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

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.

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