Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Observational antibody profiling study in adults with late-onset Pompe disease receiving enzyme replacement therapy
NL-OMON48104Active, not recruiting
- Sponsor
- Spark Therapeutics
- Start
- Mar 2020
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Open-label trial of vanglusagene ensiparvovec in Pompe disease, sponsored by Spark Therapeutics. Primary endpoint: Occurrence and titer of SPK-GAA antibodies. Started Mar 2020; status: active, not recruiting.
02
Drugs
vanglusagene ensiparvovec
03
Conditions
Pompe disease
Beyond the preview
Go deeper on NL-OMON48104.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on NL-OMON48104.