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

Status Active, not recruiting

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

Clinical Trial 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

Related Conditions & MeSH terms

Amino Acid Metabolism, Inborn Errors
Methylmalonic Acidemia

Clinical Trial Details

NCT number	NCT05506254
Study type	Observational
Source	Alexion Pharmaceuticals, Inc.
Contact
Status	Active, not recruiting
Phase
Start date	July 20, 2022
Completion date	December 31, 2037

View Details

See also
Status	Clinical Trial	Phase
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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
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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Withdrawn	`NCT01341379` - Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate	Phase 2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