Palifermin for the Reduction of Oral Mucositis in Single-... | NCT00109031

Name	Class
Amgen	INDUSTRY

Palifermin for the Reduction of Oral Mucositis in Single-... | NCT00109031 | Trialant

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Palifermin 60 µg/kg for 3 days	Active Comparator	Palifermin 60 µg/kg plus placebo to match the total volume equivalent to a 180 µg/kg dose on the 3 days prior to fractionated total body irradiation (fTBI) and palifermin 60 µg/kg on Days 0, 1 and 2 after peripheral blood progenitor cell transplantation (PBPC). Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0.	Drug: palifermin Radiation: Total Body Irradiation Drug: Cyclophosphamide Drug: Etoposide Drug: Placebo
Palifermin 180 μg/kg on Day -1	Experimental	Palifermin 180 μg/kg on Day -1 and matched placebo on Days -2 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC. Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0.	Drug: palifermin Radiation: Total Body Irradiation Drug: Cyclophosphamide Drug: Etoposide Drug: Placebo
Palifermin 180 μg/kg on Day -2	Experimental	Palifermin 180 μg/kg on Day -2 and placebo on Days -1 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC. Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0.	Drug: palifermin Radiation: Total Body Irradiation Drug: Cyclophosphamide Drug: Etoposide Drug: Placebo
Palifermin 180 μg/kg on Day -3

Interventions

Name	Type	Description	Arm Group Labels	Other Names
palifermin	Drug	Administered as one daily intravenous bolus.	Palifermin 180 μg/kg on Day -1 Palifermin 180 μg/kg on Day -2 Palifermin 180 μg/kg on Day -3

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of Participants With Severe Oral Mucositis (WHO Grade 3 and 4)	Participants underwent evaluations of oral mucosal (OM) surfaces (mucositis assessments) daily during hospitalization and daily thereafter until severe OM returned to grade ≤ 2. A trained evaluator documented the findings using the World Health Organization (WHO) oral toxicity scale according to the following: Grade 0 = None; Grade 1 = Soreness, erythema; Grade 2 = Erythema, ulcers, ability to eat solids; Grade 3 = Ulcers, requires liquid diet; Grade 4 = Alimentation not possible.	Up to Day 28

Secondary Outcomes

Measure	Description	Time Frame
Duration of Severe Oral Mucositis (WHO Grade 3 and 4)	The duration of severe oral mucositis (OM) was calculated as the number of days from the onset of severe OM (first time a WHO grade 3 or 4 was observed) to the day when severe OM was resolved (first time WHO grade 2 or less was observed after last WHO grade 3 or 4). Durations of 0 days were assigned to those participants who did not experience any WHO grade 3 or 4 during the study.

Name	Affiliation	Role
MD	Amgen	Study Director

Arm/Group Information

ID	Title	Description
FG000	Palifermin 60 µg/kg for 3 Days	Palifermin 60 µg/kg plus placebo to match the total volume equivalent to a 180 µg/kg dose on the 3 days prior to fractionated total body irradiation (fTBI) and palifermin 60 µg/kg on Days 0, 1 and 2 after peripheral blood progenitor cell transplantation (PBPC). Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0.
FG001	Palifermin 180 μg/kg on Day -1	Palifermin 180 μg/kg on Day -1 and matched placebo on Days -2 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC. Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0.
FG002	Palifermin 180 μg/kg on Day -2	Palifermin 180 μg/kg on Day -2 and placebo on Days -1 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC. Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0.
FG003	Palifermin 180 μg/kg on Day -3	Palifermin 180 μg/kg on Day -3 and placebo on Days -1 and -2 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC. Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0.

Periods

Title

Milestones

Reasons Not Completed

Overall Study

Type	Comment	Milestone Data
STARTED		FG00011 subjects FG00110 subjects FG00214 subjects FG00312 subjects
COMPLETED		FG00011 subjects FG0018 subjects FG00212 subjects FG00312 subjects
NOT COMPLETED		FG0000 subjects FG0012 subjects FG0022 subjects FG0030 subjects

Type	Comment	Reasons
Adverse Event		FG0000 subjects FG0010 subjects FG0021 subjects FG003

Baseline Characteristics Module

Arm/Group Information

ID	Title	Description
BG000	Palifermin 60 µg/kg for 3 Days	Palifermin 60 µg/kg on the 3 days prior to fractionated total body irradiation (fTBI) and palifermin 60 µg/kg on Days 0, 1 and 2 after peripheral blood progenitor cell transplantation (PBPC).
BG001	Palifermin 180 μg/kg on Day -1

