Trial Outcomes & Findings for A Phase II Study of Pegylated Interferon Alfa 2b (PEG-Intron(Trademark)) in Children With Diffuse Pontine Gliomas (NCT NCT00036569)

NCT ID: NCT00036569

Last Updated: 2012-02-13

Results Overview

Survival is measured from the date the patient is registered onto the protocol until the day of death and the date of diagnosis to the date of patient death.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 32 participants
• Primary outcome timeframe: 8 yrs 6 mo 0 days
• Results posted on: 2012-02-13

Participant Flow

Participant milestones

Measure	Interferon Alfa 0.3 mg/kg subcutaneously once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.
Overall Study STARTED	32
Overall Study COMPLETED	32
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Phase II Study of Pegylated Interferon Alfa 2b (PEG-Intron(Trademark)) in Children With Diffuse Pontine Gliomas

Baseline characteristics by cohort

Measure	Interferon Alfa n=32 Participants 0.3 mg/kg subcutaneously once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.
Age, Categorical <=18 years	32 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age Continuous	6.28 years STANDARD_DEVIATION 3.16 • n=5 Participants
Sex: Female, Male Female	19 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	2 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	23 Participants n=5 Participants
Race/Ethnicity, Customized Unknown or Not Reported	0 Participants n=5 Participants
Race/Ethnicity, Customized Black	4 Participants n=5 Participants
Race/Ethnicity, Customized Asian or Pacific Islander	2 Participants n=5 Participants
Race/Ethnicity, Customized Native Hawaiian	1 Participants n=5 Participants
Region of Enrollment United States	32 participants n=5 Participants

PRIMARY outcome

Timeframe: 8 yrs 6 mo 0 days

Survival is measured from the date the patient is registered onto the protocol until the day of death and the date of diagnosis to the date of patient death.

Outcome measures

Measure	Interferon Alfa n=32 Participants 0.3 mg/kg subcutaneously once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.	QOL Score at Follow-up 0.3 mg/kg subcutaneously once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.
Two Year Survival of Pediatric Patients With Diffuse Pontine Gliomas	14.29 Percentage of patients	—

SECONDARY outcome

Timeframe: 8 yrs 11 mo 22 days

Time between the final day of treatment to the day of disease progression.

Outcome measures

Measure	Interferon Alfa n=32 Participants 0.3 mg/kg subcutaneously once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.	QOL Score at Follow-up 0.3 mg/kg subcutaneously once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.
Median Time to Progression	235 Days	—

SECONDARY outcome

Timeframe: 8 yrs 11 mo 22 days

Here are the number of participants with adverse events. For the detailed list of adverse events see the adverse event module.

Outcome measures

Measure	Interferon Alfa n=32 Participants 0.3 mg/kg subcutaneously once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.	QOL Score at Follow-up 0.3 mg/kg subcutaneously once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.
Number of Participants With Adverse Events	32 Participants	—

SECONDARY outcome

Timeframe: once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.

QOL questionnaires will be performed prior to every cycle for patients age 6-18 years and their parents until cycle 27 and then prior to every third cycle until cycle 52 when the evaluations will become annual. The QOL (NIH Impact of Pediatric Illness Scale) is too detailed to be described and/or shown here. It is a questionnaire made up of approximately 40 questions-the answers are ranked from 1 to 5 with 5 being no impact and 1 being significant impact-For further details see the protocol.

Outcome measures

Measure	Interferon Alfa n=11 Participants 0.3 mg/kg subcutaneously once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.	QOL Score at Follow-up n=11 Participants 0.3 mg/kg subcutaneously once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.
Mean Quality of Life (QOL) Score at Baseline and Follow-Up	3.59 Units on a scale Standard Error 0.11	3.89 Units on a scale Standard Error 0.11

SECONDARY outcome

Timeframe: once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.

MRI of the brain will be performed at the NCI prior to cycles 1, 2, 3, 5, 7, and continuing every other month until cycle 27. Following cycle 27 the patient will have an MRI performed every third cycle until cycle 52 at which time they will have an MRI performed annually, and when clinically indicated. Baseline MR images are compared with MR images performed during the various cycles (e.g. cycles 1, 2, 3...) Imaging was exploratory and the degree of change that is considered clinically significant rather than technique related is still being explored.

