Decentralized Study to Assess Patient Treatment Preference Comparing Their Current Standard-of-care Wilson's Disease (WD) Treatment to a New Trientine (TETA) 4HCl Formulation.
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Orphalan
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- 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.
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
Once Daily Administration of new TETA 4HCl followed by return to standard of care
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.
干预措施: New TETA 4HCl Formulation (Drug)
Once Daily Administration of new TETA 4HCl followed by return to standard of care
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.
干预措施: Standard of Care (Drug)
结局指标
主要结局
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.
时间窗: From the screening assessment (-28 days to Day 1) to end of study at Week 8
Mean Treatment Satisfaction Questionnaire for Medication (TSQM-9) score over time including change from baseline by domain
次要结局
- 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)