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

Purpose

RATIONALE: Palifermin may help relieve or prevent oral mucositis caused by chemotherapy and radiation therapy in young patients undergoing stem cell transplant.

PURPOSE: 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.

Condition	Intervention	Phase
Breast Cancer Graft Versus Host Disease Kidney Cancer Leukemia Lymphoma Mucositis Multiple Myeloma and Plasma Cell Neoplasm Myelodysplastic Syndromes Neuroblastoma Ovarian Cancer Sarcoma Testicular Germ Cell Tumor	Biological: palifermin Other: placebo	Phase 2

Study Type:	Interventional
Study Design:	Allocation: Randomized Masking: Double-Blind Primary Purpose: Supportive Care
Official Title:	A Group-Wide Double-Blind Randomized Placebo-Controlled Trial of Palifermin to Prevent Chemotherapy and/or Radiotherapy Induced Oral Mucositis in Children Undergoing Autologous or Allogeneic Hematopoietic Stem Cell Transplantation

Resource links provided by NLM:

Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:

Incidence of WHO grade 3 or 4 oral mucositis [ Designated as safety issue: No ]

Secondary Outcome Measures:

Safety and tolerability of palifermin [ Designated as safety issue: Yes ]
Long-term disease outcome and survival [ Designated as safety issue: No ]
Duration of WHO grade 3 or 4 oral mucositis [ Designated as safety issue: No ]
Incidence, total dose, and duration of parenteral opioid analgesic use (morphine equivalents) [ Designated as safety issue: No ]
Incidence and duration of total parenteral nutrition administration [ Designated as safety issue: No ]
Incidence of febrile neutropenia and invasive bacterial infections [ Designated as safety issue: No ]
Incidence of WHO grade 3 or 4 oral mucositis among allogeneic HSCT patients receiving methotrexate as GVHD prophylaxis [ Designated as safety issue: No ]
Incidence of acute and chronic GVHD after allogeneic HSCT [ Designated as safety issue: No ]
Health care utilization [ Designated as safety issue: No ]

Estimated Enrollment:	200
Study Start Date:	January 2009
Estimated Primary Completion Date:	July 2011 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Arm I 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.	Biological: palifermin Given IV
Placebo Comparator: Arm II 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 Given IV

Detailed Description:

OBJECTIVES:

Primary

To compare whether palifermin versus placebo administered to pediatric patients three days prior to conditioning and three days after autologous or allogeneic hematopoietic stem cell transplantation (HSCT) is associated with a reduction in the incidence of WHO grade 3 or 4 oral mucositis.

Secondary

To evaluate the safety and tolerability of palifermin.
To evaluate the long-term effects of palifermin on disease outcome and survival.
To compare the incidence, total dose, and duration of parenteral opioid analgesic use (morphine equivalents), and incidence and duration of total parenteral nutrition (TPN) administration in patients treated with these regimens.
To compare the incidence of febrile neutropenia and invasive bacterial infections in patients treated with these regimens.
To determine whether palifermin versus placebo reduces the incidence of WHO grade 3 or 4 oral mucositis among allogeneic HSCT pediatric patients receiving methotrexate as graft-versus-host disease (GVHD) prophylaxis.
To determine whether palifermin versus placebo reduces acute and chronic GVHD after allogeneic HSCT.
To describe health care utilization (hospitalization duration, and administration of antibiotics, TPN, nasogastric-, nasojejunal- or gastrostomy-administered enteral nutrition, and blood products) in pediatric patients treated with these regimens.

OUTLINE: This is a multicenter study. Patients are stratified according to age in years (1 to 2 vs 3 to 11 vs 12 to 16), type of hematopoietic stem cell transplantation (HSCT) (autologous vs allogeneic), conditioning regimen (either total-body irradiation [TBI] or melphalan vs neither TBI nor melphalan). Patients are randomized to 1 of 2 treatment arms.

Arm I: 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 HSCT.
Arm II: 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 HSCT.

