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ClinicalTrials.gov public records Last synced May 22, 2026, 3:47 AM EDT

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Showing 1–4 of 4 matching trials from the live ClinicalTrials.gov search.

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Filters: Condition: Homocystinuria Due to CBS Deficiency Clear all

Recruiting No phase listed Observational

Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)

NCT02998710

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Conditions: Homocystinuria Due to CBS Deficiency
Interventions: Not listed
Lead sponsor: Travere Therapeutics, Inc.; Industry
Eligibility: 1 Year to 65 Years

Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2026
U.S. locations: 7
States / cities: Culver City, California • Aurora, Colorado • Washington D.C., District of Columbia + 4 more

Source: ClinicalTrials.gov public record

Updated Nov 21, 2024 · Synced May 22, 2026, 3:47 AM EDT

Terminated Phase 1Phase 2 Interventional

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

NCT05154890

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Conditions: Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
Interventions: Pegtarviliase IV, Pegtarviliase SC; Drug
Lead sponsor: Aeglea Biotherapeutics; Industry
Eligibility: 12 Years and older

Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 1
States / cities: Dallas, Texas

Source: ClinicalTrials.gov public record

Updated Jul 26, 2023 · Synced May 22, 2026, 3:47 AM EDT

Enrolling by invitation Phase 3 Interventional

A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)

NCT06431893

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Conditions: Homocystinuria
Interventions: Pegtibatinase; Drug
Lead sponsor: Travere Therapeutics, Inc.; Industry
Eligibility: 5 Years to 65 Years

Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 4
States / cities: Portland, Maine • New York, New York • Morrisville, North Carolina + 1 more

Source: ClinicalTrials.gov public record

Updated Apr 10, 2025 · Synced May 22, 2026, 3:47 AM EDT

Recruiting Phase 3 Interventional

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment

NCT06247085

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Conditions: Homocystinuria
Interventions: Pegtibatinase, Placebo; Drug · Other
Lead sponsor: Travere Therapeutics, Inc.; Industry
Eligibility: 12 Years to 65 Years

Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 13
States / cities: Phoenix, Arizona • New Haven, Connecticut • Atlanta, Georgia + 10 more

Source: ClinicalTrials.gov public record

Updated May 7, 2026 · Synced May 22, 2026, 3:47 AM EDT