CompletedPhase 3

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

United States119 participantsStarted 2019-12-18

Plain-language summary

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.

Who can participate

Age range18 Years

SexALL

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Exclusion criteria

✕. 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.

What they're measuring

Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug

Timeframe: Entire extension study (mean = 40.5 months on treatment)

Trial details

NCT IDNCT04138277

SponsorAmicus Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-12-31

Results submitted2025-12-19

View on ClinicalTrials.gov More Pompe Disease (Late-onset) trials