AAV Gene Therapy Study for Subjects with PKU
Public ClinicalTrials.gov record NCT04480567. 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
A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.
Study identification
- NCT ID
- NCT04480567
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 100 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 15 Years and older
- 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
- Sep 23, 2020
- Primary completion
- Nov 30, 2027
- Completion
- Nov 30, 2027
- Last update posted
- Dec 11, 2024
2020 – 2027
United States locations
- U.S. sites
- 2
- U.S. states
- 2
- U.S. cities
- 2
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of South Florida | Tampa | Florida | 33606 | — |
| Morristown Medical Center | Morristown | New Jersey | 07960 | — |
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.
Recently updated Phenylketonuria (PKU) trials
Other public records listing Phenylketonuria (PKU). Sorted by last update posted; newest first. Not a recommendation.
- NCT05270837: Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria Active, not recruiting
- NCT07694440: A Study of MZE782 in Adults With PKU Recruiting
- NCT06628128: A Long-Term Study of JNT-517 in Participants With Phenylketonuria Recruiting
- NCT05813678: A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) Recruiting
- NCT06305234: A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) Recruiting
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04480567, 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 Dec 11, 2024 · Synced Jul 30, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04480567 live on ClinicalTrials.gov.