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

This study has been completed.
Information provided by:
National Institutes of Health Clinical Center (CC) Identifier:
First received: November 11, 2005
Last updated: March 28, 2012
Last verified: March 2012

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.

Condition Intervention Phase
Neoplasm of Uncertain Malignant Potential
Unspecified Childhood Solid Tumor, Protocol Specific
Biological: PEG-interferon alfa-2a
Phase 1

Study Type: Interventional
Study Design: Primary Purpose: Treatment
Official Title: A Phase I Trial of Peginterferon Alfa-2b (PEG-Intron) for Plexiform Neurofibromas

Resource links provided by NLM:

Further study details as provided by National Institutes of Health Clinical Center (CC):

Estimated Enrollment: 36
Study Start Date: September 2005
Study Completion Date: January 2011
Detailed Description:



  • 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.


Ages Eligible for Study:   1 Year to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • 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
  Contacts and Locations
Please refer to this study by its identifier: NCT00253474

United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010-2970
United States, Illinois
Children's Memorial Hospital - Chicago
Chicago, Illinois, United States, 60614
United States, Maryland
Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support
Bethesda, Maryland, United States, 20892-1182
United States, Pennsylvania
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213
Sponsors and Collaborators
Principal Investigator: Brigitte C. Widemann, MD National Cancer Institute (NCI)
  More Information

Additional Information:
Publications: Identifier: NCT00253474     History of Changes
Obsolete Identifiers: NCT00156754
Other Study ID Numbers: 050232, 05-C-0232, NCI-P6670, CDR0000448811
Study First Received: November 11, 2005
Last Updated: March 28, 2012
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
neoplasm of uncertain malignant potential
unspecified childhood solid tumor, protocol specific

Additional relevant MeSH terms:
Neurofibroma, Plexiform
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Nervous System Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Interferon Alfa-2a
Peginterferon alfa-2a
Peginterferon alfa-2b
Antiviral Agents
Anti-Infective Agents
Therapeutic Uses
Pharmacologic Actions
Immunologic Factors
Physiological Effects of Drugs
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors
Antineoplastic Agents processed this record on April 22, 2014