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A Phase I Trial of Peginterferon Alfa-2b (PEG-Intron) for Plexiform Neurofibromas

Phase 1
1 Year
21 Years
Not Enrolling
Neoplasm of Uncertain Malignant Potential, Unspecified Childhood Solid Tumor, Protocol Specific

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Trial Information

A Phase I Trial of Peginterferon Alfa-2b (PEG-Intron) for Plexiform Neurofibromas



- 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.

Inclusion Criteria


- Diagnosis of plexiform neurofibroma which is progressive, symptomatic, or life
threatening and for which there is no other standard medical management or surgical

- 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

- 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

Type of Study:


Study Design:

Primary Purpose: Treatment

Principal Investigator

Brigitte C. Widemann, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

National Cancer Institute (NCI)


United States: Federal Government

Study ID:




Start Date:

September 2005

Completion Date:

January 2011

Related Keywords:

  • Neoplasm of Uncertain Malignant Potential
  • Unspecified Childhood Solid Tumor, Protocol Specific
  • neoplasm of uncertain malignant potential
  • unspecified childhood solid tumor, protocol specific
  • Neoplasms
  • Neurofibroma
  • Neurofibroma, Plexiform



Children's National Medical Center Washington, District of Columbia  20010-2970
Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania  15213
Children's Memorial Hospital - Chicago Chicago, Illinois  60614
Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support Bethesda, Maryland  20892-1182