Optimizing INITIation of Non-invasive Ventilation in ALS Patients
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 250
- 试验地点
- 1
- 主要终点
- Health related Quality of Life in patients with ALS
研究概览
简要总结
Objective: The primary objective in this study is to identify which (pheno)type of ALS patient has the most benefit from NIV in improving quality of life.
Study population: Adult patients with ALS, PLS (Primary Lateral Sclerosis) of PSMA (Progressive Spinal Muscular Atrophy) in the Netherlands. Patients will be included during their first visit to one of the HMV centres in the Netherlands.
Main study parameters/endpoints: The main study parameter is change in Quality of Life (QoL) defined as change in ALS Assessment Questionnaire (ALSAQ-40) in patients with ALS after initiation of NIV.
Design: Multi-centre prospective cohort study consisting of 2 non-randomized cohorts, i.e. ALS patients who start NIV and ALS patients who do not start NIV at the time of inclusion in the present study.
Duration: The total duration of the study: 3,5 years. Inclusion period: 30 months. Follow up time after initiation of NIV: 9 months. Thereafter, 6 months will be used for data analysis.
Setting: Involvement of all (4) HMV centres in the Netherlands (Groningen, Maastricht, Rotterdam, Utrecht).
Procedure: 250 ALS patients will be included. During the first regular visit to the HMV centre patients will be asked to participate in the study. Data will be recorded during regular visits to the HMV centre.
详细描述
First visit:
After informed consent the following data which are gathered as parts of usual care will be recorded in the case report form (CRF):
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Pulmonary function:
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FVC ( % predicted) upright and supine
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capillary blood gas analysis, room air (pH, pCO2, HCO3-, pO2)
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anamnestic questions (Borg scale 0-4)
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Gender
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Age
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Percutaneous Endoscopic Gastrostomy (PEG)/ Percutaneous Radiological Gastrostomy (PRG) tube: yes/no
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Medication
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Weight/ BMI
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Living situation
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Civil status
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The following clinical characteristics from their medical record (from neurologist or rehabilitation specialist):
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Date of diagnosis
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Type of ALS at onset
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ALSFRS-R at the time of diagnosis
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Pulmonary function at the time of diagnosis
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Cognitive status (ECAS, ALS-FTD-Q)
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Medical history
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Validated questionnaires (online):
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
- •Age > 18 years
- •Diagnosis of ALS, PLS or PSMA
- •Ability to give informed consent.
- •Ability to fill in the questionnaires independently or with assistance of a caregiver.
排除标准
- •Use of NIV or invasive ventilation (tracheostomy) at time of first visit to a HMV centre.
- •Pregnancy
结局指标
主要结局
Health related Quality of Life in patients with ALS
时间窗: 15 months
Assessed by Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) Min score: 0 Max score: 160 Higher scores mean a worse outcome
次要结局
- Weight(15 months)
- Overall function(15 months)
- Survival(15 months)
- Bulbar function(15 months)
- Forced vital capacity(15 months)
- Anamnestic questions(15 months)
- General health Quality of Life in patients with severe respiratory insufficiency(15 months)
- Carbondioxide(15 months)
- General health Quality of Life(15 months)
研究者
Peter Wijkstra
Prof. Dr. P.J. Wijkstra
University Medical Center Groningen