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

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

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Methylmalonic Acidemia

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Clinical Trial Site, Atlanta, Georgia, United States

About this study

Patients will be followed for a period of 15 years which includes the duration of time that the patient was in the treatment study.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Participant has previously participated in a LogicBio clinical trial and received hLB-001.

Exclusion criteria

None

Treatment and study plan

hLB-001

Drug

hLB-001 gene therapy

Primary outcomes

Incidence of treatment emergent adverse events

Time frame: 15 years

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Registry information

Official study title

A Long-term Follow-up Study of Patients in LogicBio Clinical Trials for Methylmalonic Acidemia Characterized by MMUT Mutations Who Received hLB-001

Important dates

Study start: 2022
Primary completion: 2037
Study completion: 2037
First posted: Aug 18, 2022
Registry last updated: Feb 5, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Recruiting