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

• Sponsor: LogicBio Therapeutics, Inc. (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT05506254
• Collaborator: (none)
• Enrollment: 8
• Locations: 2
• Anticipated Duration (Months): 184.4
• Patients Per Site: 4
• Patients Per Site Per Month: 0

Study Details

Study Description

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

Condition or Disease	Intervention/Treatment	Phase
Methylmalonic Acidemia	Drug: 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.

Study Design

• Study Type: Observational
• Anticipated Enrollment: 8 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: A Long-term Follow-up Study of Patients in LogicBio Clinical Trials for Methylmalonic Acidemia Characterized by MMUT Mutations Who Received hLB-001
• Actual Study Start Date: Jul 20, 2022
• Anticipated Primary Completion Date: Dec 1, 2037
• Anticipated Study Completion Date: Dec 1, 2037

Arms and Interventions

Arm	Intervention/Treatment
Patients who have previously received hLB-001	Drug: hLB-001; hLB-001 gene therapy

Outcome Measures

Primary Outcome Measures

1. Incidence of treatment emergent adverse events [15 years]

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Patient has previously participated in a LogicBio clinical trial and received hLB-001

Exclusion Criteria:

• None

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Vanderbilt Children's Hospital	Nashville	Tennessee	United States	37232
2	Seattle Children's Hospital	Seattle	Washington	United States	98105

Sponsors and Collaborators

• LogicBio Therapeutics, Inc.

Investigators

• Study Director: Daniel Gruskin, MD, LogicBio Therapeutics

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: LogicBio Therapeutics, Inc.
• ClinicalTrials.gov Identifier: NCT05506254
• Other Study ID Numbers: LB-001LT
• First Posted: Aug 18, 2022
• Last Update Posted: Aug 18, 2022
• Last Verified: Aug 1, 2022

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Amino Acid Metabolism, Inborn Errors

Study Results

No Results Posted as of Aug 18, 2022