An Efficacy and Safety Trial of Intravenous Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
1 settembre 2020 aggiornato da: Novartis Pharmaceuticals
A Multicenter, Randomized, Double-blind, Placebo Controlled Efficacy and Safety Trial of Intravenous Zoledronic Acid Twice Yearly Compared to Placebo in Osteoporotic Children Treated With Glucocorticoids.
This study was designed to evaluate the efficacy and safety of zoledronic acid compared to placebo in osteoporotic children treated with glucocorticoids
Panoramica dello studio
Stato
Completato
Condizioni
Intervento / Trattamento
Descrizione dettagliata
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.
Tipo di studio
Interventistico
Iscrizione (Effettivo)
34
Fase
- Fase 3
Contatti e Sedi
Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.
Luoghi di studio
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New South Wales
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Westmead, New South Wales, Australia, 2145
- Novartis Investigative Site
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British Columbia
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Vancouver, British Columbia, Canada, V6H 3V4
- Novartis Investigative Site
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Manitoba
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Winnipeg, Manitoba, Canada, R3E 0Z2
- Novartis Investigative Site
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Ontario
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Ottawa, Ontario, Canada, K1H 8L1
- Novartis Investigative Site
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Quebec
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Montreal, Quebec, Canada, H3T 1C5
- Novartis Investigative Site
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Montreal, Quebec, Canada, H3H 1P3
- Novartis Investigative Site
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Moscow, Federazione Russa, 119991
- Novartis Investigative Site
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Saint Petersburg, Federazione Russa, 195067
- Novartis Investigative Site
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Manchester, Regno Unito, M14 0JH
- Novartis Investigative Site
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Birmingham
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West Midlands, Birmingham, Regno Unito, B4 6NH
- Novartis Investigative Site
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Gauteng
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Soweto, Gauteng, Sud Africa, 2013
- Novartis Investigative Site
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Budapest, Ungheria, 1085
- Novartis Investigative Site
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Criteri di partecipazione
I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.
Criteri di ammissibilità
Età idonea allo studio
Da 5 anni a 17 anni (Bambino)
Accetta volontari sani
No
Sessi ammissibili allo studio
Tutto
Descrizione
Key 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
Key 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
Piano di studio
Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: Randomizzato
- Modello interventistico: Assegnazione parallela
- Mascheramento: Quadruplicare
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
|---|---|
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Sperimentale: Acido zoledronico
Infusione endovenosa di 0,05 mg/kg (max 5 mg) due volte l'anno (almeno 30 minuti) di acido zoledronico
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infusione endovenosa
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Comparatore placebo: Placebo
Twice yearly i.v of infusion of Placebo (similar dosing as active drug)
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infusione endovenosa
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 12
Lasso di tempo: Month 12
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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.
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Month 12
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Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 6
Lasso di tempo: Month 6
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Lumbar Spine Bone Mineral Density (BMD) Z-score was determined by the central imaging vendor before first treatment and at Month 6.
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.
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Month 6
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Mean Change From Baseline in Lumbar Spine BMC at Month 6 and 12
Lasso di tempo: Month 6, Month 12
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Lumbar Spine BMC was determined by the central imaging vendor before first treatment and at Months 6 and 12.
The methods to be used to measure BMC were described in the respective DXA Manuals.
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Month 6, Month 12
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Mean Change From Baseline in Total Body BMC at Month 6 and 12
Lasso di tempo: Month 6, Month 12
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Total body BMC was all determined by the central imaging vendor before first treatment and at Months 6 and 12.
The methods to be used to measure BMC were described in the respective DXA Manuals.
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Month 6, Month 12
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Mean Change From Baseline in Serum P1NP at Months 6 and 12
Lasso di tempo: Month 6, Month 12
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Serum Procollagen type 1 amino-terminal propeptide (P1NP) was collected before first treatment (baseline) and at Months 6 and Month 12 according to the instructions provided in the Laboratory Manual.
The samples were analyzed in batches at the laboratory.
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Month 6, Month 12
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Mean Change From Baseline in BSAP at Months 6 and 12
Lasso di tempo: Month 6, Month 12
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Bone specific alkaline phosphatase (BSAP) were collected before first treatment (baseline) and at Months 6 and Month 12 according to the instructions provided in the Laboratory Manual.
The samples were analyzed in batches at the laboratory.
