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

NCT07375290 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 2023-0052
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Hematopoietic Stem Cell Transplantation

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (2)

• Bone health efficiency

  Baseline, +6 months, and +12 months

• Bone substrate turnover

  Baseline and +30 days

Study Arms (1)

Zoledronate

EXPERIMENTAL

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

Interventions

Zoledronate DRUG

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

Zoledronate

Eligibility Criteria

• Age: 5 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

MeSH Terms

Interventions

Zoledronic Acid

Intervention Hierarchy (Ancestors)

Diphosphonates; Organophosphonates; Organophosphorus Compounds; Organic Chemicals; Imidazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Central Study Contacts

Jessi Anderson

CONTACT

513-803-0177; Jessica.Anderson@cchmc.org

Brady Landon

CONTACT

Brady.Landon@cchmc.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 12, 2025
• First Posted: January 29, 2026
• Study Start: February 1, 2026
• Primary Completion (Estimated): January 1, 2028
• Study Completion (Estimated): November 1, 2028
• Last Updated: January 29, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)