Myelodysplastic Syndromes Clinical Trial
Official 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
Verified date | November 2020 |
Source | Phoenix Children's Hospital |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 49 |
Est. completion date | July 1, 2019 |
Est. primary completion date | July 1, 2019 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Year to 25 Years |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | Phoenix Children's Hospital | Phoenix | Arizona |
Lead Sponsor | Collaborator |
---|---|
Phoenix Children's Hospital |
United States,
Duncan CN, Vrooman L, Apfelbaum EM, Whitley K, Bechard L, Lehmann LE. 25-hydroxy vitamin D deficiency following pediatric hematopoietic stem cell transplant. Biol Blood Marrow Transplant. 2011 May;17(5):749-53. doi: 10.1016/j.bbmt.2010.10.009. Epub 2010 Oct 15. — View Citation
Gordon CM, DePeter KC, Feldman HA, Grace E, Emans SJ. Prevalence of vitamin D deficiency among healthy adolescents. Arch Pediatr Adolesc Med. 2004 Jun;158(6):531-7. — View Citation
Hansson ME, Norlin AC, Omazic B, Wikström AC, Bergman P, Winiarski J, Remberger M, Sundin M. Vitamin d levels affect outcome in pediatric hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2014 Oct;20(10):1537-43. doi: 10.1016/j.bbmt.2014.05.030. Epub 2014 Jun 5. — View Citation
Heaney RP, Armas LA, Shary JR, Bell NH, Binkley N, Hollis BW. 25-Hydroxylation of vitamin D3: relation to circulating vitamin D3 under various input conditions. Am J Clin Nutr. 2008 Jun;87(6):1738-42. — View Citation
Holick MF, Binkley NC, Bischoff-Ferrari HA, Gordon CM, Hanley DA, Heaney RP, Murad MH, Weaver CM; Endocrine Society. Evaluation, treatment, and prevention of vitamin D deficiency: an Endocrine Society clinical practice guideline. J Clin Endocrinol Metab. 2011 Jul;96(7):1911-30. doi: 10.1210/jc.2011-0385. Epub 2011 Jun 6. Erratum in: J Clin Endocrinol Metab. 2011 Dec;96(12):3908. — View Citation
Misra M, Pacaud D, Petryk A, Collett-Solberg PF, Kappy M; Drug and Therapeutics Committee of the Lawson Wilkins Pediatric Endocrine Society. Vitamin D deficiency in children and its management: review of current knowledge and recommendations. Pediatrics. 2008 Aug;122(2):398-417. doi: 10.1542/peds.2007-1894. Review. — View Citation
Rosenblatt J, Bissonnette A, Ahmad R, Wu Z, Vasir B, Stevenson K, Zarwan C, Keefe W, Glotzbecker B, Mills H, Joyce R, Levine JD, Tzachanis D, Boussiotis V, Kufe D, Avigan D. Immunomodulatory effects of vitamin D: implications for GVHD. Bone Marrow Transplant. 2010 Sep;45(9):1463-8. doi: 10.1038/bmt.2009.366. Epub 2010 Jan 18. — View Citation
Schlereth F, Badenhoop K. [Vitamin D : More than just a bone hormone]. Internist (Berl). 2016 Jul;57(7):646-55. doi: 10.1007/s00108-016-0082-2. Review. German. — View Citation
Shepherd D, Belessis Y, Katz T, Morton J, Field P, Jaffe A. Single high-dose oral vitamin D3 (stoss) therapy--a solution to vitamin D deficiency in children with cystic fibrosis? J Cyst Fibros. 2013 Mar;12(2):177-82. doi: 10.1016/j.jcf.2012.08.007. Epub 2012 Sep 19. — View Citation
Sullivan SS, Rosen CJ, Halteman WA, Chen TC, Holick MF. Adolescent girls in Maine are at risk for vitamin D insufficiency. J Am Diet Assoc. 2005 Jun;105(6):971-4. — View Citation
Wallace G, Jodele S, Howell J, Myers KC, Teusink A, Zhao X, Setchell K, Holtzapfel C, Lane A, Taggart C, Laskin BL, Davies SM. Vitamin D Deficiency and Survival in Children after Hematopoietic Stem Cell Transplant. Biol Blood Marrow Transplant. 2015 Sep;21(9):1627-31. doi: 10.1016/j.bbmt.2015.06.009. Epub 2015 Jun 18. — View Citation
Wallace G, Jodele S, Myers KC, Dandoy CE, El-Bietar J, Nelson A, Taggart CB, Daniels P, Lane A, Howell J, Teusink-Cross A, Davies SM. Vitamin D Deficiency in Pediatric Hematopoietic Stem Cell Transplantation Patients Despite Both Standard and Aggressive Supplementation. Biol Blood Marrow Transplant. 2016 Jul;22(7):1271-1274. doi: 10.1016/j.bbmt.2016.03.026. Epub 2016 Apr 1. — View Citation
* Note: There are 12 references in all — Click here to view all references
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Safety of Stoss Therapy | 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. | 100 days | |
Primary | Efficacy of vitamin D repletion | 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. | 100 days | |
Secondary | Graft-versus-host disease | All incidences of GVHD will be recorded in the medical record throughout transplant as per standard of care for data extraction after Day +100 | 100 days | |
Secondary | Immune Recovery | Immune recovery will be obtained at Day +100 as per standard of care and recorded in the medical record | 100 days | |
Secondary | Rejection | All incidences of rejection will be recorded in the medical record throughout transplant as per standard of care for data extraction after Day +100 | 100 days | |
Secondary | Relapse | All incidences of relapse will be recorded in the medical record throughout transplant as per standard of care for data extraction after Day +100 | 100 days | |
Secondary | Infection Rates | All incidences of infection will be recorded in the medical record throughout transplant as per standard of care for data extraction after Day +100 | 100 days | |
Secondary | Mortality | All incidences of mortality will be recorded in the medical record throughout transplant as per standard of care for data extraction after Day +100 | 100 days |
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