CompletedPhase 2

Patient Preference Study: Standard of Care Versus Once-daily Trientine Tetrahydrochloride

United Kingdom10 participantsStarted 2025-07-15

Plain-language summary

Decentralized study to assess patient reported treatment satisfaction comparing their current standard-of-care Wilson's Disease (WD) treatment with a new once-daily Trientine (TETA) 4HCl formulation.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Willing and able to give informed consent for participation in the study.
✓. Proficient and fluent in English language speaker, writer and reader.
✓. Patients of any gender, aged 18 years or older as of signing the Informed Consent Form (ICF).
✓. Patients on current SOC WD maintenance treatment prescribed twice daily (or more frequently) and dose has been unchanged for at least 3-months.
✓. Women of childbearing potential and sexually active males must agree to adhere to a contraceptive method.

Exclusion criteria

✕. Major systemic disease or other illness that would, in the opinion of the investigator, compromise patient safety or interfere with the collection or interpretation of the study results.
✕. Patients with severe anaemia (e.g., Haemoglobin \<10 g/dL).
✕. Female participants who are pregnant (including a positive pregnancy test at Screening and on Day-1) or breastfeeding.
✕. Any contraindications as described in the current Investigator Brochure for TETA 4HCl.
✕. Subject receiving total daily dose of chelator as SOC greater or equal to 1200mg (trientine base or d-penicillamine).
✕. In the opinion of the investigator, the patient is likely to be a non-attender or uncooperative for routine clinical visits during the study.

What they're measuring

Assess and compare patient preference, convenience and satisfaction between current standard of care treatments for Wilson's Disease and the new TETA 4HCl formulation using patient reported outcome questionnaires.

Timeframe: From the screening assessment (-28 days to Day 1) to end of study at Week 8

Trial details

NCT IDNCT07010575

SponsorOrphalan

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-12-18

View on ClinicalTrials.gov More Wilson's Disease trials