A Phase 2 interventional study of Pegylated interferon alpha-2b in Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas, sponsored by Emory University. Completed at 1 site in United States. Open to participants aged 3 Years to 25 Years. Per ClinicalTrials.gov, last updated 2022-01-24.
Sponsored by Emory University · Phase 2, Interventional, and Treatment
This is a phase II study of the drug, pegylated interferon alfa-2b (PEG-Intron), used to treat brain tumors in a pediatric population. Researchers want to see if treatment with PEG-Intron will stop tumor growth for patients with juvenile pilocytic astrocytomas or optic pathway gliomas.
The purposes of this study are:
Low grade gliomas are the most common pediatric central nervous system malignancies and can occur in different parts of the brain. Patients who undergo gross total resection, usually those with hemispheric tumors, have an excellent prognosis with surgical resection alone. Patients for whom gross total resection is not achievable have a significant risk of disease progression. Therefore, these patients benefit from adjuvant therapy. Multiple chemotherapy regimens have shown some efficacy in residual tumor, but more than 50% of patients experience recurrences. Radiation has been shown to be an effective therapy in the treatment of these tumors. Because of concerns regarding radiation toxicity especially in young children, and progression despite chemotherapy, novel approaches are needed. This protocol represents an attempt to measure the efficacy and safety of use of pegylated interferon for patients with recurrent, refractory Juvenile Pilocytic Astrocytomas (JPA) or optic pathway gliomas. It provides a different approach to the commonly used treatment modalities. The objectives of this study are to determine the response of children with chemotherapy-refractory progressive JPA or optic pathway gliomas (OPG) to weekly pegylated interferon alpha-2b. The secondary objectives include to better identify the toxicities of weekly pegylated interferon alpha-2b (PEG-Intron™) in pediatric patients with unresectable, refractory, recurrent JPAs or optic pathway gliomas, to evaluate various magnetic resonance imaging techniques for noninvasive monitoring of metabolic and biologic changes in the tumors and to evaluate the quality of life for patients with recurrent, refractory JPAs who receive therapy with pegylated interferon alpha-2b (PEG-Intron™).
The primary end point is to determine the response rate. A two-stage design has been selected to evaluate the response rate. If the treatment demonstrates at least a 25% response rate, the researchers would consider it a promising regimen for further study. A response rate less than 5% is considered evidence of unpromising regimen. Seventeen evaluable pediatric patients with JPA or OPG will be accrued. If at least 3 responders are seen among the 17 patients, this will be considered evidence of a promising response rate for further evaluation.
1,397 studies on the registry are indexed under Glioma; 351 are open to participants now.
This study's enrollment of 9 is below the median of 32 across 1,065 interventional studies indexed under Glioma.
Browse Glioma studies →Emory University is the lead sponsor of 1,386 studies on the registry; 236 are open to participants now.
Of its 229 completed or terminated interventional studies of FDA-regulated products, 174 (76%) have results posted.
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Patients must have recovered (to Common Toxicity Criteria (CTC) v.5.0 ≤ Grade 1 unless indicated below) from the acute toxic effects of all prior chemotherapy, immunotherapy prior to entering this study, with the exception of alopecia, weight changes and Grade I or II lymphopenia
Exclusion Criteria:
Subjects will receive PEG-Intron based on their weight (1 mcg/kg/dose) once a week
Drug: Pegylated interferon alpha-2b
PEG-Intron 1mcg/kg/dose weekly through an injection under the skin on the same day each week
Also known as: Peg-Intron
Number of Children Responding to Treatment
The objective response of study participants was categorized as complete response, partial response, or stable disease. Determination of tumor response or progression is based on the pre-study baseline scan performed closest to time of study entry. The overall response assessment takes into account response in both target and non-target lesion, and the appearance of new lesions. Complete response is defined as the disappearance of all target lesions and non-target lesions, and no new lesions. Partial response is defined as ≥ 65% decrease in the sum of the products of the three perpendicular diameters of all target lesions, and no development of new lesions. Stable disease is defined as neither sufficient decrease in the sum of the products of the three perpendicular diameters of all target lesions to qualify for partial response, nor sufficient increase in a single target lesion to qualify for progressive disease, as well as no new lesions.
