A Phase II observational study of Haematopoietic Stem Cell Transplantation for highly active, treatment resistant MS
Type of Study/Intervention
Form of MS
Relapsing remitting MS
This trial is testing whether an autologous (self) haematopoietic stem cell transplant (AHSCT) for highly active, treatment resistant multiple sclerosis reduces MS disease activity over 5 years.
The treatment involves: treatment, 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.
For full details of the treatment protocol please visit Australian New Zealand Clinical Trials Registry website.
General eligibility criteria must be met for all patients entering the trial and referral from a neurologist is required. 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 55 years and ability to provide informed consent.
Specific eligibility criteria for multiple sclerosis must also be met. These are as follows:
- Patient age 18–55 years
- Expanded Disability Status Scale (EDSS) score between 2.5 and 5.5
- Less than 10 years from initiation of the first therapy with disease modifying agents
- Highly active relapsing remitting MS, despite conventional treatment within the past 2 years.
This study defines ‘highly active’ relapsing remitting MS as:
- one or more severe relapses (change in EDSS of 1 or more point, or 0.5 for those with pre relapse EDSS 5.5 or more) in motor, cerebellar or brain stem deficit (or documented changes in neurological examination consistent with these magnitudes) and/or incomplete recovery from clinically significant relapses;
- one or more gadolinium-positive (Gd+) lesion of diameter 3 mm or more on MRI within the past 6 months
- accumulation of at least 0.3 T2 lesions/month on two consecutive MRIs 6–12 months apart.
A similar study is also running in Sydney.
For more background information about AHSCT for MS please visit the MS Research Australia website www.msra.org.au/AHSCT.
For full details of inclusion and exclusion criteria please visit Australian New Zealand Clinical Trials Registry website.
Anticipated start date
Prof Richard MacDonell
Austin Health, 145 Studley Road,
Heidelberg Vic 3084, Australia
Phone: 03 9496 3705
Fax: 03 9496 2153
Full details of the trial can be found on the ANZCTR clinical trials database.