A Phase 1 Dose-escalation Study to Evaluate the Safety and Pharmacokinetics (PK) of Palifermin in Subjects With Acute Leukemias Undergoing HSCT

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Swedish Orphan Biovitrum

ClinicalTrials.gov Identifier:

NCT00460421

First received: April 12, 2007

Last updated: July 13, 2012

Last verified: July 2012

History of Changes

Results First Received: May 30, 2012

Study Type:	Interventional
Study Design:	Endpoint Classification: Safety Study; Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Prevention
Condition:	Leukemia
Intervention:	Drug: Palifermin

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
This phase 1 dose-escalation study to evaluate the safety and pharmacokinetics (PK) of palifermin in pediatric subjects with acute leukemias undergoing myeloblative therapy and allogeneic hematopoietic stem cell transplant (HSCT) was performed in 7 centers in USA between 2006 and 2011.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
Palifermin 40 µg/kg/Day	Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
Palifermin 60 µg/kg/Day	Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
Palifermin 80 µg/kg/Day	Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts

Participant Flow: Overall Study

	Palifermin 40 µg/kg/Day	Palifermin 60 µg/kg/Day	Palifermin 80 µg/kg/Day
STARTED	9	9	9
COMPLETED	9	9	9
NOT COMPLETED	0	0	0

Baseline Characteristics

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Reporting Groups

	Description
Palifermin 40 µg/kg/Day	Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
Palifermin 60 µg/kg/Day	Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
Palifermin 80 µg/kg/Day	Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
Total	Total of all reporting groups

Baseline Measures

	Palifermin 40 µg/kg/Day	Palifermin 60 µg/kg/Day	Palifermin 80 µg/kg/Day	Total
Number of Participants [units: participants]	9	9	9	27
Age [units: participants]
<=18 years	9	9	9	27
Between 18 and 65 years	0	0	0	0
>=65 years	0	0	0	0
Age [units: years] Mean ± Standard Deviation	7.4 ± 6.3	7.4 ± 5.6	8.0 ± 6.6	7.6 ± 5.9
Gender [units: participants]
Female	3	4	5	12
Male	6	5	4	15
Region of Enrollment [units: participants]
United States	9	9	9	27

Outcome Measures

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1. Primary:

Incidence of Dose Limiting Toxicities (DLTs) [ Time Frame: Approximately 1 month duration (Day -10 through Day +16) ]

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2. Secondary:

Incidence of Serum Palifermin Antibody Formation [ Time Frame: Approximately 4 month duration (Through Day + 100 (+/- 40 days)) ]

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3. Secondary:

Incidence of Severe Adverse Events (AEs) [ Time Frame: Approximately 1 1/2 months duration (Through Day +30/End of Treatment) ]

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4. Secondary:

Incidence of Laboratory Abnormalities [ Time Frame: Approximately 1 1/2 months duration (Through Day +30/End of Treatment) ]

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5. Secondary:

Pharmacokinetics of Palifermin, Clearence (CL) After the 1st Intravenous (IV) Bolus Injection for Multiple Dose Levels [ Time Frame: Day -10 ]

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6. Secondary:

Pharmacokinetics of Palifermin, Volume of Distribution at Steady State (Vss) After the 1st IV Bolus Injection for Multiple Dose Levels [ Time Frame: Day -10 ]

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7. Secondary:

Pharmacokinetics of Palifermin, Terminal Half-life (t½,z) After the 1st IV Bolus Injection for Multiple Dose Levels [ Time Frame: Day -10 ]

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8. Secondary:

Pharmacokinetics of Palifermin, t½,z After the 3rd IV Bolus Injection for Multiple Dose Levels [ Time Frame: Day -8 ]

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9. Secondary:

Pharmacokinetics of Palifermin, Area Under the Concentration Time Curve From Zero to the End of the Dosing Interval (AUCtau) After the 1st IV Bolus Injection for Multiple Dose Levels [ Time Frame: Day -10 ]

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10. Secondary:

Pharmacokinetics of Palifermin, AUCtau After the 3rd IV Bolus Injection for Multiple Dose Levels [ Time Frame: Day -8 ]

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11. Secondary:

Long-Term Follow-Up: Incidence of Secondary Malignancies [ Time Frame: Up to 4 years duration (Assessments performed on months 6, 9, 12 (+/- 30 Days) for the first year and then annually) ]

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12. Secondary:

Long-Term Follow-Up: Progression Free Survival [ Time Frame: Up to 4 years duration (Assessments performed on months 6, 9, 12 (+/- 30 Days) for the first year and then annually) ]

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13. Secondary:

Long-Term Follow-Up: Overall Survival [ Time Frame: Up to 4 years duration (Assessments performed on months 6, 9, 12 (+/- 30 Days) for the first year and then annually) ]

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Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS 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.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: The Clinical Trial Agreement generally does not restrict an investigator's discussion of trial results after completion. The Agreement permits sponsor a limited period of time to review material discussing trial results (approximately up to 90 days). Sponsor may remove confidential information, but authors have final control and approval of publication content. For multi-center studies, investigator agrees not to publish any results before the first multi-center publication.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
The original protocol included a long term follow-up (LTFU) phase up to 10 years. This was reduced in a protocol amendment to up to the time when the last enrolled subject had completed the day +100 follow-up.

Results Point of Contact:

Name/Title: Medical Program Director
Organization: Swedish Orphan Biovitrum
phone: +46 8 697 20 00
e-mail: clinical@sobi.com

No publications provided

Responsible Party:	Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier:	NCT00460421 History of Changes
Obsolete Identifiers:	NCT00896506
Other Study ID Numbers:	20010133
Study First Received:	April 12, 2007
Results First Received:	May 30, 2012
Last Updated:	July 13, 2012
Health Authority:	United States: Food and Drug Administration United States: Institutional Review Board

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