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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.
This is a single arm study where patients on Standard of Care maintenance therapy with a prescribed approved Wilson's Disease therapy administered at least twice daily will be screened for eligibility by the clinical research site either following referral from a participant identification centre (PIC) or following advertisements. An initial screening Patient Reported Outcome (PRO) assessment including the Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) and Morisky Medication Adherence Scale-8 (MMAS-8) will also be collected.
Patients who meet all the study entry criteria will be switched to a new TETA 4HCl formulation for 28 days and will be monitored using Patient Reported Outcomes and specific posology questions held within a patient questionnaire pack and blood investigations. During this treatment phase (between Day 14 and Day 28 of dosing), each participant will be interviewed to collect qualitative data on disease and therapy. Patients will then be returned to their Standard of Care treatment and followed for a further 28 days continuing to be assessed using Patient Reported Outcomes and repeat blood investigations. The safety period will be finalised with an End of Study Assessment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Assess treatment adherence and tolerability of a new TETA 4HCl formulation. | Mean Morisky Medication Adherence Scale-8 (MMAS-8) score over time including change from baseline | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Assess markers of copper balance with a new TETA 4HCl formulation. | Mean Serum NCC values as assessed by speciation assay (NCC-Sp); | 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. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Anna Davidsson | VCTC | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| VCTC | Hartshorne | Derbyshire | DE11 7AQ | United Kingdom |
| ID | Term |
|---|---|
| D006527 | Hepatolenticular Degeneration |
| ID | Term |
|---|---|
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
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| ID | Term |
|---|---|
| D059039 | Standard of Care |
| ID | Term |
|---|---|
| D019984 | Quality Indicators, Health Care |
| D011787 | Quality of Health Care |
| D006298 | Health Services Administration |
| D017530 | Health Care Quality, Access, and Evaluation |
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|
| 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. |
|
Incidence of categorical laboratory safety data and adverse events (AE) assessments during the study. |
| 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. | Mean of continuous laboratory safety data during the study | From the screening assessment (-28 days to Day 1) to end of study at Week 8 |
Cognitive validation of a novel Wilson's disease-specific patient reported outcome measure under development using qualitative patient interview data on disease and therapy. |
| From Day 14 to Day 28 |
| D002493 |
| Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008661 | Metabolism, Inborn Errors |
| D008664 | Metal Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |