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NCT Number: NCT03176849

A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT

Research has suggested that children with sufficient vitamin D levels undergoing hematopoietic stem cell transplant (HSCT) have improved outcomes, including lower incidences of infection and graft-versus-host disease (GVHD), as well as overall improved survival. However, supplementation in children undergoing HSCT has shown to be a challenge using standard or aggressive supplementation strategies. The primary objective of this study is to determine the safety and efficacy of a single, high dose oral vitamin D (Stoss Therapy) at the start of transplant followed by maintenance supplementation in children undergoing HSCT.

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Key information

Conditions

Vitamin D Deficiency Anemia Anemia, Aplastic Aplastic Anemia Avitaminosis Blood Disorder Bone Marrow Diseases Bone Marrow Failure Disorders Deficiency Diseases Hematologic Diseases Hemic and Lymphatic Diseases Immune System Diseases Immunoproliferative Disorders Leukemia Leukemia, Lymphoid Lymphatic Diseases Lymphoproliferative Disorders Malnutrition Myelodysplastic Syndromes Neoplasms Neoplasms by Histologic Type Nutrition Disorders Nutritional and Metabolic Diseases Pediatric Acute Lymphoid Leukemia Pediatric Acute Myeloid Leukemia Pediatric Cancer Precursor Cell Lymphoblastic Leukemia-Lymphoma Sickle Cell Anemia in Children Stem Cell Transplant Complications Thalassemia in Children

Age range

1 year–25 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

About this study

Comorbidities and complications including infection, organ system toxicity, graft-versus-host disease (GVHD) and disease recurrence are some of the biggest contributors to quality of life and mortality in children undergoing hematopoietic stem cell transplant (HSCT). Research has suggested that patients with sufficient vitamin D levels during transplant have improved outcomes, including lower incidences of infection and acute GVHD, as well as overall improved survival. Prior research has shown that chronically ill children are at risk for vitamin D deficiency, including those undergoing HSCT. Data has shown populations with as many as 70% of HSCT patients have insufficient levels of vitamin D at time of transplant. While several studies have attempted methods of vitamin D supplementation in this subset of patients, there has not been success with either standard or aggressive supplementation strategies.

Single high-dose oral vitamin D therapy, known as stoss therapy, has been used in other chronically ill children where adequate levels of vitamin D are difficult to attain. Stoss therapy suggests a single high-dose followed by maintenance dosing would be adequate to replete and maintain vitamin D levels in chronically ill children. While it has been shown to be effective with no evidence of toxicity in patients with rickets and cystic fibrosis, its safety and efficacy has not been studied in the transplant setting. However, there is an urgent need to identify a modifiable factor may reduce the occurrence and/or severity of HSCT associated complications. The overall objective of this study is to determine the effectiveness of a single, high dose oral vitamin D (Stoss Therapy) followed by maintenance supplementation in children undergoing HSCT. This change will result in a new and innovative approach to maintaining adequate vitamin D levels during pediatric HSCT, with the long term goal of reducing morbidity and mortality.

Our primary goal is to assess the safety and efficacy of a single, high dose of vitamin D followed by maintenance supplementation in children undergoing HSCT. Our secondary goal is to identify the effects of adequate vitamin D levels on early clinical outcomes such as cytokine levels, graft versus host disease, immune recovery, rejection, relapse, infection rates in pediatric HSCT patients.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • All pediatric patients, ages 1 to 25 years of age, undergoing hematopoietic stem cell transplant at Phoenix Children's hospital
  • Patients must sign an informed consent

Exclusion criteria

  • Prior rejection of hematopoietic stem cell transplant

Treatment and study plan

Vitamin D3

Dietary Supplement

The single, oral dose of vitamin D3 is based on patient's age and baseline 25-hydroxy-vitamin D level. Dosing is as follows: (1) For children under 3 years of age. 200000IU for those deficient, 150000IU for those insufficient, and 100000IU for those sufficient; (2) For children 3-12 years of age, 400000IU for those deficient, 350000IU for those insufficient, and 200000IU for those sufficient; (3) For children greater than 12 years of age, 600000IU for those deficient, 500000IU for those insufficient, and 300000IU for those sufficient. This is a single, one time oral dose given prior to the start of transplant.

Standard Vitamin D3 Supplementation

Dietary Supplement

Those who have sufficient vitamin D will be supplemented with 400-600IU/day of Vitamin D3 orally.

Those who have insufficient or are deficient in vitamin D will be supplemented with 50,000IU/week of Vitamin D3 orally.

Primary outcomes

  1. Safety of Stoss Therapy

    Time frame: 100 days

    In order to monitor the safety of stoss therapy, patients will be monitored for any clinical signs or symptoms of hypervitaminosis D, including abdominal pain, dehydration, and fatigue. Patients will be monitored for hypercalcemia and hyperphosphatemia with weekly complete metabolic panels and serum phosphorus during the first 100 days of transplant. Patients will have repeat measurements of serum 25(OH)D levels will be obtained at Day +30 to ensure they do not have hypervitaminosis D at that time.

  2. Efficacy of vitamin D repletion

    Time frame: 100 days

    All patients will have baseline serum 25(OH)D levels obtained prior to transplant. At baseline, patient will be classified as being sufficient (>30ng/mL), insufficient (21- 29ng/mL), or deficient (<20ng/mL) in serum vitamin D. All patients will then undergo treatment based on their trial arm and baseline levels of vitamin D. Patients will have repeat measurements of serum 25(OH)D levels will be obtained at Day +100 of transplant. At this time they will again be classified as being sufficient (>30ng/mL), insufficient (21- 29ng/mL), or deficient (<20ng/mL) in serum vitamin D following therapy to assess if the therapy was efficacious in repleting and maintaining their serum vitamin D level.

Secondary outcomes

  1. Graft-versus-host disease

    Time frame: 100 days

    All incidences of GVHD will be recorded in the medical record throughout transplant as per standard of care for data extraction after Day +100

  2. Immune Recovery

    Time frame: 100 days

    Immune recovery will be obtained at Day +100 as per standard of care and recorded in the medical record

  3. Rejection

    Time frame: 100 days

    All incidences of rejection will be recorded in the medical record throughout transplant as per standard of care for data extraction after Day +100

  4. Relapse

    Time frame: 100 days

    All incidences of relapse will be recorded in the medical record throughout transplant as per standard of care for data extraction after Day +100

  5. Infection Rates

    Time frame: 100 days

    All incidences of infection will be recorded in the medical record throughout transplant as per standard of care for data extraction after Day +100

  6. Mortality

    Time frame: 100 days

    All incidences of mortality will be recorded in the medical record throughout transplant as per standard of care for data extraction after Day +100

Sponsors and collaborators

Lead sponsor

Phoenix Children's Hospital

Other

Registry information

Official study title

A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 (Stoss Therapy) in Pediatric Patients Undergoing HSCT to Prevent Vitamin D Deficiency and Insufficiency During Transplant

Important dates

Study start
2017
Primary completion
2019
Study completion
2019
First posted
Jun 6, 2017
Registry last updated
Nov 17, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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