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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


Phase 2
1 Year
16 Years
Not Enrolling
Both
Breast Cancer, Graft Versus Host Disease, Kidney Cancer, Leukemia, Lymphoma, Mucositis, Multiple Myeloma, Plasma Cell Neoplasm, Myelodysplastic Syndromes, Neuroblastoma, Ovarian Cancer, Sarcoma, Testicular Germ Cell Tumor

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Trial Information

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


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.

Inclusion 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

Type of Study:

Interventional

Study Design:

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Supportive Care

Outcome Measure:

Incidence of WHO grade 3 or 4 oral mucositis

Outcome Description:

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.

Outcome Time Frame:

Up to day 32

Safety Issue:

Yes

Principal Investigator

Lillian Sung, MD, PhD

Investigator Role:

Study Chair

Investigator Affiliation:

The Hospital for Sick Children

Authority:

Unspecified

Study ID:

ACCL0521

NCT ID:

NCT00728585

Start Date:

January 2009

Completion Date:

January 2009

Related Keywords:

  • Breast Cancer
  • Graft Versus Host Disease
  • Kidney Cancer
  • Leukemia
  • Lymphoma
  • Mucositis
  • Multiple Myeloma
  • Plasma Cell Neoplasm
  • Myelodysplastic Syndromes
  • Neuroblastoma
  • Ovarian Cancer
  • Sarcoma
  • Testicular Germ Cell Tumor
  • 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
  • stage I ovarian epithelial cancer
  • stage II ovarian epithelial cancer
  • stage III ovarian epithelial cancer
  • stage IV ovarian epithelial cancer
  • recurrent ovarian epithelial cancer
  • recurrent ovarian germ cell tumor
  • stage IIIA breast cancer
  • stage IIIB breast cancer
  • stage IIIC breast cancer
  • stage IV breast cancer
  • recurrent breast cancer
  • 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

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