Trial Information

Randomized Double Blinded Trial of Topical Caphosol to Prevent Oral Mucositis in Children Undergoing Hematopoietic Stem Cell Transplantation

OBJECTIVES:

Primary

- 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

- 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.

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

- 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 beginning on the first day (about day -7) of the conditioning
regimen.

- Arm II: Patients rinse and gargle with placebo over 1 minute four* times daily
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.