NCT04581785

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

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

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: LogicBio Therapeutics, Inc · Industry
• Sex: All
• Age: 6 Months – 12 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Methylmalonic Acidemia

Interventions

Type	Name	Description
BIOLOGICAL	hLB-001	hLB-001 via IV infusion

Timeline

• Start date: 2021-05-29
• Primary completion: 2023-01-10
• Completion: 2023-01-10
• First posted: 2020-10-09
• Last updated: 2024-02-23
• Results posted: 2024-02-23

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study