Protocol Number: 05-C-0232

Title:

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

Number:

05-C-0232

Summary:

This study will examine whether a drug called peginterferon alfa-2b (PEG Intron) is useful in treating plexiform neurofibromas. It will determine: 1) the optimal dose of PEG Intron for children and young adults with plexiform neurofibromas; 2) the side effects the drug; and 3) whether the drug shows any indication of being helpful in treating this disease.

Plexiform neurofibromas are tumors involving a nerve and branches of the nerve. Most plexiform neurofibromas develop in people who have neurofibromatosis type I (NF1). The only known treatment for the tumors for is surgery, but often complete surgical removal of all the tumors is not possible. PEG Intron is a long-acting form of interferon-alfa-2b, a drug that is approved to treat certain diseases, such as chronic hepatitis C. PEG Intron may stop or reduce the growth of plexiform neurofibromas by blocking new blood vessel formation in the tumors.

Patients between 18 months and 21 years of age who have progressive, symptomatic, or life-threatening plexiform neurofibromas that cannot be surgically removed may be eligible for this study. Candidates are screened with a physical examination, blood and urine tests and magnetic resonance imaging (MRI) to evaluate the tumor. MRI uses a magnet and radio waves to produce detailed pictures of tissues. The patient lies on a table in a narrow cylinder containing a magnetic field, wearing ear plugs to muffle loud noises that occur with electrical switching of the magnetic fields. He or she can speak with a staff member via an intercom system at all times during the procedure.

Participants undergo the following tests and procedures:

-PEG Intron treatment: Patients receive injections of PEG Intron under the skin once a week for up to 2 years. Patients are taught to self-administer the drug, or they may choose to have a physician give the injections. Patients record each dose and any drug side effects they may experience. They take acetaminophen before starting PEG Intron, and as necessary afterwards, to prevent known side effects of the drug, such as fever and flu-like symptoms.

-Blood tests: Done every 2 weeks for the first 8 weeks, and thereafter every 3 to 6 months.

-Physical examinations: Patients have a physical examination before starting PEG Intron treatment, after 4 and 8 weeks of treatment, and then after every 3 to 6 months of treatment.

-MRI scanning: This test is done to monitor the size of the patient's plexiform neurofibroma over the course of the study. MRI is done before starting treatment with PEG Intron and is repeated after 3, 7, and 12 months of treatment and then after every 6 months.

-Worst symptom ratings: At the time of every MRI evaluation, patients report their worst symptom (what makes them feel worst) as a result of their tumors.

Sponsoring Institute:

National Cancer Institute (NCI)

Recruitment Detail

Type: Completed Study; data analyses ongoing

Gender: Male & Female

Referral Letter Required: No

Population Exclusion(s): None

Eligibility Criteria: This study is not currently recruiting new subjects. If you have questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.

Special Instructions:

Currently Not Provided

Keyword(s):

Neurofibromatosis Type 1

MRI

Toxicities

Recruitment Keyword(s):

Plexiform Neurofibroma

PN

Neurofibromatosis Type 1

NF1

Condition(s):

Plexiform neurofibromas

Investigational Drug(s):

None

Investigational Device(s):

None

Interventions:

Drug: Peginterferon Alfa-2b (PEG-Intron)

Supporting Site:

National Cancer Institute

Contact(s):

This study is not currently recruiting new subjects. If you have questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.

Citation(s):

Goldberg Y, Dibbern K, Klein J, Riccardi VM, Graham JM Jr. Neurofibromatosis type 1--an update and review for the primary pediatrician. Clin Pediatr (Phila). 1996 Nov;35(11):545-61. Review.

Hajdu SI. Peripheral nerve sheath tumors. Histogenesis, classification, and prognosis. Cancer. 1993 Dec 15;72(12):3549-52. No abstract available.

Huson SM, Harper PS, Compston DA. Von Recklinghausen neurofibromatosis. A clinical and population study in south-east Wales. Brain. 1988 Dec;111 ( Pt 6):1355-81.

• Questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.
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