Trial Outcomes & Findings for Evaluate PKs and Efficacy Assessment of Palifermin in Patients With Sarcoma (NCT NCT00267046)
NCT ID: NCT00267046
Last Updated: 2012-05-16
Results Overview
Cumulative incidence of World Health Organization (WHO) grade 2 or \> mucositis (moderate to severe) in participants completing up to 6 blinded cycles. Rate defined as participants who had Grade 2 or \> divided by total number of participants who completed up to 6 blinded cycles. WHO Criteria of Grade 1: possible buccal mucosal scalloping with/without erythema; No ulcers; swallows solid diet. Grade 2: ulcers with or without erythema; swallow solid diet. Grade 3: ulcers with/without (extensive) erythema; swallow liquid, not solid diet. Grade 4: mucositis to extent alimentation not possible.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
49 participants
Primary outcome timeframe
Within 6 blinded cycles (3-week cycles), up to 18 weeks.
Results posted on
2012-05-16
Participant Flow
Recruitment Period: 12/2005 to 02/2008. All recruitment done at UT MD Anderson Cancer Center.
Of the 49 enrolled, one participant withdrew before assignment to groups.
Participant milestones
| Measure |
Palifermin
Palifermin + Chemotherapy (AI or AP Regimen);
AI = Doxorubicin (Adriamycin) + Ifosfamide:
Palifermin 180 mcg/kg 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 intravenous (IV) for 72 hours starting Days 0 for total 90 mg/m\^2. Ifosfamide 2.5 g/m\^2 IV bolus Days 0-3 (total 10 g/m\^2); Vincristine 2 mg IV Day 0.
AP=Doxorubicin (Adriamycin) + Cisplatin:
Palifermin 180 mcg/kg 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 IV continuous infusion for 72 hours starting Day 0(total = 90 mg/m\^2); Cisplatin 120 mg/m\^2 on day 0.
|
Placebo
Placebo + Chemotherapy (AI or AP Regimen):
AI = Doxorubicin (Adriamycin) + Ifosfamide:
A single dose placebo 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 intravenous (IV) for 72 hours starting Days 0 for total 90 mg/m\^2. Ifosfamide 2.5 g/m\^2 IV bolus Days 0-3 (total 10 g/m\^2); Vincristine 2 mg IV Day 0.
AP=Doxorubicin (Adriamycin) + Cisplatin:
A single dose placebo 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 IV continuous infusion for 72 hours starting Day 0(total = 90 mg/m\^2); Cisplatin 120 mg/m\^2 on day 0.
|
|---|---|---|
|
Overall Study
STARTED
|
32
|
16
|
|
Overall Study
COMPLETED
|
20
|
5
|
|
Overall Study
NOT COMPLETED
|
12
|
11
|
Reasons for withdrawal
| Measure |
Palifermin
Palifermin + Chemotherapy (AI or AP Regimen);
AI = Doxorubicin (Adriamycin) + Ifosfamide:
Palifermin 180 mcg/kg 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 intravenous (IV) for 72 hours starting Days 0 for total 90 mg/m\^2. Ifosfamide 2.5 g/m\^2 IV bolus Days 0-3 (total 10 g/m\^2); Vincristine 2 mg IV Day 0.
AP=Doxorubicin (Adriamycin) + Cisplatin:
Palifermin 180 mcg/kg 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 IV continuous infusion for 72 hours starting Day 0(total = 90 mg/m\^2); Cisplatin 120 mg/m\^2 on day 0.
|
Placebo
Placebo + Chemotherapy (AI or AP Regimen):
AI = Doxorubicin (Adriamycin) + Ifosfamide:
A single dose placebo 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 intravenous (IV) for 72 hours starting Days 0 for total 90 mg/m\^2. Ifosfamide 2.5 g/m\^2 IV bolus Days 0-3 (total 10 g/m\^2); Vincristine 2 mg IV Day 0.