Units	Counts
Participants	BG000

Title	Description	Population Description	Parameter Type	Dispersion Type	Unit of Measure	Calculate Percentage	Denominator Units Selected	Denominators	Classes
Age, Continuous			Mean

Outcome Measures Module

Outcome Measures

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Number of Participants With Severe Oral Mucositis (WHO Grade 3 and 4)

Participants underwent evaluations of oral mucosal (OM) surfaces (mucositis assessments) daily during hospitalization and daily thereafter until severe OM returned to grade ≤ 2. A trained evaluator documented the findings using the World Health Organization (WHO) oral toxicity scale according to the following: Grade 0 = None; Grade 1 = Soreness, erythema; Grade 2 = Erythema, ulcers, ability to eat solids; Grade 3 = Ulcers, requires liquid diet; Grade 4 = Alimentation not possible.

Primary analysis set

Posted

Number

participants

Up to Day 28

ID	Title	Description
OG000	Palifermin 60 µg/kg for 3 Days	Palifermin 60 µg/kg on the 3 days prior to fractionated total body irradiation (fTBI) and palifermin 60 µg/kg on Days 0, 1 and 2 after peripheral blood progenitor cell transplantation (PBPC).

Adverse Events Module

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Palifermin 60 µg/kg for 3 Days (A)	Palifermin 60 µg/kg on the 3 days prior to fractionated total body irradiation (fTBI) and palifermin 60 µg/kg on Days 0, 1 and 2 after peripheral blood progenitor cell transplantation (PBPC).

Term	Organ System	Source Vocabulary	Assessment Type	Notes	Statistical Information
Febrile neutropenia	Blood and lymphatic system disorders	MedDRA 8.0	Systematic Assessment

Term	Organ System	Source Vocabulary	Assessment Type	Notes	Statistical Information
Anaemia	Blood and lymphatic system disorders	MedDRA 8.0	Systematic Assessment

More Info Module

Certain Agreements

Title	Organization	Phone	Extension	Email
Hans Olivecrona, MD PhD	Swedish Orphan Biovitrum	+46 8 697 20 00		hans.olivecrona@sobi.com

Condition Browse Module

ID	Term
D009369	Neoplasms
D008223	Lymphoma
D007938	Leukemia

ID	Term
D009370	Neoplasms by Histologic Type
D008232	Lymphoproliferative Disorders
D008206	Lymphatic Diseases
D006425	Hemic and Lymphatic Diseases

Intervention Browse Module

ID	Term
D051523	Fibroblast Growth Factor 7
D014916	Whole-Body Irradiation
D003520	Cyclophosphamide
D005047	Etoposide

ID	Term
D005346	Fibroblast Growth Factors
D036341	Intercellular Signaling Peptides and Proteins
D010455	Peptides
D000602	Amino Acids, Peptides, and Proteins

Race/Ethnicity, Customized

Number

participants

Title

Denominators

Categories

White or Caucasian

Title	Measurements
	BG00011 BG0018 BG00212 BG00310 BG00441

Black or African American

Title	Measurements
	BG0000 BG0012 BG0021 BG003

Hispanic or Latino

Title	Measurements
	BG0000 BG0010 BG0020 BG003

Type of diagnosis

Acute lymphoblastic leukemia., Acute myelogenous leukemia, Chronic lymphocytic leukemia, and1 ( 8) Chronic myelogenous leukemia are subsets of Leukemia.

Number

participants

Title

Denominators

Categories

Hodgkin's disease

Title	Measurements
	BG0000 BG0011 BG0024 BG0034 BG0049

Non-Hodgkin's lymphoma

Title	Measurements
	BG0009 BG0019 BG0029 BG003

Multiple Myeloma

Title	Measurements
	BG0000 BG0010 BG0020 BG003

Leukemia

Title	Measurements
	BG0002 BG0010 BG0020 BG003

Acute lymphoblastic leukemia

Title	Measurements
	BG0001 BG0010 BG0020 BG003

Acute myelogenous leukemia

Title	Measurements
	BG0001 BG0010 BG0020 BG003

Chronic lymphocytic leukemia

Title	Measurements
	BG0000 BG0010 BG0020 BG003

Chronic myelogenous leukemia

Title	Measurements
	BG0000 BG0010 BG0020 BG003

Number of days of total body irradiation

Data are reported for the safety analysis set, as treated. Seven participants received the wrong sequence of placebo and palifermin in error in. Consequently, 10, 11, 13, and 12 participants actually received treatment according in each treatment arm respectively.

