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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05506254
Recruitment Status : Active, not recruiting
First Posted : August 18, 2022
Last Update Posted : December 12, 2023
Sponsor:
Collaborator:
Alexion Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Alexion Pharmaceuticals, Inc. ( LogicBio Therapeutics, Inc )

Study Description
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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
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
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Study Type : Observational
Actual Enrollment : 4 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 : July 20, 2022
Estimated Primary Completion Date : December 31, 2037
Estimated Study Completion Date : December 31, 2037

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Groups and Cohorts
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Group/Cohort Intervention/treatment
Patients who have previously received hLB-001 Drug: hLB-001
hLB-001 gene therapy


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of treatment emergent adverse events [ Time Frame: 15 years ]

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
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.
Criteria

Inclusion Criteria:

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

Exclusion Criteria:

  • None
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05506254


Locations
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United States, Georgia
Clinical Trial Site
Atlanta, Georgia, United States, 30322
United States, Pennsylvania
Clinical Trial Site
Pittsburgh, Pennsylvania, United States, 15224
United States, Tennessee
Clinical Trial Site
Nashville, Tennessee, United States, 37232
United States, Washington
Clinical Trial Site
Seattle, Washington, United States, 98105
Sponsors and Collaborators
LogicBio Therapeutics, Inc
Alexion Pharmaceuticals, Inc.
More Information
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Responsible Party: LogicBio Therapeutics, Inc
ClinicalTrials.gov Identifier: NCT05506254    
Other Study ID Numbers: LB-001LT
First Posted: August 18, 2022    Key Record Dates
Last Update Posted: December 12, 2023
Last Verified: December 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases