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

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

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.

Study Overview

• Status: Recruiting
• Conditions: Hematopoietic Stem Cell Transplantation
• Intervention / Treatment: Drug: Zoledronate

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Jessi Anderson; Phone Number: 513-803-0177; Email: Jessica.Anderson@cchmc.org
• Study Contact Backup: Name: Brady Landon; Email: Brady.Landon@cchmc.org

Study Locations

• United States
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Recruiting
      • Cincinnati Children's Hospital Medical Center
      • Contact: Jessi Anderson; Email: Jessica.Anderson@cchmc.org
      • Principal Investigator: Jonathan Howell, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Zoledronate; Zoledronate dose will be given intravenously over 1 hour using standard infusion equipment. Patients will receive a dose of 0.025 mg/kg. Zoledronate will be infused in the inpatient setting to facilitate proper observation and data collection.	Drug: Zoledronate; Zoledronate is in the class of drugs entitled bisphosphonates which act to inhibit bone resorption by inhibiting osteoclast activity therefore reducing bone turnover.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of early intervention Zoledronate	Calcium levels following infusion of zoledronate	At the time of infusion through up to 60 days post last dose of Zoledronate
Feasibility of early intervention Zoledronate	Number of days required for inpatient stay beyond what is necessary for the patient's admission	At patient discharge, typically 1 week post infusion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Bone health efficiency	DXA scans will be measured with a z-score comparing bone density to averages for patient age and height	Baseline, +6 months, and +12 months
Bone substrate turnover	c-terminal telopeptide (CTX) and procollagen type 1 n-terminal propeptide (P1NP) measured in pg/mL	Baseline and +30 days

Collaborators and Investigators

• Sponsor: Children's Hospital Medical Center, Cincinnati

Study record dates

Study Major Dates

• Study Start (Estimated): February 1, 2026
• Primary Completion (Estimated): January 1, 2028
• Study Completion (Estimated): November 1, 2028

Study Registration Dates

• First Submitted: November 12, 2025
• First Submitted That Met QC Criteria: January 21, 2026
• First Posted (Actual): January 29, 2026

Study Record Updates

• Last Update Posted (Actual): January 29, 2026
• Last Update Submitted That Met QC Criteria: January 21, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Organic Chemicals; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Azoles; Imidazoles; Organophosphorus Compounds; Organophosphonates; Diphosphonates; Zoledronic Acid
• Other Study ID Numbers: 2023-0052

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No