First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
Pompe Disease
About this trial
This is an interventional treatment trial for Pompe Disease focused on measuring Pompe, rhGAA
Eligibility Criteria
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
Sites / Locations
- Neuromuscular Research Centre
- University of California Irvine
- University of Florida
- Emory University Division of Medical Genetics
- Infusion Associates
- Great Falls Clinic, LLP
- Rutgers New Jersey Medical School
- Duke University Medical Center
- Perelman Center for Advanced Medicine
- University of Pittsburgh
- Abramson Cancer Center Chester County Hospital
- Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)
- Womens & Childrens Hospital, Adelaide
- University Children's Hospital Department of Neuropediatrics and Inborn Metabolic Disorders, St. Josefs-Hospital
- Friedrich-Baur-Institure, Dep of Neurology - University Munich
- Erasmus Medical Center
- School of Medicine, University of Auckland
- University Hospital Birmingham NHS Foundation Trust, Queen Elizabeth Medical Center
- Salford Royal NHS Foundation Trust
Arms of the Study
Arm 1
Arm 2
Experimental
Experimental
ATB200
ATB200 + AT2221
In Stage 1, safety, tolerability, and PK will be evaluated following sequential single ascending doses of intravenously infused ATB200 for 3 dosing periods.
In Stage 2, safety, tolerability, and PK will be evaluated following single- and multiple-ascending dose combinations of ATB200 co-administered with AT2221 (Miglustat) In Stage 3, long term safety and efficacy will be assessed following 24 month treatment of ATB200 co-administered with AT2221 (Miglustat)