AP=Doxorubicin (Adriamycin) + Cisplatin:
A single dose placebo 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 IV continuous infusion for 72 hours starting Day 0(total = 90 mg/m\^2); Cisplatin 120 mg/m\^2 on day 0.
|
|---|---|---|
|
Overall Study
Disease progression
|
4
|
2
|
|
Overall Study
Chemotoxicity
|
2
|
0
|
|
Overall Study
Open-label palifermin
|
2
|
7
|
|
Overall Study
Surgery
|
3
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
|
Overall Study
Radiation
|
0
|
1
|
Baseline Characteristics
Evaluate PKs and Efficacy Assessment of Palifermin in Patients With Sarcoma
Baseline characteristics by cohort
| Measure |
Palifermin
n=32 Participants
Palifermin + Chemotherapy (AI or AP Regimen);
AI = Doxorubicin (Adriamycin) + Ifosfamide:
Palifermin 180 mcg/kg 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 intravenous (IV) for 72 hours starting Days 0 for total 90 mg/m\^2. Ifosfamide 2.5 g/m\^2 IV bolus Days 0-3 (total 10 g/m\^2); Vincristine 2 mg IV Day 0.
AP=Doxorubicin (Adriamycin) + Cisplatin:
Palifermin 180 mcg/kg 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 IV continuous infusion for 72 hours starting Day 0(total = 90 mg/m\^2); Cisplatin 120 mg/m\^2 on day 0.
|
Placebo
n=16 Participants
Placebo + Chemotherapy (AI or AP Regimen):
AI = Doxorubicin (Adriamycin) + Ifosfamide:
A single dose placebo 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 intravenous (IV) for 72 hours starting Days 0 for total 90 mg/m\^2. Ifosfamide 2.5 g/m\^2 IV bolus Days 0-3 (total 10 g/m\^2); Vincristine 2 mg IV Day 0.
AP=Doxorubicin (Adriamycin) + Cisplatin:
A single dose placebo 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 IV continuous infusion for 72 hours starting Day 0(total = 90 mg/m\^2); Cisplatin 120 mg/m\^2 on day 0.
|
Total
n=48 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Participants' Completion of 6 Blinded Cycles
Completed 6 Cycles
|
20 participants
n=99 Participants
|
5 participants
n=107 Participants
|
25 participants
n=206 Participants
|
|
Age, Customized
|
42 years
n=99 Participants
|
39 years
n=107 Participants
|
42 years
n=206 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
23 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
17 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
25 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
32 participants
n=99 Participants
|
16 participants
n=107 Participants
|
48 participants
n=206 Participants
|
|
Number of participants on two different chemotherapy regimens.
Number of participants on AI chemotherapy
|
30 Participant
n=99 Participants
|
15 Participant
n=107 Participants
|
45 Participant
n=206 Participants
|
|
Number of participants on two different chemotherapy regimens.
Number of participants on AP chemotherapy
|
2 Participant
n=99 Participants
|
1 Participant
n=107 Participants
|
3 Participant
n=206 Participants
|
|
Participants' Completion of 6 Blinded Cycles
Not Completed 6 Cycles
|
12 participants
n=99 Participants
|
11 participants
n=107 Participants
|
23 participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Within 6 blinded cycles (3-week cycles), up to 18 weeks.Population: Intention to treat (ITT).
Cumulative incidence of World Health Organization (WHO) grade 2 or \> mucositis (moderate to severe) in participants completing up to 6 blinded cycles. Rate defined as participants who had Grade 2 or \> divided by total number of participants who completed up to 6 blinded cycles. WHO Criteria of Grade 1: possible buccal mucosal scalloping with/without erythema; No ulcers; swallows solid diet. Grade 2: ulcers with or without erythema; swallow solid diet. Grade 3: ulcers with/without (extensive) erythema; swallow liquid, not solid diet. Grade 4: mucositis to extent alimentation not possible.
Outcome measures
| Measure |
Palifermin
n=32 Participants
Palifermin + Chemotherapy (AI or AP Regimen);
AI = Doxorubicin (Adriamycin) + Ifosfamide:
Palifermin 180 mcg/kg 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 intravenous (IV) for 72 hours starting Days 0 for total 90 mg/m\^2. Ifosfamide 2.5 g/m\^2 IV bolus Days 0-3 (total 10 g/m\^2); Vincristine 2 mg IV Day 0.
