A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency
NCT05154890 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 13
Last updated 2023-07-27
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.
Conditions
- Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
Interventions
- DRUG
-
Pegtarviliase IV
Administered IV
- DRUG
-
Pegtarviliase SC
Administered SC
Sponsors & Collaborators
-
Aeglea Biotherapeutics
lead INDUSTRY
Principal Investigators
-
Cortney Caudill · Aeglea Biotherapeutics
Study Design
- Allocation
- NON_RANDOMIZED
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SEQUENTIAL
Eligibility
- Min Age
- 12 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2021-05-13
- Primary Completion
- 2023-04-21
- Completion
- 2023-04-21
- FDA Drug
- Yes
Countries
- United States
- Australia
- United Kingdom
Study Locations
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