatment impact on patient self-identified worst symptoms as measured by numeric assessment tools [ Designated as safety issue: No ]

Estimated Enrollment: 35
Study Start Date: February 2001
Estimated Primary Completion Date: December 2011 (Final data collection date for primary outcome measure)
Detailed Description:


  • 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.


Ages Eligible for Study:   up to 25 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00030264

United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Jean B. Belasco, MD    215-590-3129      
Sponsors and Collaborators
Children's Hospital of Philadelphia
Study Chair: Jean B. Belasco, MD Children's Hospital of Philadelphia
  More Information

Additional Information:
No publications provided

Responsible Party: Jean Bello Belasco, Children's Hospital of Philadelphia Identifier: NCT00030264     History of Changes
Other Study ID Numbers: CDR0000069065, CHP-686, CHP-IRB-2001-2-2339, NCI-V01-1678
Study First Received: February 14, 2002
Last Updated: November 2, 2010
Health Authority: Unspecified

Keywords provided by National Cancer Institute (NCI):
plexiform neurofibroma
neurofibromatosis type 1

Additional relevant MeSH terms:
Neurofibromatosis 1
Nerve Sheath Neoplasms
Neurofibroma, Plexiform
Precancerous Conditions
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Neoplasms by Histologic Type
Neoplasms, Nerve Tissue
Neoplastic Syndromes, Hereditary
Nervous System Diseases
Nervous System Neoplasms
Neurocutaneous Syndromes
Neurodegenerative Diseases
Neuromuscular Diseases
Peripheral Nervous System Diseases
Peripheral Nervous System Neoplasms
Abortifacient Agents
Abortifacient Agents, Nonsteroidal
Antimetabolites, Antineoplastic
Antimitotic Agents
Antineoplastic Agents
Antineoplastic Agents, Phytogenic
Antirheumatic Agents
Dermatologic Agents processed this record on August 31, 2015