Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Sapropterin for Multisystem Smooth Muscle Dysfunction Syndrome (MSMDS)
NCT07574034Recruiting
- Start
- Jul 2026
- Primary completion
- Jun 2028
- Modality
- Allosteric Modulators, Metabolically Activated Prodrugs
- Countries
- 1
01
Overview
Open-label, single-arm trial of sapropterin in multisystemic smooth muscle dysfunction syndrome (MSMDS). Primary endpoints: Crossing of percentile of growth at 2 years, Increase of mean diastolic blood pressure by more than 8 mmHg at 2 years and 2 more. Started Jul 2026; status: recruiting.
02
Drugs
sapropterin
03
Conditions
multisystemic smooth muscle dysfunction syndrome (MSMDS)
Beyond the preview
Go deeper on NCT07574034.
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 NCT07574034.