Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease

Summary

This is a multicenter, randomized, open-label study with an active standard-of-care comparator (penicillamine)

Timeline

Start

2018-09-03

Primary completion

2020-08-19

Completion

2022-01-18

Outcome

Met primary endpoint

paper with the lower limit of the 95% CI within the defined non-inferiority margin. PMID 36183738 ↗

release “The CHELATE trial met its primary efficacy endpoint by demonstrating that trientine tetrahydrochloride was non-inferior to d-Penicillamine” orphalan.com ↗

release “CHELATE trial confirmed trientine tetrahydrochloride was non-inferior to d-Penicillamine and met the primary endpoint of Non-Ceruloplasmin Copper (NCC) levels” orphalan.com ↗

Publications

• Results Schilsky ML, Czlonkowska A, Zuin M, Cassiman D, Twardowschy C, Poujois A, Gondim FAA, Denk G, Cury RG, Ott P, Moore J, Ala A, D'Inca R, Couchonnal-Bedoya E, D'Hollander K, Dubois N, Kamlin COF, Weiss KH; CHELATE trial investigators. Trientine tetrahydrochloride versus penicillamine for maintenance therapy in Wilson disease (CHELATE): a randomised, open-label, non-inferiority, phase 3 trial. Lancet Gastroenterol Hepatol. 2022 Dec;7(12):1092-1102. doi: 10.1016/S2468-1253(22)00270-9. Epub 2022 Sep 30.
• Ott P, Sandahl T, Ala A, Cassiman D, Couchonnal-Bedoya E, Cury RG, Czlonkowska A, Denk G, D'Inca R, de Assis Aquino Gondim F, Moore J, Poujois A, Twardowschy CA, Weiss KH, Zuin M, Kamlin COF, Schilsky ML. Evaluation of novel assays of non-ceruloplasmin copper to monitor chelation treatment in patients with Wilson disease. JHEP Rep. 2026 Jun;8(6):101822. doi: 10.1016/j.jhepr.2026.101822. Epub 2026 Mar 12.
• Ott P, Sandahl T, Ala A, Cassiman D, Couchonnal-Bedoya E, Cury RG, Czlonkowska A, Denk G, D'Inca R, de Assis Aquino Gondim F, Moore J, Poujois A, Twardowschy CA, Weiss KH, Zuin M, Kamlin COF, Schilsky ML. Non-ceruloplasmin copper and urinary copper in clinically stable Wilson disease: Alignment with recommended targets. JHEP Rep. 2024 May 6;6(8):101115. doi: 10.1016/j.jhepr.2024.101115. eCollection 2024 Aug.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	TETA 4HCL	Unknown	—	Oral
Subject	penicillamine	Unknown	—	Oral

Indications