Long-term Follow-up Study of Patients Who Received hLB-00... | NCT05506254

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

NCT05506254

Sponsor

Alexion Pharmaceuticals, Inc.

Status

Active, not recruiting

Last Update Posted

Feb 5, 2026Actual

Enrollment

4Actual

Phase

Not provided

Conditions

Methylmalonic Acidemia

Interventions

hLB-001

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Feb 2026

Overall Recruitment Status or Expanded Access Status

Active, not recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 15, 2022Actual

Primary Completion Date

Jan 1, 2037Estimated

Completion Date

Jan 1, 2037Estimated

First Submitted Date

Aug 16, 2022

First Submission Date that Met QC Criteria

Aug 17, 2022

First Posted Date

Aug 18, 2022Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 3, 2026

Last Update Posted Date

Feb 5, 2026Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

4Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Patients who have previously received hLB-001			Drug: hLB-001

Interventions

Name	Type	Description	Arm Group Labels	Other Names
hLB-001	Drug	hLB-001 gene therapy	Patients who have previously received hLB-001

Measure	Description	Time Frame
Incidence of treatment emergent adverse events		15 years

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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

Exclusion Criteria:

None

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

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.

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Clinical Trial Site		Atlanta	Georgia	30322	United States
Clinical Trial Site

IPD Sharing Statement Module

Plan to Share IPD

Undecided

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
C537358	Methylmalonic acidemia

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided