Observational Study in Multiple Sclerosis Patients Treated With Autologous Hematopoietic Stem Cell Transplantation
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 50
- 主要终点
- Time to NEDA-3 failure
研究概览
简要总结
Observational prospective , multi-center study
Primary objective : To gain further homogenous evidence for clinical efficacy of aHSCT in patients undergoing aHSCT for MS as primary indication.
Secondary objectives:
- Safety, tolerability and toxicity of aHSCT in MS
- Quality of life and long-term disability after aHSCT
- MRI outcome after aHSCT
Primary endpoint : Time to failure to maintain a NEDA status
Secondary endpoints:
- Overall survival
- Transplant related mortality
- MRI Assessment including lesions
- Treatment-related complications
. • Quality of life through the MS QL 54 standard assessment
- Improvement of disability
Inclusion criteria:
- Diagnosis of MS according to the 2010 revision McDonald's criteria
- Availability of a detailed clinical history about MS, including progression of disability and relapse rate in the previous 2 years, previous treatments administered
- Patients aged 18yrs or over at the time of the first aHSCT
Exclusion criteria:
- Lack of one of the above criteria
- Physical, mental, or social condition which could affect the patient from returning for follow-up visits
- Patients with cognitive impairments, who are unable to provide written, informed consent prior to any testing under this protocol, including screening and baseline investigations that are considered part of routine patient care.
Recruitment: 50 patients Recruitment period: 2 years starting from the inclusion of the 1st patient Follow-up duration: 2 years
详细描述
Currently, a number of new prospective comparative trials have either started or are in preparation, and the EBMT guidelines recommend treatment on a clinical trial, where available. However, some MS patients receiving aHSCT will not be eligible for these trials, which are predominantly focussed on a tightly defined group of patients with highly active RRMS. In addition, access to such trials is not universal across EBMT and therefore there is a need for structured data collection.
The investigators propose here to conduct an observational cohort study that systematically gathers experience from MS patients, for whom an adequate set of data about their clinical history and their clinical situation at baseline is available; patients will be then be followed prospectively according to their local clinical center protocol. The expectation is that local clinical protocols will be in line with the current EBMT guidelines and recommendations. Therefore, the aim will be to prospectively and non-interventionally capture a complete dataset to safety, tolerability and efficacy outcomes for aHSCT in various sub-types of MS treated in accordance with the current EBMT guidelines and recommendations. Alongside ongoing clinical trials, the investigators hope this study will meaningfully inform the evidence-base and future guidelines and recommendations for good clinical practice.
1- Study design Multi-centre, non-interventional prospective study in patients diagnosed with Multiple Sclerosis, treated with aHSCT.
2 Aims and objectives of the study
- Primary objectives
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 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of MS according to McDonald's criteria
- •Availability of a detailed clinical history about MS, including progression of disability and relapse rate in the previous 2 years, treatments administered before aHSCT, and categorization of the sub-types of MS according to current EBMT guidelines
- •Highly active relapsing remitting MS failing DMTs
- •Progressive MS with active inflammatory component. In accordance with the EBMT guidelines, this category includes both secondary and primary progressive MS with active inflammatory component.
- •Aggressive (malignant) MS not previously treated with a full course of DMT
- •Patients aged 18 or over at time of first aHSCT
- •Signed informed consent
排除标准
- •Lack of one of the above criteria
- •Progressive MS without active inflammatory component, as this indication is assigned 'GNR' according to EBMT guidelines, irrespective of whether it is secondary or primary progressive.
- •Unwillingness or inability to comply with the requirements of this protocol including the presence of any condition (physical, mental, or social) that is likely to affect the patient be able to return for the scheduled follow-up visits.
- •Patients with cognitive impairments, who are unable to provide written, informed consent prior to any testing under this protocol, including screening and baseline investigations that are considered part of routine patient care.
结局指标
主要结局
Time to NEDA-3 failure
时间窗: 4 years
NEDA-3 is defined as a composite endpoint including survival without relapses, disability progression and MRI activity (new or enlarging T2 lesions or Gd-enhancing lesions on MRI).
次要结局
- Overall survival(4 years)
- Transplant related mortality(4 years)