Official 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)
A Study On:
Pompe Disease
Study Description
This is an open-label study, up to 12 participants will receive a single IV infusion of AB-1009. Participants will be assigned to either cohort 1 (1.0E13 vg/kg) or Cohort 2 (1.5E13 vg/kg) based on enrollment in the study.
Study duration will include a screening period of up to 75 days, primary observation of 52 weeks, and a long-term follow-up period of 4 years.
Eligibility
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Privacy Policy
University of California, Irvine - Study Information Sheet
UCI Center for Clinical Research Potential Participant Database
Lead Researcher
Eric Vilain, MD, PhD
UCI Center for Clinical Research
In the instance of parental permission, “You” refers to “Your child.”
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Future Research Use
Researchers will use your information to conduct this study. Once the study is done using your
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Data Retention - In accordance with UC Office of the President policy, information will be retained for
10 years after the end of the calendar year in which the research is completed.
If, during the course of this study, significant new information becomes available that may relate to
your willingness to continue to participate, this information will be provided to you by the research
team listed at the top of the form.
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contact the researchers listed at the top of this form
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