A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia

Clinicaltrials.gov ID# 04159103

This study is an open label clinical trial to evaluate the safety, pharmacodynamics and pharmacokinetics of mRNA-3927 given as multiple doses (up to 10 doses) in patients with propionic academia (PA).  Biomarkers, quality of life, and health care utilization will be evaluated. Eligible patients must be diagnosed with PA and will be more than 8 years old, if one of the first 2 patients enrolled in the study, or more than 1 year old after the first 2 participants are enrolled.  Exclusion criteria include: prolonged QTc, pregnancy, renal disease, heart failure, history of organ transplant, substantially abnormal screening lab values.

Recruitment status: not yet active at BCH. 2 international sites and 4 sites in USA are actively recruiting PI:  Dr. Walla Al-Hertani

Contact information: 

Dr. Walla Al-Hertani  -- Walla.Al-Hertani@childrens.harvard.edu

Lora Pixley, study coordinator – Lora.Pixley@childrens.harvard.edu