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Study of PEG-Intron for Plexiform Neurofibromas

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ClinicalTrials.gov Identifier: NCT00396019
Recruitment Status : Completed
First Posted : November 6, 2006
Results First Posted : November 6, 2017
Last Update Posted : November 6, 2017
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Jean M. Tersak, M.D., University of Pittsburgh

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Plexiform Neurofibroma
Intervention Drug: PEG-Intron
Enrollment 86
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Stratum 1 No Symptom With or Withour Tumor Growth Stratum 2 Symptoms no Tumor Growth Stratum 3 Tumor Growth With or Without Symptoms
Hide Arm/Group Description Peg-intron given every week Peg-intron given every week Peg-intron given every week
Period Title: Overall Study
Started 27 29 30
Completed 27 26 29
Not Completed 0 3 1
Arm/Group Title Stratum 1 No Symptom With or Withour Tumor Growth Stratum 2 Symptoms no Tumor Growth Stratum 3 Tumor Growth With or Without Symptoms Total
Hide Arm/Group Description Peg-intron given every week Peg-intron given every week Peg-intron given every week Total of all reporting groups
Overall Number of Baseline Participants 27 26 29 82
Hide Baseline Analysis Population Description
[Not Specified]
Age, Customized   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Less than 5 years Number Analyzed 27 participants 25 participants 29 participants 81 participants
3
  11.1%
8
  32.0%
9
  31.0%
20
  24.7%
5 to 12 years Number Analyzed 27 participants 25 participants 29 participants 81 participants
10
  37.0%
9
  36.0%
12
  41.4%
31
  38.3%
greater than or equal to 12 years Number Analyzed 27 participants 25 participants 29 participants 81 participants
14
  51.9%
8
  32.0%
8
  27.6%
30
  37.0%
[1]
Measure Analysis Population Description: Age data is missing for one subject.
Sex: Female, Male   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants 0 participants 0 participants 0 participants
Female 0
Male 0
[1]
Measure Analysis Population Description: Gender data was not captured with demographic/enrollment data.
Race and Ethnicity Not Collected   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants 0 participants 0 participants 0 participants
0
[1]
Measure Analysis Population Description: Race and Ethnicity were not collected from any participant.
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 27 participants 26 participants 29 participants 82 participants
27 26 29 82
1.Primary Outcome
Title Number of Participants With Imaging Response in Strata 1 and 2
Hide Description A response was defined as a ≥20% reduction in the sum of the volume of the "target" plexiform neurofibroma ( PN) within 12 months confirmed by a follow-up MRI after ≥4 weeks.
Time Frame MRI scans were performed at baseline and at months 4, 8, 12, 18, and 24 while on treatment and every 6 months thereafter until off study.
Hide Outcome Measure Data
Hide Analysis Population Description
Not specified
Arm/Group Title Stratum 1 No Symptom With or Withour Tumor Growth Stratum 2 Symptoms no Tumor Growth
Hide Arm/Group Description:
Peg-intron given every week
Peg-intron given every week
Overall Number of Participants Analyzed 27 26
Measure Type: Number
Unit of Measure: participants
Confirmed Response 0 1
Unconfirmed Response 1 0
2.Primary Outcome
Title Clinical Response in Stratum 2
Hide Description Clinical response was defined as a protocol-specified improvement in ophthalmologic evaluation, an improvement of at least one level in performance status (PS) ≥50% decrease in the amount of pain medications required per week compared with baseline or ability to change from a narcotic to a nonnarcotic analgesic, sustained for at least one month.
Time Frame baseline, week 6, months 4, 8, 12, 18, and 24 while on treatment and every 6 months thereafter until off study
Hide Outcome Measure Data
Hide Analysis Population Description
Only participants in Stratum 2 contributed data for this Outcome Measure
Arm/Group Title Stratum 2 Symptoms on Tumor Growth
Hide Arm/Group Description:
Peg-intron given every week
Overall Number of Participants Analyzed 26
Measure Type: Number
Unit of Measure: participants
3
3.Primary Outcome
Title Time to Progression (TTP) in Stratum 3
Hide Description TTP was estimated by the Kaplan-Meter method
Time Frame baseline, week 6, months 4, 8, 12, 18, and 24 while on treatment and every 6 months thereafter until off study
Hide Outcome Measure Data
Hide Analysis Population Description
Only participants in Stratum 3 contributed data for this Outcome Measure
Arm/Group Title Stratum 3: Tumor Growth With or Without Symptoms
Hide Arm/Group Description:
Peg-intron given every week
Overall Number of Participants Analyzed 29
Median (Full Range)
Unit of Measure: months
29.4 [1] 
(7.1 to NA)
[1]
¹not everyone experienced endpoint
4.Secondary Outcome
Title Number of Participants With Imaging Response in Stratum 3
Hide Description A Response was defined as a ≥ 20% reduction in the sum of the volume of the "target" plexiform neurofibroma (PN) within 12 months, confirmed by a follow-up MRI after ≥ 24 weeks
Time Frame MRI scans were performed at baseline and at months 4, 8, 12, 18, and 24 while on treatment and every 6 months thereafter until off study
Hide Outcome Measure Data
Hide Analysis Population Description
Not Specified
Arm/Group Title Stratum 3: Tumor Growth With or Without Symptoms
Hide Arm/Group Description:
Peg-intron given every week
Overall Number of Participants Analyzed 29
Measure Type: Number
Unit of Measure: participants
Confirmed Response 1
Unconfirmed Response 1
Time Frame Adverse event data were collected during clinic visits at baseline, week 6, months 4, 8, 12, 18, and 24 while on treatment and every 6 months thereafter until off study
Adverse Event Reporting Description Other
 