Blood samples are collected at baseline, 32 days, and 100 days after HSCT to evaluate the immunogenicity of palifermin. Oral mucositis is assessed at baseline, daily for 8 days prior to and 32 days after HSCT, or until oral mucositis has resolved by the WHO Mucositis Scale, Oral Mucositis Assessment Scale (OMAS), modified Walsh mucositis scale, Oral Mucositis Daily Questionnaire (OMDQ), and the pain categorical rating scale.

After completion of HSCT, patients are followed periodically for up to 10 years.

Eligibility

Ages Eligible for Study:	1 Year to 16 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

DISEASE CHARACTERISTICS:

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:
- HLA-matched sibling or parent
- Partially matched family donor (mismatched for a single HLA locus [class I])
- Fully matched unrelated marrow or peripheral blood stem cell donor
- HLA-matched or partially mismatched (at least 4 of 6 match) cord blood (class I or II)

PATIENT CHARACTERISTICS:

Not pregnant or nursing
Negative pregnancy test
Fertile patients must use effective contraception
No HIV positivity
No known sensitivity to any E. coli-derived products
- Known grade 1 to 2 allergic reactions to asparaginase allowed
- No prior grade 3-4 allergies to asparaginase or pegaspargase

PRIOR CONCURRENT THERAPY:

See Disease Characteristics
More than 30 days since prior and no concurrent treatment with any of the following therapies:
- Oral cryotherapy
- Glutamine as an oral supplement
- Traumeel®
- Gelclair®
- Oral vancomycin paste
- Low-level laser therapy
- 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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00728585

Sponsors and Collaborators

Children's Oncology Group

National Cancer Institute (NCI)

Investigators

Study Chair:

Lillian Sung, MD, PhD

The Hospital for Sick Children

More Information

No publications provided

ClinicalTrials.gov Identifier:	NCT00728585 History of Changes
Other Study ID Numbers:	CDR0000588622, COG-ACCL0521
Study First Received:	August 5, 2008
Last Updated:	May 26, 2009
Health Authority:	Unspecified

Keywords provided by National Cancer Institute (NCI):

mucositis
graft versus host disease
recurrent childhood large cell lymphoma
recurrent childhood lymphoblastic lymphoma
recurrent childhood small noncleaved cell lymphoma
recurrent/refractory childhood Hodgkin lymphoma
accelerated phase chronic myelogenous leukemia
blastic phase chronic myelogenous leukemia
childhood acute lymphoblastic leukemia in remission
childhood acute myeloid leukemia in remission
childhood chronic myelogenous leukemia
chronic phase chronic myelogenous leukemia
juvenile myelomonocytic leukemia
recurrent childhood acute lymphoblastic leukemia
recurrent childhood acute myeloid leukemia

secondary acute myeloid leukemia
childhood myelodysplastic syndromes
de novo myelodysplastic syndromes
disseminated neuroblastoma
previously treated childhood rhabdomyosarcoma
previously treated myelodysplastic syndromes
recurrent Wilms tumor and other childhood kidney tumors
recurrent childhood rhabdomyosarcoma
recurrent neuroblastoma
recurrent malignant testicular germ cell tumor
secondary myelodysplastic syndromes
stage I multiple myeloma
stage II multiple myeloma
stage III multiple myeloma
refractory multiple myeloma

Additional relevant MeSH terms:

Breast Neoplasms
Neoplasms
Carcinoma, Renal Cell
Kidney Neoplasms
Graft vs Host Disease
Leukemia
Lymphoma
Multiple Myeloma
Neoplasms, Plasma Cell
Plasmacytoma
Myelodysplastic Syndromes
Preleukemia
Neuroblastoma
Ovarian Neoplasms
Stomatitis

Neoplasms, Germ Cell and Embryonal
Mucositis
Sarcoma
Neoplasms by Site
Breast Diseases
Skin Diseases
Adenocarcinoma
Carcinoma
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Urologic Neoplasms
Urogenital Neoplasms
Kidney Diseases
Urologic Diseases
Immune System Diseases

ClinicalTrials.gov processed this record on May 19, 2013