A Phase II Trial of Peginterferon Alfa-2b (PEG-Intron) for Plexiform Neurofibromas
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.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
The primary outcomes for the the study will be response or progression free survival, depending on the stratum in which the patient has enrolled.
Regina Jakacki, M.D.
Children's Hospital of Pittsburgh
United States: Institutional Review Board
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