Doxycycline to Upgrade Organ Response in Light Chain (AL) Amyloidosis (DUAL) Trial: A Phase II Open Label Study of Oral Doxycycline Administered as an Adjunct to Plasma Cell Directed Therapy in Light Chain (AL) Amyloidosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 32
- 试验地点
- 1
- 主要终点
- Hematologic Response
研究概览
简要总结
In this study the investigators want to find out more about the addition of the antibiotic, doxycycline, to standard anti-amyloid therapy in people with amyloidosis. The investigators want to find out whether doxycycline improves the response to standard anti-amyloid therapy and whether it causes any problems (side effects).
详细描述
Organ response to anti-plasma cell therapy in AL amyloidosis tends to lags behind hematologic response as chemotherapy may not clear pre-formed organ amyloid. Doxycycline has been shown to have inhibitory effects on amyloid fibril formation as well as de-fibrillogenic effects and shown to be beneficial in in vitro, murine models and other preclinical studies. The investigators will prospectively evaluate the safety and efficacy of doxycycline in AL amyloidosis patients when used in conjunction with anti-plasma cell chemotherapy.
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
- •Patients with biopsy proven AL amyloidosis.
- •Patients ≥ 18 years of age are eligible.
- •Patient must provide informed consent.
- •All patients must have measurable amyloid organ involvement of a vital organ (eg. heart, liver, kidneys). Localized amyloidosis will also be eligible as long as the amyloid involvement is radiologically measurable.
- •A negative pregnancy test will be required for all women of child bearing potential. Breast feeding is not permitted.
- •Patients who have previously been taking doxycycline will be eligible as long as there is no contraindication to stay on doxycycline 100 mg twice daily (BID) for 1 year in the opinion of the treating physician.
- •Creatinine clearance of >25 ml/min.
排除标准
- •Patients with severe malabsorption syndrome precluding absorption of oral agents will be excluded.
- •Known intolerance or allergic reactions with doxycycline.
- •Previous chemotherapy for AL amyloidosis.
研究组 & 干预措施
Doxycycline
Doxycycline will be administered at dose of 100mg orally twice daily for 1 year.
干预措施: Doxycycline (Drug)
结局指标
主要结局
Hematologic Response
时间窗: 1 year
This measure will record the number of subjects for each grade of response to therapy for subjects with Systematic Disease (hematologic) only. Hematologic response will be determined as follows. Complete response (CR) - Negative serum/urine immunofixation with normal Free Light Chain (FLC) ratio Very Good Partial Response (VGPR)- Difference between involved and uninvolved FLCs (dFLC) \< 40 mg/L Partial Response (PR)- dFLC decrease \> 50% No Response (NR)- less then PR.
次要结局
- Mortality(Baseline, 3 months, 6 months, 1 year)
- Amyloid Organ Response(6 months and 1 year)
- Patient-reported Health Quality of Life(Baseline, 3, 6, 9 and 12 months)
- Patient-reported Mental Quality of Life(Baseline, 3, 6, 9 and 12 months)
研究者
Anita D'Souza
Assistant Professor
Medical College of Wisconsin