Study of PEG-Intron for Plexiform Neurofibromas - Full Text View

Sponsors and Collaborators:	Children's Hospital of Pittsburgh National Cancer Institute (NCI)
Information provided by:	Children's Hospital of Pittsburgh
ClinicalTrials.gov Identifier:	NCT00396019

Purpose

This study is for slow growing tumors called plexiform neurofibromas (PNF) which are a relatively common problem in people with neurofibromatosis type 1 (NF1). These tumors are benign but as they grow, they can become disfiguring as well as disabling or even life threatening. They often cause pain, difficulty using arms or legs because of spinal cord compression, and/or nerve damage. At present, the only available therapy for plexiform neurofibromas is to try to surgically remove as much of the tumor as is possible. Because these tumors grow into the surrounding areas, total surgical resection is often impossible. Most tumors will re-grow after surgery if the entire tumor cannot be removed. To date, other treatments including chemotherapy and radiotherapy have not been able to shrink these tumors.

Interferon is a drug that is used for different types of tumors as well as for hepatitis. It has been used in the treatment of plexiform neurofibromas (PNF) with some subjects showing improvement in symptoms and/or a decrease in the size of the tumor. Most subjects had no further growth of their tumor while on the PEG-Intron. The drug used in this study is PEG (pegylated)-Intron. PEG-Intron is a long acting form of interferon which keeps the drug from being broken down in the body for a longer period of time and potentially could be more effective than the short-acting interferon. PEG-Intron has been approved by the Food and Drug Administration (FDA) for the treatment of Hepatitis C.

The goals of this study are:

To determine how your child's plexiform neurofibroma responds to PEG-Intron when given weekly.
To determine the side effects of PEG-Intron when given weekly to participants with plexiform neurofibromas.
To evaluate a new method of measuring changes in the size of tumors called volume analysis. This method measures the entire volume of a tumor in three dimensions. The standard method of measuring tumors uses only the length and/or width of the tumor. By studying the different ways of measuring tumors we hope to be able to determine which method is the most accurate and useful.

Condition	Intervention	Phase
Plexiform Neurofibroma	Drug: PEG-Intron	Phase II

MedlinePlus related topics: Cancer

Drug Information available for: Interferon alfa-2b Peginterferon Alfa-2b

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Historical Control, Single Group Assignment, Safety/Efficacy Study
Official Title:	A Phase II Trial of Peginterferon Alfa-2b (PEG-Intron) for Plexiform Neurofibromas

Further study details as provided by Children's Hospital of Pittsburgh:

Primary Outcome Measures:

The primary outcomes for the the study will be response or progression free survival, depending on the stratum in which the patient has enrolled. [ Time Frame: 5 years ] [ Designated as safety issue: No ]

Estimated Enrollment:	87
Study Start Date:	November 2006
Estimated Study Completion Date:	November 2011
Estimated Primary Completion Date:	November 2013 (Final data collection date for primary outcome measure)

Detailed Description:

PEG-Intron will be given every week through a small needle under the skin, in the same way that insulin is given to people with diabetes. Subjects will be taught to do this at home. As long as the tumor isn't growing and the side effects are tolerable, the injections will be given once a week for 2 years.

Children will be enrolled on the study into one of three strata (patient groups): strata 1 includes children who do not have any symptoms associated with their PNF and MRI scans over the past year may or may not show tumor growth, strata 2 includes children who have symptoms associated with the PNF but MRI scans over the past year have not shown growth and strata 3 includes children who have shown an increase in the size of the PNF on MRI scans over the past year, with or without any symptoms.

Eligibility

Ages Eligible for Study:	18 Months to 21 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Age > or equal to 18 months to 21 years
Diagnosis Patients with NF1 and progressive, symptomatic or life-threatening plexiform neurofibromas which are not surgically resectable. Patients without biopsy-proof of plexiform must have at least one other diagnostic criteria for NF1: 6 or more cafe-au-lait spots, freckling in the axilla or groin, optic glioma, 2 or more Lisch nodules, distinctive bony lesion, first degree relative with NF1.
Only eligible if complete tumor resection is not feasible, or if a patient with a surgical option refuses surgery.
Patients may be treated on this trial without having received prior therapy. If patients have received prior therapy, they must have recovered from all toxic effects prior to entering this study.
Must have life expectancy of at least 12 months and a performance score (Karnofsky or Lansky) of > or equal to 50.
Must have adequate liver, kidney and bone marrow function

Exclusion Criteria:

Clinically significant unrelated systemic illness.
Received an investigational agent within the past 30 days.
Evidence of active visual pathway glioma
History of malignant peripheral nerve sheath tumor or ther cancer other than surgically cured non-melanoma skin cancer or cervical carcinoma in situ
Ongoing radiation therapy, chemotherapy, hormonal therapy directed at the tumor, or immunotherapy.
Inability to return for follow-up visits or obtain follow-up studies
Severe cardiovascular disease
Pre-existing sever psychiatric condition or history of psychiatric disorder requiring hospitalization or a history of suicidal ideation or attempt.
Thyroid dysfunction not responsive to therapy.
Uncontrolled diabetes mellitus
History of seropositivity for HIV
Subjects who are pregnant, lactating, or of reproductive potential and not practicing an effective means of contraception.
Any medical condition requiring chronic systemic corticosteroids.
Subjects who are known to be actively abusing alcohol or drugs.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00396019

Locations

United States, Pennsylvania
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213

Sponsors and Collaborators

Children's Hospital of Pittsburgh

National Cancer Institute (NCI)

Investigators

Study Chair:

Regina Jakacki, M.D.

Children's Hospital of Pittsburgh

More Information

Responsible Party:	Children's Hospital of Pittsburgh ( Regina Jakacki, MD )
Study ID Numbers:	UPCI 06-116
Study First Received:	November 2, 2006
Last Updated:	January 3, 2008
ClinicalTrials.gov Identifier:	NCT00396019
Health Authority:	United States: Institutional Review Board

Study placed in the following topic categories:

Neuromuscular Diseases
Neurofibroma
Peripheral Nervous System Diseases
Peginterferon alfa-2b
Peripheral Nervous System Neoplasms

Neurofibroma, Plexiform
Interferon Alfa-2b
Nervous System Neoplasms
Nerve Sheath Neoplasms

Additional relevant MeSH terms:

Anti-Infective Agents
Neoplasms
Neoplasms by Histologic Type
Therapeutic Uses

Nervous System Diseases
Neoplasms, Nerve Tissue
Antiviral Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 14, 2009