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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
Enrolling by invitation Phase 3 Interventional

A 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
View directory record Official record
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 – 2030
U.S. locations
13
States / cities
Phoenix, Arizona • New Haven, Connecticut • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Jul 21, 2026, 11:36 PM 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
View directory record Official record
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 Jul 21, 2026, 11:36 PM EDT
Recruiting No phase listed Observational

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

NCT02998710
View directory record Official record
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 Jul 21, 2026, 11:36 PM EDT
Terminated Phase 1Phase 2 Interventional

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

NCT05154890
View directory record Official record
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 Jul 21, 2026, 11:36 PM EDT