Phase 3
Completed N=119
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)
Pompe Disease (Late-onset)
Source: ClinicalTrials.gov NCT04138277 ↗
Enrolled (actual)
119
Serious AEs
22.9%
Results posted
Jan 2026
Primary outcomePrimary: Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug — 80; 37; 17; 10 Participants
◆ Published Evidence
Established
27citations · ~14 / year
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).
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.
Linked Publications (3)
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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).
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Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe disease.
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Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug |
80; 37; 17; 10; 6; 2 | — |
| SECONDARY Change From Baseline in 6-Minute Walk Distance (6MWD) |
-33.3; -8.6 | — |
| SECONDARY Change From Baseline in Sitting % Predicted Forced Vital Capacity (FVC) |
-3.8; -0.5 | — |
| SECONDARY Change From Baseline in Manual Muscle Testing (MMT) Lower Extremity Score |
0.6; 0.4 | — |
| SECONDARY Change From Baseline in the Total Score for Patient-reported Outcomes Measurement Information System (PROMIS®) - Physical Function |
-2.7; -2.5 | — |
| SECONDARY Change From Baseline in the Total Score for PROMIS® - Fatigue |
1.5; 1.1 | — |
| SECONDARY Change From Baseline in Gait, Stairs, Gower, Chair (GSGC) Test |
0.5; 0.3 | — |
| SECONDARY Change From Baseline in EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) Responses |
27; 12; 4; 1; 0; 0 | — |
| SECONDARY Change From Baseline in Overall Physical Well-being (Subject's Global Impression of Change [SGIC], Question 1) |
0; 0; 0; 1; 12; 2 | — |
| SECONDARY Change From Baseline in Physician's Global Impression of Change (PGIC) Overall Status |
0; 0; 0; 1; 9; 3 | — |
| SECONDARY Percent Change From Baseline in Creatine Kinase (U/L) |
-17.8; -29.0 | — |
| SECONDARY Percent Change From Baseline in Urine Hex4 (mmol/Mol Creatinine) |
-16.7; -62.4 | — |
| SECONDARY Proportion of Subjects With Positive Anti-drug Antibodies at Baseline and Week 208 |
63; 30; 34; 17 | — |
Eligibility Criteria
Inclusion Criteria
- 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.
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.
Data sourced from ClinicalTrials.gov (NCT04138277) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.