NCT03539952

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

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 77 (actual)
• Sponsor: Orphalan · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

This is a multicenter, randomized, open label study with an active standard-of-care comparator. Stable patients who are already considered to be stable on their standard-of-care penicillamine chelation therapy for at least 1 year will enroll in the study and enter a 12-week Penicillamine Baseline Period comprising of 1 month (4 weeks) run-in period followed by a 2 month (8 weeks) evaluation period. During this time all patients will continue to take their current penicillamine under study conditions. At the end of the Penicillamine Baseline Period, patients who fulfill the protocol definition of being adequately controlled and tolerating penicillamine will be randomized in a 1:1 ratio to receive either TETA 4HCl or to continue to receive penicillamine. There is then a 24-week Post-randomization Phase comprising of a 1 month (4 weeks) run-in period for both treatment arms and a 5 month (20 weeks) evaluation period. Patients who successfully complete the 24-week Post-randomization Phase of the study will have the opportunity to enter an Extension Phase. In the first version of the clinical trial protocol, the intention was to have an 18 month (72 weeks) Extension Phase. During the first 24 weeks of the Extension Phase, subjects would continue receiving their allocated TETA 4HCl or penicillamine (i.e., up to Week 60 of the study). Thereafter all patients were receiving TETA 4HCl for a further 48 weeks (i.e., from Week 60 to Week 108). Study clinic visits occur were scheduled every 6 months in the Extension Phase. With the final version of the protocol, the Extension Phase stopped at Week 60. Patients who already passed the Week 60 visit were allowed to end the study at the next planned visit. As a consequece end of treatment varied Week 60 and Week 108

Conditions

• Wilson Disease

Interventions

Type	Name	Description
DRUG	Penicillamine (D1-W12)	Penicillamine during baseline period (D1-W12)
DRUG	TETA 4HCL (W12-60)	TETA 4HCL during post randomisation and 1st extension period (W12-W60)
DRUG	Penicillamine (W12-W60)	Penicillamine during rondomisation and 1st extension period period (W12-W60)
DRUG	TETA 4HCL (60-<W108)	TETA 4HCL during 2nd extension period (W60-\<W108)

Timeline

• Start date: 2018-09-03
• Primary completion: 2020-08-19
• Completion: 2022-01-18
• First posted: 2018-05-30
• Last updated: 2025-07-02
• Results posted: 2023-04-20

Locations

15 sites across 9 countries: United States, Belgium, Brazil, Denmark, France, Germany, Italy, Poland, United Kingdom

Regulatory

• FDA-regulated drug study