Trial Outcomes & Findings for Pegylated Interferon ALFA-2b in Children With Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas (NCT NCT02343224)

Last Updated: 2022-01-24

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

Month 12

Results posted on

2022-01-24

Participant Flow

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.

Participant milestones

Participant milestones
Measure	PEGINTRON Participants with juvenile pilocytic astrocytomas or optic pathway gliomas receiving PEGINTRON based on their weight (1 mcg/kg/dose) once a week, for up to two years
Overall Study STARTED	9
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	9

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pegylated Interferon ALFA-2b in Children With Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	PEGINTRON n=9 Participants Participants with juvenile pilocytic astrocytomas or optic pathway gliomas receiving PEGINTRON based on their weight (1 mcg/kg/dose) once a week, for up to two years
Age, Categorical <=18 years	6 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=99 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants
Age, Continuous	11 years n=99 Participants
Sex: Female, Male Female	4 Participants n=99 Participants
Sex: Female, Male Male	5 Participants n=99 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=99 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=99 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	3 Participants n=99 Participants
Race (NIH/OMB) White	6 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Region of Enrollment United States	9 Participants n=99 Participants

PRIMARY outcome

Timeframe: Month 12

Outcome measures

Outcome measures
Measure	PEGINTRON n=9 Participants Participants with juvenile pilocytic astrocytomas or optic pathway gliomas receiving PEGINTRON based on their weight (1 mcg/kg/dose) once a week, for up to two years
Number of Children Responding to Treatment Complete Response	0 Participants
Number of Children Responding to Treatment Partial Response	0 Participants
Number of Children Responding to Treatment Stable Disease	5 Participants

SECONDARY outcome

Timeframe: Month 12, Month 24

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.

Outcome measures

Outcome measures
Measure	PEGINTRON n=9 Participants Participants with juvenile pilocytic astrocytomas or optic pathway gliomas receiving PEGINTRON based on their weight (1 mcg/kg/dose) once a week, for up to two years
Number of Participants Meeting Event Free Survival Criteria Month 12	7 Participants
Number of Participants Meeting Event Free Survival Criteria Month 24	7 Participants

SECONDARY outcome

Timeframe: Month 12, Month 24

Overall survival is measured as the time from study enrollment to death from any cause.

Outcome measures

Outcome measures
Measure	PEGINTRON n=9 Participants Participants with juvenile pilocytic astrocytomas or optic pathway gliomas receiving PEGINTRON based on their weight (1 mcg/kg/dose) once a week, for up to two years
Number of Participants Meeting Overall Survival Criteria Month 12	7 Participants
Number of Participants Meeting Overall Survival Criteria Month 24	7 Participants

Adverse Events

PEGINTRON

Serious events: 1 serious events

Other events: 9 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	PEGINTRON n=9 participants at risk Participants with juvenile pilocytic astrocytomas or optic pathway gliomas receiving PEGINTRON based on their weight (1 mcg/kg/dose) once a week, for up to two years
General disorders Anorexia, grade 3	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.

Other adverse events

Other adverse events
Measure	PEGINTRON n=9 participants at risk Participants with juvenile pilocytic astrocytomas or optic pathway gliomas receiving PEGINTRON based on their weight (1 mcg/kg/dose) once a week, for up to two years
Gastrointestinal disorders Abdominal pain, grade 1	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Hepatobiliary disorders Increased alanine transaminase (ALT), grade 1	55.6% 5/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Decreased absolute neutrophil count (ANC), grade 1	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
General disorders Anorexia, grade 2	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Hepatobiliary disorders Increased aspartate aminotransferase (AST), grade 1	22.2% 2/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Gastrointestinal disorders Constipation, grade 2	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Respiratory, thoracic and mediastinal disorders Cough, grade 1	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
General disorders Dizziness, grade 1	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Skin and subcutaneous tissue disorders Dry skin, grade 1	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Anemia, grade 1	44.4% 4/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Gastrointestinal disorders Dyspepsia, grade 2	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
General disorders Fatigue, grade 1	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
General disorders Headache, grade 1	33.3% 3/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Hypermagnesemia, grade 1	22.2% 2/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Hypertriglyceridemia, grade 2	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Hyperuricemia, grade 1	33.3% 3/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Hypoalbuminemia, grade 1	44.4% 4/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Hypocalcemia, grade 1	66.7% 6/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Hypokalemia, grade 1	33.3% 3/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Hypophosphatemia, grade 1	33.3% 3/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Infections and infestations Infection, grade 2	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Decreased lymphocyte count, grade 1	22.2% 2/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
General disorders Nausea, grade 1	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Decreased neutrophil count, grade 1	33.3% 3/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Injury, poisoning and procedural complications Pain at injection site, grade 1	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Decreased platelet count, grade 1	33.3% 3/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Skin and subcutaneous tissue disorders Right forearm eczema, grade 1	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Gastrointestinal disorders Vomiting, grade 2	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
General disorders Weight loss, grade 2	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Decreased white blood cell count, grade 1	66.7% 6/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Hepatobiliary disorders Increased ALT, grade 2	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Anemia, grade 2	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
General disorders Fatigue, grade 2	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Hypophosphatemia, grade 2	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Decreased lymphocyte count, grade 2	44.4% 4/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Decreased neutrophil count, grade 2	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Decreased neutrophil count, grade 3	22.2% 2/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Decreased platelet count, grade 3	11.1% 1/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.
Blood and lymphatic system disorders Decreased white blood cell count, grade 2	22.2% 2/9 • 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). Information on serious adverse events and adverse events that were related to the study treatment were collected.

Additional Information

Dolly Aguilera MD

Emory University

Phone: 404-785-3515

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place