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

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

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT05506254

Enrollment

Registered

2022-08-18

Start date

2022-07-15

Completion date

2037-01-01

Last updated

2026-02-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Methylmalonic Acidemia

Brief 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

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.

Interventions

DRUGhLB-001

hLB-001 gene therapy

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

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

Exclusion criteria

* None

Design outcomes

Primary

Measure	Time frame
Incidence of treatment emergent adverse events	15 years

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 6, 2026