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

1 de setembro de 2020 atualizado por: 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

Visão geral do estudo

Status

Concluído

Condições

Intervenção / Tratamento

Descrição detalhada

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 de estudo

Intervencional

Inscrição (Real)

34

Estágio

  • Fase 3

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Locais de estudo

  • Austrália
    • New South Wales
      • Westmead, New South Wales, Austrália, 2145
        • Novartis Investigative Site
  • Canadá
    • British Columbia
      • Vancouver, British Columbia, Canadá, V6H 3V4
        • Novartis Investigative Site
    • Manitoba
      • Winnipeg, Manitoba, Canadá, R3E 0Z2
        • Novartis Investigative Site
    • Ontario
      • Ottawa, Ontario, Canadá, K1H 8L1
        • Novartis Investigative Site
    • Quebec
      • Montreal, Quebec, Canadá, H3T 1C5
        • Novartis Investigative Site
      • Montreal, Quebec, Canadá, H3H 1P3
        • Novartis Investigative Site
  • Federação Russa
      • Moscow, Federação Russa, 119991
        • Novartis Investigative Site
      • Saint Petersburg, Federação Russa, 195067
        • Novartis Investigative Site
  • Hungria
      • Budapest, Hungria, 1085
        • Novartis Investigative Site
  • Reino Unido
      • Manchester, Reino Unido, M14 0JH
        • Novartis Investigative Site
    • Birmingham
      • West Midlands, Birmingham, Reino Unido, B4 6NH
        • Novartis Investigative Site
  • África do Sul
    • Gauteng
      • Soweto, Gauteng, África do Sul, 2013
        • Novartis Investigative Site

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

5 anos a 17 anos (Filho)

Aceita Voluntários Saudáveis

Não

Gêneros Elegíveis para o Estudo

Tudo

Descrição

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

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: Randomizado
  • Modelo Intervencional: Atribuição Paralela
  • Mascaramento: Quadruplicar

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Experimental: Ácido zoledrônico
Duas vezes por ano 0,05 mg/kg (max 5 mg) infusão i.v (pelo menos 30 minutos) de ácido zoledrônico
Medicamento: Ácido zoledrônico
infusão intravenosa
Comparador de Placebo: Placebo
Twice yearly i.v of infusion of Placebo (similar dosing as active drug)
Medicamento: Placebo
infusão intravenosa

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 12
Prazo: 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

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 6
Prazo: Month 6
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.
Month 6
Mean Change From Baseline in Lumbar Spine BMC at Month 6 and 12
Prazo: Month 6, Month 12
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.
Month 6, Month 12
Mean Change From Baseline in Total Body BMC at Month 6 and 12
Prazo: Month 6, Month 12
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.
Month 6, Month 12
Mean Change From Baseline in Serum P1NP at Months 6 and 12
Prazo: Month 6, Month 12
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.
Month 6, Month 12
Mean Change From Baseline in BSAP at Months 6 and 12
Prazo: Month 6, Month 12
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.
Month 6, Month 12
Mean Change From Baseline in Serum NTX at Months 6 and 12
Prazo: Month 6, Month 12
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.
Month 6, Month 12
Mean Change From Baseline in Serum TRAP-5b at Months 6 and 12
Prazo: Month 6, Month 12
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.
Month 6, Month 12
Number of Participants With New Vertebral Fractures at Month 12
Prazo: Month 12
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.
Month 12
Mean Change From Baseline in Vertebral Morphometry at Month 12
Prazo: Month 12
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.
Month 12
Percentage of Patients With Reduction in Pain at Months 3, 6, 9 and 12
Prazo: Month 3, Month 6, Month 9 and Month 12
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'.
Month 3, Month 6, Month 9 and Month 12
Mean Change From Baseline in 2nd Metacarpal Cortical Width at Month 12
Prazo: Month 12
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.
Month 12
Urinary Concentration of Zoledronic Acid at Month 12
Prazo: Month 12
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.
Month 12
Safety of Zoledronic Acid for the Treatment of Osteoporotic Children Treated With Glucocorticoids
Prazo: Baseline through Month 12
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.
Baseline through Month 12

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Novartis Pharmaceuticals

Publicações e links úteis

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Publicações Gerais

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Real)

4 de dezembro de 2008

Conclusão Primária (Real)

5 de março de 2018

Conclusão do estudo (Real)

5 de março de 2018

Datas de inscrição no estudo

Enviado pela primeira vez

26 de novembro de 2008

Enviado pela primeira vez que atendeu aos critérios de CQ

26 de novembro de 2008

Primeira postagem (Estimativa)

27 de novembro de 2008

Atualizações de registro de estudo

Última Atualização Postada (Real)

2 de setembro de 2020

Última atualização enviada que atendeu aos critérios de controle de qualidade

1 de setembro de 2020

Última verificação

1 de setembro de 2020

Mais Informações

Termos relacionados a este estudo

Palavras-chave

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • CZOL446H2337
  • 2008-001252-52 (Número EudraCT)

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

INDECISO

Descrição do plano 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.

Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .

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