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

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Children's Oncology Group
ClinicalTrials.gov Identifier:
NCT01305200
First received: February 25, 2011
Last updated: August 14, 2014
Last verified: August 2014
  Purpose

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.


Condition Intervention Phase
Childhood Acute Lymphoblastic Leukemia in Remission
Childhood Acute Myeloid Leukemia in Remission
Childhood Chronic Myelogenous Leukemia
Childhood Myelodysplastic Syndromes
Chronic Eosinophilic Leukemia
Chronic Myelomonocytic Leukemia
Chronic Neutrophilic Leukemia
de Novo Myelodysplastic Syndromes
Disseminated Neuroblastoma
Juvenile Myelomonocytic Leukemia
Mucositis
Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable
Previously Treated Childhood Rhabdomyosarcoma
Previously Treated Myelodysplastic Syndromes
Recurrent Childhood Acute Lymphoblastic Leukemia
Recurrent Childhood Acute Myeloid Leukemia
Recurrent Childhood Large Cell Lymphoma
Recurrent Childhood Lymphoblastic Lymphoma
Recurrent Childhood Rhabdomyosarcoma
Recurrent Childhood Small Noncleaved Cell Lymphoma
Recurrent Malignant Testicular Germ Cell Tumor
Recurrent Wilms Tumor and Other Childhood Kidney Tumors
Recurrent/Refractory Childhood Hodgkin Lymphoma
Relapsing Chronic Myelogenous Leukemia
Secondary Acute Myeloid Leukemia
Secondary Myelodysplastic Syndromes
Unspecified Childhood Solid Tumor, Protocol Specific
Drug: supersaturated calcium phosphate rinse
Other: placebo
Other: questionnaire administration
Procedure: quality-of-life assessment
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver)
Primary Purpose: Supportive Care
Official Title: A Randomized Double Blinded Trial of Topical Caphosol to Prevent Oral Mucositis in Children Undergoing Hematopoietic Stem Cell Transplantation

Resource links provided by NLM:


Further study details as provided by Children's Oncology Group:

Primary Outcome Measures:
  • Duration of severe oral mucositis (WHO grade 3 or 4) [ Time Frame: Up to day 20 ] [ Designated as safety issue: Yes ]
    Will be compared between the groups in a 2 sample t-test. Descriptive statistics will be used to summarize the duration of severe mucositis in each arm.


Secondary Outcome Measures:
  • Incidence of severe oral mucositis (WHO grade 3 or 4) [ Time Frame: Up to day 20 ] [ Designated as safety issue: Yes ]
    The groups will be compared using the Chi square test or Fisher's exact test. Comparison of such incidences between the 2 groups by Cochran-Mantel-Haenszel test with adjustment for the stratification factors will also be performed, as well as by logistic regression models with potential adjustment for the stratification factors and other confounders.

  • Severity of mucositis according to pain categorical rating scale and modified OMDQ [ Time Frame: Up to day 20 ] [ Designated as safety issue: Yes ]
    Will be compared between the groups using a linear mixed model with repeated measures and also by comparing the area under the curve (AUC) in a 2-sample t test or Wilcoxon rank sum test.

  • Incidence, total dose, and duration of parenteral opioid analgesic use [ Time Frame: Up to day 20 ] [ Designated as safety issue: No ]
    The groups will be compared using the Chi square test or Fisher's exact test. Comparison of such incidences between the 2 groups by Cochran-Mantel-Haenszel test with adjustment for the stratification factors will also be performed, as well as by logistic regression models with potential adjustment for the stratification factors and other confounders.

  • Incidence and duration of TPN use [ Time Frame: Up to day 20 ] [ Designated as safety issue: No ]
    The groups will be compared using the Chi square test or Fisher's exact test. Comparison of such incidences between the 2 groups by Cochran-Mantel-Haenszel test with adjustment for the stratification factors will also be performed, as well as by logistic regression models with potential adjustment for the stratification factors and other confounders.

  • Incidence of febrile neutropenia [ Time Frame: Up to day 20 ] [ Designated as safety issue: No ]
    The groups will be compared using the Chi square test or Fisher's exact test. Comparison of such incidences between the 2 groups by Cochran-Mantel-Haenszel test with adjustment for the stratification factors will also be performed, as well as by logistic regression models with potential adjustment for the stratification factors and other confounders.

  • Incidence of invasive bacterial infections [ Time Frame: Up to day 20 ] [ Designated as safety issue: No ]
    The groups will be compared using the Chi square test or Fisher's exact test. Comparison of such incidences between the 2 groups by Cochran-Mantel-Haenszel test with adjustment for the stratification factors will also be performed, as well as by logistic regression models with potential adjustment for the stratification factors and other confounders.

  • ChIMES score [ Time Frame: Up to day 20 ] [ Designated as safety issue: No ]
    The Spearman correlation coefficients will be described using all evaluations but in order to account for the same child providing multiple measures, we will obtain the P values using a linear mixed model with repeated measures.


Enrollment: 226
Study Start Date: March 2011
Estimated Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Arm I (supersaturated calcium phosphate rinse)
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.
Drug: supersaturated calcium phosphate rinse
Mouth rinse
Other Name: Caphosol
Other: questionnaire administration
Ancillary studies
Procedure: quality-of-life assessment
Ancillary studies
Other Name: quality of life assessment
Placebo Comparator: Arm II (placebo)
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.
Other: placebo
Mouth rinse
Other Name: PLCB
Other: questionnaire administration
Ancillary studies
Procedure: quality-of-life assessment
Ancillary studies
Other Name: quality of life assessment

Detailed 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.

  Eligibility

Ages Eligible for Study:   4 Years to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01305200

  Show 35 Study Locations
Sponsors and Collaborators
Children's Oncology Group
Investigators
Principal Investigator: Nathaniel Treister, MD Children's Oncology Group
  More Information

No publications provided

Responsible Party: Children's Oncology Group
ClinicalTrials.gov Identifier: NCT01305200     History of Changes
Other Study ID Numbers: ACCL1031, NCI-2011-02635, CDR0000695718, ACCL1031, COG-ACCL1031, ACCL1031, U10CA095861
Study First Received: February 25, 2011
Last Updated: August 14, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Hodgkin Disease
Hypereosinophilic Syndrome
Kidney Neoplasms
Leukemia
Leukemia, Lymphoid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Leukemia, Myelomonocytic, Acute
Leukemia, Myelomonocytic, Chronic
Leukemia, Myelomonocytic, Juvenile
Leukemia, Neutrophilic, Chronic
Lymphoma
Lymphoma, Non-Hodgkin
Mucositis
Myelodysplastic Syndromes
Myelodysplastic-Myeloproliferative Diseases
Myeloproliferative Disorders
Neoplasm Metastasis
Neoplasms
Neoplasms, Germ Cell and Embryonal
Neuroblastoma
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Preleukemia
Rhabdomyosarcoma
Rhabdomyosarcoma, Embryonal
Stomatitis
Syndrome
Testicular Neoplasms
Wilms Tumor

ClinicalTrials.gov processed this record on October 20, 2014