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Clinical trial

Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease

NCT04093349 is registered in ClinicalTrials.gov with Spark Therapeutics, Inc. as lead sponsor.

NCT NCT04093349
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Registry record

As filed in ClinicalTrials.gov.

Title
Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease
Lead sponsor
Spark Therapeutics, Inc.
Phase
Phase 1/2
Overall status
Active, not recruiting
Start date
October 1, 2020
Completion date
April 30, 2032
Enrollment
4 (actual)
Sites
29

Conditions

As the registry lists them; a condition with its own page is linked.

acid maltase deficiency, glycogen storage disease type 2, glycogen storage disease type ii, lopd, lysosomal storage diseases, pompe disease, pompe disease (late-onset)