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Peginterferon Alfa-2b in Younger Patients With Craniopharyngioma That is Recurrent or Cannot Be Removed By Surgery

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ClinicalTrials.gov Identifier: NCT01964300
Recruitment Status : Terminated (Lack of efficacy (stratum 2); slow accrual (stratum 1))
First Posted : October 17, 2013
Results First Posted : February 24, 2020
Last Update Posted : February 24, 2020
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Pediatric Brain Tumor Consortium

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Childhood Craniopharyngioma
Interventions Biological: peginterferon alfa-2b
Other: laboratory biomarker analysis
Enrollment 19
Recruitment Details Patients 18 months to 25 years with histologically confirmed craniopharyngioma were enrolled at Pediatric Brain Tumor Consortium (PBTC) member institutions. The first patient was enrolled on 4/1/2014 and the last patient was enrolled on 11/20/2017. Accrual was halted early due to lack of efficacy in stratum 2 and slow accrual in stratum 1.
Pre-assignment Details Stratum 1 patients had progressive unresectable or recurrent craniopharyngiomas treated with surgery alone. Stratum 2 patients had progressive/recurrent craniopharyngiomas following radiation. 19 patients were enrolled (7 stratum 1, 12 stratum 2); one of the stratum 2 patients was ineligible leaving 18 eligible patients (7 stratum 1, 11 stratum 2).
Arm/Group Title Stratum 1 Stratum 2
Hide Arm/Group Description Stratum 1 patients are those with progressive unresectable or recurrent craniopharyngiomas treated with surgery alone, who had not received radiation therapy. Treatment consisted of Peginterferon alfa-2b (Sylatron) subcutaneously weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity. Stratum 2 patients are those with patients with progressive or recurrent craniopharyngiomas following radiation therapy. Treatment consisted of Peginterferon alfa-2b (Sylatron) subcutaneously weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity.
Period Title: Overall Study
Started 7 12
Completed 1 1
Not Completed 6 11
Reason Not Completed
Ineligible after enrollment             0             1
Adverse Event             1             1
Lack of Efficacy             3             5
Withdrawal by Subject             0             1
To receive alternative therapy             1             2
Enlargement of cyst requiring drainage             1             0
Weight criteria not met             0             1
Arm/Group Title Stratum 1 Stratum 2 Total
Hide Arm/Group Description Stratum 1 patients are those with progressive unresectable or recurrent craniopharyngiomas treated with surgery alone, who had not received radiation therapy. Treatment consisted of Peginterferon alfa-2b (Sylatron) subcutaneously weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity. Stratum 2 patients are those with patients with progressive or recurrent craniopharyngiomas following radiation therapy. Treatment consisted of Peginterferon alfa-2b (Sylatron) subcutaneously weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity. Total of all reporting groups
Overall Number of Baseline Participants 7 11 18
Hide Baseline Analysis Population Description
The one stratum 2 patient found to be ineligible after enrollment was excluded. Summary statistics for baseline characteristics are shown for eligible patients only (n=18).
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 11 participants 18 participants
<=18 years
7
 100.0%
5
  45.5%
12
  66.7%
Between 18 and 65 years
0
   0.0%
6
  54.5%
6
  33.3%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 7 participants 11 participants 18 participants
10.6
(2.0 to 17.0)
20.5
(6.1 to 25.0)
13.1
(2.0 to 25.0)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 11 participants 18 participants
Female
4
  57.1%
6
  54.5%
10
  55.6%
Male
3
  42.9%
5
  45.5%
8
  44.4%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 11 participants 18 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
2
  18.2%
2
  11.1%
White
4
  57.1%
8
  72.7%
12
  66.7%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
3
  42.9%
1
   9.1%
4
  22.2%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 7 participants 11 participants 18 participants
7 11 18
1.Primary Outcome
Title Rate of Disease Stabilization at 1 Year (i.e., 9 Courses of Treatment) (for Stratum 1 Patients Only)
Hide Description The percentage of stratum 1 patients with disease stabilization at 1 year is reported, along with a 95% exact confidence interval for the estimate of the true 1-year disease stabilization rate.
Time Frame Up to 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
This primary outcome measure only relates to stratum 1 patients.
Arm/Group Title Stratum 1 Stratum 2
Hide Arm/Group Description:
Stratum 1 patients are those with progressive unresectable or recurrent craniopharyngiomas treated with surgery alone, who had not received radiation therapy. Treatment consisted of Peginterferon alfa-2b (Sylatron) subcutaneously weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity.
