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

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ClinicalTrials.gov Identifier: NCT01305200
Recruitment Status : Completed
First Posted : February 28, 2011
Results First Posted : May 9, 2017
Last Update Posted : September 17, 2019
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Children's Oncology Group

Tracking Information
First Submitted Date  ICMJE February 25, 2011
First Posted Date  ICMJE February 28, 2011
Results First Submitted Date  ICMJE November 30, 2016
Results First Posted Date  ICMJE May 9, 2017
Last Update Posted Date September 17, 2019
Study Start Date  ICMJE March 2011
Actual Primary Completion Date June 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 28, 2017)
Duration of Severe Oral Mucositis (WHO Grade 3 or 4) [ Time Frame: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation. ]
Mean days of severe (WHO Grade 3 or 4) Mucositis.
Original Primary Outcome Measures  ICMJE
 (submitted: February 25, 2011)
Duration of Severe Oral Mucositis (WHO Grade 3 or 4)
Change History Complete list of historical versions of study NCT01305200 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 28, 2017)
  • Incidence of Severe Oral Mucositis [ Time Frame: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation. ]
    Percentage of patients with Severe Oral Mucositis (WHO Grade 3 or 4) per arm.
  • Oral Mucositis Daily Questionnaire (OMDQ) [ Time Frame: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation ]
    Area under the curve (AUC) of the Oral Mucositis Daily Questionnaire (OMDQ) subscales
  • Incidence of Parenteral Opioid Analgesic Use (Morphine Equivalents). [ Time Frame: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation. ]
    Opioid Administration = yes
  • Duration of Parenteral Opioid Analgesic Use (Morphine Equivalents). [ Time Frame: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation. ]
    Mean days of parenteral opioid analgesic use.
  • Total Dose of Parenteral Opioid Analgesic Used (Morphine Equivalents). [ Time Frame: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation. ]
    Morphine equivalent dose in mg/kg/day
  • Incidence of Total Parenteral Nutrition (TPN) Administration. [ Time Frame: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation. ]
    Total Parenteral Nutrition = yes
  • Duration of Total Parenteral Nutrition (TPN) Administration. [ Time Frame: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation. ]
    Mean days of total parenteral nutrition (TPN) administration.
  • Incidence of Febrile Neutropenia [ Time Frame: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation. ]
    Fever and Neutropenia = yes
  • Incidence of Invasive Bacterial Infections [ Time Frame: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation. ]
    Invasive Bacterial Infection = yes
  • Severity of Mucositis [ Time Frame: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation ]
    Area Under the Curve of Severity of Mucositis. According to mouth pain categorical rating scale ranges 0-10 with higher scores reflecting more severe pain.
Original Secondary Outcome Measures  ICMJE
 (submitted: February 25, 2011)
  • Incidence of severe oral mucositis (WHO grade 3 or 4)
  • Severity of mucositis according to pain categorical rating scale and modified OMDQ
  • Incidence, total dose, and duration of parenteral opioid analgesic use
  • Incidence and duration of TPN use
  • Incidence of Febrile Neutropenia
  • Incidence of Invasive Bacterial Infections
  • ChIMES score
Current Other Pre-specified Outcome Measures
 (submitted: March 28, 2017)
Ancillary Validation Study of ChIMES [ Time Frame: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation. ]
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Supersaturated Calcium Phosphate Rinse in Preventing Oral Mucositis in Young Patients Undergoing Autologous or Donor Stem Cell Transplant
Official Title  ICMJE A Randomized Double Blinded Trial of Topical Caphosol to Prevent Oral Mucositis in Children Undergoing Hematopoietic Stem Cell Transplantation
Brief Summary 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.
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Care Provider)
Primary Purpose: Supportive Care
Condition  ICMJE
  • 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
Intervention  ICMJE
  • Drug: supersaturated calcium phosphate rinse
    Mouth rinse
    Other Name: Caphosol
  • 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
Study Arms  ICMJE
  • Placebo Comparator: Arm I (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.
    Interventions:
    • Other: placebo
    • Other: questionnaire administration
    • Procedure: quality-of-life assessment
  • Experimental: Arm II (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.
    Interventions:
    • Drug: supersaturated calcium phosphate rinse
    • Other: questionnaire administration
    • Procedure: quality-of-life assessment
Publications * Treister N, Nieder M, Baggott C, Olson E, Chen L, Dang H, Krailo M, August A, Sung L. Caphosol for prevention of oral mucositis in pediatric myeloablative haematopoietic cell transplantation. Br J Cancer. 2017 Jan 3;116(1):21-27. doi: 10.1038/bjc.2016.380. Epub 2016 Nov 22.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 14, 2014)
226
Original Estimated Enrollment  ICMJE
 (submitted: February 25, 2011)
200
Actual Study Completion Date  ICMJE June 30, 2015
Actual Primary Completion Date June 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 4 Years to 21 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01305200
Other Study ID Numbers  ICMJE ACCL1031
NCI-2011-02635 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
CDR0000695718 ( Other Identifier: Clinical Trials.gov )
ACCL1031 ( Other Identifier: Children's Oncology Group )
COG-ACCL1031 ( Other Identifier: DCP )
ACCL1031 ( Other Identifier: CTEP )
U10CA095861 ( U.S. NIH Grant/Contract )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Children's Oncology Group
Study Sponsor  ICMJE Children's Oncology Group
Collaborators  ICMJE National Cancer Institute (NCI)
Investigators  ICMJE
Principal Investigator: Nathaniel Treister, MD Children's Oncology Group
PRS Account Children's Oncology Group
Verification Date November 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP