Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia
Study Identifier:
LB001-001
ClinicalTrials.gov Identifier:
EudraCT Identifier:
N/A
Sponsor:
Alexion Pharmaceuticals
Study Contact Information:
N/A
Recruitment Complete
Study Details
Medical Condition
- Unmapped
Study Drug
- Biological: hLB-001
Date
May 2021 - Dec 2023
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 6 Months - 12 Years
Requirements Information
Sex
Female & Male
Age
6 Months - 12 Years
Study Details
Medical Condition
- Unmapped
Study Drug
- Biological: hLB-001
Date
May 2021 - Dec 2023
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 6 Months - 12 Years years
Requirements Information
Protocol 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.
Trial Locations
Location
Status
Location
Clinical Trial Site
Atlanta, Georgia, United States, 30322
Status
N/A
Location
Clinical Trial Site
Pittsburgh, Pennsylvania, United States, 15224
Status
N/A
Location
Clinical Trial Site
Nashville, Tennessee, United States, 37232
Status
N/A
Location
Clinical Trial Site
Seattle, Washington, United States, 98105
Status
N/A