A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD
- Registration Number
- NCT04138277
- Lead Sponsor
- Amicus Therapeutics
- Brief 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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 119
- 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.
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description ATB200/AT2221 AT2221 Participants received ATB200 co-administered with AT2221 capsule (Miglustat) ATB200/AT2221 ATB200 Participants received ATB200 co-administered with AT2221 capsule (Miglustat)
- Primary Outcome Measures
Name Time Method Proportion of participants with Treatment Emergent Adverse Events (TEAE) baseline, up to approximately 4 years
- Secondary Outcome Measures
Name Time Method 6-Minute Walk Test baseline, up to approximately 4 years Change in 6MWD from baseline to assess the efficacy of ATB200/AT2221 co-administration
Pulmonary Function - Forced vital capacity (FVC) baseline, up to approximately 4 years Change from baseline in FVC (sitting) to assess the efficacy of ATB200/AT2221 co-administration
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 baseline, up to approximately 4 years Change in R-Pact from baseline to assess the efficacy of ATB200/AT2221 co-administration
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) questionnaires baseline, up to approximately 4 years Change from baseline in scores of EQ-5D-5L questionnaire to assess the efficacy of ATB200/AT2221 co-administration.
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 baseline, up to approximately 4 years Change from baseline in GSGC score to assess the efficacy of ATB200/AT2221 co-administration.
Physician's Global Impression of Change baseline, up to approximately 4 years Change in the Physician's Global Impression of Change (PGIC) evaluation to assess the efficacy of ATB200/AT2221 co-administration.
Subject's Global Impression of Change baseline, up to approximately 4 years Change from baseline in scores of Subject's Global Impression of Change (SGIC) questionnaire to assess the efficacy of ATB200/AT2221 co-administration.
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
Trial Locations
- Locations (60)
Alberta Children's Hospital, Heritage Medical Research Clinic
🇨🇦Calgary, Alberta, Canada
University of Arkansas for Medical Sciences
🇺🇸Little Rock, Arkansas, United States
University of California, Irvine
🇺🇸Irvine, California, United States
University of Florida Clinical Research Center
🇺🇸Gainesville, Florida, United States
Emory Clinic
🇺🇸Atlanta, Georgia, United States
IU Health Neuroscience Center
🇺🇸Indianapolis, Indiana, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
Billings Clinic
🇺🇸Billings, Montana, United States
Hackensack University Medical Center
🇺🇸Hackensack, New Jersey, United States
Northwell Health
🇺🇸Great Neck, New York, United States
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