PEG-Interferon Alfa-2b in Treating Young Patients With Plexiform Neurofibroma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00253474
Recruitment Status : Completed
First Posted : November 15, 2005
Last Update Posted : March 29, 2012
National Cancer Institute (NCI)
Information provided by:
National Institutes of Health Clinical Center (CC)

November 11, 2005
November 15, 2005
March 29, 2012
September 2005
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Complete list of historical versions of study NCT00253474 on Archive Site
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PEG-Interferon Alfa-2b in Treating Young Patients With Plexiform Neurofibroma
A Phase I Trial of Peginterferon Alfa-2b (PEG-Intron) for Plexiform Neurofibromas

RATIONALE: PEG-interferon alfa-2b may interfere with the growth of tumor cells.

PURPOSE: This phase I trial is studying the side effects and best dose of PEG-interferon alfa-2b in treating young patients with plexiform neurofibroma.



  • Determine the maximum tolerated dose of PEG-interferon alfa-2b in patients with unresectable plexiform neurofibroma. (Dose escalation portion of study closed to accrual as of 2/2005.)
  • Determine the toxicity profile of this drug in these patients.


  • Obtain, preliminary, information about the efficacy of this drug in these patients.
  • Evaluate the growth rate of plexiform neurofibroma using volumetric MRI analysis in patients treated with this drug.
  • Evaluate the impact of this drug, in terms of "worst symptom" score, in these patients.

OUTLINE: This is a dose-escalation, multicenter study. (Dose-escalation portion of the study closed to accrual as of 2/2005.)

Patients receive PEG-interferon alfa-2b subcutaneously once weekly for 2 years in the absence of disease progression or unacceptable toxicity.

Cohorts of 3-6 patients receive escalating doses of PEG-interferon alfa-2b until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 1 of 6 patients experience dose-limiting toxicity. A total of 12 patients receive treatment at the MTD.

After completion of study treatment, patients are followed every 6 months.

PROJECTED ACCRUAL: A total of 36 patients will be accrued for this study.

Phase 1
Primary Purpose: Treatment
  • Neoplasm of Uncertain Malignant Potential
  • Unspecified Childhood Solid Tumor, Protocol Specific
Biological: PEG-interferon alfa-2a
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Jakacki RI, Dombi E, Potter DM, Goldman S, Allen JC, Pollack IF, Widemann BC. Phase I trial of pegylated interferon-alpha-2b in young patients with plexiform neurofibromas. Neurology. 2011 Jan 18;76(3):265-72. doi: 10.1212/WNL.0b013e318207b031.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
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January 2011
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  • Diagnosis of plexiform neurofibroma which is progressive, symptomatic, or life threatening and for which there is no other standard medical management or surgical option
  • Histologic confirmation of tumor is not required in the presence of consistent clinical and radiographic findings provided the following are true:

    • No clinical observation or scan suggestive of malignant transformation
    • Meets ≥ 1 of the following diagnostic criteria for neurofibroma type 1 (NF1):

      • Six or more cafe-au-lait spots (> 0.5 cm in prepubertal patients or > 1.5 cm in post pubertal patients)
      • Freckling in axilla or groin
      • Optic glioma
      • Two or more Lisch nodules
      • A distinctive bony lesion (e.g., dysplasia of the sphenoid bone, dysplasia, or thinning of long bone cortex)
      • A first degree relative with NF1
  • No history of malignant peripheral nerve sheath tumor
  • No active visual pathway glioma
  • No active brain tumor or brain metastases


Performance status

  • ECOG 0-2

Life expectancy

  • At least 12 months


  • Absolute neutrophil count > 1,500/mm^3
  • Hemoglobin > 10 g/dL
  • Platelet count > 100,000/mm^3


  • Bilirubin < 1.5 mg/dL
  • SGPT ≤ 2 times upper limit of normal
  • No significant hepatic dysfunction


  • Creatinine based on age as follows:

    • ≤ 0.8 mg/dL (for patients age 5 years and under)
    • ≤ 1.0 mg/dL (for patients age 6 to 10 years)
    • ≤ 1.2 mg/dL (for patients age 11 to 15 years)
    • ≤ 1.5 mg/dL (for patients age 16 to 21 years) OR
  • Creatinine clearance ≥ 70 mL/min


  • No significant cardiac dysfunction
  • No severe cardiovascular disease
  • No cardiac arrhythmia requiring chronic treatment
  • No congestive heart failure
  • No symptomatic ischemic heart disease


  • No significant pulmonary dysfunction


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • No serious infection
  • No other significant unrelated systemic illness
  • No significant organ dysfunction
  • No other malignancy except surgically cured nonmelanoma skin cancer or carcinoma in situ of the cervix
  • No history of severe psychiatric condition or psychiatric disorder requiring hospitalization
  • No history of suicidal ideation or attempt
  • No thyroid dysfunction unresponsive to therapy
  • No uncontrolled diabetes mellitus
  • No history of HIV positivity
  • No alcohol or drug abuse


Biologic therapy

  • No concurrent immunotherapy
  • No concurrent colony-stimulating factors (e.g., erythropoietin or filgrastim [G-CSF])


  • No concurrent chemotherapy for this disease

Endocrine therapy

  • No concurrent chronic systemic corticosteroids
  • No concurrent hormonal therapy for this disease


  • No concurrent radiotherapy for this disease


  • Prior surgery allowed provided it has been at least 21 days since surgery and there is presence of residual tumor


  • Recovered from prior therapy
  • More than 30 days since prior investigational agents
Sexes Eligible for Study: All
1 Year to 21 Years   (Child, Adult)
Contact information is only displayed when the study is recruiting subjects
United States
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National Institutes of Health Clinical Center (CC)
National Cancer Institute (NCI)
Principal Investigator: Brigitte C. Widemann, MD National Cancer Institute (NCI)
National Institutes of Health Clinical Center (CC)
March 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP