Methylmalonic and Propionic Acidemias Treatment with BBP-671 – Phase I

A First-in-human, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BBP-671 in Healthy Subjects and Patients with Propionic Acidemia or Methylmalonic Acidemia

Protocol Description

The goal of this study is to see how well people with methylmalonic acidemia (MMA) and propionic acidemia (PA) accept treatment with the investigational drug BBP-671. The study will evaluate the effects of BBP- 671 on certain biomarkers and will determine if BBP-671 is safe and tolerable. Through this study, researchers hope to further understand BBP-671 and these rare genetic diseases that impair the proper digestion of fats and proteins, leading to a buildup of toxic acidic substances in the blood and altered energy metabolism.

Study Level: Phase I

Eligibility Criteria

Subject to certain exclusion criteria, this study is accepting patients, ages 18 through 55 years, who have been diagnosed with methylmalonic acidemia or propionic acidemia.
Males and Females: Ages 18 through 55 years


Following screening and a run-in period that includes physicals and blood testing, participants will be randomly divided into groups that will either receive the study drug or a placebo. The study drug will be taken twice daily for a 6-week period, with clinic visits scheduled at specific intervals for physical examinations, laboratory tests including blood collection and electrocardiograms.
Visits: 12
Duration: 4 months

Status: Open to Enrollment

Source(s) of Support

CoA Therapeutics, Inc.

Primary Investigator

Gerard Vockley, MD, PhD

Contact Information

For more information about the study or enrollment, please contact:
Michele Graham, RN