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
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
  Hide Participant Flow

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
No text entered.

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
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

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
  Show All Outcome Measures

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

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

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

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

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

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 ]

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

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

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 ]

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

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) ]

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) ]

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) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide 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.


  More Information
  Hide More Information

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:
unchecked 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.
unchecked 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.


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