AP=Doxorubicin (Adriamycin) + Cisplatin:
Palifermin 180 mcg/kg 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 IV continuous infusion for 72 hours starting Day 0(total = 90 mg/m\^2); Cisplatin 120 mg/m\^2 on day 0.
|
Placebo
n=16 Participants
Placebo + Chemotherapy (AI or AP Regimen):
AI = Doxorubicin (Adriamycin) + Ifosfamide:
A single dose placebo 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 intravenous (IV) for 72 hours starting Days 0 for total 90 mg/m\^2. Ifosfamide 2.5 g/m\^2 IV bolus Days 0-3 (total 10 g/m\^2); Vincristine 2 mg IV Day 0.
AP=Doxorubicin (Adriamycin) + Cisplatin:
A single dose placebo 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 IV continuous infusion for 72 hours starting Day 0(total = 90 mg/m\^2); Cisplatin 120 mg/m\^2 on day 0.
|
|---|---|---|
|
Cumulative Incidence Rate of Oral Mucositis
Cumulative incidence of WHO grade 2 or higher muco
|
44 Percentage of Participants
Interval 24.0 to 62.0
|
88 Percentage of Participants
Interval 52.0 to 97.0
|
|
Cumulative Incidence Rate of Oral Mucositis
Cumulative Incidence of WHO Grade 3 or 4 Mucositis
|
13 Percentage of Participants
Interval 4.0 to 27.0
|
51 Percentage of Participants
Interval 23.0 to 73.0
|
PRIMARY outcome
Timeframe: Within the first 2 blinded cycles (3-week cycles), up to 6 weeks.Population: Intention to treat (ITT).
Median maximum score (0 to 10, with 10 being the worst) for participant-reported outcomes in the first 2 blinded cycles for Mouth Pain, Overall Mouth and Throat Soreness, and Rectal Soreness while median maximum score for Swallowing, Drinking and Eating Difficulty (0 to 4, with 4 being the difficult).
Outcome measures
| Measure |
Palifermin
n=32 Participants
Palifermin + Chemotherapy (AI or AP Regimen);
AI = Doxorubicin (Adriamycin) + Ifosfamide:
Palifermin 180 mcg/kg 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 intravenous (IV) for 72 hours starting Days 0 for total 90 mg/m\^2. Ifosfamide 2.5 g/m\^2 IV bolus Days 0-3 (total 10 g/m\^2); Vincristine 2 mg IV Day 0.
AP=Doxorubicin (Adriamycin) + Cisplatin:
Palifermin 180 mcg/kg 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 IV continuous infusion for 72 hours starting Day 0(total = 90 mg/m\^2); Cisplatin 120 mg/m\^2 on day 0.
|
Placebo
n=16 Participants
Placebo + Chemotherapy (AI or AP Regimen):
AI = Doxorubicin (Adriamycin) + Ifosfamide:
A single dose placebo 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 intravenous (IV) for 72 hours starting Days 0 for total 90 mg/m\^2. Ifosfamide 2.5 g/m\^2 IV bolus Days 0-3 (total 10 g/m\^2); Vincristine 2 mg IV Day 0.
AP=Doxorubicin (Adriamycin) + Cisplatin:
A single dose placebo 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 IV continuous infusion for 72 hours starting Day 0(total = 90 mg/m\^2); Cisplatin 120 mg/m\^2 on day 0.
