NCT05560555已完成不适用
Tafamidis 61mg, Outcomes in ATTR Amyloidosis With Neurologic and Multisystemic Involvement - TRAMA
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 5
- 试验地点
- 3
- 主要终点
- Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv
研究概览
简要总结
A study of patients with hereditary transthyretin amyloidosis (ATTRv) and wild-type transthyretin amyloidosis (ATTRwt) that have been enrolled in B3461028 and B3461045 studies in Spain - exposed to tafamidis 61mg for ≥12 months with polyneuropathy (PN) have kept going to their multisystemic follow-ups (neuro/ophthalmo/gastrointestinal) ≥12 months.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Retrospective
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
- •Treatment with tafamidis 61 mg ≥ 12 months
- •Neurological follow up ≥ 12 months
- •Diagnosis of transthyretin amyloidosis with polyneuropathy (ATTR-PN) based on one of the following:
- •Amplitude reduction in, at least, 2 nerves under normal value, excluding median nerve OR 50% amplitude reduction in, at least, 2 nerves on the basal value of the patient, excluding median nerve OR 2 abnormal tests detecting thin fibers alterations (through Sudo scan, RR Interval analysis, etc..)
排除标准
- •Treatment with tafamidis 61 mg < 12 months
- •Neurological follow up < 12 months
- •Other diagnosis for polyneuropathy
研究组 & 干预措施
Retrospective cohort ATTRv and ATTRwt patients enrolled in B3461028 and B3461045 studies in Spain
干预措施: Tafamidis (Drug)
结局指标
主要结局
Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv
时间窗: Baseline and Month 12 (data collected and analyzed over 22 days)
NIS (Neuropathy Impairment Score) is a clinically important, sensitive measure of individual neurological function, assessing sensory function, reflexes, and muscle weakness. NIS score ranged from 0 to 244, with higher score indicating greater disability or impairment. The rate of change was calculated from last follow-up.
次要结局
- Change in Familial Amyloid Polyneuropathy Specific Rasch-Built Overall Disability Scale (FAP-RODs) for ATTRv(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
- Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv(Month 18, 24 and 30 months after the start of treatment (data collected and analyzed over 22 days))
- Change in NIS for ATTRv(Baseline, Month 6, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
- Change in COMPASS-31 for ATTRv(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
- Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
- Change in Norfolk Quality of Life- Diabetic Neuropathy (Norfolk QOL-DN) for ATTRv(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
- Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRv(From Baseline to Month 30 (data collected and analyzed over 22 days))
- Number of Participants With Symptoms of Autonomic Neuropathy(Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
- Number of Participants With Symptoms of Peripheral Neuropathy(At baseline (data collected and analyzed over 22 days))
- Percentage of Responders to Treatment for ATTRv(Month 12 (data collected and analyzed over 22 days))
- Ulnar/Sural Sensory Nerve Action Potential Amplitude (SNAP) for ATTRv(Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
- Change in NIS for ATTRwt(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
- Change in NIS-LL for ATTRwt(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
- Number of Participants With R-R Interval Variability for ATTRv(Month 18, 24 and 30 (data collected and analyzed over 22 days))
- Number of Participants With R-R Interval Variability for ATTRwt(Month 18, 24 and 30 (data collected and analyzed over 22 days))
- mBMI for ATTRwt(Month 18, 24 and 30 after start of treatment (data collected and analysed over 22 days))
- Ulnar/Sural SNAP Score for ATTRwt(Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
- Ulnar/Peroneal CMAP Score for ATTRwt(Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
- Number of Participants With Carpal Tunnel Syndrome(At baseline (data collected and analyzed over 22 days))
- Number of Participants With Lumbar Stenosis(At baseline (data collected and analyzed over 22 days))
- Modified Body Mass Index (mBMI) for ATTRv(Month 18, 24 and 30 after start of treatment (data collected and analysed over 22 days))
- Ulnar/Peroneal Compound Muscle Action Potential Amplitude (CMAP) for ATTRv(Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
- Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRwt(From Baseline to Month 30 (data collected and analysed over 22 days))
- Percentage of Responders to Treatment for ATTRwt(Month 12 (data collected and analyzed over 22 days))
- Change in Norfolk QOL-DN for ATTRwt(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
- Change in COMPASS-31 for ATTRwt(Baseline, Month 6, 12, 18, 24 and 36 after treatment initiation)
- Change in FAP-RODs for ATTRwt(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
- Number of Participants According to FAP Stage for ATTRwt(Month 18, 24 and 30 months after the start of treatment (data collected and analyzed over 22 days))
- Number of Participants With Gastrointestinal Disturbances(Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
- Number of Participants With Unintentional Weight Loss(Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
- Number of Participants With Urological Disturbances(Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
- Number of Participants With Ophthalmological Disturbances(Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
- Number of Participants With Central Nervous System (CNS) Disturbances(Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
研究者
研究点 (3)
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