Zoledronate to Prevent Bone Health Complications in Pediatric Hematopoietic Stem Cell Transplant Survivors

Официальное название

Early Intervention With Zoledronate to Safely Prevent Bone Health Complications in Pediatric Hematopoietic Stem Cell Transplant Survivors

Обзор

The purpose of this pilot study is to investigate the safety and preliminarily assess efficacy of early intervention with zoledronate in high risk pediatric hematopoietic stem cell transplantation (HSCT) patients to prevent the development of bone disease and fractures and reduce potential pain and suffering.

Подробное описание

Bone disease, including low bone density and fragility fractures (osteoporosis), is common among survivors of pediatric hematopoietic stem cell transplantation (HSCT). Further, patients who develop graft-versus-host disease (GVHD) following HSCT or who have high cumulative doses of glucocorticoids are at even higher risk to develop bone complications. Recent data also suggest that a large number of HSCT candidates arrive to transplantation already with low bone mineral density, adding to the potential risk of developing bone disease following HSCT. Typically, treatment for osteoporosis in children using bisphosphonates, such as zoledronate, is recommended only after the development of fragility fractures.

The investigators propose to study the safety and efficacy of a novel method of early intervention with zoledronate in high risk pediatric HSCT patients to prevent the development of bone disease and fractures in order to reduce potential pain and suffering.

Вмешательства

• Препарат Zoledronate
  Zoledronate is in the class of drugs entitled bisphosphonates which act to inhibit bone resorption by inhibiting osteoclast activity therefore reducing bone turnover.

Первичные конечные точки

• Safety of early intervention Zoledronate [Срок оценки: At the time of infusion through up to 60 days post last dose of Zoledronate]
• Feasibility of early intervention Zoledronate [Срок оценки: At patient discharge, typically 1 week post infusion]

Вторичные конечные точки (2)

• Bone health efficiency [Срок оценки: Baseline, +6 months, and +12 months]
• Bone substrate turnover [Срок оценки: Baseline and +30 days]

Критерии участия

Критерии включения

• Patients ≥5 and ≤18 years old who are preparing for HSCT with a height-for-age corrected DXA Z-score of <-2.0 and admitted to a CCHMC inpatient unit.
• Patients ≥5 and ≤18 years old recovering from HSCT and who have developed de novo acute or chronic GVHD and are admitted to a CCHMC inpatient unit.

Критерии исключения

• Age <5 years and >18 years
• Patients with Fanconi anemia or other radiation-sensitive syndromes with increased malignancy risk
• history of prior bisphosphonate use
• low 25-OH vitamin D levels (<20 ng/mL)
• active febrile illness
• uncontrolled infection
• Elevated creatinine at the time of enrollment, history or renal failure, or documented low glomerular filtration rate (GFR≤90)
• Active bone disease including history of abnormal PTH level for any reason, active bone fracture/healing, or primary disorder of bone development or metabolism.
• Women who are pregnant or breast feeding.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Профилактика

Центры проведения

США · 1 центр

• Cincinnati Children's Hospital Medical Center — Cincinnati

Идентификаторы

NCT: NCT07375290 · 2023-0052

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