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

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

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Key information

Conditions

Oral Mucositis Hematopoietic Stem Cell Transplantation Mouth Diseases Stomatitis Stomatognathic Diseases

Age range

2 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Palifermin

Drug

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

Other names: Kepivance®

Primary outcomes

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

Sponsors and collaborators

Lead sponsor

St. Jude Children's Research Hospital

Other

Registry information

Official study title

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

Important dates

Study start: 2007
Primary completion: 2011
Study completion: 2012
First posted: Jun 19, 2008
Registry last updated: Mar 23, 2012

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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