Resumen

Se trata de un estudio multicéntrico de fase 3, aleatorizado, doble ciego y controlado con placebo para evaluar la eficacia y seguridad del comprimido de liberación prolongada de deucrictibant administrado una vez al día por vía oral en comparación con placebo, para la profilaxis y prevención de los ataques de angioedema en participantes de ≥ 12 años con angioedema hereditario.

Descripción detallada

El estudio consta de un Período de Tamizaje durante el cual se confirma la elegibilidad, un Período de Tratamiento de 24 semanas y un Período de Seguimiento de un máximo de 4 semanas; alternativamente, los participantes podrán incorporarse al estudio de etiqueta abierta PHA022121-C307 (CHAPTER-4). Durante el Período de Tratamiento, los participantes recibirán el fármaco del estudio enmascarado (deucrictibant o placebo, aleatorizados en proporción 2:1). Los participantes se someterán a evaluaciones periódicas de eficacia y seguridad, completarán un diario electrónico a diario y responderán cuestionarios en momentos predefinidos del estudio.

Elegibilidad

Edad mínima: 12 YearsSexo: ALL

Criterios de inclusión

Provision of written informed consent/assent.
Male or female, aged ≥12 years at the time of providing written informed consent/assent.
Diagnosis of hereditary angioedema (HAE)
History of at least 3 HAE attacks within the 3 consecutive months prior to Screening Visit
Predefined number of attacks during the Screening Period
Reliable access and ability to use standard of care on-demand treatments to effectively manage acute HAE attacks.
Willing and able to adhere to all protocol requirements, including eDiary and ePRO data recording.
Female participants of childbearing potential must agree to the protocol specified pregnancy testing and contraception methods.

Criterios de exclusión

Any diagnosis of angioedema other than HAE
Participation in a clinical study with any other investigational drug within the last 30 days or within 5 half-lives of the investigational drug at Screening (whichever is longer)
Has received prior prophylactic treatment with deucrictibant
Exposure to ACE inhibitors or any estrogen-containing medications with systemic absorption within 4 weeks of Screening
Prior gene therapy for any indication at any time
Use of prophylactic treatment for HAE within 2 weeks of Screening for C1INH, oral kallikrein inhibitors, or anti-fibrinolytics; within 4 weeks of Screening for attenuated androgens; within 5 half-lives of Screening for monoclonal antibodies, or within 7 days of Screening for short-term prophylaxis
Any females who are pregnant, plan to become pregnant, or are currently breast-feeding
Abnormal hepatic function
Moderate or severe renal impairment
Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study.
History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse
Use of medications that are moderate and strong inhibitors or strong inducers of CYP3A4 within the last 30 days or within 5 half-lives (whichever is longer) of the time of randomization
Known hypersensitivity to deucrictibant or any of the excipients of the study drug

Estudio de fase 3, aleatorizado, doble ciego, controlado con placebo para evaluar la eficacia y seguridad del comprimido de liberación prolongada de deucrictibant administrado por vía oral para la profilaxis de los ataques de angioedema en adolescentes y adultos con angioedema hereditario

Resumen

Descripción detallada

Elegibilidad

Criterios de inclusión

Criterios de exclusión

Intervenciones

Deucrictibant

Placebo

Ubicaciones

Study Site

Study Site

Patrocinadores