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

NCT04138277Phase 3Completed

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Lead sponsor
Amicus Therapeutics (Industry)
Enrollment
119 (actual)
Start date
18 Dec 2019
Primary completion
31 Dec 2024 (actual)
Study completion
31 Dec 2024
Last update posted
5 Mar 2026

Design

Study type
Interventional
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None
Arms
1

Interventions

  • AT2221 Drug
  • ATB200 Biological

Conditions and primary outcome

  • Pompe Disease (Late-onset)

Primary outcome

Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug (Entire extension study (mean = 40.5 months on treatment))

Posted results on ClinicalTrials.gov

Source: ClinicalTrials.gov results for NCT04138277 (opens in a new tab)· Licence: public-domain

First posted · Last update posted · Imported from ClinicalTrials.gov download 2026-10-25

Primary outcomes with a statistical analysis

No primary outcome with a statistical analysis is posted for this study.

Serious adverse events

Time frame: Adverse event data collected over duration of the study. Duration of participation varied by patient (range: 1 to 58 months).

Participants with serious adverse events per group for NCT04138277, as posted
GroupAffectedAt risk
Treatment Continued Group1781
Treatment Switched Group1037

Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately.

Source: ClinicalTrials.gov, U.S. National Library of Medicine. Listing a study does not mean NLM has evaluated it.

Results from company and journal sources

No editor-confirmed results are on record for this study.

Publications

3 on PubMed

  1. Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe disease.

    Therapeutic advances in rare disease · 2026 Jan-Dec

    PubMed 41769220 (opens in a new tab)DOI 10.1177/26330040261425686 (opens in a new tab)

  2. Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials.

    Therapeutic advances in rare disease · 2026 Jan-Dec

    PubMed 41631150 (opens in a new tab)DOI 10.1177/26330040261416943 (opens in a new tab)

  3. 104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).

    Journal of neurology · 2024 May

    PubMed 38418563 (opens in a new tab)DOI 10.1007/s00415-024-12236-0 (opens in a new tab)

Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies.

CT-VCA trial quality

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Data integrity

Compare what the registry, company releases, FDA documents and publications say about this study.

Drugs on HealthTrade

Source

Source: ClinicalTrials.gov, U.S. National Library of Medicine. Listing a study does not mean NLM has evaluated it.

Nothing on this page is medical or investment advice.