AAV Gene Therapy Study for Subjects with PKU (NCT04480567)
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
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of South Florida — Tampa, Florida
Age range: 15 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (3 Sites)
- University of South Florida — Tampa, Florida
- Morristown Medical Center — Morristown, New Jersey
- University Hospital Birmingham NHS Foundation Trust — Birmingham,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Phenylketonuria (PKU)
- Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria — Phoenix, Arizona
- A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) — Aurora, Colorado
- A Long-Term Study of JNT-517 in Participants With Phenylketonuria — Gainesville, Florida
- A Study of MZE782 in Adults With PKU — Oakland, California
- A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) — Aurora, Colorado
Explore by condition: All Phenylketonuria (PKU) clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.