Brief summary

This trial is testing autologous (self) haematopoetic stem cells transplant for a variety of severe autoimmune diseases, including multiple sclerosis.

The treatment involves: treatment and collection and freezing of stem cells from the patient’s blood; chemotherapy (BEAM-ATG) to destroy the immune system; reinfusion of the patient’s own stem cells.

Full details of the treatment protocol can be found here in the Australian New Zealand Clinical Trials Registry.

Inclusion criteria

General eligibility criteria must be met for all patients entering the trial. This includes general health criteria relating to cardiovascular, lung and liver health, absence of serious infectious diseases, negative pregnancy test for women, age between 18 and 65 years, and the ability to provide informed consent. Specific eligibility criteria must also be met for each disease type. For multiple sclerosis the eligibility criteria are as follows:

1. Diagnosis of relapsing remitting MS (RRMS) made by a Neurologist

2. EDSS score 0-6.5 (this corresponds to being able to walk, needing at most bilateral assistance to walk 20m without resting)

-Patients with an EDSS of 0-2 will require a second independent physician to assess the patient’s suitability for HSCT.

-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 HSCT.

3. Disease duration of at least 15 years from diagnosis of MS

4. New MRI activity within last 12 months. Inflammatory active MS as defined by at least 1 Gd+ (>3mm) lesion (off steroids for one month) or at least 2 new T2 lesions on MRI within the last 12 months, compared to a reference scan not older than 36 months and preferably within the last 24 months from the date of eligibility review

5. Patients with RRMS who have failed at least one licensed disease modifying drug of high efficacy because of demonstrated lack of efficacy.

NB: The trial includes consideration of patients with highly active disease (determined by previous MRI prior to commencement of high efficacy treatment), where the long term immunosuppressive risk on treatment is determined to be of significance to patient’s morbidity/mortality as determined by a panel of physicians.

For full details of inclusion and exclusion criteria please visit the ANZCTR trial listing.

Exclusion criteria

A full list of exclusion criteria can be found on the ANZCTR clinical trials database.

Anticipated start date

Started

Recruitment contacts

Dr John Moore
St Vincent’s Hospital, Sydney
390 Victoria St, Darlinghurst NSW 2010, Australia
Email: svhcancer_research@stvincents.com.au
Phone: 02 9355 5656
Fax: 02 9355 5735

Further details

For further details visit the ANZCTR trial listing.

Region

NSW

Last updated

2/03/2023

Trial number

ACTRN12613000339752