A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
AskBio Inc
Study ID
NCT07282847
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • LOPD
  • Pompe Disease (Late-onset)
  • Pompe Disease Late-Onset

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • AB-1009 (GAA Gene) — GENETIC
    A single intravenous infusion of AB-1009

Study Details

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).

Key Dates

Start date
Apr 15, 2026
Status verified
Feb 2026
Primary completion
Sep 30, 2028
Completion
Sep 30, 2032

Study Design

Enrollment
12 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1
    1.0E13 vg/kg
  • Experimental: Cohort 2
    1.5E13 vg/kg

Primary Outcome Measure

Incidence of treatment-emergent adverse events (TEAEs) [ Time Frame: From Day 1 (Dosing) to Week 52 (the end of the primary observation period) ]

Central Contacts

Locations (9)

FacilityCityStateZIPSite coordinators
Barrow Neurological InstitutePhoenixArizona85013
Shafeeq Ladha, MD (PRINCIPAL_INVESTIGATOR)
University of California, Irvine (UCI)IrvineCalifornia92697
UCI Alpha Clinic
[email protected]
949-824-3990
Tahseen Mozaffar, MD (PRINCIPAL_INVESTIGATOR)
Stanford Neuroscience Health CenterPalo AltoCalifornia94304
John Day, MD, PhD (PRINCIPAL_INVESTIGATOR)
NYU LangoneNew YorkNew York10016
Nicolas Abreu, MD (PRINCIPAL_INVESTIGATOR)
Duke UniversityDurhamNorth Carolina27705
Ming Xu, RN, MSN
[email protected]
919-681-1945
Natalie Katz, MD, PhD (PRINCIPAL_INVESTIGATOR)
Hospital of the University of PennsylvaniaPhiladelphiaPennsylvania19104
Amanda Hicks
[email protected]
267-496-3772
Abigail Browngoehl
[email protected]
Paul McIntosh, MD (PRINCIPAL_INVESTIGATOR)
University of Pittsburgh Medical Center (UPMC)PittsburghPennsylvania15213
Paula Clemens, MD (PRINCIPAL_INVESTIGATOR)
University of Texas Southwest Medical CenterDallasTexas75235
Markey McNutt, MD, PhD
[email protected]
214-645-7411
Juana Luevao
[email protected]
214-645-7411
Markey McNutt, MD, PhD (PRINCIPAL_INVESTIGATOR)
Virginia Commonwealth University (VCU)RichmondVirginia23298
Susan Sparks, MD, PhD (PRINCIPAL_INVESTIGATOR)

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By research site
Barrow Neurological Institute· Phoenix, AZUniversity of California, Irvine (UCI)· Irvine, CAStanford Neuroscience Health Center· Palo Alto, CANYU Langone· New York, NYDuke University· Durham, NCHospital of the University of Pennsylvania· Philadelphia, PA

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