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drugset / Trial / NCT05154890

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

NCT05154890 ↗

Phase 1/2 Terminated 13 enrolled Aeglea Biotherapeutics
Non-randomizedSequentialOpen-labelTreatment

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.

Timeline

Start
2021-05-13
Primary completion
2023-04-21
Completion
2023-04-21

Outcome

Outcome not reported

Stopped (Business): “Sponsor Decision”

Drugs

EvaluationDrugModalityDoseRoute
Subject Pegtarviliase Protein / enzyme biologic — Intravenous

Indications

classic homocystinuria