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BONAPH1DE

Overview

Información sobre este estudio

This is a global, prospective, observational, longitudinal study designed to characterize the long-term safety of lumasiran in PH1 patients in the real-world setting. The data collected in this
study will also be utilized to further characterize the natural history and real-world clinical management of patients diagnosed with PH1, as well as the long-term effectiveness of lumasiran. Patients will be managed and treated per routine clinical practice. This protocol does not recommend the use of any specific treatments, visits, or procedures. No medication is provided as part of study participation.


Patients are expected to contribute data for the duration of the study or until study discontinuation (e.g., due to death, withdrawal of consent, loss to follow-up, study termination, enrollment in a clinical trial with an investigational treatment during the study). Study data will be collected at the time of a routine clinical encounter, or by referencing the medical record, and entered into the electronic data capture (EDC) at least once every 12 months using electronic case report forms (eCRFs). If patients attend more than 1 visit per year, sites can enter multiple follow-up visits. In addition to the prospective data collection, a chart review of up to 5 years relative to the time of enrollment in the study will be conducted to collect retrospective data, where available. In cases where diagnosis was more than 5 years prior to enrollment data will also be collected at the time of diagnosis, if available.

Elegibilidad para la participación

Los requisitos de elegibilidad de los participantes incluyen la edad, el sexo, el tipo y el estadio de la enfermedad, y los problemas de salud o tratamientos previos. Las pautas difieren de un estudio a otro e identifican quiénes pueden o no pueden participar. No hay garantía de que cada persona elegible que desee participar en un ensayo se inscribirá. Comunícate con el equipo del estudio para analizar la elegibilidad del estudio y la posible participación.

Inclusion Criteria:

  •  Documented diagnosis of PH1, per physician’s determination.
  • Written patient consent (per local regulations or ethics committee requirements) obtained prior to enrollment. If the patient is under the age of legal consent, written consent must be obtained from their legal guardian, and the patient should provide assent per local and national requirements. Should they become adults during their participation in the study, they will review and sign the adult consent, as applicable. Consent may be obtained from a legal representative if required and permitted by country-specific regulations or requirements.

Exclusion Criteria:

  • Currently enrolled in a clinical trial for any investigational agent.

Sedes participantes de Mayo Clinic

Los estatus de los estudios cambian con frecuencia. Comunícate con el equipo del estudio para obtener la información más actualizada acerca de la posibilidad de participar.

Sede de Mayo Clinic Estatus Contacto

Rochester, Minn.

Investigador principal de Mayo Clinic

John Lieske, M.D.

Abierto para la inscripción

Contact information:

Carly Banks CCRP

(507) 255-4347

Banks.Carly@mayo.edu

More information

Publicaciones

Publications are currently not available

Otros temas en investigación

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CLS-20541448

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