|
|---|---|---|
|
Median Maximum Score for Patient Reported Outcomes in 2 Blinded Cycles
Overall Mouth and Throat Soreness
|
1 Units on a scale
Interval 0.0 to 9.0
|
5.5 Units on a scale
Interval 0.0 to 9.0
|
|
Median Maximum Score for Patient Reported Outcomes in 2 Blinded Cycles
Mouth Pain
|
1 Units on a scale
Interval 0.0 to 7.0
|
5 Units on a scale
Interval 0.0 to 10.0
|
|
Median Maximum Score for Patient Reported Outcomes in 2 Blinded Cycles
Rectal Soreness
|
0 Units on a scale
Interval 0.0 to 9.0
|
4 Units on a scale
Interval 0.0 to 10.0
|
|
Median Maximum Score for Patient Reported Outcomes in 2 Blinded Cycles
Drinking Difficulty
|
0 Units on a scale
Interval 0.0 to 3.0
|
1.5 Units on a scale
Interval 0.0 to 3.0
|
|
Median Maximum Score for Patient Reported Outcomes in 2 Blinded Cycles
Eating Difficulty
|
0 Units on a scale
Interval 0.0 to 4.0
|
2 Units on a scale
Interval 0.0 to 4.0
|
|
Median Maximum Score for Patient Reported Outcomes in 2 Blinded Cycles
Swallowing Difficulty
|
0 Units on a scale
Interval 0.0 to 3.0
|
2 Units on a scale
Interval 0.0 to 4.0
|
Adverse Events
Palifermin
Serious events: 0 serious events
Other events: 31 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Palifermin
n=32 participants at risk
Palifermin + Chemotherapy (AI or AP Regimen);
AI = Doxorubicin (Adriamycin) + Ifosfamide:
Palifermin 180 mcg/kg 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 intravenous (IV) for 72 hours starting Days 0 for total 90 mg/m\^2. Ifosfamide 2.5 g/m\^2 IV bolus Days 0-3 (total 10 g/m\^2); Vincristine 2 mg IV Day 0.
AP=Doxorubicin (Adriamycin) + Cisplatin:
Palifermin 180 mcg/kg 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 IV continuous infusion for 72 hours starting Day 0(total = 90 mg/m\^2); Cisplatin 120 mg/m\^2 on day 0.
|
Placebo
n=16 participants at risk
Placebo + Chemotherapy (AI or AP Regimen):
AI = Doxorubicin (Adriamycin) + Ifosfamide:
A single dose placebo 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 intravenous (IV) for 72 hours starting Days 0 for total 90 mg/m\^2. Ifosfamide 2.5 g/m\^2 IV bolus Days 0-3 (total 10 g/m\^2); Vincristine 2 mg IV Day 0.
AP=Doxorubicin (Adriamycin) + Cisplatin:
A single dose placebo 3 days prior to chemotherapy; Adriamycin 30 mg/m\^2 IV continuous infusion for 72 hours starting Day 0(total = 90 mg/m\^2); Cisplatin 120 mg/m\^2 on day 0.
|
|---|---|---|
|
General disorders
Increased thickness of oral mucosa
|
93.8%
30/32 • 2 years and 3 months.
Adverse events reported were found in the first two blinded cycles.
|
31.2%
5/16 • 2 years and 3 months.
Adverse events reported were found in the first two blinded cycles.
|
|
General disorders
Altered taste
|
62.5%
20/32 • 2 years and 3 months.
Adverse events reported were found in the first two blinded cycles.
|
25.0%
4/16 • 2 years and 3 months.
Adverse events reported were found in the first two blinded cycles.
|
|
General disorders
Flushing
|
50.0%
16/32 • 2 years and 3 months.
Adverse events reported were found in the first two blinded cycles.
|
18.8%
3/16 • 2 years and 3 months.
Adverse events reported were found in the first two blinded cycles.
|
|
General disorders
Film coating in mouth
|
53.1%
17/32 • 2 years and 3 months.
Adverse events reported were found in the first two blinded cycles.
|
18.8%
3/16 • 2 years and 3 months.
Adverse events reported were found in the first two blinded cycles.
|
|
General disorders
Increased sensitivity in tongue or gums
|
46.9%
15/32 • 2 years and 3 months.
Adverse events reported were found in the first two blinded cycles.
|
12.5%
2/16 • 2 years and 3 months.
Adverse events reported were found in the first two blinded cycles.
|
|
General disorders
Warm sensation
|
34.4%
11/32 • 2 years and 3 months.
Adverse events reported were found in the first two blinded cycles.
|
0.00%
0/16 • 2 years and 3 months.
Adverse events reported were found in the first two blinded cycles.
|
|
General disorders
Increased saliva
|
25.0%
8/32 • 2 years and 3 months.
Adverse events reported were found in the first two blinded cycles.
|
6.2%
1/16 • 2 years and 3 months.
Adverse events reported were found in the first two blinded cycles.
|
Additional Information
Saroj Vadhan-Raj, MD/Professor
UT MD Anderson Cancer Center
Phone: 713-792-7966
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place