Number

participants

Title

Denominators

Categories

≤ 2 days

Title	Measurements
	BG0000 BG0011 BG0021 BG0030 BG0042

3 days

Title	Measurements
	BG0004 BG0014 BG0026 BG003

4 days

Title	Measurements
	BG0006 BG0016 BG0026 BG003

≥ 5 days

Title	Measurements
	BG0000 BG0010 BG0020 BG003

Secondary

Duration of Severe Oral Mucositis (WHO Grade 3 and 4)

The duration of severe oral mucositis (OM) was calculated as the number of days from the onset of severe OM (first time a WHO grade 3 or 4 was observed) to the day when severe OM was resolved (first time WHO grade 2 or less was observed after last WHO grade 3 or 4). Durations of 0 days were assigned to those participants who did not experience any WHO grade 3 or 4 during the study.

Primary analysis set

Posted

Mean

Standard Deviation

days

Up to Day 28

ID	Title	Description
OG000	Palifermin 60 µg/kg for 3 Days	Palifermin 60 µg/kg on the 3 days prior to fractionated total body irradiation (fTBI) and palifermin 60 µg/kg on Days 0, 1 and 2 after peripheral blood progenitor cell transplantation (PBPC).
OG001	Palifermin 180 μg/kg on Day -1	Palifermin 180 μg/kg on Day -1 and placebo on Days -2 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.
OG002	Palifermin 180 μg/kg on Day -2	Palifermin 180 μg/kg on Day -2 and placebo on Days -1 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.
OG003	Palifermin 180 μg/kg on Day -3	Palifermin 180 μg/kg on Day -3 and placebo on Days -1 and -2 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.

Units	Counts
Participants	OG00011 OG00110 OG00213 OG003

Title

Denominators

Categories

Title	Measurements
	OG0006.0± 4.1 OG0014.4± 5.4 OG0021.9± 3.6 OG003

Secondary

Area Under the Curve (AUC) of Mouth and Throat Soreness Score

The Oral Mucositis Daily Questionnaire (OMDQ) is a self-reported tool that evaluates overall health, mouth and throat soreness (MTS) and activity limitations due to MTS. The OMDQ was completed once daily beginning with the first day of study drug administration through Day 28. The area under the curve of mouth and throat soreness score was assessed from the question "How much mouth and throat soreness did you experience in the past 24 hours?" Participants answered on a scale from 0 (no soreness) to 4 (extreme soreness). A higher value in MTS AUC indicates worse self-assessed MTS.

Primary analysis set with available MTS data

Posted

Mean

Standard Deviation

MTS score * days

From the first day of study drug administration through Day 28

ID	Title	Description
OG000	Palifermin 60 µg/kg for 3 Days	Palifermin 60 µg/kg on the 3 days prior to fractionated total body irradiation (fTBI) and palifermin 60 µg/kg on Days 0, 1 and 2 after peripheral blood progenitor cell transplantation (PBPC).
OG001	Palifermin 180 μg/kg on Day -1	Palifermin 180 μg/kg on Day -1 and placebo on Days -2 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.
OG002	Palifermin 180 μg/kg on Day -2	Palifermin 180 μg/kg on Day -2 and placebo on Days -1 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.
OG003	Palifermin 180 μg/kg on Day -3	Palifermin 180 μg/kg on Day -3 and placebo on Days -1 and -2 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.

Units	Counts
Participants	OG00011 OG00110 OG00213 OG003

Title

Denominators

Categories

Title	Measurements
	OG00026.4± 22.4 OG00145.1± 14.4 OG00230.4± 18.6 OG003

Secondary

Number of Participants With Parenteral or Transdermal Opioid Analgesic Use

Includes nonprophylactic intravenous opioid analgesics (fentanyl, morphine, morphine sulphate, hydromorphone, meperidine) and transdermal opioid analgesics (fentanyl patch) for the indication of oral mucositis and dysphagia.

Primary analysis set

Posted

Number

participants

Up to Day 28

ID	Title	Description
OG000	Palifermin 60 µg/kg for 3 Days	Palifermin 60 µg/kg on the 3 days prior to fractionated total body irradiation (fTBI) and palifermin 60 µg/kg on Days 0, 1 and 2 after peripheral blood progenitor cell transplantation (PBPC).
OG001	Palifermin 180 μg/kg on Day -1	Palifermin 180 μg/kg on Day -1 and placebo on Days -2 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.
OG002	Palifermin 180 μg/kg on Day -2	Palifermin 180 μg/kg on Day -2 and placebo on Days -1 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.
OG003	Palifermin 180 μg/kg on Day -3	Palifermin 180 μg/kg on Day -3 and placebo on Days -1 and -2 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.

