Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency

Conditions

• Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency

Eligibility Criteria

Sex

ALL

Age

N/A - N/A

Healthy Volunteers

Not accepted

Interventions

• Pyridoxal Phosphate — DRUG
  Oral tablets 50 mg

Study Details

The proposed clinical study is intended to evaluate oral P5P for the treatment of patients confirmed to have Pyridox(am)ine 5'-Phosphate Oxidase (PNPO) deficiency via genetic analysis. There is an unmet clinical need for pharmaceutical grade P5P, as to date none has been made commercially available. Patients will receive pharmaceutical grade P5P according to their normal oral P5P dosing regimen, as previously established by their physicians.

Key Dates

Start date

Feb 16, 2024

Status verified

Oct 2025

Primary completion

Nov 30, 2026

Completion

Nov 30, 2026

Study Design

Enrollment

15 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Single Arm Active
  Pyridoxal 5'-Phosphate

Primary Outcome Measure

Overall survival time (time to death), including incidence of death at 12 months [ Time Frame: 12 months ]

Central Contacts

• Laura Cole, Ph.D.
  204-487-7412
  [email protected]

Locations (6)

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