Pediatric Oncology Nutrition Intervention Trial

NCT ID: NCT06175273

Last Updated: 2025-09-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 19 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-01-31
• Study Completion Date: 2025-08-04

Brief Summary

Nearly 60% of pediatric patients diagnosed with cancer develop malnutrition caused by a combination of disease burden, side effects of chemotherapy, and the intensity of cancer treatment. These patients are known to have an increased risk of infection, treatment-related toxicity, inferior clinical outcomes, and increased risk of mortality. Malnutrition may progress to cancer cachexia, characterized by anorexia, increased inflammation, decreased fat, and decreased muscle mass with subsequent weight loss, which is associated with decreased overall survival.

The goal of the proposed research is to determine changes in body composition, weight status, and nutritional status between common nutrition interventions including oral nutrition supplements (ONS), appetite stimulants, and enteral nutrition (EN) among pediatric cancer patients. A secondary goal of this research is to utilize the findings to develop clinical nutrition guidelines for this patient population. The specific objective of the research proposed is to solve the lack of evidence to adequately treat nutritional deficits in the pediatric oncology population. Without this data, there is a lack of clinical consistency in the initiation and selection of appropriate nutrition interventions to provide a more definitive pathway of care. This study can help formulate a clinical guideline for this patient population before, during, and after treatment.

Detailed Description

The investigators plan to enroll a minimum of 45 subjects between the ages of 2-18 years old, over the course of one year, including males and females of all ethnicities. The subject will be a newly diagnosed pediatric patient with cancer who will actively receive chemotherapy at UK Healthcare's Kentucky Children's Hospital (KCH).

All those who enroll will have a baseline ultrasound and biomarkers taken at enrollment. Participants will also receive 'standard of care' medical nutrition therapy (MNT) counseling at diagnosis for following a high protein/high-calorie diet. If any of the enrolled participants lose >10% of the diagnosis weight, the participants will then be randomized to receive (1) oral nutrition supplements, (2) age-appropriate appetite stimulants, or (3) enteral nutrition via a feeding tube. At the point of randomization, participants will receive another ultrasound and biomarkers. Those randomized will then receive additional ultrasounds and biomarkers at 1 month and 3 months after randomization. The remaining patients that have either weight maintenance or weight gain, will be utilized as a comparator group and will receive an ultrasound at 3 months and 6 months from enrollment/diagnosis. Each group will have a goal of at least 10 patients per group.

Those receiving ONS will be prescribed a standard ONS (1.0 kcal per mL) that will meet a minimum of 50% of estimated energy needs to promote weight gain but allow for continued regular dietary intakes. The ONS will be prescribed as 8oz (240mL) per dose at least once a day, but up to six times per day depending on estimated nutrient needs. Those receiving age-appropriate appetite stimulants will be provided either cyproheptadine or olanzapine. Subjects will be given cyproheptadine if the participants fall between the ages of 2-12 years of age and olanzapine if the participants are >12 years of age or older. Table 1 summarizes the medication dosage forms, initial and dose titration options, and precautions/contraindications of each appetite stimulant. Medication adjustments will be made within 2-4 weeks of initiation due to the variable peak effect times, and chemotherapy treatment plans dictating clinic follow-up. Those randomized to EN will have a feeding tube placed and started on tube feeds via a designated pump for overnight feeds, meeting at least 50% of estimated energy needs to allow for continued regular dietary intakes during the day. After placement of a feeding tube, an abdominal x-ray will be obtained per hospital policy to confirm the appropriate placement of the feeding tube prior to use to ensure the safety of using said feeding tube.

