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NCT04706013 · ClinicalTrials.gov registry record · Phase 3
Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
A Phase 3 study of Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency, sponsored by Medicure.
- Recruiting
- Registry status
- Phase 3
- Development phase
- 15
- Enrollment target
- 7
- Study locations
NCT04706013: Recruiting Phase 3 study of Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency, sponsored by Medicure.
NCT04706013 is a Phase 3 study of Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency that is actively recruiting participants, run by Medicure. The registered enrollment target is 15 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (98% lower). The trial reports 7 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04706013, a Phase 3 study of Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency, is actively recruiting participants, sponsored by Medicure.
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 15 participants
- Enrollment target
- 7
- Study locations
Study Summary
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.
Primary Outcome
The study group will be compared to a historical control group (without active treatment) for overall survival by using the method of survival analysis
Conditions Studied
Interventions
- DRUG Pyridoxal Phosphate
Study Locations (7)
Alabama
- Children's Hospital of Alabama - Birmingham
California
- Lucile Packard Children's Hospital - Palo Alto
Colorado
- Children's Hospital Colorado - Aurora
Massachusetts
- Boston Children's Hospital - Boston
North Carolina
- Duke Children's Hospital - Durham
Ohio
- Akron's Children's Hospital - Akron
Queensland
- Queensland Children's Hospital - South Brisbane
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2024-02-16 |
| Est. Completion | 2026-11 |
| Phase | Phase 3 |
What NCT04706013 shows while recruiting
NCT04706013 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 15 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (98% lower).
The record links to 1 condition, with Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency appearing as the primary indexed condition, and to 1 intervention - of which Pyridoxal Phosphate is the first listed.
NCT04706013 lists 7 locations in 7 states (Alabama, California, Colorado).
Frequently Asked Questions
What is clinical trial NCT04706013 about?
NCT04706013 is a clinical study titled "Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency". 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....
What is the current status of trial NCT04706013?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 15 participants. The study started on 2024-02-16. Estimated completion is 2026-11.
What conditions does trial NCT04706013 study?
This clinical trial studies the following conditions: Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency.
What interventions are being tested in trial NCT04706013?
The interventions under investigation include: Pyridoxal Phosphate (DRUG).
Who is sponsoring clinical trial NCT04706013?
This trial is sponsored by Medicure, which has 5 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04706013 being conducted?
This trial has 7 study locations across Alabama, California, Colorado, Massachusetts, North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04706013's enrollment target sits among peer trials
15 1978th of 2000 higher than 18 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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