Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas
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|ClinicalTrials.gov Identifier: NCT00030264|
Recruitment Status : Completed
First Posted : January 27, 2003
Results First Posted : August 8, 2018
Last Update Posted : August 8, 2018
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 or disease||Intervention/treatment||Phase|
|Neurofibromatosis Type 1 Precancerous Condition||Drug: Methotrexate Drug: Vinblastine||Phase 2|
- 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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||23 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Vinblastine/Methotrexate For Severe Progressive Plexiform Neurofibromas: A Phase II Study|
|Study Start Date :||February 2001|
|Actual Primary Completion Date :||December 2013|
|Actual Study Completion Date :||March 2016|
Experimental: Methotrexate & Vinblastine
Methotrexate and Vinblastine will be given once a week for the first 26 weeks and then every two weeks for the next 26 weeks or until disease progression (whichever occurs first).
Methotrexate will be given at a dose of 30mg/m2/week intramuscular (IM) or intravenous (IV) for the first 26 weeks, then every 2 weeks for the next 26 weeks or until disease progression (whichever occurs first)
Vinblastine will be given at a dose of 6mg/m2/week intravenous (IV) for for the first 26 weeks, then every 2 weeks for the next 26 weeks or until disease progression (whichever occurs first). Maximum actual dose may not exceed 10mg.
- Time to Disease Progression [ Time Frame: 6 months ]
Disease progression was assessed both radiographically and clinically.
Tumor assessments to assess for radiographic disease progression were assessed by magnetic resonance imaging (MRI) measurement whenever possible or computed tomography (CT) scan and/or tumor measurement during physical examination of palpable lesions. Clinical assessments for clinical progression of disease were assessed by treating physician or designee.
Progressive disease as measured by the appearance of new lesions; an increased size of index tumor(s) by >/= 25% of the sum of the products of baseline measurements; and/or by increase in symptoms.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00030264
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|Principal Investigator:||Jean B. Belasco, MD||Children's Hospital of Philadelphia|