A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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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

Conditions
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Pompe Disease (Late-onset)

Intervention / Treatment
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* Drug: AT2221
* Biological: ATB200


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 1. Subject must have completed Study ATB200-03. 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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ATB200/AT2221<br>Participants received ATB200 co-administered with AT2221 capsule (Miglustat) | Drug: AT2221<br>* Participants received ATB200 co-administered with AT2221 (Miglustat)<br>* Other names: Miglustat;Biological: ATB200<br>* Enzyme Replacement Therapy via intravenous infusion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of participants with Treatment Emergent Adverse Events (TEAE) |  | baseline, up to approximately 4 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 6-Minute Walk Test | Change in 6MWD from baseline to assess the efficacy of ATB200/AT2221 co-administration | baseline, up to approximately 4 years | 
| Pulmonary Function - Forced vital capacity (FVC) | Change from baseline in FVC (sitting) to assess the efficacy of ATB200/AT2221 co-administration | baseline, up to approximately 4 years | 
| Change from baseline in muscle strength measured by Quantitative Muscle Strength testing |  | baseline, up to approximately 4 years | 
| Change from baseline in muscle strength measured by Manual Muscle Strength testing |  | baseline, up to approximately 4 years | 
| The Rasch-built Pompe-specific activity (R-PAct) questionnaires | Change in R-Pact from baseline to assess the efficacy of ATB200/AT2221 co-administration | baseline, up to approximately 4 years | 
| EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) questionnaires | Change from baseline in scores of EQ-5D-5L questionnaire to assess the efficacy of ATB200/AT2221 co-administration. | baseline, up to approximately 4 years | 
| Change from baseline in scores of PROMIS - physical function questionnaire |  | baseline, up to approximately 4 years | 
| Change from baseline in scores of PROMIS - fatigue questionnaire |  | baseline, up to approximately 4 years | 
| Change from baseline in scores of PROMIS - dyspnea questionnaire |  | baseline, up to approximately 4 years | 
| Change from baseline in scores of PROMIS - upper extremity questionnaire |  | baseline, up to approximately 4 years | 
| Motor Function - Gait, Stairs, Gower, Chair (GSGC) test | Change from baseline in GSGC score to assess the efficacy of ATB200/AT2221 co-administration. | baseline, up to approximately 4 years | 
| Physician's Global Impression of Change | Change in the Physician's Global Impression of Change (PGIC) evaluation to assess the efficacy of ATB200/AT2221 co-administration. | baseline, up to approximately 4 years | 
| Subject's Global Impression of Change | Change from baseline in scores of Subject's Global Impression of Change (SGIC) questionnaire to assess the efficacy of ATB200/AT2221 co-administration. | baseline, up to approximately 4 years | 
| Change from baseline Biomarker -CK |  | baseline, up to approximately 4 years | 
| Change from baseline Biomarker -uHex4 |  | baseline, up to approximately 4 years | 
| Immunogenicity: Incidence of neutralizing |  | baseline, up to approximately 4 years | 
| Immunogenicity: anti-drug antibodies |  | baseline, up to approximately 4 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pompe, rhGAA