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Genetics in Predicting Risk of Bisphosphonate-Related Osteonecrosis of the Jaw in Patients With Cancer Receiving Zoledronic Acid

Not Applicable
Withdrawn
Conditions
Musculoskeletal Complications
Malignant Neoplasm
Interventions
Other: pharmacological study
Registration Number
NCT02069340
Lead Sponsor
University of Southern California
Brief Summary

This randomized clinical trial studies genetics in predicting risk of bisphosphonate-related osteonecrosis of the jaw in patients with cancer receiving zoledronic acid. Zoledronic acid is an anti-resorptive drug used as part of cancer treatment. A serious side effect of these drugs is death of the jawbone, commonly called bisphosphonate-related osteonecrosis of the jaw (BRONJ). Genetic research may help doctors understand risk factors for BRONJ or who is more likely to get BRONJ and why.

Detailed Description

PRIMARY OBJECTIVES:

I. To develop a pharmacometric model to predict jawbone zoledronic acid (Zol) concentrations in oncologic patients by conducting a prospective cohort study of Zol pharmacometrics in BRONJ patients, measuring drug in plasma, urine, and jawbone tissue obtained during surgical treatment for BRONJ.

SECONDARY OBJECTIVES:

I. To clinically assess and validate our predictive pharmacometric model, and develop a risk model for BRONJ in oncologic patients receiving intravenous Zol.

OUTLINE: Patients are randomized to 1 of 2 treatment arms.

ARM I: Patients receive zoledronic acid intravenously (IV) over 15 minutes on day 1.

ARM II: Patients receive zoledronic acid IV over 30 minutes on day 1.

After completion of study treatment, patients are followed up for 1 month.

Recruitment & Eligibility

Status
WITHDRAWN
Sex
All
Target Recruitment
Not specified
Inclusion Criteria
  • PATIENTS WITH BRONJ:
  • All cancer patients > 18 years of any ethnicity who have been treated with intravenous zoledronate (zoledronic acid) for >=1 year duration
  • Clinical diagnosis of BRONJ subsequent to oral surgery as established by standard clinical protocol per American Association of Oral and Maxillofacial Surgeons (AAOMS) diagnostic criteria
  • Willingness to have photographs taken to document lesions
  • Consent for sample collection for urine, hematology, histopathology and microbial profiling
  • Cognitively able and willing to provide consent
  • Have a World Health Organization (WHO)/Eastern Cooperative Oncology Group (ECOG) performance score =< 2 and life expectancy > 6 months
  • PATIENTS WITHOUT BRONJ:
  • Cancer patients without BRONJ who have been treated with intravenous zoledronate for >= 1 year duration
  • No signs or symptoms of BRONJ
  • Willingness to provide consent for sample collection for blood, urine and saliva
Exclusion Criteria
  • WHO/ECOG performance score > 2 and life expectancy of < 6 months
  • Coagulopathy
  • Active systemic infection or autoimmune disease
  • Currently pregnant or within 3 months post-partum, or unwilling to undergo pregnancy testing or report possible pregnancy promptly
  • Severe cardiovascular, pulmonary or other systemic conditions that prevent participation in the study
  • Salivary gland hypofunction regardless of underlying pathology
  • Neutropenia (serum absolute neutrophil count [ANC] < 1,000/uL)
  • Cognitive, language or hearing problems
  • Renal disease, and we will use a calculated serum creatinine clearance over 30 ml/min at the screening appointment as an exclusion criteria
  • Participation in another research project that might interfere with completion of this study
  • Patients undergoing active antibiotic therapy

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Arm II (zoledronic acid over 30 minutes)pharmacological studyPatients receive zoledronic acid IV over 30 minutes on day 1.
Arm I (zoledronic acid over 15 minutes)pharmacological studyPatients receive zoledronic acid IV over 15 minutes on day 1.
Arm II (zoledronic acid over 30 minutes)zoledronic acidPatients receive zoledronic acid IV over 30 minutes on day 1.
Arm I (zoledronic acid over 15 minutes)zoledronic acidPatients receive zoledronic acid IV over 15 minutes on day 1.
Primary Outcome Measures
NameTimeMethod
Jawbone tissue concentrations of Zol collected during surgical treatment for BRONJUp to 1 month

Data will be iteratively fit to the model using non-parametric modeling, simulation, and clinical dosing software. The parameters will be estimated, as well as their relationships to each other. Each measured patient concentration is Fisher weighted.

Plasma concentrations of Zol collected at visits 2, 3, 4, and 5Up to 1 month

Data will be iteratively fit to the model using non-parametric modeling, simulation, and clinical dosing software. The parameters will be estimated, as well as their relationships to each other. Each measured patient concentration is Fisher weighted.

Urine concentrations of Zol collected at visits 2, 3, 4, and 5Up to 1 month

Data will be iteratively fit to the model using non-parametric modeling, simulation, and clinical dosing software. The parameters will be estimated, as well as their relationships to each other. Each measured patient concentration is Fisher weighted.

Secondary Outcome Measures
NameTimeMethod
Identify potential risk factors for BRONJUp to1 month

The magnitude of associations between the study variables and BRONJ status will be estimated. For categorical variables, the univariate association with each variable and with BRONJ will be determined using Wald's test of association. For continuous variables, the association with each variable and BRONJ will be determined using Wald's test. Logistic regression will be used to evaluate the risk of BRONJ for development of the final risk model.

Trial Locations

Locations (2)

USC Norris Comprehensive Cancer Center

🇺🇸

Los Angeles, California, United States

J.Craig Venter Institute-San Diego

🇺🇸

San Diego, California, United States

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