Baseline demographics will be collected, including gender, age, diagnosis, QMLT ultrasound measurement, subjective global assessment (SGA) malnutrition risk, pediatric z-scores, and activity levels. Investigators will assess the subjects' nutritional intake, body composition via QMLT ultrasound, mid-upper arm circumference, pediatric z-scores (weight, length, and body mass index), biomarkers (leptin, IL-6, Lipid profiles, Cystatin-C, Vitamin D, and C-reactive protein), compliance and tolerance to nutrition intervention, physical activity, and clinical outcomes (rates of infection, number of hospital days for non-chemotherapy administration, number of clinic visits for non-chemotherapy administration, chemotherapy toxicity CTCAE grade, rate or relapse, and 1-year survival rates) at multiple time points throughout the study. Nutritional assessment will involve a 24-hour food recall which will be performed at diagnosis, randomization, and then every 1-2 weeks once randomized to an intervention. Weight loss and pediatric z-scores (weight-for-age, length-for-age, and BMI-for-age) will be assessed every 1-2 weeks throughout the study enrollment. Ultrasound assessment will be of the QMLT by measuring at the midpoint between the ASIS and the upper pole of the patella. The measurements will be taken bilaterally, three times, and averaged together for the final measurement. Ultrasound assessment will occur at enrollment when weight loss criteria are met for randomization, and then at 1 month and 3 months after randomization (or when the weight lost is regained).

Pediatric physical activity will be assessed every 1-2 weeks in combination with ultrasound measurements. Adherence and adverse effects to medications, tube feeds, or oral nutrition supplements will be assessed at each clinic visit. Biomarkers will be analyzed at randomization, 1 month, and 3 months after randomization.

Standard of care for patients are to receive MNT education at diagnosis. For those that lose weight, standard of care is to receive the nutrition interventions listed above (oral nutrition supplements, appetite stimulants, or enteral nutrition via feeding tube). The standard of care is to generally go from a least invasive to a more invasive approach. The research components of this study include being randomized to a nutrition intervention rather than a non-validated step-wise approach, the ultrasound measurements, and the biomarkers. These components are not considered standard of care and are research procedures. Baseline demographics will be collected, including gender, age, diagnosis, QMLT ultrasound measurement, subjective global assessment (SGA) malnutrition risk, pediatric z-scores, and activity levels. The team will assess the subjects' nutritional intake, body composition via QMLT ultrasound, mid-upper arm circumference, pediatric z-scores (weight, length, and body mass index), biomarkers (leptin, IL-6, Lipid profiles, Cystatin-C, Vitamin D, and C-reactive protein), compliance and tolerance to nutrition intervention, physical activity, and clinical outcomes (rates of infection, number of hospital days for non-chemotherapy administration, number of clinic visits for non-chemotherapy administration, chemotherapy toxicity CTCAE grade, rate or relapse, and 1-year survival rates) at multiple timepoints throughout the study. Nutritional assessment will involve a 24-hour food recall which will be performed at diagnosis, randomization, and then every 1-2 weeks once randomized to an intervention. Weight loss and pediatric z-scores (weight-for-age, length-for-age, and BMI-for-age) will be assessed every 1-2 weeks throughout the study enrollment. Ultrasound assessment will be of the QMLT by measuring at the midpoint between the ASIS and the upper pole of the patella, a total of 4 ultrasounds will be conducted during the study. The measurements will be taken bilaterally, three times, and averaged together for the final measurement. Ultrasound assessment will occur at enrollment when weight loss criteria are met for randomization, and then every 1-2 weeks for up to 8 weeks after randomization. Pediatric physical activity will be assessed every 1-2 weeks in combination with ultrasound measurements. Adherence and adverse effects to medications, tube feeds, or oral nutrition supplements will be assessed at each clinic visit.

Biomarkers will be analyzed at randomization, and weeks 4 and 8 after randomization. Additionally, measurements of resource utilization will be measured such as the number of hospital days for non-chemotherapy-related problems, the number of sick visits to the clinic, dietitian visits, infections, and rates of relapse or overall survival. Biomarkers will be drawn when the children are in the clinic for chemotherapy or lab work. The participants will already have their central venous line accessed by a qualified clinic RN and labs will be drawn at that time to ensure no extra harm to the patient. Additionally, all patients will have EMLA cream used to numb the area prior to having a clinic RN who is trained in accessing ports and has accessed the ports during previous clinic visits and will continue to do so throughout therapy.

