Autologous Haematopoietic Stem Cell Transplant in Multiple Sclerosis

St Vincent's Hospital, Sydney0 个研究点目标入组 30 人开始时间: 2022年4月5日最近更新: 2023年12月4日

适应症

试验速览

阶段: 2 期
状态: 招募中
发起方
入组人数: 30

研究概览研究设计入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional
分配方式: Non-randomised trial
主要目的: Treatment
盲法: Open (masking not used)

入排标准

年龄范围: 18 Years 至 70 Years（—）
性别: All

入选标准

•HSCT patients with MS
•-Age 18-65
•-Age 65-70 (may be considered only if HCT-CI<3 and deemed fit both physically and cognitively by at least two investigators)
•-Adequate organ function as measured by:
•oCardiac LV Ejection Fraction > 45%
•oTotal Lung Capacity > 60%
•oDLCO/VA > 50%.
•oNegative serology for active HBV, active HCV and HIV.
•oNegative CT skeletal survey in patients with CIDP and a para-protein
•oSerological assessments of haematology, liver, kidney and thyroid function reviewed by transplant physician and specialty input sought were required.
•-No evidence of chronic infection or significant systemic illness where a treating specialist has concerns about HSCT.
•-Clearance from treating physician in the case of prior or co-existent malignancy
•-No current history of substance abuse (drug or alcohol) or other factor (eg: serious psychiatric impairment) that may interfere with patient’s ability to comply with the study procedure and follow up.
•-Negative pregnancy test.
•-Sperm collection or ova cryopreservation is to be offered prior to HSCT in those of child-bearing age.
•-Patients must agree to use a form of effective contraception (either i.e. partner) during and for 3 months after HSCT (females that are either post-menopausal for 12 months prior to randomization or surgically sterile [through hysterectomy or bilateral oophorectomy] are not required to use birth control).
•-Able to provide informed consent and the absence of mental and cognitive deficits which can interfere with the capability of providing the informed consent.
•-AHSCT deemed an appropriate high-intensity immunotherapeutic treatment in the opinion of the referring physician.
•-Diagnosis of relapsing MS made by a neurologist according to the 2017 revised McDonald’s criteria
•-EDSS score 0-6.5
•-Patients with an EDSS 6.5 – 8 may be considered eligible if an increase in EDSS of >2 points occurred in the preceding 3 months in the context of an acute, radiologically proven MS relapse. If EDSS is 6.5-8 a second independent neurologist will be required to assess the patient’s suitability for AHSCT.
•-Active MS despite the use of high efficacy disease modifying therapy* for >3 months prior to the relapse. ‘Active MS’ defined as:
•o>1 clinical relapse in the opinion of the referring neurologist
•oEvidence of radiological disease activity (T1 lesion, T2/FLAIR lesion, Gd+ lesion) and evidence that this new activity did not preclude commencement of high-efficacy DMT.
•-Patients with a history of highly active disease (as determined by clinical history/previous MRI) prior to commencement of high efficacy treatment, where the risk of continuing the treatment is determined to be significant (eg: patients who are stable on Natalizumab with a JC virus antibody positive status) may be eligible for AHSCT without evidence of current disease activity.
•oConfirmation regarding suitability for AHSCT will be required by an external MS neurologist in this case (in addition to the referring neurologist and trial neurologist)
•*High efficacy DMT currently includes: natalizumab, ocrelizumab, ofatumumab, alemtuzumab, fingolimod and cladribine. Future DMT’s of a similar class/mechanisms of action will also be considered high efficacy eg: future CD-20 monoclonal antibodies (mAbs) for MS
•Inclusion criteria for Multiple Sclerosis patients NOT receiving AHSCT who will be enrolled in the comparator arms (observational and tissue banking study)
•-Age 18-65

排除标准

•-Any patient during the screening phase whilst being considered for HSCT arm who does not meet inclusion criteria.
•-Any patient on the study treatment arm deemed not suitable for transplant by a consensus of HSCT specialists as determined at the HSCT MDT.
•-Any patient unable to understand the purpose and risks of the study or adhere to the post-transplant management including medication adherence and appointment attendance.
•-Patients with a predominately progressive form of MS (‘primary’ or ‘inactive secondary’ progressive MS).
•-Patients where MS mimics have not been adequately excluded.
•-Patients unable to undergo MRI scans.
•-Patients with advanced disease where the risks of transplant are deemed to outweigh potential benefits.

研究者

发起方

St Vincent's Hospital, Sydney

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