Study of Oral MC-1 for the Treatment of Patients with PNPO Deficiency

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2025-522346-46-00

Acronym

16002

Enrollment

Registered

2025-11-21

Start date

Unknown

Completion date

Unknown

Last updated

2025-11-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Seizures associated with Pyridox(am)ine 5’-Phosphate Oxidase (PNPO) deficiency

Brief summary

Incidence of death at 12 months. The incidence of death at 12 months will be compared between the study group and an external control group (without any P5P treatment).

Detailed description

Frequency of seizures (including but not limited to status epilepticus)

Interventions

None listed

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Incidence of death at 12 months. The incidence of death at 12 months will be compared between the study group and an external control group (without any P5P treatment).	—

Secondary

Measure	Time frame
Frequency of seizures (including but not limited to status epilepticus)	—

Countries

Poland

Outcome results