Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

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

This study has been completed.
Sponsor:
Collaborator:
Information provided by:
National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier:
NCT00253474
First received: November 11, 2005
Last updated: March 28, 2012
Last verified: March 2012
  Purpose

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:

OBJECTIVES:

Primary

  • 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.

Secondary

  • 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.

  Eligibility

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

DISEASE CHARACTERISTICS:

  • 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

PATIENT CHARACTERISTICS:

Performance status

  • ECOG 0-2

Life expectancy

  • At least 12 months

Hematopoietic

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

Hepatic

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

Renal

  • 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

Cardiovascular

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

Pulmonary

  • No significant pulmonary dysfunction

Other

  • 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

PRIOR CONCURRENT THERAPY:

Biologic therapy

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

Chemotherapy

  • No concurrent chemotherapy for this disease

Endocrine therapy

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

Radiotherapy

  • No concurrent radiotherapy for this disease

Surgery

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

Other

  • Recovered from prior therapy
  • More than 30 days since prior investigational agents
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00253474

Locations
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
Investigators
Principal Investigator: Brigitte C. Widemann, MD National Cancer Institute (NCI)
  More Information

Additional Information:
Publications:
ClinicalTrials.gov 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:
Neoplasms
Neurofibroma
Neurofibroma, Plexiform
Neoplasms by Histologic Type
Neoplasms, Nerve Tissue
Nerve Sheath Neoplasms
Nervous System Diseases
Nervous System Neoplasms
Neuromuscular Diseases
Peripheral Nervous System Diseases
Peripheral Nervous System Neoplasms
Interferon-alpha
Interferons
Peginterferon alfa-2a
Anti-Infective Agents
Antineoplastic Agents
Antiviral Agents
Immunologic Factors
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on November 24, 2014