Ignite Creation Date:
2024-05-06 @ 3:17 PM
Last Modification Date:
2024-10-26 @ 1:46 PM
Study NCT ID:
NCT04581785
Status:
TERMINATED
Last Update Posted:
2024-02-23
First Post:
2020-10-02
Brief Title:
Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia
Sponsor:
LogicBio Therapeutics Inc
Organization:
Alexion Pharmaceuticals Inc
Study Overview
Official Title:
A Phase 12 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations
Status:
TERMINATED
Status Verified Date:
2024-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Due to low likelihood of clinical benefit in treated participants
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
SUNRISE
Brief Summary:
The SUNRISE trial is a first-in-human FIH open-label Phase 12 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
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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None