NCT00799266

Brief Summary

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

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
34

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Dec 2008

Longer than P75 for phase_3

Geographic Reach
6 countries

12 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 26, 2008

Completed
1 day until next milestone

First Posted

Study publicly available on registry

November 27, 2008

Completed
7 days until next milestone

Study Start

First participant enrolled

December 4, 2008

Completed
9.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 5, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 5, 2018

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

July 5, 2019

Completed
Last Updated

September 2, 2020

Status Verified

September 1, 2020

Enrollment Period

9.3 years

First QC Date

November 26, 2008

Results QC Date

September 4, 2018

Last Update Submit

September 1, 2020

Conditions

Osteoporosis

Keywords

Osteoporosis, children and adolescents, zoledronic acid, chronic inflammation, Duchenne muscular dystrophy, glucocorticoids, chronic inflammatory conditions

Outcome Measures

Primary Outcomes (1)

  • Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 12

    Lumbar Spine Bone Mineral Density (BMD) Z-score was determined by the central imaging vendor before first treatment and at Month 12. The methods to be used to measure Lumbar Spine BMD Z-score were described in the respective DXA Manuals provided by central imaging vendor. Positive changes from baseline indicated an improvement in condition.

    Month 12

Secondary Outcomes (13)

  • Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 6

    Month 6

  • Mean Change From Baseline in Lumbar Spine BMC at Month 6 and 12

    Month 6, Month 12

  • Mean Change From Baseline in Total Body BMC at Month 6 and 12

    Month 6, Month 12

  • Mean Change From Baseline in Serum P1NP at Months 6 and 12

    Month 6, Month 12

  • Mean Change From Baseline in BSAP at Months 6 and 12

    Month 6, Month 12

  • +8 more secondary outcomes

Study Arms (2)

Zoledronic acid

EXPERIMENTAL

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

Drug: Zoledronic acid

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

Zoledronic acidDRUG

intravenous infusion

Zoledronic acid
PlaceboDRUG

intravenous infusion

Placebo

Eligibility Criteria

Age5 Years - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

Novartis Investigative Site

Westmead, New South Wales, 2145, Australia

Location

Novartis Investigative Site

Vancouver, British Columbia, V6H 3V4, Canada

Location

Novartis Investigative Site

Winnipeg, Manitoba, R3E 0Z2, Canada

Location

Novartis Investigative Site

Ottawa, Ontario, K1H 8L1, Canada

Location

Novartis Investigative Site

Montreal, Quebec, H3H 1P3, Canada

Location

Novartis Investigative Site

Montreal, Quebec, H3T 1C5, Canada

Location

Novartis Investigative Site

Budapest, 1085, Hungary

Location

Novartis Investigative Site

Moscow, 119991, Russia

Location

Novartis Investigative Site

Saint Petersburg, 195067, Russia

Location

Novartis Investigative Site

Soweto, Gauteng, 2013, South Africa

Location

Novartis Investigative Site

West Midlands, Birmingham, B4 6NH, United Kingdom

Location

Novartis Investigative Site

Manchester, M14 0JH, United Kingdom

Location

Related Publications (1)

  • 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.

    PMID: 34228102DERIVED

MeSH Terms

Conditions

OsteoporosisMuscular Dystrophy, Duchenne

Interventions

Zoledronic Acid

Condition Hierarchy (Ancestors)

Bone Diseases, MetabolicBone DiseasesMusculoskeletal DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesMuscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

DiphosphonatesOrganophosphonatesOrganophosphorus CompoundsOrganic ChemicalsImidazolesAzolesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Results Point of Contact

Title
Study Director
Organization
Novartis Pharmaceuticals
Novartis.email@novartis.com
862-778-8300

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 26, 2008

First Posted

November 27, 2008

Study Start

December 4, 2008

Primary Completion

March 5, 2018

Study Completion

March 5, 2018

Last Updated

September 2, 2020

Results First Posted

July 5, 2019

Record last verified: 2020-09

Locations

GB(2)AU(1)CA(5)HU(1)RU(2)ZA(1)