Randomized Double Blinded Trial of Topical Caphosol to Prevent Oral Mucositis in Children Undergoing Hematopoietic Stem Cell Transplantation
- 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.
- 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
- 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.
Allocation: Randomized, Masking: Double-Blind, Primary Purpose: Supportive Care
Duration of severe oral mucositis (WHO Grade 3 or 4)
Lillian Sung, MD, PhD
The Hospital for Sick Children
|Children's Hospital of Philadelphia||Philadelphia, Pennsylvania 19104|
|Mayo Clinic Cancer Center||Rochester, Minnesota 55905|
|University of Chicago Cancer Research Center||Chicago, Illinois 60637|
|New York Medical College||Valhalla, New York 10595|
|Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill||Chapel Hill, North Carolina 27599-7570|
|Children's National Medical Center||Washington, District of Columbia 20010-2970|
|Miami Children's Hospital||Miami, Florida 33155-4069|
|All Children's Hospital||St. Petersburg, Florida 33701|
|Children's Memorial Hospital - Chicago||Chicago, Illinois 60614|
|Children's Hospital of New Orleans||New Orleans, Louisiana 70118|
|City of Hope Comprehensive Cancer Center||Duarte, California 91010|
|Kosair Children's Hospital||Louisville, Kentucky 40202-3830|
|Midwest Children's Cancer Center at Children's Hospital of Wisconsin||Milwaukee, Wisconsin 53226|
|Methodist Children's Hospital of South Texas||San Antonio, Texas 78229-3993|
|Children's Hospital and Research Center Oakland||Oakland, California 94609|
|Rady Children's Hospital - San Diego||San Diego, California 92123-4282|
|UCSF Helen Diller Family Comprehensive Cancer Center||San Francisco, California 94115|
|AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus||Atlanta, Georgia 30322|
|Dana-Farber/Harvard Cancer Center at Dana-Farber Cancer Institute||Boston, Massachusetts 02115|
|C.S. Mott Children's Hospital at University of Michigan Medical Center||Ann Arbor, Michigan 48109-0286|
|University of Mississippi Cancer Clinic||Jackson, Mississippi 39216-4505|
|Nationwide Children's Hospital||Columbus, Ohio 43205-2696|
|Oklahoma University Cancer Institute||Oklahoma City, Oklahoma 73104|
|Children's Hospital of Pittsburgh of UPMC||Pittsburgh, Pennsylvania 15213|
|Riley's Children Cancer Center at Riley Hospital for Children||Indianapolis, Indiana 46202-5225|