Gene Therapy for Pompe Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new gene therapy called AB-1009 for individuals with late-onset Pompe disease, a condition affecting muscle strength and function. The study evaluates the safety and effectiveness of a single intravenous infusion of the treatment. Participants will be grouped into two different dose levels to assess how the body tolerates varying amounts of the medication. Eligible participants must have a Pompe disease diagnosis, have been on enzyme replacement therapy for at least six months, and be able to walk at least 100 meters (about the length of a football field) with or without a walking aid. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new therapy.
Do I have to stop taking my current medications for the trial?
The trial requires participants to stay on their current enzyme replacement treatment (ERT) until close to dosing. Other medications may need to be stopped if they are investigational drugs or could affect the study, as evaluated by the investigator.
Is there any evidence suggesting that AB-1009 is likely to be safe for humans?
Research shows that AB-1009 is undergoing testing to determine its safety for treating late-onset Pompe disease, a rare condition affecting muscles and nerves. Earlier studies on similar treatments have demonstrated that gene therapy can be safe for patients. However, some individuals might experience side effects, such as mild reactions like fever or headache after treatment. Serious side effects are less common but can occur. As this trial is in its early stages, the primary goal is to ensure the treatment's safety for participants. This step is crucial in understanding the treatment's effectiveness and the body's response.12345
Why do researchers think this study treatment might be promising?
Researchers are excited about AB-1009 for Pompe disease because it uses a novel gene therapy approach to tackle the root cause of the condition, unlike traditional enzyme replacement therapies. Most current treatments for Pompe disease focus on supplementing the enzyme that patients lack, but AB-1009 aims to deliver a functional gene directly to patients' cells, potentially offering a more lasting solution. This gene therapy could lead to more efficient production of the enzyme within the body, reducing the need for frequent treatments and potentially improving overall quality of life for patients.
What evidence suggests that AB-1009 might be an effective treatment for Pompe disease?
Research has shown that AB-1009, a gene therapy, could be promising for treating late-onset Pompe disease. The trial will evaluate AB-1009 in two different dosage cohorts. This treatment provides a working copy of a gene that helps break down glycogen, a type of sugar that accumulates in the muscles of people with Pompe disease. Early results suggest that AB-1009 might improve muscle function by addressing the root cause of the disease. Although human studies have provided limited information, this approach aims to offer longer-lasting benefits than current treatments. More research is needed to confirm these early findings, but the potential for better outcomes is encouraging.16789
Are You a Good Fit for This Trial?
Adults with late-onset Pompe disease, a genetic enzyme disorder affecting muscles and nerves, can join this trial. Participants must have a confirmed diagnosis and meet specific health criteria not detailed here.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single intravenous infusion of AB-1009
Primary Observation
Participants are observed for safety, tolerability, and efficacy of the treatment
Long-term Follow-up
Participants are monitored for long-term safety and efficacy
What Are the Treatments Tested in This Trial?
Interventions
- AB-1009
Trial Overview
The study is testing AB-1009 gene therapy given through an IV. It's an open-label trial where everyone gets the treatment, and doses increase over time to find the safest effective amount.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
1.5E13 vg/kg
1.0E13 vg/kg
Find a Clinic Near You
Who Is Running the Clinical Trial?
AskBio Inc
Lead Sponsor
Bayer
Industry Sponsor
Bill Anderson
Bayer
Chief Executive Officer since 2023
BSc in Chemical Engineering from the University of Texas, MSc in Chemical Engineering and Management from MIT
Michael Devoy
Bayer
Chief Medical Officer since 2014
MD, PhD
Citations
A Study to Evaluate Safety, Tolerability, and Efficacy of AB ...
This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB ...
STIG study: real-world data of long-term outcomes of adults ...
STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa.
Bayer Backs AskBio Gene Therapy Push in Pompe Disease
The treatment being tested is AB-1009, a gene therapy given as a single intravenous infusion. It is designed to deliver a working copy of the ...
Real-world outcomes from a series of patients with late ...
Pompe disease, also known as glycogen storage disease type II (OMIM #232300), is an inherited, progressive neuromuscular disorder caused by ...
(PDF) Real‐life effectiveness 1 year after switching to ...
Discussion At the group level, gait parameters improved slightly with a stabilization of previous worsening, but respiratory parameters showed limited changes.
Glycogen Storage Disease Type II - StatPearls - NCBI Bookshelf
Glycogen storage disease type II, or Pompe disease, is a rare inherited disorder caused by a deficiency of the enzyme acid α-glucosidase (GAA).
Pompe Disease (Glycogen Storage Disease Type 2)
The study was undertaken to evaluate the safety and efficacy of aglucosidase alfa in juvenile and adult patients with Pompe disease. LOTS ...
Type II Glycogen Storage Disease (Pompe Disease)
Pompe disease (type II glycogen storage disease) is an inherited enzyme defect that usually manifests in childhood.
Glycogen Storage Disease Type II
Glycogen storage disease type II (GSD II) (Pompe disease) is a severe, progressive, autosomal recessive metabolic disorder. It is the only ...
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