An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease

ATB200-02

This study is an international, multi-center, study of Pompe disease patients that are currently receiving enzyme-replacement therapy (ERT). The purpose of this study is to find out if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.

2016-01-01

2024-06-01

2024-06-01

Active (not recruiting)

Early phase I

32

Key Inclusion Criteria: * Male and female subjects between 18 and 75years of age, inclusive * Diagnosis of Pompe disease Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory): * Has received ERT with alglucosidase alfa for the previous 2-6 years, inclusive * Subject is currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency of once every other week * Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT ) * Has upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value ERT-experienced subjects (non-ambulatory): * Has received ERT with alglucosidase alfa (Myozyme/Lumizyme) for ≥2 years * Is wheelchair-bound ERT-naïve subjects (ambulatory): * Must be able to walk 200-500 meters on the 6MWT * Has upright FVC must be 30% to 80% of predicted normal value * Subject has never received alglucosidase alfa Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory): * Has received ERT with alglucosidase alfa for \>7years, inclusive * Subject is currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency of once every other week * Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT ) * Has upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value Exclusion Criteria: * Subject has received treatment with prohibited medications within 30 days of Baseline Visit * Subject, if female, is pregnant or breastfeeding at screening * Subject, whether male or female, planning to conceive a child during the study * Subject has a medical or any other extenuating condition or circumstance that may, in opinion of investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with protocol requirements * Subject has a history of allergy or sensitivity to miglustat or other iminosugars * Subjects with active systemic autoimmune disease such as lupus, scleroderma, or rheumatoid arthritis. All subjects with autoimmune disease must be discussed with the Amicus Medical Monitor * Subjects with active bronchial asthma. All subjects with bronchial asthma must be discussed with the Amicus Medical Monitor

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 32, 'type': 'ESTIMATED'}}

2024-04-04