An Efficacy and Safety Trial of Intravenous Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids

NCT ID: NCT00799266

Last Updated: 2020-09-02

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

34 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-12-04

Study Completion Date

2018-03-05

Brief Summary

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This study was designed to evaluate the efficacy and safety of zoledronic acid compared to placebo in osteoporotic children treated with glucocorticoids

Detailed Description

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In March 2017, Novartis stopped enrollment as the study was not feasible to be conducted due to low enrollment and other recruitment challenges. Patients receiving the treatment continued to receive the treatment per protocol.

Conditions

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Osteoporosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Zoledronic acid

Twice yearly 0.05 mg/kg (max 5 mg) i.v infusion (at least 30 minutes) of zoledronic acid

Group Type EXPERIMENTAL

Zoledronic acid

Intervention Type DRUG

intravenous infusion

Placebo

Twice yearly i.v of infusion of Placebo (similar dosing as active drug)

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

intravenous infusion

Interventions

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Zoledronic acid

intravenous infusion

Intervention Type DRUG

Placebo

intravenous infusion

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* A diagnosis of chronic rheumatologic conditions or inflammatory bowel disease or Duchenne muscular dystrophy requiring systemic glucocorticoids (i.v. or oral) within 12 months prior to screening
* Lumbar Spine BMDZ-score of -0.5 or worse
* Evidence of at least at least 1 vertebral compression fracture of Genant Grade 1 or higher (or radiographic signs of vertebral fracture) within 1 month from Screening visit OR One or more, low-trauma, lower extremity long-bone fracture which occurred sometime within the 2 years PRECEDING enrollment in the study OR Two or more, low-trauma, upper extremity long-bone fractures which occurred sometime within the 2 years PRECEDING enrollment in the study
* Consent/assent to study participation

Exclusion Criteria

* History of primary bone disease (OI, Idiopathic Juvenile Osteoporosis, Rickets/Osteomalacia)
* Any medical condition that might have interfered with the evaluation of lumbar spine BMD, such as severe scoliosis or spinal fusion. Patients with less than 3 evaluable vertebrae by Dual Energy X-ray Absorptiometry (DXA) evaluation in the region of interest lumbar 1 (L1) to lumbar 4 (L4),
* Hypocalcemia and hypophosphatemia
* Serum 25-hydroxy vitamin D concentrations of \<20 ng/mL or \<50 nmol/L
* estimated glomerular filtration rate (GFR) \<60 mL/min/1.73 m2
* serum creatinine increase between Visit 1 and Visit 2 \>0.5 mg/dL (44.2 μmol/L)
* Uncontrolled symptoms of cardiac failure or arrhythmia
* Any prior use of bisphosphonates, or high dose sodium fluoride
Minimum Eligible Age

5 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Novartis Investigative Site

Westmead, New South Wales, Australia

Site Status

Novartis Investigative Site

Vancouver, British Columbia, Canada

Site Status

Novartis Investigative Site

Winnipeg, Manitoba, Canada

Site Status

Novartis Investigative Site

Ottawa, Ontario, Canada

Site Status

Novartis Investigative Site

Montreal, Quebec, Canada

Site Status

Novartis Investigative Site

Montreal, Quebec, Canada

Site Status

Novartis Investigative Site

Budapest, , Hungary

Site Status

Novartis Investigative Site

Moscow, , Russia

Site Status

Novartis Investigative Site

Saint Petersburg, , Russia

Site Status

Novartis Investigative Site

Soweto, Gauteng, South Africa

Site Status

Novartis Investigative Site

West Midlands, Birmingham, United Kingdom

Site Status

Novartis Investigative Site

Manchester, , United Kingdom

Site Status

Countries

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Australia Canada Hungary Russia South Africa United Kingdom

References

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Ward LM, Choudhury A, Alos N, Cabral DA, Rodd C, Sbrocchi AM, Taback S, Padidela R, Shaw NJ, Hosszu E, Kostik M, Alexeeva E, Thandrayen K, Shenouda N, Jaremko JL, Sunkara G, Sayyed S, Aftring RP, Munns CF. Zoledronic Acid vs Placebo in Pediatric Glucocorticoid-induced Osteoporosis: A Randomized, Double-blind, Phase 3 Trial. J Clin Endocrinol Metab. 2021 Nov 19;106(12):e5222-e5235. doi: 10.1210/clinem/dgab458.

Reference Type DERIVED
PMID: 34228102 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2008-001252-52

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CZOL446H2337

Identifier Type: -

Identifier Source: org_study_id

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