The purpose of this study is to obtain information pertaining to the occurrence of antibodies to investigational SPK-3006 capsid and GAA, GAA activity and GAA antigen levels in the usual care setting of Late-Onset Pompe Disease (LOPD) participants on an enzyme replacement regimen. Additionally, a careful evaluation of laboratory and functional testing in patients with LOPD may provide information to better understand the disease features and better drive the design of a future interventional investigational gene therapy trial. An understanding of the underlying status of liver and muscle health in individuals with LOPD may also inform best surveillance during the conduct of gene therapy trials.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
SCREENING
Masking
NONE
Enrollment
61
Collected during a single study visit to establish the occurrence of neutralizing antibodies to SPK-3006 capsid in participants with LOPD on an enzyme replacement regimen.
Barrow Neurological Institute
Phoenix, Arizona, United States
University of California Irvine Health
Orange, California, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
University of Minnesota Medical School
Minneapolis, Minnesota, United States
Oregon Health & Science University
Portland, Oregon, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Hôpital Raymond-Poincaré
Garches, Hauts-de-Seine, France
Assistance Publique Hôpitaux de Marseille
Marseille, France
CHU Nice
Nice, France
Klinikum der Universität München
München, Germany
...and 8 more locations
Neutralizing Antibodies Titer to SPK-3006 capsid
The neutralizing antibodies titer to SPK-3006 is measured once prospectively at one site visit.
Time frame: 1 day
Occurrence of Neutralizing Antibodies to SPK-3006 capsid
The proportion of participants who have Neutralizing Antibodies to SPK-3006 capsid.
Time frame: 1 day
Anti-GAA binding antibodies Titer
Anti-GAA binding antibodies titer is measured once prospectively at one site visit.
Time frame: 1 day
Occurrence of Anti-GAA binding antibodies across participants
The proportion of participants who have Anti-GAA binding antibodies.
Time frame: 1 day
Neutralizing antibodies to circulating GAA Titer
Neutralizing antibodies to circulating GAA titer is measured once prospectively at one site visit and, if available, retrospectively from medical records that are within 24 months of signing the informed consent.
Time frame: 1 day
Occurrence of Neutralizing antibodies to circulating GAA
The proportion of participants who have neutralizing antibodies to circulating GAA.
Time frame: 1 day
GAA activity level
GAA activity level (percent of normal) is measured once prospectively at one site visit and, if available, retrospectively from medical records that are within 24 months of signing the informed consent.
Time frame: 1 day
GAA antigen level
GAA antigen level (percent of normal) is measured once prospectively at one site visit and, if available, retrospectively from medical records that are within 24 months of signing the informed consent.
Time frame: 1 day
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