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

NCT07010575 | Completed Phase 2

• 1 other identifier: ORPH-131-015
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Wilson's Disease

Outcome Measures

Primary Outcomes (2)

• 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.

  Mean Treatment Satisfaction Questionnaire for Medication (TSQM-9) score over time including change from baseline by domain

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

• 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.

  Incidence of categorical posology questions over time

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

Secondary Outcomes (3)

• Assess treatment adherence and tolerability of a new TETA 4HCl formulation.

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

• Assess treatment adherence and tolerability of a new TETA 4HCl formulation.

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

• Assess treatment adherence and tolerability of a new TETA 4HCl formulation.

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

Other Outcomes (2)

• Assess markers of copper balance with a new TETA 4HCl formulation.

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

• Qualitative data analysis following a semi-structured patient interview designed to develop a WD-specific PRO measure.

  From Day 14 to Day 28

Study Arms (1)

Once Daily Administration of new TETA 4HCl followed by return to standard of care

EXPERIMENTAL

The new formulation of TETA 4HCl will be administered once a day for 28 days. Each film-coated tablet contains 300 mg of trientine base. Once completed patients will return to their standard of care Wilson's Disease treatment and be followed for a further 28 days.

Drug: New TETA 4HCl Formulation; Drug: Standard of Care

Interventions

New TETA 4HCl Formulation DRUG

Individual patient doses will depend on the Standard of Care (SOC) therapy at study entry and guided by recommended dosing switch schedule outlined in the study protocol. The dose may subsequently be titrated based on clinical response per the investigator's judgement.

Once Daily Administration of new TETA 4HCl followed by return to standard of care

Standard of Care DRUG

Patients will be returned to their approved Wilson's Disease SOC therapy (dose and frequency) at study entry as prescribed by their treating Wilson's Disease physician.

Once Daily Administration of new TETA 4HCl followed by return to standard of care

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Orphalan: lead

Study Sites (1)

VCTC

Hartshorne, Derbyshire, DE11 7AQ, United Kingdom

Location

MeSH Terms

Conditions

Hepatolenticular Degeneration

Interventions

Standard of Care

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Study Officials

• Anna Davidsson

  VCTC

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 12, 2025
• First Posted: June 8, 2025
• Study Start: July 15, 2025
• Primary Completion: December 18, 2025
• Study Completion: December 18, 2025
• Last Updated: January 9, 2026

Record last verified: 2026-01

Locations

GB (1)