Units	Counts
Participants	OG00011 OG00110 OG00213 OG003

Title

Denominators

Categories

Yes

Title	Measurements
	OG0007 OG0017 OG0027 OG003

Secondary

Number of Participants With WHO Grades 2, 3 or 4 Oral Mucositis

Participants underwent evaluations of oral mucosal (OM) surfaces (mucositis assessments) daily during hospitalization and daily thereafter until OM returned to grade ≤ 2. A trained evaluator documented the findings using the World Health Organization (WHO) oral toxicity scale according to the following: Grade 0 = None; Grade 1 = Soreness, erythema; Grade 2 = Erythema, ulcers, ability to eat solids; Grade 3 = Ulcers, requires liquid diet; Grade 4 = Alimentation not possible.

Primary analysis set

Posted

Number

participants

Up to Day 28

ID	Title	Description
OG000	Palifermin 60 µg/kg for 3 Days	Palifermin 60 µg/kg on the 3 days prior to fractionated total body irradiation (fTBI) and palifermin 60 µg/kg on Days 0, 1 and 2 after peripheral blood progenitor cell transplantation (PBPC).
OG001	Palifermin 180 μg/kg on Day -1	Palifermin 180 μg/kg on Day -1 and placebo on Days -2 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.
OG002	Palifermin 180 μg/kg on Day -2	Palifermin 180 μg/kg on Day -2 and placebo on Days -1 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.
OG003	Palifermin 180 μg/kg on Day -3	Palifermin 180 μg/kg on Day -3 and placebo on Days -1 and -2 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.

Units	Counts
Participants	OG00011 OG00110 OG00213 OG003

Title

Denominators

Categories

Yes

Title	Measurements
	OG00010 OG0018 OG0029 OG003

Secondary

Duration of WHO Grade 2, 3 or 4 Oral Mucositis

The duration of grade 2, 3 or 4 oral mucositis (OM) was calculated as the number of days from the onset of grade 2, 3 or 4 OM (first time a WHO grade 2, 3 or 4 was observed) to the day when WHO grade 2 - 4 OM was resolved (first time WHO grade less than 2 was observed after last WHO grade 2, 3 or 4). Durations of 0 days were assigned to those participants who did not experience any WHO grade 2, 3 or 4 during the study.

OM was evaluated using the World Health Organization (WHO) oral toxicity scale according to the following: Grade 0 = None; Grade 1 = Soreness, erythema; Grade 2 = Erythema, ulcers, ability to eat solids; Grade 3 = Ulcers, requires liquid diet; Grade 4 = Alimentation not possible.

Primary analysis set with available OM assessment data

Posted

Mean

Standard Deviation

days

Up to Day 28

ID	Title	Description
OG000	Palifermin 60 µg/kg for 3 Days	Palifermin 60 µg/kg on the 3 days prior to fractionated total body irradiation (fTBI) and palifermin 60 µg/kg on Days 0, 1 and 2 after peripheral blood progenitor cell transplantation (PBPC).
OG001	Palifermin 180 μg/kg on Day -1	Palifermin 180 μg/kg on Day -1 and placebo on Days -2 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.
OG002	Palifermin 180 μg/kg on Day -2	Palifermin 180 μg/kg on Day -2 and placebo on Days -1 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.
OG003	Palifermin 180 μg/kg on Day -3	Palifermin 180 μg/kg on Day -3 and placebo on Days -1 and -2 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.

Units	Counts
Participants	OG00011 OG0019 OG00213 OG003

Title

Denominators

Categories

Title	Measurements
	OG00010.3± 4.9 OG0019.0± 8.2 OG0024.7± 4.2 OG003

Secondary

Number of Participants With WHO Grade 4 Oral Mucositis

Primary analysis set

Posted

Number

participants

Up to Day 28

ID	Title	Description
OG000	Palifermin 60 µg/kg for 3 Days	Palifermin 60 µg/kg on the 3 days prior to fractionated total body irradiation (fTBI) and palifermin 60 µg/kg on Days 0, 1 and 2 after peripheral blood progenitor cell transplantation (PBPC).
OG001	Palifermin 180 μg/kg on Day -1	Palifermin 180 μg/kg on Day -1 and placebo on Days -2 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.
OG002	Palifermin 180 μg/kg on Day -2	Palifermin 180 μg/kg on Day -2 and placebo on Days -1 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.
OG003	Palifermin 180 μg/kg on Day -3	Palifermin 180 μg/kg on Day -3 and placebo on Days -1 and -2 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC.

Units	Counts
Participants	OG00011 OG00110 OG00213 OG003

Title

Denominators

Categories

Yes

Title	Measurements
	OG0005 OG0015 OG0022 OG003