RecruitingPhase 2ACTRN12619000348156

Autologous Haematopoietic Stem Cell Transplantation for highly active treatment resistant multiple sclerosis.

A study to evaluate the safety of Autologous Haematopoietic Stem Cell Transplantation in patients with highly active treatment resistant multiple sclerosis.

Sponsor

Alfred Health

Enrollment

50 participants

Start Date

May 6, 2019

Study Type

Interventional

Conditions

Relapsing Remitting Multiple Sclerosis

Summary

We propose to study the benefits and risks of Autologous Haematopoetic Stem Cell Transplant (AHSCT) in people who have an aggressive form of MS not controlled by conventional treatment. Participants will have AHSCT at The Alfred hospital, and they will be closely monitored for 5 years post the transplant to ensure their safety, and also level of Multiple Sclerosis disease activity.

Eligibility

Sex: Both males and femalesMin Age: 18 YearssMax Age: 60 Yearss

Plain Language Summary

Simplified for easier understanding

Multiple sclerosis (MS) is a condition where the immune system mistakenly attacks the brain and spinal cord, leading to disability over time. For most people, disease-modifying drugs slow this process — but for some, MS remains highly active and destructive despite treatment. For these individuals, autologous haematopoietic stem cell transplantation (AHSCT) offers a different approach: the immune system is essentially 'reset' using the patient's own stem cells. This study involves harvesting stem cells from the patient's blood, then using chemotherapy to eliminate the malfunctioning immune system, and finally re-infusing the stem cells to rebuild a healthier immune system from scratch. Participants are monitored closely for five years after the procedure. You may be eligible if you are aged 18–60, have relapsing-remitting MS (not progressive MS), and your disease remains highly active — meaning significant relapses and new lesions on MRI — despite at least two years of conventional treatment. People with certain heart, lung, liver, or kidney conditions are not eligible. This is a significant procedure and would be discussed in detail with your treating team.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

Autologous Haematopoietic Stem Cell Transplantation Participants are assessed using inclusion and exclusion criteria specific for highly active, treatment resistant multiple sclerosis in order to det

Autologous Haematopoietic Stem Cell Transplantation Participants are assessed using inclusion and exclusion criteria specific for highly active, treatment resistant multiple sclerosis in order to determine whether they are eligible for an Autologous Stem cell transplant as part of the study. Participants will undergo stem cell collection following intravenous infusion 2g/m2 cyclophosphamide. Intravenous fluids 0.9% Normal Saline(one litre every 6 hours) and mesna (dose 3g/m2) over 5 hours commencing one hour prior to cyclophosphamide, will be prescribed to run concurrently as per current standard practice in the Haematology Unit for malignant conditions. The intravenous fluids will run for 24 hours. This dose of Cyclophosphamide is given as a day patient in day oncology HOC unit (unless admission is determined to be necessary). Other supportive medications eg. anti-emetics as per local guidelines From day 5 onwards daily GCSF 5mcg/kg twice daily for at least 7 days will be administered sub-cutaneously. The maximum duration for GCSF is 9 days. The duration of GCSF is determined by the level of stem cells in the blood. Haematopoietic stem cells will then be collected and cryopreserved as per standard operating procedures in the Haematology Department. Within 4-8 weeks from the collection of stem cells, the participant is hospitalized for the immune ablative and transplantation procedure. The timing of the transplantation procedure is determined by participant health and wellbeing, and availability of resources.The immune ablative regime consists of cyclophosphamide 50mg/kg (total of 200mg/kg) from day -5 to day -2 before transplantation, and rabbit antithymocyte globulin ATG (Thymoglobuline®) 0.5mg/kg intravenous infusion on day -5, 1.0mg/kg on day -4 and 1.5mg/kg pm days -3,-2 and -1. Methylprednisolone 1000mg intravenous infusion is to be infused 30minutes prior to rabbit ATG infusions. An additional 250mg of methylprednisolone should be used in the setting of ATG induced fever. Give mesna IV (40% of the cyclophosphamide dose) in 100 mL of normal saline over 30 minutes before the infusion of cyclophosphamide. Then commence mesna (120% of cyclophosphamide dose) in 1 litre of normal saline over 24 hours at the same time as cyclophosphamide, to finish 24 hours after the last dose of cyclophosphamide (on D-5, D-4, D-3 and D-2). The collection and transplantation procedures will be performed as per The Alfred hospital's Standard of Care in the Haematology Department ward under the care of Haematologists, and haematology trained nursing staff. The minimum target dose of stem cells is 2 x 10^6 cells. The cells are administered by intravenous infusion.