Trial Outcomes & Findings for Study of PEG-Intron for Plexiform Neurofibromas (NCT NCT00396019)
NCT ID: NCT00396019
Last Updated: 2017-11-06
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
86 participants
Primary outcome timeframe
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.
Results posted on
2017-11-06
Participant Flow
Participant milestones
| Measure |
Stratum 1 No Symptom With or Withour Tumor Growth
Peg-intron given every week
|
Stratum 2 Symptoms no Tumor Growth
Peg-intron given every week
|
Stratum 3 Tumor Growth With or Without Symptoms
Peg-intron given every week
|
|---|---|---|---|
|
Overall Study
STARTED
|
27
|
29
|
30
|
|
Overall Study
COMPLETED
|
27
|
26
|
29
|
|
Overall Study
NOT COMPLETED
|
0
|
3
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Age data is missing for one subject.
Baseline characteristics by cohort
| Measure |
Stratum 1 No Symptom With or Withour Tumor Growth
n=27 Participants
Peg-intron given every week
|
Stratum 2 Symptoms no Tumor Growth
n=26 Participants
Peg-intron given every week
|
Stratum 3 Tumor Growth With or Without Symptoms
n=29 Participants
Peg-intron given every week
|
Total
n=82 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Customized
Less than 5 years
|
3 Participants
n=27 Participants • Age data is missing for one subject.
|
8 Participants
n=25 Participants • Age data is missing for one subject.
|
9 Participants
n=29 Participants • Age data is missing for one subject.
|
20 Participants
n=81 Participants • Age data is missing for one subject.
|
|
Age, Customized
5 to 12 years
|
10 Participants
n=27 Participants • Age data is missing for one subject.
|
9 Participants
n=25 Participants • Age data is missing for one subject.
|
12 Participants
n=29 Participants • Age data is missing for one subject.
|
31 Participants
n=81 Participants • Age data is missing for one subject.
|
|
Age, Customized
greater than or equal to 12 years
|
14 Participants
n=27 Participants • Age data is missing for one subject.
|
8 Participants
n=25 Participants • Age data is missing for one subject.
|
8 Participants
n=29 Participants • Age data is missing for one subject.
|
30 Participants
n=81 Participants • Age data is missing for one subject.
|
|
Sex: Female, Male
Female
|
—
|
—
|
—
|
0 Participants
Gender data was not captured with demographic/enrollment data.
|
|
Sex: Female, Male
Male
|
—
|
—
|
—
|
0 Participants
Gender data was not captured with demographic/enrollment data.
|
|
Race and Ethnicity Not Collected
|
—
|
—
|
—
|
0 Participants
Race and Ethnicity were not collected from any participant.
|
|
Region of Enrollment
United States
|
27 participants
n=27 Participants
|
26 participants
n=26 Participants
|
29 participants
n=29 Participants
|
82 participants
n=82 Participants
|
PRIMARY outcome
Timeframe: 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.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.
Outcome measures
| Measure |
Stratum 1 No Symptom With or Withour Tumor Growth
n=27 Participants
Peg-intron given every week
|
Stratum 2 Symptoms no Tumor Growth
n=26 Participants
Peg-intron given every week
|
|---|---|---|
|
Number of Participants With Imaging Response in Strata 1 and 2
Confirmed Response
|
0 participants
|
1 participants
|
|
Number of Participants With Imaging Response in Strata 1 and 2
Unconfirmed Response
|
1 participants
|
0 participants
|
PRIMARY outcome
Timeframe: baseline, week 6, months 4, 8, 12, 18, and 24 while on treatment and every 6 months thereafter until off studyPopulation: Only participants in Stratum 2 contributed data for this Outcome Measure
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.
Outcome measures
| Measure |
Stratum 1 No Symptom With or Withour Tumor Growth
n=26 Participants
Peg-intron given every week
|
Stratum 2 Symptoms no Tumor Growth
Peg-intron given every week
|
|---|---|---|
|
Clinical Response in Stratum 2
|
3 participants
|
—
|
PRIMARY outcome
Timeframe: baseline, week 6, months 4, 8, 12, 18, and 24 while on treatment and every 6 months thereafter until off studyPopulation: Only participants in Stratum 3 contributed data for this Outcome Measure
TTP was estimated by the Kaplan-Meter method
Outcome measures
| Measure |
Stratum 1 No Symptom With or Withour Tumor Growth
n=29 Participants
Peg-intron given every week
|
Stratum 2 Symptoms no Tumor Growth
Peg-intron given every week
|
|---|---|---|
|
Time to Progression (TTP) in Stratum 3
|
29.4 months
Interval 7.1 to
¹not everyone experienced endpoint
|
—
|
SECONDARY outcome
Timeframe: 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 studyA 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
Outcome measures
| Measure |
Stratum 1 No Symptom With or Withour Tumor Growth
n=29 Participants
Peg-intron given every week
|
Stratum 2 Symptoms no Tumor Growth
Peg-intron given every week
|
|---|---|---|
|
Number of Participants With Imaging Response in Stratum 3
Confirmed Response
|
1 participants
|
—
|
|
Number of Participants With Imaging Response in Stratum 3
Unconfirmed Response
|
1 participants
|
—
|
Adverse Events
Stratum 1 No Symptom With or Withour Tumor Growth
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Stratum 2 Symptoms no Tumor Growth
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Stratum 3 Tumor Growth With or Without Symptoms
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Stratum 1 No Symptom With or Withour Tumor Growth
n=27 participants at risk
Peg-intron given every week
|
Stratum 2 Symptoms no Tumor Growth
n=26 participants at risk
Peg-intron given every week
|
Stratum 3 Tumor Growth With or Without Symptoms
n=29 participants at risk
Peg-intron given every week
|
|---|---|---|---|
|
General disorders
Fatigue
|
7.4%
2/27 • 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
Other
|
15.4%
4/26 • 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
Other
|
6.9%
2/29 • 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
Other
|
|
Psychiatric disorders
Behavior
|
7.4%
2/27 • 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
Other
|
11.5%
3/26 • 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
Other
|
6.9%
2/29 • 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
Other
|
|
General disorders
Allergic Reaction
|
3.7%
1/27 • 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
Other
|
0.00%
0/26 • 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
Other
|
0.00%
0/29 • 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
Other
|
|
Psychiatric disorders
Depression
|
7.4%
2/27 • 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
Other
|
3.8%
1/26 • 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
Other
|
0.00%
0/29 • 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
Other
|
|
Investigations
Transaminitis
|
0.00%
0/27 • 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
Other
|
0.00%
0/26 • 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
Other
|
6.9%
2/29 • 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
Other
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place