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Dose Escalation Study Of Palifermin in Pediatric Research Participants Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Primary Purpose

Oral Mucositis, Hematopoietic Stem Cell Transplantation

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Palifermin

About this trial

This is an interventional treatment trial for Oral Mucositis focused on measuring Mucositis, Oral, Hematopoietic Stem Cell Transplantation, Allogeneic Transplantation, Pharmacokinetics, Maximum Tolerated Dose

Eligibility Criteria

2 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Age greater than or equal to 2 years and less than 18 years of age.
Diagnosis of a hematologic malignancy.
Has not received a prior autologous or allogeneic HSCT.
Is scheduled to receive either a matched family member or MUD bone marrow stem cell graft at St. Jude Children's Research Hospital. A matched family member donor is defined as a sibling matched at 5 or 6 HLA loci or another related donor matched at 6 HLA loci.
Is scheduled to receive a myeloablative preparative regimen (cyclophosphamide/TBI based) prior to the infusion of the allogeneic graft.
Cardiac shortening fraction greater than or equal to 25%.
Serum creatinine is less than twice the upper limit of normal for age.
Bilirubin less than 3.0 mg/dl.
Aspartate transaminase (AST) less than 500 IU/ml.
Alanine transaminase (ALT) less than 500 IU/ml.
Amylase less than 1.5 times the upper limits of normal for age.
Lipase less than 1.5 times the upper limits of normal for age.
Forced vital capacity (FVC) greater than or equal to 40% of predicted value or pulse oximetry greater than or equal to 92% on room air.
No known hypersensitivity to E coli-derived proteins or palifermin.
No active or recent (within 30 days prior to enrollment) gastrointestinal bleeding.
No active or recent (within 30 days prior to enrollment) oral ulcerations.
No active fungal infection, bacteremia or viremia within two weeks prior to enrollment.

Exclusion Criteria:

Female - pregnant (negative serum or urine pregnancy test within 14 days prior to enrollment).
Female - lactating.

Sites / Locations

St. Jude Children's Research Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

Dose Level 1

Dose Level 2

Dose Level 3

Arm Description

Palifermin 40 mcg/kg/day intravenous

Palifermin 60 mcg/kg/day intravenous

Palifermin 90 mcg/kg/day intravenous

Outcomes

Primary Outcome Measures

To determine the maximum tolerated dose and describe the toxicity profile of palifermin in pediatric research participants with hematologic malignancies undergoing first HSCT.

Secondary Outcome Measures

Full Information

NCT ID

NCT00701688

First Posted

June 17, 2008

Last Updated

March 22, 2012

Sponsor

St. Jude Children's Research Hospital

1. Study Identification

Unique Protocol Identification Number

NCT00701688

Brief Title

Dose Escalation Study Of Palifermin in Pediatric Research Participants Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Official Title

Phase I Study Of The Use Of Palifermin in Pediatric Research Participants Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Study Type

Interventional

2. Study Status

Record Verification Date

March 2012

Overall Recruitment Status

Completed

Study Start Date

September 2007 (undefined)

Primary Completion Date

September 2011 (Actual)

Study Completion Date

February 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

St. Jude Children's Research Hospital

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

Mucositis is a well-known complication of both autologous and allogeneic hematopoietic stem cell transplantation (HSCT). Many who suffer this disorder require total parental nutrition and intravenous narcotics for pain control. Palifermin (Kepivance[TM]) is a human keratinocyte growth factor that is produced by recombinant DNA technology in E. coli. Palifermin is a FDA-approved, commercially available pharmacologic agent that is manufactured by Amgen. As keratinocyte growth factor receptors have been found within the epithelium of gastric mucosa, the use of palifermin has been proven to decrease the frequency and duration of severe mucositis in adult studies. Whereas the appropriate dosing regimen has been determined for adults at 60mcg/kg/day, the dosing of palifermin has not been established in the pediatric setting. This initial pediatric study of palifermin will determine the maximum tolerated dose, evaluating the use of this agent at three dose levels, below, at, and above the recommended adult dose. Non-hematologic, life-threatening NCI grade IV or grade V toxicities definitely related to the administration of palifermin from the first infusion until day +6 after HSCT (post palifermin administration day +3) will comprise the safety endpoints of the study. The study is designed to evaluate palifermin at 3 dose levels. The study population will be recipients of either a matched family member donor or matched unrelated donor HSCT. The pharmacokinetics of palifermin at each dose level will be described to help determine the appropriate dose for future studies, which will evaluate efficacy

