Esta página se tradujo automáticamente y no se garantiza la precisión de la traducción. por favor refiérase a versión inglesa para un texto fuente.

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

1 de septiembre de 2020 actualizado 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

Descripción general del estudio

Estado

Terminado

Condiciones

Intervención / Tratamiento

Descripción detallada

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 estudio

Intervencionista

Inscripción (Actual)

34

Fase

  • Fase 3

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

  • Australia
    • New South Wales
      • Westmead, New South Wales, Australia, 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
  • Federación Rusa
      • Moscow, Federación Rusa, 119991
        • Novartis Investigative Site
      • Saint Petersburg, Federación Rusa, 195067
        • Novartis Investigative Site
  • Hungría
      • Budapest, Hungría, 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
  • Sudáfrica
    • Gauteng
      • Soweto, Gauteng, Sudáfrica, 2013
        • Novartis Investigative Site

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

5 años a 17 años (Niño)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Descripción

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

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: Aleatorizado
  • Modelo Intervencionista: Asignación paralela
  • Enmascaramiento: Cuadruplicar

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Ácido zoledrónico
Dos veces al año 0,05 mg/kg (máx. 5 mg) en perfusión i.v (al menos 30 minutos) de ácido zoledrónico
Droga: Ácido zoledrónico
infusión intravenosa
Comparador de placebos: Placebo
Twice yearly i.v of infusion of Placebo (similar dosing as active drug)
Droga: Placebo
infusión intravenosa

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 12
Periodo de tiempo: 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 resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 6
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Novartis Pharmaceuticals

Publicaciones y enlaces útiles

La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.

Publicaciones Generales

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

4 de diciembre de 2008

Finalización primaria (Actual)

5 de marzo de 2018

Finalización del estudio (Actual)

5 de marzo de 2018

Fechas de registro del estudio

Enviado por primera vez

26 de noviembre de 2008

Primero enviado que cumplió con los criterios de control de calidad

26 de noviembre de 2008

Publicado por primera vez (Estimar)

27 de noviembre de 2008

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

2 de septiembre de 2020

Última actualización enviada que cumplió con los criterios de control de calidad

1 de septiembre de 2020

Última verificación

1 de septiembre de 2020

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

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

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

INDECISO

Descripción del plan 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.

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

Suscribir