Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy
February 3, 2026 updated by: Alexion Pharmaceuticals, Inc.
A Long-term Follow-up Study of Patients in LogicBio Clinical Trials for Methylmalonic Acidemia Characterized by MMUT Mutations Who Received hLB-001
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
Study Overview
Status
Active, not recruiting
Conditions
Intervention / Treatment
Detailed Description
Patients will be followed for a period of 15 years which includes the duration of time that the patient was in the treatment study.
Study Type
Observational
Enrollment (Actual)
4
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Georgia
-
Atlanta, Georgia, United States, 30322
- Clinical Trial Site
-
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Pennsylvania
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Pittsburgh, Pennsylvania, United States, 15224
- Clinical Trial Site
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Tennessee
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Nashville, Tennessee, United States, 37232
- Clinical Trial Site
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Washington
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Seattle, Washington, United States, 98105
- Clinical Trial Site
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-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Sampling Method
Non-Probability Sample
Study Population
This is a non-interventional long-term follow-up study of participants who have participated in LogicBio studies for the treatment of MMA and received hLB-001.
Description
Inclusion Criteria:
- Participant has previously participated in a LogicBio clinical trial and received hLB-001.
Exclusion Criteria:
- None
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
|---|---|
|
Patients who have previously received hLB-001
|
hLB-001 gene therapy
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
|---|---|
|
Incidence of treatment emergent adverse events
Time Frame: 15 years
|
15 years
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
July 15, 2022
Primary Completion (Estimated)
January 1, 2037
Study Completion (Estimated)
January 1, 2037
Study Registration Dates
First Submitted
August 16, 2022
First Submitted That Met QC Criteria
August 17, 2022
First Posted (Actual)
August 18, 2022
Study Record Updates
Last Update Posted (Actual)
February 5, 2026
Last Update Submitted That Met QC Criteria
February 3, 2026
Last Verified
February 1, 2026
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- LB-001LT
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
UNDECIDED
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.