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 and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) during the primary observation period
Time frame: Day 1 (Dosing) through Week 52 (the end of the primary observation period)
Area under the curve (AUC) of GAA activity in serum
Time frame: Baseline through Week 52
Viral shedding (whole blood, saliva, urine)
Time frame: Day 1 through Week 24 (or until 3 consecutive data points are at or below the limit of detection)
AUC of urinary Glc4
Time frame: Baseline during the primary observation period (through Week 52)
Change from baseline in GAA activity in muscle biopsy tissue at Week 52
Time frame: Baseline and Week 52
Change from baseline in muscle biopsy glycogen content at Week 52
Time frame: Baseline and Week 52
Change from baseline in forced vital capacity (FVC) (% predicted) at Week 24 and Week 52
Time frame: Baseline, Week 24, and Week 52
Change from baseline in maximum inspiratory pressure (MIP) at Week 24 and Week 52
Time frame: Baseline, Week 24, and Week 52
Change from baseline in maximum expiratory pressure (MEP) at Week 24 and Week 52
Time frame: Baseline, Week 24, and Week 52
Change from baseline in distance walked in the 6-Minute Walk Test (6MWT) at Week 24 and Week 52
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Stanford Neuroscience Health Center
Palo Alto, California, United States
NYU Langone
New York, New York, United States
NOT_YET_RECRUITINGDuke University
Durham, North Carolina, United States
RECRUITINGHospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States
RECRUITINGUniversity of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, United States
NOT_YET_RECRUITINGUniversity of Texas Southwest Medical Center
Dallas, Texas, United States
RECRUITINGVirginia Commonwealth University (VCU)
Richmond, Virginia, United States
NOT_YET_RECRUITINGTime frame: Baseline, Week 24, and Week 52
Patient's Global Impression of Change (PGI-C) at Week 24 and Week 52
Score range: 1-7; lower scores indicate greater improvement and a better outcome.
Time frame: Week 24 and Week 52
Clinical Global Impression of Change (CGI-C) at Week 24 and Week 52
Score range: 1-7; lower scores indicate greater improvement and a better outcome
Time frame: Week 24 and Week 52
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) for physical function changes at Week 24 and Week 52
PROMIS Short Form v2.0- Physical Function 20a consists of 20 questions that are scored on a scale of 1 to 5. The total score (i.e. sum) ranges between 20 and 100, with a higher score indicating better physical functioning.
Time frame: Baseline, Week 24, and Week 52
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) for fatigue changes at Week 24 and Week 52
PROMIS Short Form v1.0- Fatigue 8a consists of eight questions, scored on a scale of 1 to 5. The total score (i.e. sum) ranges between 8 and 40, with lower scores indicating less fatigue.
Time frame: Baseline, Week 24, and Week 52