Stratum 2 patients are those with patients with progressive or recurrent craniopharyngiomas following radiation therapy. Treatment consisted of Peginterferon alfa-2b (Sylatron) subcutaneously weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 7 0
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
28.6
(5.3 to 65.9)
2.Primary Outcome
Title Sustained Objective Response (PR+CR) Rate Observed During the First Year of Treatment (for Stratum 2 Patients Only)
Hide Description Objective responses had to be sustained for 3 months. The percentage of participants with sustained objective responses is reported with an exact 95% confidence interval of the true sustained objective response rate.
Time Frame Up to 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
Only eligible stratum 2 patients are included in this analysis. This is a *secondary* outcome measure for stratum 1 patients and is therefore reported as a separate outcome measure.
Arm/Group Title Stratum 1 Stratum 2
Hide Arm/Group Description:
Stratum 1 patients are those with progressive unresectable or recurrent craniopharyngiomas treated with surgery alone, who had not received radiation therapy. Treatment consisted of Peginterferon alfa-2b (Sylatron) subcutaneously weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity.
Stratum 2 patients are those with patients with progressive or recurrent craniopharyngiomas following radiation therapy. Treatment consisted of Peginterferon alfa-2b (Sylatron) subcutaneously weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 0 11
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
0.0
(0.0 to 25.0)
3.Secondary Outcome
Title Sustained Objective Response (PR+CR) Rate Observed During the First Year of Treatment (for Stratum 1 Patients Only)
Hide Description Objective responses had to be sustained for 3 months. The percentage of participants with sustained objective responses is reported with an exact 95% confidence interval of the true sustained objective response rate.
Time Frame Up to 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
The seven stratum 1 patients are included in this analysis. This outcome measure was a *primary* outcome measure for stratum 2 patients and is therefore reported as a separate outcome measure for stratum 2.
Arm/Group Title Stratum 1 Stratum 2
Hide Arm/Group Description:
Stratum 1 patients are those with progressive unresectable or recurrent craniopharyngiomas treated with surgery alone, who had not received radiation therapy. Treatment consisted of Peginterferon alfa-2b (Sylatron) subcutaneously weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity.
Stratum 2 patients are those with patients with progressive or recurrent craniopharyngiomas following radiation therapy. Treatment consisted of Peginterferon alfa-2b (Sylatron) subcutaneously weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 7 0
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
14.3
(0.01 to 55.4)
4.Secondary Outcome
Title Progression-free Survival (PFS)
Hide Description PFS estimates for each stratum were estimated using the method of Kaplan and Meier. PFS was defined as the time interval from date on treatment to the earliest date of disease progression, second malignancy, or death; or to date of last contact for patients without events. One- and two-year PFS estimates are reported by stratum. Only eligible patients were included in this analysis (7 stratum 1 patients and 11 stratum 2 patients).
Time Frame 2 years after treatment start
Hide Outcome Measure Data
Hide Analysis Population Description
The one ineligible stratum 2 patient enrolled on the study was excluded from this analysis, leaving 7 stratum 1 patients and 11 stratum 2 patients.
Arm/Group Title Stratum 1 Stratum 2
Hide Arm/Group Description:
Stratum 1 patients are those with progressive unresectable or recurrent craniopharyngiomas treated with surgery alone, who had not received radiation therapy. Treatment consisted of Peginterferon alfa-2b (Sylatron) subcutaneously weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity.
Stratum 2 patients are those with patients with progressive or recurrent craniopharyngiomas following radiation therapy. Treatment consisted of Peginterferon alfa-2b (Sylatron) subcutaneously weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 7 11
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
PFS at 1 year
83.3
(50.0 to 100.0)
68.2
(39.8 to 96.6)
PFS at 2 years
27.8
(0.0 to 60.5)
34.1
(0.0 to 72.5)
Time Frame Up to 2 years after start of treatment
Adverse Event Reporting Description Adverse events were graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. All grades 3 through 5 adverse events while on treatment or within 30 days of treatment were to be collected, in addition to grades 1 and 2 adverse events if the attribution was at least possibly related. All adverse events collected and reported in the study database are reported here.
 
Arm/Group Title Stratum 1 Stratum 2
Hide Arm/Group Description Stratum 1 patients are those with progressive unresectable or recurrent craniopharyngiomas treated with surgery alone, who had not received radiation therapy. Treatment consisted of Peginterferon alfa-2b (Sylatron) subcutaneously weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity.

