Tumor-induced Osteomalacia Disease Monitoring Program (TIO DMP)

Activo, no reclutaClinicalTrials.gov

ID: NCT04783428Tipo: OBSERVATIONALInicio: 31 de ene de 2022Fin estimado: 28 de feb de 2032

Traducción no disponible, mostrando original

Resumen

The objectives of this observational study are to assess the long-term safety and long-term effectiveness of burosumab in patients with TIO who are being treated with burosumab as prescribed by their physician and to monitor the course of the underlying phosphaturic mesenchymal tumor (PMT) overtime in patients with TIO irrespective of their treatment status.

Descripción detallada

Enrolled patients may or may not be treated with commercially available burosumab during the TIO DMP at the discretion of their treating physician. Given the observational nature of the TIO DMP, specific treatments or supportive management will not be provided as part of the study.

Elegibilidad

Sexo: ALL

Criterios de inclusión

Have a clinical diagnosis of TIO based on the presence of an underlying PMT (confirmed by imaging) AND/OR historical documentation. Note: For adult patients with TIO in whom the causative PMT has never been located, and all pediatric patients, documented evidence of negative genetic testing for other hereditary hypophosphatemic disorders is necessary
For patient safety, all participating female patients of child-bearing potential must be willing to have pregnancy tests prior to certain assessments performed as part of the DMP
Be willing to provide access to prior medical records including tumor pathology reports and biopsy slides, imaging, biochemical, and diagnostic, medical, and surgical history data, if available
Be willing and able to provide informed consent after the nature of the study has been explained, and prior to any research-related procedures
Be willing and able to comply with the study visit schedule and study procedures

Criterios de exclusión

Have a clinical diagnosis of TIO deemed to be caused by a tumor other than a PMT
Serious medical or psychiatric comorbidity that, in the opinion of the Investigator, would present a concern for patient safety or compromise the ability to provide consent or comply with the study visit schedule and study procedures
Less than 1 year of life expectancy (for any cause) in the opinion of the Investigator
Concurrent enrollment in a clinical trial without prior approval from the TIO DMP Sponsor
Undergoing treatment with burosumab for an unapproved indication

Intervenciones

other

No intervention

Access to any treatment is through authorized commercial use and not as part of this DMP

Ubicaciones

IDIM - Instituto de Diagnóstico e Investigaciones Metabólicas

Buenos Aires, Argentina

Patrocinadores

PrincipalUltragenyx Pharmaceutical Inc

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