ACTUS-101 for Pompe Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
Open-label, ascending dose trial of ACTUS-101 administered intravenously.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications. However, you must have been on a stable dose of enzyme replacement therapy (ERT) for at least 52 weeks before starting the trial.
How is the treatment ACTUS-101 for Pompe Disease different from other treatments?
ACTUS-101 uses a gene therapy approach with an adeno-associated virus (AAV) to deliver a gene that helps the liver produce the enzyme needed to break down glycogen, potentially eliminating the need for regular enzyme replacement therapy (ERT). This is different from traditional treatments that require frequent ERT infusions.12345
What data supports the effectiveness of the treatment AAV2/8-LSPhGAA, ACTUS-101 for Pompe Disease?
Who Is on the Research Team?
Edward Smith, MD
Principal Investigator
Duke University
Are You a Good Fit for This Trial?
This trial is for adults over 18 with Pompe disease, confirmed by specific tests and genetic markers. Participants must be able to walk at least 100 meters and have been on enzyme replacement therapy (ERT) for two years, with a stable dose in the last year. They should have certain lung function levels but can't join if they've used other investigational drugs recently, started or stopped respiratory training near enrollment, have certain infections or liver disease, are pregnant/nursing, need invasive ventilation, or had a live vaccine recently.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a one-time intravenous infusion of ACTUS-101 at varying dose levels
Follow-up
Participants are monitored for safety and effectiveness after treatment, including assessments of GAA bioactivity, muscle status, pulmonary function, and antibody formation
What Are the Treatments Tested in This Trial?
Interventions
- AAV2/8-LSPhGAA
- ACTUS-101
Find a Clinic Near You
Who Is Running the Clinical Trial?
AskBio Inc
Lead Sponsor
Asklepios Biopharmaceutical, Inc.
Lead Sponsor