Clinical trial NCT00051636: Safety and Efficacy Trial With Zoledronic Acid for the Treatment of Paget's Disease of Bone, Including an Extended Observation Period
Official title: Randomized, Double-blind, Safety and Efficacy Trial With Intravenous Zoledronic Acid for the Treatment of Paget's Disease of Bone Using Risedronate as a Comparator, Including an Extended Observational Period
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Paget's Disease of Bone
Keywords: Bisphosphonate
SAP
SAP excess
non-inferiority
therapeutic response
extended observation period
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2003-01-15
Enrollment: 172
Sex: ALL
Minimum age (years): 30
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The core study looked at the effect of Zoledronic Acid given once as an intravenous (i.v.) infusion compared to 60 days of oral Risedronate in patients with Paget's disease of bone. The effect was demonstrated in the reduction of serum alkaline phosphatase (SAP). The extended observation period included participants of the core study who responded to treatment.


Eligibility criteria: Inclusion Criteria:

* 30 years or older
* Serum alkaline phosphatase (SAP) 2 times upper limit normal (ULN)
* Confirmed diagnosis of Paget's disease of the bone (by x-ray, magnetic resonance imaging, computerized tomography, radioisotope imaging, etc.).
* 90 days washout calcitonin
* 180 day washout bisphosphonate

Exclusion Criteria:

* Allergic reaction to bisphosphonates
* History of upper gastrointestinal disorders
* History of iritis, uveitis
* Calculated creatinine clearance \< 30 ml/min at baseline
* Evidence of vitamin D deficiency

Other protocol-defined inclusion/exclusion criteria applied.

Primary outcomes:
measure: Number of Patients Who Achieve Therapeutic Response at 6 Months.
timeFrame: 6 months
Secondary outcomes:
measure: Relative Change in Serum Alkaline Phosphatase (SAP) in Units Per Liter (U/L) at Day 28
timeFrame: Baseline and day 28
measure: Relative Change in Serum C-telopeptide (CTx) in ng/mL at Day 10
timeFrame: Baseline and day 10
measure: Relative Change in Urine Alpha C-telopeptide (α-CTx) in ug/mmol at Day 10
timeFrame: Baseline and day 10
measure: Time to First Therapeutic Response
timeFrame: 182 days
measure: Number of Patients Who Achieved Serum Alkaline Phosphatase Normalization at Day 28 Relative to Baseline
timeFrame: Baseline and day 28
measure: Change in Pain Severity Score
timeFrame: Baseline and day 182
measure: Change in Pain Interference Score
timeFrame: Baseline and day 182
measure: Number of Participants With a Loss of Therapeutic Response During the Extended Observation Period
timeFrame: 8 years was the maximum
measure: Number of Participants With a Partial Disease Relapse During the Extended Observation Period
timeFrame: 8 years was the maximum
measure: Number of Participants With a Disease Relapse During the Extended Observation Period
timeFrame: 8 years was the maximum
Other outcomes:
Locations:
city: Santa Monica
country: United States
facility: Novartis Investigative Site
state: California
city: Lakewood
country: United States
facility: Novartis Investigative Site
state: Colorado
city: Boca Raton
country: United States
facility: Novartis Investigative Site
state: Florida
city: Miami
country: United States
facility: Novartis Investigative Site
state: Florida
city: Maywood
country: United States
facility: Novartis Investigative site
state: Illinois
city: Worcester
country: United States
facility: Novartis investigative site
state: Massachusetts
city: Detroit
country: United States
facility: Novartis investigative site
state: Michigan
city: New York
country: United States
facility: Novartis Investigative Site
state: New York
city: Syracuse
country: United States
facility: Novartis investigative site
state: New York
city: Durham
country: United States
facility: Novartis investigative site
state: North Carolina
city: Medford
country: United States
facility: Novartis investigative site
state: Oregon
city: Providence
country: United States
facility: Novartis investigative site
state: Rhode Island
city: Concord
country: Australia
facility: Novartis Investigative Site
city: Fitzroy
country: Australia
facility: Novartis Investigative Site
city: Geelong
country: Australia
facility: Novartis Investigative site
city: Kogarah
country: Australia
facility: Novartis Investigative Site
city: Maroochydore
country: Australia
facility: Novartis Investigative site
city: Nedlands
country: Australia
facility: Novartis Investigative site
city: Calgary
country: Canada
facility: Novartis Investigative Site
city: London
country: Canada
facility: Novartis Investigative Site
city: Montreal
country: Canada
facility: Novartis Investigative Site
city: Ste-Foy
country: Canada
facility: Novartis Investigative Site
city: Toronto
country: Canada
facility: Novartis Investigative Site
city: Auckland
country: New Zealand
facility: Novartis Investigative site
city: Christchurch
country: New Zealand
facility: Novartis Investigative Site
city: Salamanca
country: Spain
facility: Novartis Investigative site
city: Liverpool
country: United Kingdom
facility: Novartis Investigative site
city: Manchester
country: United Kingdom
facility: Novartis Investigative Site
city: Nottingham
country: United Kingdom
facility: Novartis Investigative site
city: Oxford
country: United Kingdom
facility: Novartis Investigative Site
city: Penarth
country: United Kingdom
facility: Novartis Investigative site
city: Pernarth
country: United Kingdom
facility: Novartis investigative site
city: Stanmore
country: United Kingdom
facility: Novartis Investigative Site
city: Vale of Glamorgan
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
References: