Methylmalonic Acidemia Treatment with hLB-001 Gene Therapy: The SUNRISE Trial – Phase I/II

Study of hLB-001 Gene Therapy in Pediatric Patients with Methylmalonic Acidemia Characterized by MMUT Mutations

Protocol Description

This study uses a type of gene therapy to address methylmalonic acidemia (MMA), a rare genetic defect that causes problems with digestion of fats and proteins resulting in a buildup of methylmalonic acid in tissues and blood. While certain therapies can address symptoms, there presently is no cure for MMA. Through this trial, researchers will assess effects of a single intravenous infusion of hLB-001 in pediatric patients with MMA characterized by mutation of the methylmalonyl-CoA mutase gene (MMUT).

Study Level: Phase I/II

Eligibility Criteria

This study is enrolling candidates, ages 6 months to 12 years of age, who have MMA characterized by the MMUT mutation.
Boys and Girls: 6 months to 12 years of age

Requirements

Following a 16-day screening/run-in period for testing, participants will receive the study drug via intravenous infusion at the hospital. Participants will remain in the hospital for at least 2 days for testing and observation before being discharged. They will be followed by the research team for about 12 months and will be invited to participate in a longer-term follow-up study that tracks them for up to 15 years.
Visits: Requires hospitalization for the infusion and observation
Duration: 12 months

Status: Enrolling by Invitation

Source(s) of Support

LogicBio Therapeutics

Primary Investigator

Gerard Vockley, MD, PhD

Contact Information

For more information about the study or enrollment, please contact: 
Mark Tumblin, Clinical Research Coordinator
412-692-5969