Childhood Acute Lymphoblastic Leukemia in RemissionPhase 3NCT01305200
Supersaturated Calcium Phosphate Rinse in Preventing Oral Mucositis in Young Patients Undergoing Autologous or Donor Stem Cell Transplant
A Phase 3 study testing supersaturated calcium phosphate rinse, questionnaire administration and quality-of-life assessment in people with Childhood Acute Lymphoblastic Leukemia in Remission and Childhood Acute Myeloid Leukemia in Remission, run by Children's Oncology Group at 35 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 21 years
- Conditions the study is for
- Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia
- What is being tested?
- supersaturated calcium phosphate rinse, questionnaire administration and quality-of-life assessment
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 226 participants
- Where
- 35 sitesin 3 countries
- Study dates
- Mar 2011to 30 Jun 2015
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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:
Show all 14Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
35 sites in 3 countries.
35 sites in 3 countries
United States31 sites
C S Mott Children's Hospital
Ann Arbor, Michigan, 48109
Children's Oncology Group
Arcadia, California, 91006-3776
Children's Healthcare of Atlanta - Egleston
Atlanta, Georgia, 30322
Floating Hospital for Children at Tufts Medical Center
Boston, Massachusetts, 02111
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
Lurie Children's Hospital-Chicago
Chicago, Illinois, 60614
University of Chicago
Chicago, Illinois, 60637
Nationwide Children's Hospital
Columbus, Ohio, 43205
City of Hope
Duarte, California, 91010
Riley Hospital for Children
Indianapolis, Indiana, 46202
University of Mississippi Medical Center
Jackson, Mississippi, 39216
The Childrens Mercy Hospital
Kansas City, Missouri, 64108
Kosair Children's Hospital
Louisville, Kentucky, 40202
Miami Children's Hospital
Miami, Florida, 33155
Midwest Children's Cancer Center
Milwaukee, Wisconsin, 53226
Children's Hospital-Main Campus
New Orleans, Louisiana, 70118
Columbia University Medical Center
New York, New York, 10032
Children's Hospital and Research Center at Oakland
Oakland, California, 94609-1809
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Mayo Clinic
Rochester, Minnesota, 55905
Methodist Children's Hospital of South Texas
San Antonio, Texas, 78229
Rady Children's Hospital - San Diego
San Diego, California, 92123
University of California San Francisco Medical Center-Parnassus
San Francisco, California, 94143
All Children's Hospital
St. Petersburg, Florida, 33701
Montefiore Medical Center
The Bronx, New York, 10467-2490
Ny Cancer%
Valhalla, New York, 10595
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Alfred I duPont Hospital for Children
Wilmington, Delaware, 19803
Australia2 sites
Royal Childrens Hospital
Herston, Queensland, 4029
Princess Margaret Hospital for Children
Perth, Western Australia, 6008
Canada2 sites
The Montreal Children's Hospital of the MUHC
Montreal, Quebec, H3H 1P3
Hospital for Sick Children
Toronto, Ontario, M5G 1X8
About this study
Official title: A Randomized Double Blinded Trial of Topical Caphosol to Prevent Oral Mucositis in Children Undergoing Hematopoietic Stem Cell Transplantation
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.
Read the detailed description
PRIMARY OBJECTIVES:
- 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:
- 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.
- Conditions
- 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
- Being tested
- supersaturated calcium phosphate rinse, placebo, questionnaire administration, quality-of-life assessment
- Medical subject headings
- Pdgfra-Associated Chronic Eosinophilic Leukemia, Leukemia, Myelomonocytic, Chronic, Leukemia, Neutrophilic, Chronic, Leukemia, Myelomonocytic, Juvenile, Mucositis, Myeloproliferative Disorders, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Dendritic Cell Sarcoma, Interdigitating
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
supersaturated calcium phosphate rinseDrug
Also called Caphosol
Mouth rinse
placeboOther
Also called PLCB
Mouth rinse
questionnaire administrationOther
Ancillary studies
quality-of-life assessmentProcedure or surgery
Also called quality of life assessment
Ancillary studies
| Study group | Role | What they receive |
|---|---|---|
| Arm I (placebo) | Placebo comparator | 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. Receives: Other: placebo; Other: questionnaire administration; Procedure: quality-of-life assessment |
| Arm II (supersaturated calcium phosphate rinse) | Experimental | 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. Receives: Drug: supersaturated calcium phosphate rinse; Other: questionnaire administration; Procedure: quality-of-life assessment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Duration of Severe Oral Mucositis (WHO Grade 3 or 4)
Mean days of severe (WHO Grade 3 or 4) Mucositis.
Measured: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation.
Secondary outcomes (10)
Incidence of Severe Oral Mucositis
Percentage of patients with Severe Oral Mucositis (WHO Grade 3 or 4) per arm.
Measured: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation.
Oral Mucositis Daily Questionnaire (OMDQ)
Area under the curve (AUC) of the Oral Mucositis Daily Questionnaire (OMDQ) subscales
Measured: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation
Incidence of Parenteral Opioid Analgesic Use (Morphine Equivalents).
Opioid Administration = yes
Measured: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation.
Duration of Parenteral Opioid Analgesic Use (Morphine Equivalents).
Mean days of parenteral opioid analgesic use.
Measured: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation.
Total Dose of Parenteral Opioid Analgesic Used (Morphine Equivalents).
Morphine equivalent dose in mg/kg/day
Measured: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation.
Incidence of Total Parenteral Nutrition (TPN) Administration.
Total Parenteral Nutrition = yes
Measured: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation.
Duration of Total Parenteral Nutrition (TPN) Administration.
Mean days of total parenteral nutrition (TPN) administration.
Measured: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation.
Incidence of Febrile Neutropenia
Fever and Neutropenia = yes
Measured: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation.
Incidence of Invasive Bacterial Infections
Invasive Bacterial Infection = yes
Measured: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation.
Severity of Mucositis
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.
Measured: Day -1 (day prior to stem cell infusion) to Day 20 following transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2011 (estimated)
- Study startMar 2011
- Primary completionJun 2015
- Study completion30 Jun 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Childhood Acute Lymphoblastic Leukemia in RemissionStudies of the same condition, open and under way.
- More trials run by Children's Oncology GroupThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Care provider
- Enrolment
- 226 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Oncology Group
- Sponsor type
- Research network
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Nathaniel Treister, MD, Principal investigator, Children's Oncology Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01305200
- Sponsor's protocol ID
- ACCL1031
- Other IDs
- NCI-2011-02635, CDR0000695718, ACCL1031, COG-ACCL1031, ACCL1031, U10CA095861
- Status last verified
- Nov 2016
- First submitted
- 25 Feb 2011
- Last updated
- 17 Sep 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
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.
PubMed 27875526