Trials / Completed
CompletedNCT03893240
Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
A Multi-Center, Low-Interventional Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
- Status
- Completed
- Phase
- N/A
- Study type
- Interventional
- Enrollment
- 61 (actual)
- Sponsor
- Spark Therapeutics, Inc. · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
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.
Conditions
- Pompe Disease
- Pompe Disease (Late-onset)
- Glycogen Storage Disease Type 2
- LOPD
- Lysosomal Storage Diseases
- Acid Maltase Deficiency
Interventions
| Type | Name | Description |
|---|---|---|
| DIAGNOSTIC_TEST | Neutralizing Antibody to SPK-3006 capsid | 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. |
Timeline
- Start date
- 2019-06-12
- Primary completion
- 2020-10-27
- Completion
- 2020-10-27
- First posted
- 2019-03-28
- Last updated
- 2023-03-24
Locations
18 sites across 6 countries: United States, France, Germany, Italy, Netherlands, United Kingdom
Source: ClinicalTrials.gov record NCT03893240. Inclusion in this directory is not an endorsement.