Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
High-dose oral genistein aglycone in Sanfilippo syndrome (MPS III)
2013-001479-18Phase 3Completed
- Sponsor
- Manchester University NHS Foundation Trust
- Start
- May 2014
- Modality
- Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial in Sanfilippo syndrome, mucopolysaccharidosis type III A, mucopolysaccharidosis type IIIB and 1 more comparing Genistein aglycone with Placebo (2 arms). Primary endpoint: Primary: CSF heparan sulphate at 52 weeks. Started May 2014; status: completed.
02
Drugs
genistein
03
Conditions
Sanfilippo syndromemucopolysaccharidosis type III Amucopolysaccharidosis type IIIBmucopolysaccharidosis type IIIC
Beyond the preview
Go deeper on 2013-001479-18.
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 2013-001479-18.