A Phase II Trial of Peginterferon Alfa-2b (PEG-Intron) for Plexiform Neurofibromas
18 Months
21 Years
Both
Plexiform Neurofibroma
Trial Information
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.
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.
Type of Study:
Interventional
Study Design:
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
The primary outcomes for the the study will be response or progression free survival, depending on the stratum in which the patient has enrolled.
Outcome Time Frame:
5 years
Safety Issue:
No
Principal Investigator
Regina Jakacki, M.D.
Investigator Role:
Study Chair
Investigator Affiliation:
Children's Hospital of Pittsburgh
Authority:
United States: Institutional Review Board
Study ID:
UPCI 06-116
NCT ID:
NCT00396019
Start Date:
November 2006
Completion Date:
November 2013
Related Keywords:
- Plexiform Neurofibroma
- Neurofibroma
- Neurofibroma, Plexiform
Name | Location |
|---|---|
| Children's Hospital of Pittsburgh | Pittsburgh, Pennsylvania 15213 |
