NCT05359276已完成不适用
Acid Sphingomyelinase Deficiency (ASMD): Data Analysis of Adult and Pediatric Patients on Early Access to Olipudase Alfa in France
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
- Sanofi
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Change in pulmonary function diffusion capacity of lung for carbon monoxide (DLco)
研究概览
简要总结
Primary Objective:
To describe the lung, spleen and liver outcomes of olipudase alfa
Secondary Objectives:
- To describe the patient's characteristics
- To describe conditions of olipudase alfa use
- To describe safety data related to the use of olipudase alfa
- To describe complementary effectiveness outcomes parameters
详细描述
Approximate duration of enrollment: 30 months
Total study duration: approximately 30 months
This is a national, multicenter observational retrospective and prospective cohort data collection study. Retrospective is defined as collection of data from all patients, including deceased patients, who were already on early access olipudase alfa in France before the start of this study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The patient, or the patient's parent(s)/guardian(s), has signed written informed consent.
- •Patients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France (ie, nominative compassionate use, pre marketing authorization early access, post marketing authorization early access).
- •The patient has documented deficiency of acid sphingomyelinase in peripheral leukocytes, lymphocytes, or cultured fibroblasts.
- •Male and female patients of all ages.
排除标准
- •The patient or legal guardian(s) who has not received information notice or who opposes to data collection.
- •Patient who died before study initiation and who was opposed to data collection for research purpose when he/she was alive.
- •The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
研究组 & 干预措施
Cohort 1
Patients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France
干预措施: Olipudase alfa (Drug)
结局指标
主要结局
Change in pulmonary function diffusion capacity of lung for carbon monoxide (DLco)
时间窗: From baseline to 24 months
Change in spleen size
时间窗: From baseline to 24 months
Change in liver size
时间窗: From baseline to 24 months
次要结局
- Baseline patient characteristics(At baseline)
- Condition of olipudase alfa use(From baseline up to 3 years)
- Safety: AE(From baseline up to 3 years)
- Safety: immunogenicity(From baseline up to 3 years)
- Complementary effectiveness: change in pulmonary function DLco(From baseline to 12 months and 36 months)
- Complementary effectiveness: change in spleen size(From baseline to 12 months and 36 months)
- Complementary effectiveness: change in liver size(FFrom baseline to 12 months and 36 months)
- Change in interstitial pulmonary infiltration based on lung imaging (thoracic CT-scan)(From baseline to 12 months and 24 months)
- Change in platelet count(From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years)
- Change in biomarkers (chitotriosidase and lysosphingomyelin) plasma levels(From baseline at 3, 6, 9, 12 months and every year up to 3 years)
- Change in liver function(From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years)
- Change in lipid profile(From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years)
- Change in growth curve for pediatric patient(From baseline at 6, 12, 24 months and every year up to 3 years)
- Change in weight(From baseline to 12 months and 36 months)
- Number of Participants with Evolution of Comorbidities(From baseline to 12 months, 24 months and 36 months)
研究者
研究点 (1)
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