Study of PEG-Intron for Plexiform Neurofibromas
Condition: Plexiform Neurofibroma
MeSH terms: Neurofibroma; Neurofibromatoses; Neurofibroma, Plexiform
Intervention: PEG-Intron
Intervention MeSH terms: Peginterferon alfa-2b
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 86.0
Sex: All
Minimum age: 18 Months
Maximum age: 21 Years
Healthy volunteers: No

Summary:

      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:

        1. To determine how your child's plexiform neurofibroma responds to PEG-Intron when given
           weekly.

        2. To determine the side effects of PEG-Intron when given weekly to participants with
           plexiform neurofibromas.

        3. 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 the investigators hope to be able to
           determine which method is the most accurate and useful.
    

Eligibility 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.