A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease (LOPD)

CompletadoFase 3ClinicalTrials.gov

ID: NCT04138277Tipo: INTERVENTIONALInicio: 18 de dic de 2019Fin estimado: 31 de dic de 2024

Traducción no disponible, mostrando original

Resumen

This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.

Descripción detallada

This is an open-label extension study for subjects who completed the ATB200-03 study. The subjects will stay in this study until regulatory approval or marketing authorization and/or commercialization in the participating subject's country.

Elegibilidad

Edad mínima: 18 YearsSexo: ALL

Criterios de inclusión

\. Subject must have completed Study ATB200-03.
Note: Subjects who were forced to withdraw from Study ATB200-03 for a logistical reason not related to the efficacy or safety of cipaglucosidase alfa/miglustat (eg, hospitalization for a car accident, COVID-19 pandemic, or emergency surgery) that resulted in several consecutive missed doses may have been eligible to participate in this study upon approval by the Amicus medical monitor.

Criterios de exclusión

Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.
Subject, if female, is pregnant or breastfeeding.
Subject, whether male or female, is planning to conceive a child during the study.
Subject had a hypersensitivity to any of the excipients in cipaglucosidase alfa or miglustat, or had a medical condition or any other extenuating circumstance that may have, in the opinion of the investigator or medical monitor, posed an undue safety risk to the subject or may have compromised his/her ability to comply with or adversely impacted protocol requirements.

Intervenciones

drug

AT2221

Participants received ATB200 co-administered with AT2221 (miglustat)

biological

ATB200

Enzyme Replacement Therapy via intravenous infusion

Ubicaciones

Hospital Universitario Austral

Buenos Aires, Argentina

Patrocinadores

PrincipalAmicus Therapeutics

Aviso: La información de este ensayo proviene de fuentes públicas y tiene carácter exclusivamente informativo. No constituye asesoramiento médico, regulatorio ni de ningún otro tipo. Las traducciones son automatizadas y no poseen validez oficial. Consultá siempre con un profesional de la salud. Ver Términos de Uso.

A Phase 3 Open-label Extension Study to Assess the Long-t... | EligiMed