A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

NCTID	NCT04093349 (View at clinicaltrials.gov)
Description	The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts. (Show More)
Development Status	Inactive
Indication	Glycogen Storage Disease Type 2 (Pompe Disease)
Disease Ontology Term	DOID:2752
Compound Name	SPK-3006
Sponsor	Spark Therapeutics, Inc.
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	4
Results Posted	Not Available

Target Gene/Variant	GAA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV
Editor Type	none
Dose 1	Undisclosed dose escalation (unknown doses, unknown number of cohorts)
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	29
Locations	Canada,Netherlands,United States,Denmark,Italy,United Kingdom,France,Germany

Has US IND	True
FDA Designations
Recent Updates	Roche acquired Spark in 2019, announced they were discontinuing this program mid 2024, only enrolled 4 patients