Outcome measures

Measure	Interferon Alfa n=32 Participants 0.3 mg/kg subcutaneously once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.	QOL Score at Follow-up 0.3 mg/kg subcutaneously once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.
Number of Participants With a Metabolic and Biological Change in the Brainstem Through Magnetic Resonance Imaging (MRI) Techniques	32 Participants	—

Adverse Events

Interferon Alfa

Serious events: 32 serious events

Other events: 32 other events

Deaths: 0 deaths

Serious adverse events

Measure	Interferon Alfa n=32 participants at risk 0.3 mg/kg subcutaneously once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.
Investigations ALT	21.9% 7/32 • Number of events 8
Nervous system disorders Ataxia	3.1% 1/32 • Number of events 1
Eye disorders Diplopia	9.4% 3/32 • Number of events 3
General disorders Fatigue	6.2% 2/32 • Number of events 2
General disorders Fever	3.1% 1/32 • Number of events 1
Infections and infestations Infection	3.1% 1/32 • Number of events 1
Psychiatric disorders Insomnia	6.2% 2/32 • Number of events 2
Investigations Leukopenia	28.1% 9/32 • Number of events 16
Investigations Lymphopenia	28.1% 9/32 • Number of events 20
Gastrointestinal disorders Nausea	3.1% 1/32 • Number of events 1
Nervous system disorders Neuropathy-cranial	37.5% 12/32 • Number of events 42
Nervous system disorders Neuropathy-motor	9.4% 3/32 • Number of events 3
Investigations Neutropenia	37.5% 12/32 • Number of events 32
Nervous system disorders Pyramidal tract dysfunction	3.1% 1/32 • Number of events 1
Nervous system disorders Seizure	3.1% 1/32 • Number of events 1
Gastrointestinal disorders Vomiting	3.1% 1/32 • Number of events 1

Other adverse events

Measure	Interferon Alfa n=32 participants at risk 0.3 mg/kg subcutaneously once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.
Gastrointestinal disorders Abdominal pain	3.1% 1/32 • Number of events 1
Investigations Alanine aminotransferase (ALT)	37.5% 12/32 • Number of events 24
Metabolism and nutrition disorders Anorexia	6.2% 2/32 • Number of events 2
Musculoskeletal and connective tissue disorders Arthralgia	3.1% 1/32 • Number of events 1
Investigations Aspartate aminotransferase (AST)	21.9% 7/32 • Number of events 17
Nervous system disorders Ataxia	0.00% 0/32
Investigations Bilirubin	6.2% 2/32 • Number of events 2
Injury, poisoning and procedural complications Bruising	9.4% 3/32 • Number of events 4
Eye disorders Diplopia	0.00% 0/32
General disorders Fatigue	9.4% 3/32 • Number of events 4
General disorders Fever	6.2% 2/32 • Number of events 2
Nervous system disorders Headache	6.2% 2/32 • Number of events 4
Investigations Hemoglobin	18.8% 6/32 • Number of events 26
Infections and infestations Infection	0.00% 0/32
General disorders Injection site reaction	6.2% 2/32 • Number of events 3
Psychiatric disorders Insomnia	3.1% 1/32 • Number of events 3
Investigations Leukopenia	40.6% 13/32 • Number of events 41
Investigations Lymphopenia	28.1% 9/32 • Number of events 20
Gastrointestinal disorders Nausea	3.1% 1/32 • Number of events 1
Nervous system disorders Neuropathy-cranial	0.00% 0/32
Nervous system disorders Neuropathy-motor	9.4% 3/32 • Number of events 4
Investigations Neutropenia	25.0% 8/32 • Number of events 18
Psychiatric disorders Personality change	3.1% 1/32 • Number of events 1
Nervous system disorders Seizure	0.00% 0/32
Investigations Thrombocytopenia	6.2% 2/32 • Number of events 2
Nervous system disorders Pyramidal tract dysfunction	28.1% 9/32 • Number of events 15
Gastrointestinal disorders Vomiting	3.1% 1/32 • Number of events 1

Additional Information

Kathy Warren, M.D.

National Cancer Institute

Phone: 301-435-4683

Email: warrenk@mail.nih.gov

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place