Time frame: Month 12
Number of Participants Meeting Event Free Survival Criteria
Event free survival is the time to treatment failure from the time of study enrollment to tumor progression, tumor recurrence, death from any cause or occurrence of a second malignant neoplasm.
Time frame: Month 12, Month 24
Number of Participants Meeting Overall Survival Criteria
Overall survival is measured as the time from study enrollment to death from any cause.
Time frame: Month 12, Month 24
Participants were enrolled at Children's Healthcare of Atlanta, in Atlanta, Georgia. Enrollment began November 2014 and all follow up was complete by November 11, 2020.
| Milestone | PEGINTRON |
|---|---|
| Started | 9 |
| Completed | 0 |
| Not completed | 9 |
The objective response of study participants was categorized as complete response, partial response, or stable disease. Determination of tumor response or progression is based on the pre-study baseline scan performed closest to time of study entry. The overall response assessment takes into account response in both target and non-target lesion, and the appearance of new lesions. Complete response is defined as the disappearance of all target lesions and non-target lesions, and no new lesions. Partial response is defined as ≥ 65% decrease in the sum of the products of the three perpendicular diameters of all target lesions, and no development of new lesions. Stable disease is defined as neither sufficient decrease in the sum of the products of the three perpendicular diameters of all target lesions to qualify for partial response, nor sufficient increase in a single target lesion to qualify for progressive disease, as well as no new lesions.
| Participants | PEGINTRON |
|---|---|
| Complete Response | 0 |
| Partial Response | 0 |
| Stable Disease | 5 |
Event free survival is the time to treatment failure from the time of study enrollment to tumor progression, tumor recurrence, death from any cause or occurrence of a second malignant neoplasm.
| Participants | PEGINTRON |
|---|---|
| Month 12 | 7 |
| Month 24 | 7 |
Overall survival is measured as the time from study enrollment to death from any cause.
| Participants | PEGINTRON |
|---|---|
| Month 12 | 7 |
| Month 24 | 7 |
Collected over Data collection for adverse events began at the Baseline (Day 0) study visit and continued through the time when the participant was removed from the study intervention (up to 2 years).. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| PEGINTRON | 2/9 (22.2%) | 1/9 (11.1%) | 9/9 (100%) |
| Event | PEGINTRON |
|---|---|
| Anorexia, grade 3General disorders | 1/9 |
| Event | PEGINTRON |
|---|---|
| Hypocalcemia, grade 1Blood and lymphatic system disorders | 6/9 |
| Decreased white blood cell count, grade 1Blood and lymphatic system disorders | 6/9 |
| Increased alanine transaminase (ALT), grade 1Hepatobiliary disorders | 5/9 |
| Anemia, grade 1Blood and lymphatic system disorders | 4/9 |
| Hypoalbuminemia, grade 1Blood and lymphatic system disorders | 4/9 |
| Decreased lymphocyte count, grade 2Blood and lymphatic system disorders | 4/9 |
| Headache, grade 1General disorders | 3/9 |
| Hyperuricemia, grade 1Blood and lymphatic system disorders | 3/9 |
| Hypokalemia, grade 1Blood and lymphatic system disorders | 3/9 |
| Hypophosphatemia, grade 1Blood and lymphatic system disorders | 3/9 |
| Age, Categorical(Participants) | PEGINTRON |
|---|---|
| <=18 years | 6 |
| Between 18 and 65 years | 3 |
| >=65 years | 0 |
| Age, Continuous(years) | PEGINTRON |
|---|---|
| Mean | 11 (5 to 21) |
| Sex: Female, Male(Participants) | PEGINTRON |
|---|---|
| Female | 4 |
| Male | 5 |
| Ethnicity (NIH/OMB)(Participants) | PEGINTRON |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 9 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | PEGINTRON |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 3 |
| White | 6 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(Participants) | PEGINTRON |
|---|---|
| United States | 9 |
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