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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)

NCT07282847 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-05-06

No results posted yet for this study

Summary

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).

Conditions

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

Interventions

GENETIC

AB-1009 (GAA Gene)

A single intravenous infusion of AB-1009

Sponsors & Collaborators

  • Bayer

    collaborator INDUSTRY

  • AskBio Inc

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-04-15
Primary Completion
2028-09-30
Completion
2032-09-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07282847 on ClinicalTrials.gov