Early zoledronate to prevent bone problems after pediatric stem cell transplant

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

This pilot tests whether giving zoledronate early can prevent bone loss and fractures in children and teens undergoing or recovering from hematopoietic stem cell transplant who are at high risk.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 5 Years to 18 Years
• Sex: All
• Sponsor: Children's Hospital Medical Center, Cincinnati (other)
• Drugs / interventions: radiation
• Locations: 1 site (Cincinnati, Ohio)
• Trial ID: NCT07375290 on ClinicalTrials.gov

What this trial studies

This Phase 1 pilot will give zoledronate to high-risk pediatric HSCT patients either around the time of transplant or after they develop graft-versus-host disease, with the primary goal of characterizing safety and looking for preliminary signals of benefit. Eligible patients are 5–18 years old with a height-adjusted DXA Z-score < -2.0 prior to transplant or those who develop de novo acute or chronic GVHD after transplant; major exclusions include prior bisphosphonate use, low 25-OH vitamin D, renal dysfunction, active infection, and radiation-sensitive syndromes. Treatment is administered during an inpatient admission at Cincinnati Children's Hospital Medical Center and participants will be monitored for adverse events, renal function, mineral metabolism, and changes in bone mineral density. Outcomes include safety endpoints and early measures of bone density and fracture prevention over follow-up.

Who should consider this trial

Good fit: Ideal candidates are children and adolescents aged 5–18 admitted to Cincinnati Children's for HSCT who have a height-adjusted DXA Z-score < -2.0 before transplant or who develop new acute or chronic GVHD after transplant, and who do not have exclusion conditions such as low vitamin D, renal impairment, or prior bisphosphonate exposure.

Not a fit: Patients younger than 5 or older than 18, those with prior bisphosphonate use, vitamin D deficiency, significant renal impairment, radiation-sensitive syndromes (e.g., Fanconi anemia), active febrile illness or uncontrolled infection, pregnancy or breastfeeding, or those without low bone density/high risk are unlikely to benefit from this intervention.

Why it matters

Potential benefit: If successful, early zoledronate could reduce the development of osteoporosis and fractures after pediatric HSCT, lowering pain and long-term bone complications.

How similar studies have performed: Bisphosphonates including zoledronate have been effective for treating established pediatric osteoporosis, but prophylactic early use around HSCT is novel and has limited prior evidence.

Eligibility criteria

Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Where this trial is running

Cincinnati, Ohio

• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (RECRUITING)

Study contacts

• Study coordinator: Jessi Anderson

Conditions: Hematopoietic Stem Cell Transplantation