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NCT05506254 Clinical Trial

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

Methylmalonic Acidemia Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data
NCT number NCT05506254
Other study ID # LB-001LT
Secondary ID
Status Active, not recruiting
Phase
First received
Last updated
Start date July 20, 2022
Est. completion date December 31, 2037

Study information
Verified date December 2023
Source Alexion Pharmaceuticals, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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

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.

Recruitment information / eligibility
Status Active, not recruiting
Enrollment 4
Est. completion date December 31, 2037
Est. primary completion date December 31, 2037
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility Inclusion Criteria: - Participant has previously participated in a LogicBio clinical trial and received hLB-001. Exclusion Criteria: - None

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• hLB-001
hLB-001 gene therapy

Locations
Country Name City State
United States Clinical Trial Site Atlanta Georgia
United States Clinical Trial Site Nashville Tennessee
United States Clinical Trial Site Pittsburgh Pennsylvania
United States Clinical Trial Site Seattle Washington

Sponsors (2)
Lead Sponsor Collaborator
LogicBio Therapeutics, Inc Alexion Pharmaceuticals, Inc.

Country where clinical trial is conducted

United States, 

Outcome
Type Measure Description Time frame Safety issue
Primary Incidence of treatment emergent adverse events 15 years
See also
  Status Clinical Trial Phase
Enrolling by invitation NCT05842837 - A Non-Interventional Post-Authorization Study of Carbaglu for the Treatment of Hyperammonemia Due to MMA and PA
Completed NCT01599286 - Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia Phase 2
Completed NCT02426775 - Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia Phase 3
Terminated NCT04581785 - Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia Phase 1/Phase 2
Recruiting NCT05295433 - An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705 Phase 1/Phase 2
Terminated NCT04836494 - A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia Phase 1
Completed NCT03484767 - "The MaP Study": Mapping the Patient Journey in MMA and PA
Terminated NCT04732429 - Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia Phase 2
Recruiting NCT00078078 - Clinical and Laboratory Study of Methylmalonic Acidemia
Recruiting NCT05040178 - An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
Recruiting NCT04176523 - Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
Recruiting NCT01289158 - Combined Malonic and Methylmalonic Aciduria (CMAMMA): Gene Identification and Outcome Study N/A
Withdrawn NCT01341379 - Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate Phase 2
Recruiting NCT04899310 - A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia Phase 1/Phase 2
Terminated NCT05438485 - Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia