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Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia — SUNRISE  

Methylmalonic Acidemia Clinical Trial

Official title:
A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations

Clinical Trial Summary

The SUNRISE trial is a first-in-human (FIH), open-label, Phase 1/2 clinical trial designed to assess the safety, tolerability and preliminary efficacy of a single intravenous infusion of hLB-001 in pediatric patients with MMA characterized by methylmalonyl-CoA mutase gene (MMUT) mutations. hLB-001 is a liver-targeted, recombinant engineered adeno-associated viral (rAAV) vector utilizing the LK03 capsid (rAAV-LK03), designed to non-disruptively integrate the human methylmalonyl-CoA mutase gene at the albumin locus. The trial is expected to enroll pediatric patients with ages ranging from 6 months to 12 years, initially starting with 3 to 12 year-old patients and then adding patients aged 6 months to 2 years.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04581785
Study type Interventional
Source Alexion Pharmaceuticals, Inc.
Contact
Status Terminated
Phase Phase 1/Phase 2
Start date May 29, 2021
Completion date January 10, 2023
View Details
See also
  Status Clinical Trial Phase
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Completed NCT02426775 - Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia Phase 3
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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
Active, not recruiting NCT05506254 - Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy
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