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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
Sponsor:
Information provided by (Responsible Party):
Children's Hospital of Philadelphia

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Neurofibromatosis Type 1
Precancerous Condition
Interventions Drug: Methotrexate
Drug: Vinblastine
Enrollment 23
Recruitment Details 23 eligible subjects were enrolled at the Children's Hospital of Philadelphia and Texas Children's Hospital between March 2001 and June 2011
Pre-assignment Details  
Arm/Group Title Methotrexate & Vinblastine
Hide Arm/Group Description

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: 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: 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.

Period Title: Overall Study
Started 23
Completed 23
Not Completed 0
Arm/Group Title Methotrexate & Vinblastine
Hide Arm/Group Description

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: 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: 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.

Overall Number of Baseline Participants 23
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 23 participants
<=18 years
19
  82.6%
Between 18 and 65 years
4
  17.4%
>=65 years
0
   0.0%
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 23 participants
5.56
(0.34 to 23.99)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 23 participants
Female
13
  56.5%
Male
10
  43.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 23 participants
Hispanic or Latino
1
   4.3%
Not Hispanic or Latino
13
  56.5%
Unknown or Not Reported
9
  39.1%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 23 participants
American Indian or Alaska Native
0
   0.0%
Asian
2
   8.7%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
2
   8.7%
White
17
  73.9%
More than one race
0
   0.0%
Unknown or Not Reported
2
   8.7%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 23 participants
23
 100.0%
1.Primary Outcome
Title Time to Disease Progression
Hide Description

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.

Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
20 subjects were analyzed for time to progression. 3 subjects were not included in the response analysis due to lost to follow up (N=2) or progression of comorbid condition which required early termination (N=1)
Arm/Group Title Methotrexate & Vinblastine
Hide Arm/Group Description:

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: 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: 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.

Overall Number of Participants Analyzed 20
Mean (Full Range)
Unit of Measure: Months
Total Number Analyzed 20 participants
25.8
(0.2 to 85.0)
Radiographic Progression Number Analyzed 6 participants
29.2
(3.1 to 58.5)
Clinical Progression Number Analyzed 14 participants
25.8
(0.2 to 85.0)
Time Frame [Not Specified]
Adverse Event Reporting Description For non-serious events, CTCAE Grade 3 and 4 were collected.
 
Arm/Group Title Methotrexate & Vinblastine
Hide Arm/Group Description

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: 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: 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.

All-Cause Mortality
Methotrexate & Vinblastine
Affected / at Risk (%)
Total   3/23 (13.04%)    
Hide Serious Adverse Events
Methotrexate & Vinblastine
Affected / at Risk (%) # Events
Total   6/23 (26.09%)    
Gastrointestinal disorders   
Anorexia  1/23 (4.35%)  1
Hepatobiliary disorders   
Hepatic - Other - Cholecytitis 1 [1]  1/23 (4.35%)  1
Infections and infestations   
Infection - Bacterial 1 [2]  1/23 (4.35%)  1
Infection - Viral 1 [3]  1/23 (4.35%)  1
Infection 1 [4]  1/23 (4.35%)  1
Investigations   
Febrile neutropenia 1 [5]  1/23 (4.35%)  1
Fever 1 [6]  1/23 (4.35%)  1
Fever 1 [7]  1/23 (4.35%)  1
Respiratory, thoracic and mediastinal disorders   
Pulmonary - Other - Respiratory Distress  1/23 (4.35%)  1
1
Term from vocabulary, CTCAE (2.0)
[1]
Acute cholecystitis with fever, nausea and vomiting, elevated liver tests
[2]
Fever, otitis externa, otitis media, cellulitis
[3]
Parainfluenza with grade 4 ANC
[4]
Chicken Pox
[5]
fever with neutropenia
[6]
Fever with upper respiratory infection symptoms
[7]
Fever NOS
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Methotrexate & Vinblastine
Affected / at Risk (%) # Events
Total   8/23 (34.78%)    
Blood and lymphatic system disorders   
Neutrophils/granulocytes (ANC/AGC)  8/23 (34.78%)  15
Leukocytes (total WBC)  2/23 (8.70%)  3
Lymphopenia  1/23 (4.35%)  1
Infections and infestations   
Infection - Viral 1 [1]  1/23 (4.35%)  1
Investigations   
SGPT (ALT) (serum glutamic pyruvic transaminase) 1 [2]  1/23 (4.35%)  1
1
Term from vocabulary, CTCAE (2.0)
[1]
Parainfluenza
[2]
ALT increased
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Ratnakar Patti
Organization: Children's Hospital of Philadelphia
Phone: 267-426-5503
Layout table for additonal information
Responsible Party: Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT00030264    
Other Study ID Numbers: 07-2339
CHP-686 ( Other Identifier: Children's Hospital of Philadelphia )
First Submitted: February 14, 2002
First Posted: January 27, 2003
Results First Submitted: April 9, 2018
Results First Posted: August 8, 2018
Last Update Posted: August 8, 2018