Stratum 2 patients are those with patients with progressive or recurrent craniopharyngiomas following radiation therapy. Treatment consisted of Peginterferon alfa-2b (Sylatron) subcutaneously weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity.

Since the ineligible patient received 2 days of Peginterferon alfa-2b, that patient is included in all adverse event reporting results.

All-Cause Mortality
Stratum 1 Stratum 2
Affected / at Risk (%) Affected / at Risk (%)
Total   0/7 (0.00%)      1/12 (8.33%)    
Hide Serious Adverse Events
Stratum 1 Stratum 2
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/7 (14.29%)      5/12 (41.67%)    
Gastrointestinal disorders     
Nausea   0/7 (0.00%)  0 1/12 (8.33%)  1
Infections and infestations     
Catheter related infection   1/7 (14.29%)  1 0/12 (0.00%)  0
Injury, poisoning and procedural complications     
Fall   0/7 (0.00%)  0 1/12 (8.33%)  1
Investigations     
Neutrophil count decreased   0/7 (0.00%)  0 1/12 (8.33%)  1
Metabolism and nutrition disorders     
Anorexia   0/7 (0.00%)  0 1/12 (8.33%)  1
Hypernatremia   0/7 (0.00%)  0 1/12 (8.33%)  1
Hyponatremia   0/7 (0.00%)  0 1/12 (8.33%)  1
Musculoskeletal and connective tissue disorders     
Generalized muscle weakness   0/7 (0.00%)  0 1/12 (8.33%)  1
Muscle weakness right-sided   1/7 (14.29%)  1 0/12 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Treatment related secondary malignancy   0/7 (0.00%)  0 1/12 (8.33%)  1
Nervous system disorders     
Ataxia   0/7 (0.00%)  0 1/12 (8.33%)  2
Dizziness   1/7 (14.29%)  1 0/12 (0.00%)  0
Dysarthria   0/7 (0.00%)  0 1/12 (8.33%)  2
Encephalopathy   0/7 (0.00%)  0 1/12 (8.33%)  1
Facial nerve disorder   1/7 (14.29%)  1 0/12 (0.00%)  0
Headache   1/7 (14.29%)  1 2/12 (16.67%)  2
Somnolence   0/7 (0.00%)  0 1/12 (8.33%)  1
Tremor   1/7 (14.29%)  1 0/12 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Dyspnea   0/7 (0.00%)  0 1/12 (8.33%)  1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Stratum 1 Stratum 2
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/7 (100.00%)      12/12 (100.00%)    
Blood and lymphatic system disorders     
Anemia   3/7 (42.86%)  3 3/12 (25.00%)  4
Eye disorders     
Blurred vision   0/7 (0.00%)  0 1/12 (8.33%)  1
Floaters   0/7 (0.00%)  0 1/12 (8.33%)  1
Optic nerve disorder   0/7 (0.00%)  0 1/12 (8.33%)  1
Photophobia   0/7 (0.00%)  0 1/12 (8.33%)  1
Gastrointestinal disorders     
Abdominal pain   1/7 (14.29%)  1 0/12 (0.00%)  0
Diarrhea   0/7 (0.00%)  0 3/12 (25.00%)  4
Gastrointestinal disorders - Other, specify   2/7 (28.57%)  2 0/12 (0.00%)  0
Mucositis oral   1/7 (14.29%)  1 0/12 (0.00%)  0
Nausea   1/7 (14.29%)  1 4/12 (33.33%)  5
Vomiting   1/7 (14.29%)  1 1/12 (8.33%)  2
General disorders     
Chills   3/7 (42.86%)  8 0/12 (0.00%)  0
Fatigue   5/7 (71.43%)  9 3/12 (25.00%)  4
Fever   7/7 (100.00%)  11 2/12 (16.67%)  4
Flu like symptoms   0/7 (0.00%)  0 2/12 (16.67%)  2
General disorders and administration site conditions - Other, specify   1/7 (14.29%)  1 0/12 (0.00%)  0
Hypothermia   0/7 (0.00%)  0 1/12 (8.33%)  1
Injection site reaction   0/7 (0.00%)  0 1/12 (8.33%)  1