Arm/Group Title Stratum 1 No Symptom With or Withour Tumor Growth Stratum 2 Symptoms no Tumor Growth Stratum 3 Tumor Growth With or Without Symptoms
Hide Arm/Group Description Peg-intron given every week Peg-intron given every week Peg-intron given every week
All-Cause Mortality
Stratum 1 No Symptom With or Withour Tumor Growth Stratum 2 Symptoms no Tumor Growth Stratum 3 Tumor Growth With or Without Symptoms
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/27 (0.00%)   0/26 (0.00%)   0/29 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Stratum 1 No Symptom With or Withour Tumor Growth Stratum 2 Symptoms no Tumor Growth Stratum 3 Tumor Growth With or Without Symptoms
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/27 (0.00%)   0/26 (0.00%)   0/29 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Stratum 1 No Symptom With or Withour Tumor Growth Stratum 2 Symptoms no Tumor Growth Stratum 3 Tumor Growth With or Without Symptoms
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   7/27 (25.93%)   8/26 (30.77%)   6/29 (20.69%) 
General disorders       
Fatigue  1  2/27 (7.41%)  4/26 (15.38%)  2/29 (6.90%) 
Allergic Reaction  3 [1]  1/27 (3.70%)  0/26 (0.00%)  0/29 (0.00%) 
Investigations       
Transaminitis  [2]  0/27 (0.00%)  0/26 (0.00%)  2/29 (6.90%) 
Psychiatric disorders       
Behavior  2 [3]  2/27 (7.41%)  3/26 (11.54%)  2/29 (6.90%) 
Depression  4 [4]  2/27 (7.41%)  1/26 (3.85%)  0/29 (0.00%) 
1
Term from vocabulary, Fatugue
2
Term from vocabulary, Worsening behavior
3
Term from vocabulary, Allergic Reaction
4
Term from vocabulary, Worsening depression
Indicates events were collected by systematic assessment
[1]
Allergic Reaction
[2]
Transaminitis
[3]
Behavior
[4]
Worsening depression
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
Name/Title: Dr. Regina Jakacki
Organization: AstraZeneca
Responsible Party: Jean M. Tersak, M.D., University of Pittsburgh
ClinicalTrials.gov Identifier: NCT00396019     History of Changes
Obsolete Identifiers: NCT00678951
Other Study ID Numbers: UPCI 06-116
First Submitted: November 2, 2006
First Posted: November 6, 2006
Results First Submitted: August 9, 2017
Results First Posted: November 6, 2017
Last Update Posted: November 6, 2017