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

• Register: ClinicalTrials.gov
• Last refreshed on: 1 April 2024
• Main ID: NCT04138277
• Date of registration: 18/10/2019
• Prospective Registration: Yes
• Primary sponsor: Amicus Therapeutics
• Public title: A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD
• Scientific title: 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
• Date of first enrolment: December 18, 2019
• Target sample size: 110
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04138277
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Austria, Belgium, Bosnia and Herzegovina, Canada, Denmark, France, Germany, Greece, Hungary, Italy, Japan, Korea, Republic of, Netherlands, New Zealand, Poland, Slovenia, Spain, Sweden, Taiwan, United Kingdom, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Subject must have completed Study ATB200-03.

Exclusion Criteria

1. Subject plans to receive gene therapy or participate in another interventional study
for Pompe disease.

2. Subject, if female, is pregnant or breastfeeding.

3. Subject, whether male or female, is planning to conceive a child during the study.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Pompe Disease (Late-onset)

Intervention(s)

Biological: ATB200

Drug: AT2221

Primary Outcome(s)

Proportion of participants with Treatment Emergent Adverse Events (TEAE) [Time Frame: baseline, up to approximately 4 years]

Secondary Outcome(s)

Change from baseline in scores of PROMIS - fatigue questionnaire [Time Frame: baseline, up to approximately 4 years]

Change from baseline in scores of PROMIS - upper extremity questionnaire [Time Frame: baseline, up to approximately 4 years]

EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) questionnaires [Time Frame: baseline, up to approximately 4 years]

Immunogenicity: anti-drug antibodies [Time Frame: baseline, up to approximately 4 years]

Motor Function - Gait, Stairs, Gower, Chair (GSGC) test [Time Frame: baseline, up to approximately 4 years]

The Rasch-built Pompe-specific activity (R-PAct) questionnaires [Time Frame: baseline, up to approximately 4 years]

6-Minute Walk Test [Time Frame: baseline, up to approximately 4 years]

Change from baseline Biomarker -CK [Time Frame: baseline, up to approximately 4 years]

Change from baseline in muscle strength measured by Manual Muscle Strength testing [Time Frame: baseline, up to approximately 4 years]

Change from baseline Biomarker -uHex4 [Time Frame: baseline, up to approximately 4 years]

Change from baseline in muscle strength measured by Quantitative Muscle Strength testing [Time Frame: baseline, up to approximately 4 years]

Physician's Global Impression of Change [Time Frame: baseline, up to approximately 4 years]

Change from baseline in scores of PROMIS - dyspnea questionnaire [Time Frame: baseline, up to approximately 4 years]

Pulmonary Function - Forced vital capacity (FVC) [Time Frame: baseline, up to approximately 4 years]

Change from baseline in scores of PROMIS - physical function questionnaire [Time Frame: baseline, up to approximately 4 years]

Subject's Global Impression of Change [Time Frame: baseline, up to approximately 4 years]

Immunogenicity: Incidence of neutralizing [Time Frame: baseline, up to approximately 4 years]

Secondary ID(s)

ATB200-07

Source(s) of Monetary Support

Please refer to primary and secondary sponsors