Palifermin in Preventing Oral Mucositis Caused by Chemotherapy and/or Radiation Therapy in Young Patients Undergoing Stem Cell Transplant

NCT00728585 | Withdrawn Phase 2

• 4 other identifiers: ACCL0521NCI-2009-00329CDR0000588622U10CA095861
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This randomized phase II trial is studying palifermin to see how well it works compared with a placebo in preventing oral mucositis caused by chemotherapy and/or radiation therapy in young patients undergoing stem cell transplant. Palifermin may help relieve or prevent oral mucositis caused by chemotherapy and radiation therapy in young patients undergoing stem cell transplant.

Conditions

Gastrointestinal Mucositis; Malignant Neoplasm

Outcome Measures

Primary Outcomes (1)

• Incidence of WHO grade 3 or 4 oral mucositis

  The incidence of WHO grade 3 or 4 mucositis, the palifermin and placebo groups, will be compared using a generalized Cochran-Mantel-Haenszel method for general association as the primary analysis. In addition, this outcome will be examined using a logistic regression model; both approaches will account for the randomization strata. Potential confounders will be examined using multiple logistic regression models.

  Up to day 32

Secondary Outcomes (10)

• Incidence of adverse events and laboratory abnormalities of palifermin according to using Common Terminology Criteria for Adverse Events (CTCAE) v 3.0

  Up to 100 days post-HSCT

• Long-term effects of palifermin on disease outcome and survival

  Up to 10 years

• Duration of WHO grade 3 or 4 oral mucositis

  Up to day 32

• Daily OMAS scores

  Up to day 32

• Daily modified Walsh mucositis scores

  Up to day 32

Study Arms (2)

Arm I (palifermin)

EXPERIMENTAL

Patients receive palifermin IV once daily for 3 days prior to chemotherapy and/or radiotherapy in the absence of unacceptable toxicity. Patients then receive palifermin IV on days 0, 1, and 2 after autologous or allogeneic hematopoietic stem cell transplantation.

Drug: Palifermin

Arm II (placebo)

PLACEBO COMPARATOR

Patients receive placebo IV once daily for 3 days prior to chemotherapy and/or radiotherapy in the absence of unacceptable toxicity. Patients then receive placebo IV on days 0, 1, and 2 after autologous or allogeneic hematopoietic stem cell transplantation.

Other: Placebo

Interventions

Palifermin DRUG

Given IV

Also known as: Growth Factor, Recombinant Human Keratinocyte, Kepivance, Keratinocyte Growth Factor, Recombinant Human, Recombinant Human Keratinocyte Growth Factor, rhKGF, rhu Keratinocyte Growth Factor

Arm I (palifermin)

Placebo OTHER

Given IV

Also known as: placebo therapy, PLCB, sham therapy

Arm II (placebo)

Eligibility Criteria

• Age: 1 Year - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

Criteria: * Patients undergoing myeloablative autologous or allogeneic hematopoietic stem cell transplantation (HSCT) for any indication * Any type of myeloablative HSCT conditioning regimen allowed * Patients undergoing allogeneic HSCT may undergo 1 of the following types of donor stem cells: 1. HLA-matched sibling or parent 2. Partially matched family donor (mismatched for a single HLA locus \[class I\]) 3. Fully matched unrelated marrow or peripheral blood stem cell donor 4. HLA-matched or partially mismatched (at least 4 of 6 match) cord blood (class I or II) * Fertile patients must use effective contraception * No HIV positivity * No known sensitivity to any E. coli-derived products 1. Known grade 1 to 2 allergic reactions to asparaginase allowed 2. No prior grade 3-4 allergies to asparaginase or pegaspargase * More than 30 days since prior and no concurrent treatment with any of the following therapies: 1. Oral cryotherapy 2. Glutamine as an oral supplement 3. Traumeel 4. Gelclair 5. Oral vancomycin paste 6. Low-level laser therapy 7. An investigational product or device in another clinical trial * No prior palifermin or other keratinocyte growth factors * No other concurrent cytotoxic drugs for conditioning or graft-vs-host disease prophylaxis (intrathecal methotrexate or cytarabine for CNS involvement allowed) * Not pregnant or nursing

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Children's Oncology Group: lead
• National Cancer Institute (NCI): collaborator

MeSH Terms

Conditions

Mucositis; Neoplasms

Interventions

Fibroblast Growth Factor 7

Condition Hierarchy (Ancestors)

Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Mouth Diseases; Stomatognathic Diseases

Intervention Hierarchy (Ancestors)

Fibroblast Growth Factors; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Lillian Sung

  Children's Oncology Group

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 5, 2008
• First Posted: August 6, 2008
• Study Start: March 13, 2008
• Primary Completion: May 8, 2009
• Last Updated: August 7, 2018

Record last verified: 2013-04