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

NCTID	NCT04581785 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Inactive
Indication	Methylmalonic Acidemia
Disease Ontology Term	DOID:0060740
Compound Name	LB-001
Compound Description	ALB-2A-MMUT
Sponsor	LogicBio Therapeutics, Inc
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	4
Results Posted	View Results

Target Gene/Variant	MMUT
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell	Hepatocyte
Delivery System	Viral transduction
Vector Type	AAV-LK03
Editor Type
Dose 1	5E13 vg/kg
Dose 2	1E14 vg/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	4
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Program was terminated 4/25/23 due to low likelihood of clinical benefit