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Pirfenidone in Treating Young Patients With Neurofibromatosis Type I and Recurrent or Progressive Plexiform Neurofibromas

This study is ongoing, but not recruiting participants.
Study NCT00078936.   Last updated on October 12, 2008.   Information provided by National Cancer Institute (NCI)

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Descriptive Information Fields
Brief Title  Pirfenidone in Treating Young Patients With Neurofibromatosis Type I and Recurrent or Progressive Plexiform Neurofibromas
Official Title  Phase II Trial Of Pirfenidone In Children, Adolescents, And Young Adults With Neurofibromatosis Type I And Progressive Plexiform Neurofibromas
Brief Summary

RATIONALE: Some tumors need growth factors produced by the body's white blood cells to keep growing. Pirfenidone may interfere with growth factors and stop the tumor from growing.

PURPOSE: Phase II trial to study the effectiveness of pirfenidone in treating young patients who have neurofibromatosis type 1 and recurrent or progressive plexiform neurofibroma.

Detailed Description

OBJECTIVES:

Primary

  • Determine the time to disease progression in pediatric patients with neurofibromatosis type 1 (NF1) and recurrent or progressive plexiform neurofibroma treated with pirfenidone.
  • Determine the objective response rate in patients treated with this drug.
  • Determine the toxicity of this drug in these patients.

Secondary

  • Determine the quality of life of patients treated with this drug.

OUTLINE: This is an open-label, multicenter study.

Patients receive oral pirfenidone three times daily continuously for a course of 28 days. Courses repeat in the absence of disease progression or unacceptable toxicity.

For patients 6 to 18 years of age, quality of life is assessed at baseline, before courses 4 and 7, and then after every 6 courses.

PROJECTED ACCRUAL: A total of 36 patients will be accrued for this study within 12-14 months.

Study Phase Phase II
Study Type  Interventional
Study Design  Treatment, Open Label
Primary Outcome Measure  Time to disease progression [ Designated as safety issue: No ]
Objective response rate [ Designated as safety issue: No ]
Toxicity [ Designated as safety issue: Yes ]
Secondary Outcome Measure  Quality of life [ Designated as safety issue: No ]
Condition  Neurofibromatosis Type 1 (nf1)
Precancerous/Nonmalignant Condition
Intervention  Drug: pirfenidone
MEDLINE PMIDs
Links Clinical trial summary from the National Cancer Institute's PDQ® database This link exits the ClinicalTrials.gov site
Recruitment Information Fields
Recruitment Status  Active, not recruiting
Enrollment  36
Start Date  October 2005
Completion Date
Eligibility Criteria 

DISEASE CHARACTERISTICS:

  • All of the following:

    • Diagnosis of neurofibromatosis type 1 (NF1)
    • Histologically confirmed OR consistent clinical and radiographic findings of plexiform neurofibroma (defined as neurofibroma that has grown along the length of a nerve and may involve multiple fascicles and branches)

      • Recurrent disease (presence of new lesion) or progressive disease as documented on last 2 consecutive MRI or CT scans or within the past year by 1 of the following:

        • At least 20% increase in volume
        • At least 13% increase in the product of the 2 longest perpendicular diameters
        • At least 6% increase in the longest diameter
      • Measurable lesion at least 3 cm in 1 dimension
    • Meets 1 or more of the following other diagnostic criteria for NF1:

      • At least 6 cafe-au-lait spots

        • At least 0.5 cm in prepubertal patients
        • At least 1.5 cm in postpubertal patients
      • Freckling in the axilla or groin
      • Optic glioma
      • At least 2 Lisch nodules
      • One of the following distinctive bony lesions:

        • Dysplasia of the sphenoid bone
        • Dysplasia of the long bone cortex
        • Thinning of the long bone cortex
      • One first-degree relative with NF1
  • Ineligible for or refused complete resection of plexiform neurofibroma

    • Prior surgery for progressive disease allowed provided the plexiform neurofibroma was incompletely resected and is measurable
  • No evidence of malignant glioma or malignant peripheral nerve sheath tumor

PATIENT CHARACTERISTICS:

Age

  • 3 to 21

Performance status

  • Karnofsky 50-100% (over 10 years of age) OR
  • Lansky 50-100% (10 years of age and under)

Life expectancy

  • At least 12 months

Hematopoietic

  • Absolute granulocyte count ≥ 1,500/mm^3*
  • Hemoglobin ≥ 9 g/dL*
  • Platelet count ≥ 150,000/mm^3* NOTE: *Transfusion independent

Hepatic

  • Bilirubin normal (except for patients with Gilbert's syndrome)
  • SGPT ≤ 2 times upper limit of normal
  • No significant hepatic dysfunction

Renal

  • Creatinine normal OR
  • Creatinine clearance ≥ 70 mL/min

Cardiovascular

  • No significant cardiac dysfunction

Pulmonary

  • No significant pulmonary dysfunction

Other

  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception during and for 2 months after study treatment
  • Able to take pirfenidone by mouth
  • Able to undergo MRI
  • No clinically significant unrelated systemic illness that would preclude study participation
  • No serious infection
  • No other significant organ dysfunction
  • No other cancer requiring treatment with chemotherapy or radiotherapy

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • At least 1 week since prior filgrastim (G-CSF)
  • No prior pirfenidone
  • No concurrent immunotherapy
  • No concurrent biologic therapy (e.g., interferon)
  • No concurrent hematopoietic growth factors

Chemotherapy

  • At least 4 weeks since prior chemotherapy
  • No concurrent chemotherapy

Endocrine therapy

  • Concurrent corticosteroids allowed
  • 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 prior therapy (toxicity level less than grade 2)
  • More than 30 days since prior investigational agents
  • No other concurrent investigational agents
Gender Both
Ages 3 Years to 21 Years
Accepts Healthy Volunteers No
Contacts ††
Location Countries  United States
Administrative Information Fields
NCT ID  NCT00078936
Organization ID CDR0000353200
Secondary IDs †† NCI-04-C-0080
Study Sponsor  National Cancer Institute (NCI)
Collaborators ††
Investigators 
Principal Investigator:     Brigitte C. Widemann, MD     NCI - Pediatric Oncology Branch    
Information Provided By National Cancer Institute (NCI)
Verification Date October 2008
First Received Date  March 8, 2004
Last Updated Date October 12, 2008

 †    Required WHO trial registration data element.
††   WHO trial registration data element that is required only if it exists.




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