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Month 6, Month 12
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Mean Change From Baseline in Serum NTX at Months 6 and 12
Lasso di tempo: Month 6, Month 12
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Serum Cross linked N-telopeptide (NTX) were collected before first treatment (baseline) and at Months 6 and Month 12 according to the instructions provided in the Laboratory Manual.
The samples were analyzed in batches at the laboratory.
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Month 6, Month 12
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Mean Change From Baseline in Serum TRAP-5b at Months 6 and 12
Lasso di tempo: Month 6, Month 12
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Serum Tartrate-resistant acid phosphatase isoform 5b (TRAP 5b) was collected before first treatment (baseline) and at Months 6 and Month 12 according to the instructions provided in the Laboratory Manual.
The samples were analyzed in batches at the laboratory.
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Month 6, Month 12
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Number of Participants With New Vertebral Fractures at Month 12
Lasso di tempo: Month 12
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New vertebral fractures were defined as fractures of Genant Grade 1 or higher that occurred at lumbar or thoracic spine from first dose infusion to the end of the study.
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Month 12
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Mean Change From Baseline in Vertebral Morphometry at Month 12
Lasso di tempo: Month 12
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Vertebral morphometry (or concave index) was calculated using the average ratio between mid-height and posterior height from L1 to L4 and performed by a central reader.
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Month 12
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Percentage of Patients With Reduction in Pain at Months 3, 6, 9 and 12
Lasso di tempo: Month 3, Month 6, Month 9 and Month 12
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Pain was evaluated at each visit (in office and telephone visit) at randomization, Months 3, 6, 9 and 12 using the Faces Pain Scale-Revised (FPS-R).
Children were selecting the face that best fits their pain.
The pain score ranged from 0 (No Pain) to 10 (Very Much Pain).
The reduction in pain from baseline by visit was evaluated based on whether or not patients had a decrease in their FPS-R from baseline.
If pain remained the same or worsened from baseline a patient was classified as '0' and if the pain scale decreased then the patient was classified as '1'.
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Month 3, Month 6, Month 9 and Month 12
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Mean Change From Baseline in 2nd Metacarpal Cortical Width at Month 12
Lasso di tempo: Month 12
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Left posteroanterior (PA) hand/wrist X-ray were taken at Visit 1 and at the Month 12 visit to assess bone age and the between-treatment differences for change in 2nd metacarpal cortical width at Month 12 relative to baseline.
If a fracture of the left upper extremity precluded radiographic imaging, then the right hand was evaluated for this purpose.
In this case, the right hand was be imaged at both Visit 1 and at Month 12.
The information was used in the assessment of bone density.
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Month 12
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Urinary Concentration of Zoledronic Acid at Month 12
Lasso di tempo: Month 12
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Urine was collected overnight or for at least 4 waking hours from all patients able to provide specimens, to measure urinary concentration of zoledronic acid at Month 12.
Only descriptive analysis done.
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Month 12
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Safety of Zoledronic Acid for the Treatment of Osteoporotic Children Treated With Glucocorticoids
Lasso di tempo: Baseline through Month 12
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Analysis of absolute and relative frequencies for treatment emergent Adverse Event (AE), Serious Adverse Event (SAE) and Deaths by primary System Organ Class (SOC) to demonstrate that zoledronic acid is safe for the treatment of osteoporotic children treated with glucocorticoids through the monitoring of relevant clinical and laboratory safety parameters.
Only descriptive analysis done.
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Baseline through Month 12
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Collaboratori e investigatori
Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.
Sponsor
Pubblicazioni e link utili
La persona responsabile dell'inserimento delle informazioni sullo studio fornisce volontariamente queste pubblicazioni. Questi possono riguardare qualsiasi cosa relativa allo studio.
Studiare le date dei record
Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.
Studia le date principali
Inizio studio (Effettivo)
4 dicembre 2008
Completamento primario (Effettivo)
5 marzo 2018
Completamento dello studio (Effettivo)
5 marzo 2018
Date di iscrizione allo studio
Primo inviato
26 novembre 2008
Primo inviato che soddisfa i criteri di controllo qualità
26 novembre 2008
Primo Inserito (Stima)
27 novembre 2008
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
2 settembre 2020
Ultimo aggiornamento inviato che soddisfa i criteri QC
1 settembre 2020
Ultimo verificato
1 settembre 2020
Maggiori informazioni
Termini relativi a questo studio
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
- CZOL446H2337
- 2008-001252-52 (Numero EudraCT)
Piano per i dati dei singoli partecipanti (IPD)
Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?
INDECISO
Descrizione del piano IPD
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .