PEG-interferon alfa-2a in Unspecified Childhood Solid Tumor, Protocol Specific and Neoplasm of Uncertain Malignant Potential - Clinical Trials Registry

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Biological: PEG-interferon alfa-2a

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.

Study Type

Interventional

Enrollment (Anticipated)

36

Phase

Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Primary Purpose: Treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Institutes of Health Clinical Center (CC)

Collaborators

National Cancer Institute (NCI)

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2005

Study Completion (Actual)

January 1, 2011

Study Registration Dates

First Submitted

November 11, 2005

First Submitted That Met QC Criteria