🏛️ Verified NIH Record • Updated 2026-02-03
Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy (NCT05506254)
A Long-term Follow-up Study of Patients in LogicBio Clinical Trials for Methylmalonic Acidemia Characterized by MMUT Mutations Who Received hLB-001
🔵 Active, Not Recruiting Interventional Protocol 📊 Observational Clinical Research Sponsor: Alexion Pharmaceuticals, Inc.
🩺 Plain-English Summary (8th-Grade Level)
This is a non-interventional long-term follow-up study of patients who have participated in LogicBio studies for the treatment of MMA and received hLB-001
Who can join: Review the eligibility criteria below.
Location: Clinical Trial Site — Atlanta, Georgia
Age range: 18 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 (4 Sites)
- Clinical Trial Site — Atlanta, Georgia
- Clinical Trial Site — Pittsburgh, Pennsylvania
- Clinical Trial Site — Nashville, Tennessee
- Clinical Trial Site — Seattle, Washington
💰 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.
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💡 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.