RecruitingACTRN12622001186741

Autologous Stem Cell Transplant in Neuro-Inflammatory diseases other than multiple sclerosis

A phase II interventional study: Safety and tolerability of Haematopoietic Stem Cell Transplantation for neuro-inflammatory disease


Sponsor

The Alfred- Melbourne

Enrollment

5 participants

Start Date

Oct 30, 2022

Study Type

Interventional

Conditions

Summary

Autologous haematopoietic stem cell transplantation (AHSCT) is a procedure that is typically used to give high doses of chemotherapy to patients with blood cancers, followed by stem cell infusion, so that a new blood and immune system can regrow. Stem cells are collected from the blood following stimulation with intravenous cyclophosphamide chemotherapy and granulocyte colony stimulating factor (GCSF) injections. Patients are then admitted into hospital to receive high doses of chemotherapy that intensely suppresses their immune system. Subsequently they have their stem cells transfused to re-grow a new immune system and provide protection from the toxic effects of the chemotherapy. It takes about 14 days for the new stem cells to grow and all patients are monitored carefully over the subsequent months and years to see if the immunosuppression controls their auto-immune disease. Over the last 15 years AHSCT become much safer, and evidence has accumulated suggesting a proportion of patients with severe autoimmune conditions will respond to AHSCT. Often these patients had prolonged remissions of their AID. In well selected patients there is a possibility to establish a prolonged period of disease remission.


Eligibility

Sex: Both males and femalesMin Age: 18 YearssMax Age: 70 Yearss

Plain Language Summary

Simplified for easier understanding

Some severe autoimmune neurological diseases — conditions where the immune system mistakenly attacks the nervous system — do not respond well to standard medications. For patients with these treatment-resistant conditions, a procedure called autologous haematopoietic stem cell transplantation (AHSCT) may offer a chance at long-term remission. The procedure involves collecting your own stem cells, then using high-dose chemotherapy to essentially "reset" the immune system, before reinfusing your own stem cells to rebuild it from scratch. This study is offering AHSCT to patients with severe neuro-inflammatory conditions (excluding multiple sclerosis, which has its own trials). The goal is to track whether this intense treatment leads to lasting improvement in neurological symptoms, and to carefully monitor safety. Over the past 15 years, AHSCT has become significantly safer, and selected patients with autoimmune diseases often experience long periods of remission. You may be eligible if you are aged 18 to 65 (or up to 70 in some cases if you are in good health), have a severe autoimmune neurological condition that has not responded to conventional treatment, have adequate heart, lung, kidney, and liver function, and are negative for HIV and active hepatitis. The study is conducted at The Alfred Hospital in Melbourne and requires commitment to the full transplant process and long-term follow-up.

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 via subcutaneous injection. 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.


Locations(1)

The Alfred - Melbourne

VIC, Australia

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ACTRN12622001186741