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
Sponsor:
Information provided by:
National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:
NCT00030264
First received: February 14, 2002
Last updated: November 2, 2010
Last verified: June 2009
  Purpose

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:

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.

  Eligibility

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

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00030264

Locations
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
Investigators
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
ClinicalTrials.gov 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:
Neurofibromatoses
Neurofibromatosis 1
Neurofibroma
Osteitis Fibrosa Cystica
Precancerous Conditions
Neurofibroma, Plexiform
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplasms
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Nervous System Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Bone Diseases, Endocrine
Bone Diseases
Musculoskeletal Diseases
Methotrexate
Vinblastine
Abortifacient Agents, Nonsteroidal
Abortifacient Agents
Reproductive Control Agents
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 23, 2014