Detailed Description

Secondary objectives of this study include exploring the pharmacoeconomics of palifermin in this particular patient population through an assessment of inpatient days, intravenous nutrition, and analgesia requirements within 100 days post-transplantation. We will also explore the research participants' rates of immune reconstitution, specifically T and B-lymphocytes and NK cells, within the first year of HSCT.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Oral Mucositis, Hematopoietic Stem Cell Transplantation

Keywords

Mucositis, Oral, Hematopoietic Stem Cell Transplantation, Allogeneic Transplantation, Pharmacokinetics, Maximum Tolerated Dose

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

19 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Dose Level 1

Arm Type

Experimental

Arm Description

Palifermin 40 mcg/kg/day intravenous

Arm Title

Dose Level 2

Arm Type

Experimental

Arm Description

Palifermin 60 mcg/kg/day intravenous

Arm Title

Dose Level 3

Arm Type

Experimental

Arm Description

Palifermin 90 mcg/kg/day intravenous

Intervention Type

Drug

Intervention Name(s)

Palifermin

Other Intervention Name(s)

Kepivance®

Intervention Description

Dose level 1 40 mcg/kg/day intravenous Dose level 2 60 mcg/kg/day intravenous Dose level 3 90 mcg/kg/day intravenous

Primary Outcome Measure Information:

Title

To determine the maximum tolerated dose and describe the toxicity profile of palifermin in pediatric research participants with hematologic malignancies undergoing first HSCT.

Time Frame

30 days

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Age greater than or equal to 2 years and less than 18 years of age. Diagnosis of a hematologic malignancy. Has not received a prior autologous or allogeneic HSCT. Is scheduled to receive either a matched family member or MUD bone marrow stem cell graft at St. Jude Children's Research Hospital. A matched family member donor is defined as a sibling matched at 5 or 6 HLA loci or another related donor matched at 6 HLA loci. Is scheduled to receive a myeloablative preparative regimen (cyclophosphamide/TBI based) prior to the infusion of the allogeneic graft. Cardiac shortening fraction greater than or equal to 25%. Serum creatinine is less than twice the upper limit of normal for age. Bilirubin less than 3.0 mg/dl. Aspartate transaminase (AST) less than 500 IU/ml. Alanine transaminase (ALT) less than 500 IU/ml. Amylase less than 1.5 times the upper limits of normal for age. Lipase less than 1.5 times the upper limits of normal for age. Forced vital capacity (FVC) greater than or equal to 40% of predicted value or pulse oximetry greater than or equal to 92% on room air. No known hypersensitivity to E coli-derived proteins or palifermin. No active or recent (within 30 days prior to enrollment) gastrointestinal bleeding. No active or recent (within 30 days prior to enrollment) oral ulcerations. No active fungal infection, bacteremia or viremia within two weeks prior to enrollment. Exclusion Criteria: Female - pregnant (negative serum or urine pregnancy test within 14 days prior to enrollment). Female - lactating.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Ashok Srinivasan, MD

Organizational Affiliation

St. Jude Children's Research Hospital

Official's Role

Principal Investigator

Facility Information:

Facility Name

St. Jude Children's Research Hospital

City

Memphis

State/Province

Tennessee

ZIP/Postal Code

38105

Country

United States

12. IPD Sharing Statement

Links:

URL

http://www.stjude.org

Description

St. Jude Children's Research Hospital

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Dose Escalation Study Of Palifermin in Pediatric Research Participants Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

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