Alternate Title
Basic Trial Information
Objectives
Entry Criteria
Expected Enrollment
Outcomes
Outline
Trial Contact Information
Registry Information

Alternate Title

Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas

Basic Trial Information

Phase	Type	Status	Age	Sponsor	Protocol IDs
Phase II	Treatment	Active	25 and under	Other	CHP-686 CHP-IRB-2001-2-2339, NCI-V01-1678, NCT00030264

Objectives

1. 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.
2. Determine the objective response rate in patients treated with this regimen.
3. Determine the toxic effects of this regimen in these patients.
4. Determine the quality of life of patients treated with this regimen.

Entry Criteria

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

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

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/mm³
  • Platelet count greater than 100,000/mm³

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

Expected Enrollment

A total of 35 patients will be accrued for this study within approximately 3 years.

Outcomes

Time to disease progression after 6 months

Objective response rate
Toxicity
Quality of life parameters as measured by standard, validated, age-calibrated performance, pain, and mood scales
Perception of treatment impact on patient self-identified worst symptoms as measured by numeric assessment tools

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.

Trial Contact Information

Trial Lead Organizations

Children's Hospital of Philadelphia

Jean Belasco, MD, Protocol chair		Ph: 215-590-2800

U.S.A.
Pennsylvania
	Philadelphia
	Children's Hospital of Philadelphia
		Jean Belasco, MD	Ph:  215-590-3129

Registry Information
Official Title		Vinblastine/Methotrexate For Severe Progressive Plexiform Neurofibromas: A Phase II Study
Trial Start Date		2001-02-28
Trial Completion Date		2011-12-01 (estimated)
Registered in ClinicalTrials.gov		NCT00030264
Date Submitted to PDQ		2001-10-02
Information Last Verified		2009-06-28

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