Infections and infestations     
Urinary tract infection   0/7 (0.00%)  0 1/12 (8.33%)  1
Investigations     
Alanine aminotransferase increased   4/7 (57.14%)  8 8/12 (66.67%)  14
Alkaline phosphatase increased   0/7 (0.00%)  0 3/12 (25.00%)  5
Aspartate aminotransferase increased   3/7 (42.86%)  5 10/12 (83.33%)  15
Investigations - Other, specify   2/7 (28.57%)  2 0/12 (0.00%)  0
Lymphocyte count decreased   1/7 (14.29%)  3 4/12 (33.33%)  6
Neutrophil count decreased   3/7 (42.86%)  10 8/12 (66.67%)  18
Platelet count decreased   0/7 (0.00%)  0 4/12 (33.33%)  6
Weight loss   4/7 (57.14%)  5 2/12 (16.67%)  2
White blood cell decreased   6/7 (85.71%)  14 8/12 (66.67%)  12
Metabolism and nutrition disorders     
Anorexia   4/7 (57.14%)  5 2/12 (16.67%)  2
Hyperglycemia   1/7 (14.29%)  1 0/12 (0.00%)  0
Hyperkalemia   1/7 (14.29%)  2 2/12 (16.67%)  2
Hypermagnesemia   2/7 (28.57%)  3 1/12 (8.33%)  1
Hypernatremia   1/7 (14.29%)  1 3/12 (25.00%)  3
Hyperuricemia   0/7 (0.00%)  0 1/12 (8.33%)  1
Hypoalbuminemia   0/7 (0.00%)  0 1/12 (8.33%)  2
Hypocalcemia   1/7 (14.29%)  1 6/12 (50.00%)  8
Hypoglycemia   0/7 (0.00%)  0 1/12 (8.33%)  1
Hypokalemia   2/7 (28.57%)  5 1/12 (8.33%)  2
Hypomagnesemia   0/7 (0.00%)  0 1/12 (8.33%)  1
Hyponatremia   2/7 (28.57%)  2 1/12 (8.33%)  1
Hypophosphatemia   1/7 (14.29%)  1 1/12 (8.33%)  1
Metabolism and nutrition disorders - Other, specify   2/7 (28.57%)  2 0/12 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Back pain   1/7 (14.29%)  1 0/12 (0.00%)  0
Myalgia   0/7 (0.00%)  0 1/12 (8.33%)  1
Pain in extremity   1/7 (14.29%)  1 1/12 (8.33%)  1
Nervous system disorders     
Abducens nerve disorder   0/7 (0.00%)  0 1/12 (8.33%)  1
Cognitive disturbance   0/7 (0.00%)  0 1/12 (8.33%)  1
Dizziness   0/7 (0.00%)  0 1/12 (8.33%)  1
Headache   2/7 (28.57%)  5 2/12 (16.67%)  2
Seizure   0/7 (0.00%)  0 1/12 (8.33%)  1
Somnolence   0/7 (0.00%)  0 1/12 (8.33%)  1
Psychiatric disorders     
Depression   1/7 (14.29%)  1 1/12 (8.33%)  1
Insomnia   0/7 (0.00%)  0 3/12 (25.00%)  3
Personality change   0/7 (0.00%)  0 1/12 (8.33%)  1
Skin and subcutaneous tissue disorders     
Alopecia   0/7 (0.00%)  0 1/12 (8.33%)  1
Pruritus   1/7 (14.29%)  1 0/12 (0.00%)  0
Rash maculo-papular   1/7 (14.29%)  1 0/12 (0.00%)  0
Vascular disorders     
Hot flashes   0/7 (0.00%)  0 1/12 (8.33%)  1
Hypotension   0/7 (0.00%)  0 1/12 (8.33%)  1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Catherine Billups
Organization: Pediatric Brain Tumor Consortium
Phone: 901-595-3709
EMail: catherine.billups@stjude.org
Layout table for additonal information
Responsible Party: Pediatric Brain Tumor Consortium
ClinicalTrials.gov Identifier: NCT01964300    
Other Study ID Numbers: PBTC-039
NCI-2013-01639 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
PBTC-039 ( Other Identifier: Pediatric Brain Tumor Consortium )
PBTC-039 ( Other Identifier: CTEP )
U01CA081457 ( U.S. NIH Grant/Contract )
First Submitted: October 14, 2013
First Posted: October 17, 2013
Results First Submitted: October 17, 2019
Results First Posted: February 24, 2020
Last Update Posted: February 24, 2020