Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Personalized 3D-printed biotin formulations in children with rare metabolic diseases
2024-519378-37-00Phase 3Authorised
- Sponsor
- Idis
- Start
- Mar 2025
- Modality
- Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
- Countries
- 1
01
Overview
Phase 3, single-arm trial of biotin in 3-methylcrotonylglycinuria (3-MCG) and biotinidase deficiency (BTD), sponsored by Idis. Primary endpoint: Patient-reported acceptability and symptoms. Planned start Mar 2025; status: authorised.
02
Drugs
biotin
03
Conditions
3-methylcrotonylglycinuria (3-MCG)biotinidase deficiency (BTD)
Beyond the preview
Go deeper on 2024-519378-37-00.
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 2024-519378-37-00.