The Effect of Diflunisal on Familial Transthyretin Amyloidosis: An Open Label Extension Study of "the Diflunisal Trial" (IND 68092), and an Open Label Observational Study on Previously Untreated Patients With Familial Transthyretin Amyloidosis.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 55
- 试验地点
- 3
- 主要终点
- Changes in the Kumamoto scale
研究概览
简要总结
An ongoing trial of diflunisal has been closed for enrollment, thus, patients suitable for the study can no longer participate or receive treatment by diflunisal; and patients, who have participated in the trial can not continue their treatment. The investigators want to continue to monitor the effect of the drug on transthyretin (TTR) amyloidosis in an open label observational study.
Primary endpoint will be a composite score of the manifestations of the disease (Kumamoto scale) and secondary end points will be measurements of neurological impairment, heart involvement and nutritional status.
详细描述
Duration of treatment in this study is dependent of the results from the ongoing IND 68092-study, which are planned to be presented 2013.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •Biopsy and genetically proven systemic transthyretin amyloidosis caused by a TTR gene mutation. The amyloid shall be proven to be of transthyretin type, and the fibril composition settled.
- •Age ≥ 18 years.
- •Negative pregnancy test and contraception for sexually active women of child bearing potential.
排除标准
- •Concomitant use of non-study non-steroidal anti-inflammatory drugs (NSAIDs)
- •Heart failure with symptoms at daily activities (NYHA class ≥III)
- •Renal insufficiency (creatinine clearance < 30 ml calculated from the Cockcroft-Gault formula)
- •Active non-haemorrhoidal bleeding within the last 18 month.
- •Non-treated peptic ulcer disease.
- •Anticoagulation therapy, low dose ASA permitted.
- •Non-steroidal or aspirin allergy/hypersensitivity
- •Thrombocytopenia (< 100,000 platelets/mm3)
- •Inability or unwillingness of subject to give written informed consent
- •By the investigator regarded as unable to follow the study guidelines and scheduled controls.
结局指标
主要结局
Changes in the Kumamoto scale
时间窗: Enrollment, 12 month and annual follow-up
Composite score of the manifestations of the disease (Kumamoto Scale). Results at enrollment will be compared to results at 12 months and annual follow-ups.
次要结局
- Changes in modified body mass index (mBMI)(Enrollment, 12 month and annual follow-up)
- Changes in paraneoplastic neurological disorders (PND) scale(Enrollment, 12 month and annual follow-up)
- Changes in cardiac function(Enrollment, 1 month, 2 month, 3 month, 6 month, 9 month 12 month, 18 month and annual follow-up)
- Safety follow-up Blood Work(1 month, 3 month, 6 month, 9 month, 12 month and follow-up every 6 months)
研究者
Ole B Suhr, Professor, MD, PhD
Professor MD PhD
Umeå University