Ireland
--:--IST
Latest
Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide
Clinical Trials in the USA / NCT05506254
Active, not recruiting Observational

Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy

NCT05506254 · tracked via the Priya Life Science USA tracker
Sponsor
Alexion Pharmaceuticals, Inc
Phase
Observational
Started
2022-07-15
Last updated
2026-02-05

Condition(s) studied

Methylmalonic Acidemia

Investigational drug(s) / intervention(s)

hLB-001

hLB-001: hLB-001 gene therapy

Study summary

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

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: * Participant has previously participated in a LogicBio clinical trial and received hLB-001. Exclusion Criteria: * None

Primary outcome measure(s)

  • Incidence of treatment emergent adverse events — 15 years

Trial sites (4)

FacilityCityRegionStatus
Clinical Trial Site Atlanta Georgia
Clinical Trial Site Pittsburgh Pennsylvania
Clinical Trial Site Nashville Tennessee
Clinical Trial Site Seattle Washington

More Alexion Pharmaceuticals, Inc. trials in the USA

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES) Active, not recruiting
A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab Recruiting
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy Recruiting
Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa Active, not recruiting
A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis. Recruiting

Other trials for the same condition

An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics Recruiting
An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705 Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05506254 on ClinicalTrials.gov ↗ ← All trials in the USA