An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)

CompletadoFase 3ClinicalTrials.gov

ID: NCT04185363Tipo: INTERVENTIONALInicio: 8 de ene de 2020Fin estimado: 23 de abr de 2025

Traducción no disponible, mostrando original

Resumen

The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.

Descripción detallada

The study will be conducted at multiple sites in North America, Europe, Asia, and South America.

Elegibilidad

Edad mínima: 1 YearEdad máxima: 18 YearsSexo: ALL

Criterios de inclusión

Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
Completion of study MRX-502

Criterios de exclusión

Any female who is pregnant or lactating or who is planning to become pregnant
Administration of prohibited medication between the MRX-502 EOT visit and the MRX 503 Baseline Visit (Day 0)
History of non-compliance in study MRX-502, non-adherence to medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to non-adherence with the study protocol based on Investigator judgment
Experienced an adverse event (AE) or serious adverse event (SAE) related to maralixibat during the MRX-502 study that led to permanent discontinuation of the subject from maralixibat
Any other conditions or laboratory abnormalities that, in the opinion of the Investigator or Sponsor Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study

Intervenciones

drug

Maralixibat

All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram \[mcg/kg\]) twice daily

Ubicaciones

Hospital Italiano de Buenos Aires

Buenos Aires, Argentina

Patrocinadores

PrincipalMirum Pharmaceuticals, Inc.

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