| Imperiale, Michael |
HARMONY, NCT06247085: 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 |
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| Active, not recruiting | 3 | 70 | US | Pegtibatinase, OT-058, TVT-058, Placebo | Travere Therapeutics, Inc. | Homocystinuria | 12/25 | 01/26 | | |
ENSEMBLE, NCT06431893: A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥12 to ≤65 Years of Age with Classical Homocystinuria (HCU) |
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| Enrolling by invitation | 3 | 90 | US | Pegtibatinase, OT-58, TVT-058 | Travere Therapeutics, Inc. | Homocystinuria | 01/27 | 01/27 | | |
NCT03406611: Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE) |
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| Active, not recruiting | 1/2 | 32 | US | Pegtibatinase, TVT-058, OT-58, PEG modified CBS, PEG htCBS C15S, htCBS C15S ME-200GS, Placebo | Travere Therapeutics, Inc. | Homocystinuria | 06/26 | 12/26 | | |
NCT02998710: Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA) |
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| Recruiting | N/A | 150 | Europe, US, RoW | | Travere Therapeutics, Inc. | Homocystinuria Due to CBS Deficiency | 04/26 | 08/26 | | |
| Vaidya, Sagar A |
ENSEMBLE, NCT06431893: A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥12 to ≤65 Years of Age with Classical Homocystinuria (HCU) |
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| Enrolling by invitation | 3 | 90 | US | Pegtibatinase, OT-58, TVT-058 | Travere Therapeutics, Inc. | Homocystinuria | 01/27 | 01/27 | | |
NCT03406611: Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE) |
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| Active, not recruiting | 1/2 | 32 | US | Pegtibatinase, TVT-058, OT-58, PEG modified CBS, PEG htCBS C15S, htCBS C15S ME-200GS, Placebo | Travere Therapeutics, Inc. | Homocystinuria | 06/26 | 12/26 | | |
NCT02998710: Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA) |
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| Recruiting | N/A | 150 | Europe, US, RoW | | Travere Therapeutics, Inc. | Homocystinuria Due to CBS Deficiency | 04/26 | 08/26 | | |
NCT03115086: The REPLACE Registry for Cholbam® (Cholic Acid) |
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| Active, not recruiting | N/A | 55 | US | Cholbam | Mirum Pharmaceuticals, Inc. | Bile Acid Synthesis Disorders | 07/38 | 07/39 | | |
| Slawka, Sergio |
HARMONY, NCT06247085: 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 |
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| Active, not recruiting | 3 | 70 | US | Pegtibatinase, OT-058, TVT-058, Placebo | Travere Therapeutics, Inc. | Homocystinuria | 12/25 | 01/26 | | |
