Purpose of this Study
The person in the study will take part for about 5 years and 2 months. During this time, doctors will check their health in many ways. These checks include a physical exam, blood tests, breathing tests, heart tests, muscle scans, and other special tests.
The study has four parts:
1. First, there is a screening visit to make sure the person can join.
2. Next, the person will get the study medicine.
3. Then, there is an active observation period for 1 year. During this time, there will be about 19 visits at home and at the clinic.
4. Finally, there is a long-term follow-up for 4 years. This part has 8 visits.
Who Can Participate?
Eligibility
To join the study, a person must meet these rules:
1. The person must be 18 years old or older when they sign the consent form.
2. The person must have a problem with the GAA enzyme or have changes in both copies of the GAA gene.
3. The person must have had enzyme replacement treatment for at least 6 months and at least 10 treatments before signing the consent form.
4. The person’s breathing test (FVC) must be between 30% and 80% of what is expected.
5. The person must be able to walk at least 100 meters in a walking test. They can use a cane or walker if needed.
Age Range
18-100
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
Description
The goal of this study is to find out if a medicine called AB-1009 is safe and works well for people with Pompe disease. AB-1009 is a new treatment that has not been approved by the FDA yet. It is a type of gene therapy. This means it gives the body a healthy copy of a gene called GAA. People with Pompe disease have a changed GAA gene, and that change causes the disease.
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
Yes
Spanish Materials Available
No
Study Details
Full Title
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)
Principal Investigator
Natalie
Katz
Protocol Number
PRO00118845
NCT ID
NCT07282847
Phase
I/II
Enrollment Status
Pending Open to Enrollment