CompletedPhase 3

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

United States, Belgium, Brazil77 participantsStarted 2018-09-03

Plain-language summary

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

Age range18 Years – 75 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Patient is able to provide, and has provided, written informed consent
✓. Written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable, including: For US sites: Authorization for Use and Release of Health Research Study Information and for EU sites: Data Protection Consent
✓. Male or female, aged ≥ 18 and ≤ 75 years of age at time of consent
✓. Patient has a diagnosis of Wilson's disease, as defined by a prior or current Leipzig score of ≥ 4
✓. Patient's Wilson's disease is clinically stable, in the opinion of the investigator, and being treated with penicillamine for at least 1 year (52 weeks) prior to the screening/enrolment visit
✓. Patient is on a stable dose and regimen of penicillamine for at least 4 months (16 weeks) prior to the screening/enrolment visit (other prescribed treatments for Wilson's disease not permitted during this study)
✓. No anticipated need that patient will require additional pharmacological therapies other than study medication, including prescribed zinc therapy, for the management of copper levels during the study
✓. Patient must be willing to maintain stable diet throughout the study, and avoid foods with high copper content, including the Penicillamine Baseline Period

✕. Patient is in 'de-coppering' phase of treatment for Wilson's disease, in the opinion of the investigator
✕. Patient evidence of uncontrolled liver disease, including but not limited to:
✕. Modified Nazer score of \> 4 (result may not be available until after start of run in period since based on lab results\*)
✕. decompensated cirrhosis
✕. acute hemolytic anemia
✕. acute hepatitis
✕. hepatic malignancy
✕. evidence of acute liver failure

Serum NCC Concentration

Timeframe: Week 36

NCT IDNCT03539952

SponsorOrphalan

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-08-19

Results submitted2023-01-19

Who can participate

Age range18 Years – 75 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Patient is able to provide, and has provided, written informed consent
✓. Written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable, including: For US sites: Authorization for Use and Release of Health Research Study Information and for EU sites: Data Protection Consent
✓. Male or female, aged ≥ 18 and ≤ 75 years of age at time of consent
✓. Patient has a diagnosis of Wilson's disease, as defined by a prior or current Leipzig score of ≥ 4
✓. Patient's Wilson's disease is clinically stable, in the opinion of the investigator, and being treated with penicillamine for at least 1 year (52 weeks) prior to the screening/enrolment visit
✓. Patient is on a stable dose and regimen of penicillamine for at least 4 months (16 weeks) prior to the screening/enrolment visit (other prescribed treatments for Wilson's disease not permitted during this study)
✓. No anticipated need that patient will require additional pharmacological therapies other than study medication, including prescribed zinc therapy, for the management of copper levels during the study
✓. Patient must be willing to maintain stable diet throughout the study, and avoid foods with high copper content, including the Penicillamine Baseline Period

✕. Patient is in 'de-coppering' phase of treatment for Wilson's disease, in the opinion of the investigator
✕. Patient evidence of uncontrolled liver disease, including but not limited to:
✕. Modified Nazer score of \> 4 (result may not be available until after start of run in period since based on lab results\*)
✕. decompensated cirrhosis
✕. acute hemolytic anemia
✕. acute hepatitis
✕. hepatic malignancy
✕. evidence of acute liver failure