Clinical trials: Organic Acidemia

Methylmalonic Acidemia

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

• Terminated
• Age: 6 months to 12 years
• Phase 1/2
• Results posted

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…

Trial name

SUNRISE (NCT04581785)

Testing

hLB-001

Where

4 sites in United States

Volunteers

4 people

Sponsor

LogicBio Therapeutics, Inc