Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
Public ClinicalTrials.gov record NCT04706013. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Study of Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT04706013
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 15 participants
Conditions and interventions
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Feb 16, 2024 (Actual)
- Primary completion
- Nov 2026 (Estimated)
- Completion
- Nov 2026 (Estimated)
- Last update posted
- Oct 16, 2025
2024 – 2026
United States locations
- U.S. sites
- 6
- U.S. states
- 6
- U.S. cities
- 6
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital of Alabama | Birmingham | Alabama | 35233 | Recruiting |
| Lucile Packard Children's Hospital | Palo Alto | California | 94304 | Recruiting |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | Recruiting |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | Recruiting |
| Duke Children's Hospital | Durham | North Carolina | 27705 | Active, not recruiting |
| Akron's Children's Hospital | Akron | Ohio | 44308 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04706013, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 16, 2025 · Synced Oct 25, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04706013 live on ClinicalTrials.gov.