Supersaturated Calcium Phosphate Rinse in Preventing Oral Mucositis in Young Patients Undergoing Autologous or Donor Stem Cell Transplant

Children's Oncology Group

Status and phase: Completed; Phase 3

Conditions: Chronic Neutrophilic Leukemia; Chronic Eosinophilic Leukemia; Recurrent Childhood Acute Lymphoblastic Leukemia; Previously Treated Myelodysplastic Syndromes; Relapsing Chronic Myelogenous Leukemia; Childhood Myelodysplastic Syndromes; Mucositis; Recurrent Childhood Acute Myeloid Leukemia; Recurrent/Refractory Childhood Hodgkin Lymphoma; Chronic Myelomonocytic Leukemia; Childhood Chronic Myelogenous Leukemia; de Novo Myelodysplastic Syndromes; Childhood Acute Myeloid Leukemia in Remission; Recurrent Malignant Testicular Germ Cell Tumor; Juvenile Myelomonocytic Leukemia; Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable; Secondary Myelodysplastic Syndromes; Recurrent Wilms Tumor and Other Childhood Kidney Tumors; Disseminated Neuroblastoma; Childhood Acute Lymphoblastic Leukemia in Remission; Recurrent Childhood Lymphoblastic Lymphoma; Secondary Acute Myeloid Leukemia; Recurrent Childhood Large Cell Lymphoma; Recurrent Childhood Small Noncleaved Cell Lymphoma; Previously Treated Childhood Rhabdomyosarcoma; Unspecified Childhood Solid Tumor, Protocol Specific; Recurrent Childhood Rhabdomyosarcoma

Treatments: Other: placebo; Other: questionnaire administration; Procedure: quality-of-life assessment; Drug: supersaturated calcium phosphate rinse

Study type: Interventional

Funder types: NETWORK; NIH

Identifiers

NCT01305200

NCI-2011-02635 (Registry Identifier)

CDR0000695718 (Other Identifier)

COG-ACCL1031 (Other Identifier)

U10CA095861 (U.S. NIH Grant/Contract)

ACCL1031 (Other Identifier)

Details and patient eligibility

About

This randomized phase III trial is studying how well Caphosol rinse works in preventing mucositis in young patients undergoing autologous or donor stem cell transplant. Supersaturated calcium phosphate (Caphosol) rinse may be able to prevent mucositis, or mouth sores, in patients undergoing stem cell transplant.

Full description

PRIMARY OBJECTIVES:

I. To determine if topically administered supersaturated calcium phosphate (Caphosol), rinsed orally four times daily at the initiation of conditioning for hematopoietic stem cell transplantation (HSCT), reduces oral mucositis as demonstrated by a decrease in duration of severe oral mucositis (World Health Organization [WHO] grade 3 or 4), compared to placebo.

SECONDARY OBJECTIVES:

I. To determine whether Caphosol administration, when compared to placebo, reduces oral mucositis as demonstrated by a decrease in incidence of severe oral mucositis (WHO grade 3 or 4); severity of mucositis according to mouth pain categorical rating scale and Oral Mucositis Daily Questionnaire (OMDQ); incidence, total dose, and duration of parenteral opioid analgesic use (morphine equivalents); and incidence and duration of total parenteral nutrition (TPN) administration.

II. To determine whether Caphosol administration, when compared to placebo, reduces the incidence of febrile neutropenia and invasive bacterial infections.

III. To validate a new pediatric measure of oral mucositis termed the Children's International Mucositis Evaluation Scale (ChIMES).

OUTLINE: This is a multicenter study. Patients are stratified according to conditioning regimen (total-body irradiation (TBI) or melphalan vs neither TBI nor melphalan) and hematopoietic stem cell transplantation (HSCT) (autologous vs allogeneic). Patients are randomized to 1 of 2 treatment arms.

ARM I: Patients rinse and gargle with supersaturated calcium phosphate rinse over 1 minute four* times daily (QID) beginning on the first day (about day -7) of the conditioning regimen.

ARM II: Patients rinse and gargle with placebo over 1 minute QID* beginning the first day (about day -7) of the conditioning regimen.

NOTE: * Patients who reach WHO grade 3 or 4 mucositis have the option to request a total of 6 rinses daily.

In both arms, treatment continues until day 20 post-transplantation OR until mucositis resolves to WHO grade =< 2 for two consecutive days OR on day 12 in patients who do not experience oral mucositis of at least WHO grade >= 1. Patients are assessed daily by trained healthcare professionals using the Oral Mucositis Daily Questionnaire (OMDQ), the Pain Rating Scale, the WHO Mucositis Scale, and the Children's International Mucositis Evaluation Scale (ChIMES) from day -1 and continuing until day 20. Patients are also observed for the incidence of total dose and duration of parenteral opioid analgesic use, duration of total parenteral nutrition (TPN) administration, febrile neutropenia, and invasive bacterial infections.

After completion of study therapy, patients are followed up for 30 days.

Enrollment: 226 patients

Sex: All

Ages: 4 to 21 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients undergoing myeloablative autologous or allogeneic hematopoietic stem cell transplantation (HSCT) for any indication

• One or more of the following donor stem cell sources (autologous or allogeneic):

  • Bone marrow
  • Placental blood (umbilical cord blood)
  • Cytokine-mobilized peripheral blood

• Patients eligible for allogeneic HSCT must have one of the following types of donor stem cells:

  • Human leukocyte antigen (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)

• Patients expecting to receive any type of myeloablative HSCT conditioning regimen are eligible

  • No non-myeloablative or reduced-intensity conditioning regimens

• Eligible patients must not have received palifermin within 30 days prior to enrollment

• Eligible patients must not have received prior treatment with Caphosol

Exclusion criteria

• Females of childbearing potential must have a negative pregnancy test; patients must agree to use an effective birth control method; lactating patients must agree not to nurse a child while on this trial

Trial design

Primary purpose: Supportive Care

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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226 participants in 2 patient groups, including a placebo group

Arm I (placebo)

Placebo Comparator group

Description:

Patients rinse and gargle with placebo over 1 minute QID beginning the first day (about day -7) of the conditioning regimen. Treatment continues until day 20 post-transplantation.

Treatment:

Other: questionnaire administration

Procedure: quality-of-life assessment

Other: placebo

Arm II (supersaturated calcium phosphate rinse)

Experimental group

Description:

Patients rinse and gargle with supersaturated calcium phosphate rinse over 1 minute QID beginning on the first day (about day -7) of the conditioning regimen. Treatment continues until day 20 post-transplantation.

Treatment:

Drug: supersaturated calcium phosphate rinse

Other: questionnaire administration

Procedure: quality-of-life assessment