NCT04836494

A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia

A First-in-human, Randomized, Placebo-controlled, Single and Multiple Ascending Dose Escalation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BBP-671 in Healthy Subjects and In Patients With Propionic Acidemia or Methylmalonic Acidemia

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 79 (actual)
• Sponsor: CoA Therapeutics, Inc., a BridgeBio company · Industry
• Sex: All
• Age: 15 Years – 55 Years
• Healthy volunteers: Accepted

Summary

The purpose of this study is to assess the safety, tolerability, PK and PD of BBP-671 in healthy volunteers and patients with Propionic Acidemia or Methylmalonic Acidemia.

Detailed description

This is the first-in-human study with BBP-671 and is designed to provide healthy subjects single- and multiple-dose and patient multidose safety, tolerability, PK, and PD data regarding BBP-671 for future clinical studies.

Conditions

• Healthy Volunteers
• Propionic Acidemia
• Methylmalonic Acidemia
• Organic Acidemia

Interventions

Type	Name	Description
DRUG	BBP-671	BBP-671, oral suspension
DRUG	Placebo	Placebo matching BBP-671
DRUG	BBP-671	BBP-671, tablet

Timeline

• Start date: 2021-03-25
• Primary completion: 2023-11-20
• Completion: 2023-11-20
• First posted: 2021-04-08
• Last updated: 2023-12-21

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study