Conditions

Pediatric Cancer; Nutrition Related Cancer; Nutrition Aspect of Cancer; Muscle Loss; Malnutrition, Child

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: NONE

Study Groups

Control

Standard of Care who have either weight gain, weight maintenance, or weight loss \<10%.

Group Type: ACTIVE_COMPARATOR

Standard of Care

Intervention Type: OTHER

Diet education on high protein, high calorie food consumption

Oral Nutrition Supplements

Those receiving ONS will be prescribed a standard ONS (1.0 kcal per mL) that will meet a minimum of 50% of estimated energy needs to promote weight gain but allow for continued regular dietary intakes. The ONS will be prescribed as 8oz (240mL) per dose at least once a day, but up to six times per day depending on estimated nutrient needs.

Group Type: EXPERIMENTAL

Oral Nutrition Supplement

Intervention Type: DIETARY_SUPPLEMENT

8oz (240mL) per dose at least once a day, but up to six times per day depending on estimated nutrient needs. The formula will equate to 1.0kcal/mL

Enteral Nutrition

Those randomized to EN will have a feeding tube placed and started on tube feeds via a designated pump for overnight feeds, meeting at least 50% of estimated energy needs to allow for continued regular dietary intakes during the day.

Group Type: EXPERIMENTAL

Tube Feed

Intervention Type: DIETARY_SUPPLEMENT

EN will have a feeding tube placed and started on tube feeds via a designated pump for overnight feeds, meeting at least 50% of estimated energy needs

Appetite Stimulants

Those receiving age-appropriate appetite stimulants will be provided either cyproheptadine or olanzapine. Subjects will be given cyproheptadine if they fall between the ages of 2-12 years of age and olanzapine if they are \>12 years of age or older.

Group Type: EXPERIMENTAL

Cyproheptadine

Intervention Type: DRUG

0.25mg/kg/d divided BID. May titrate by 4mg increments to a max of 12mg/day.

Olanzapine

Intervention Type: DRUG

2.5mg once a day (at night). May titrate by 2.5mg increments to max of 10mg/day

Interventions

Oral Nutrition Supplement

8oz (240mL) per dose at least once a day, but up to six times per day depending on estimated nutrient needs. The formula will equate to 1.0kcal/mL

Intervention Type: DIETARY_SUPPLEMENT

Tube Feed

EN will have a feeding tube placed and started on tube feeds via a designated pump for overnight feeds, meeting at least 50% of estimated energy needs

Intervention Type: DIETARY_SUPPLEMENT

Cyproheptadine

0.25mg/kg/d divided BID. May titrate by 4mg increments to a max of 12mg/day.

Intervention Type: DRUG

Olanzapine

2.5mg once a day (at night). May titrate by 2.5mg increments to max of 10mg/day

Intervention Type: DRUG

Standard of Care

Diet education on high protein, high calorie food consumption

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• New diagnosis of pediatric cancer

Exclusion Criteria

• Current use or history of enteral nutrition, oral supplement, or orexigenic/anorectic use.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 19 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Center for Advancing Translational Sciences (NCATS)

NIH

Sponsor Role: collaborator

Corey Hawes

OTHER

Sponsor Role: lead

Responsible Party

Corey Hawes

Clinical Nutrition Professor

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Corey J Hawes, DCN, RD, CSO, CNSC, LD

Role: PRINCIPAL_INVESTIGATOR

University of Kentucky, Kentucky Children's Hospital

Locations

University of Kentucky

Lexington, Kentucky, United States

Countries

United States

Provided Documents

Document Type: Informed Consent Form

View Document

Other Identifiers

UL1TR001998

Identifier Type: NIH

Identifier Source: secondary_id

View Link

89377

Identifier Type: -

Identifier Source: org_study_id