NCT05154890

A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency

A Phase 1/2 Multiple Ascending-Dose Study in Subjects With Homocystinuria Due to Cystathionine β-Synthase (CBS) Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of ACN00177

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Aeglea Biotherapeutics · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety and tolerability of pegtarviliase in approximately 36 subjects with homocystinuria due to CBS deficiency.

Detailed description

The purpose of this Phase 1/2 study is to evaluate the safety, pharmacokinetics and pharmacodynamics of multiple ascending doses of pegtarviliase in subjects with homocystinuria due to CBS deficiency. The study is composed of 2 parts: Part 1: a single IV (intravenous) cohort with 4 once-weekly (QW) doses of study drug and Part 2: three SC (subcutaneous) cohorts with 4 QW doses of study drug, with an optional fifth.

Conditions

• Homocystinuria Due to Cystathionine Beta-Synthase Deficiency

Interventions

Type	Name	Description
DRUG	Pegtarviliase IV	Administered IV
DRUG	Pegtarviliase SC	Administered SC

Timeline

• Start date: 2021-05-13
• Primary completion: 2023-04-21
• Completion: 2023-04-21
• First posted: 2021-12-13
• Last updated: 2023-07-27

Locations

9 sites across 3 countries: United States, Australia, United Kingdom

Regulatory

• FDA-regulated drug study