This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult participants with late-onset Pompe disease (LOPD).
This is an open-label study, up to 12 participants will receive a single IV infusion of AB-1009. Participants will be assigned to either cohort 1 (1.0E13 vg/kg) or Cohort 2 (1.5E13 vg/kg) based on enrollment in the study. Study duration will include a screening period of up to 75 days, primary observation of 52 weeks, and a long-term follow-up period of 4 years.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
12
A single intravenous infusion of AB-1009
Barrow Neurological Institute
Phoenix, Arizona, United States
NOT_YET_RECRUITINGUniversity of California, Irvine (UCI)
Irvine, California, United States
RECRUITINGIncidence of treatment-emergent adverse events (TEAEs)
Time frame: From Day 1 (Dosing) to Week 52 (the end of the primary observation period)
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Stanford Neuroscience Health Center
Palo Alto, California, United States
NOT_YET_RECRUITINGNYU Langone
New York, New York, United States
NOT_YET_RECRUITINGDuke University
Durham, North Carolina, United States
NOT_YET_RECRUITINGOregon Health and Science University (OHSU)
Portland, Oregon, United States
NOT_YET_RECRUITINGUniversity of Pennsylvania
Philadelphia, Pennsylvania, United States
NOT_YET_RECRUITINGUniversity of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, United States
NOT_YET_RECRUITINGUniversity of Texas Southwest Medical Center
Dallas, Texas, United States
NOT_YET_RECRUITINGVirginia Commonwealth University (VCU)
Richmond, Virginia, United States
NOT_YET_RECRUITING