Autoimmune Diseases

Ongoing Studies

last updated 16 January, 2007

Safety phase I/II studies:

The results of retrospective analyses of autologous HSCT for RA, SSc, MS, SLE and JIA have been published.

Phase II/III Trials: Prospective, Randomised, Controlled

ASTIS Trial - Autologous Stem cell Transplantation International Scleroderma Trial

• HSCT versus monthly pulse Cy 750 mg/m2
• The Trial is currently running. 84 patients have been enrolled. The second safety interim analysis on the first 80 patients has just been completed and the preparations are being made for the 3rd safety interim-analysis
• Study Chair: J M van Laar, D Farge, A Tyndall
• Statistician: J Sont
• Study manager: Ms Ingeborg de Jonge
• Study Administration: Department of Clinical Epidemiology, Leiden University Medical Center, The Netherlands
• Web site: www.astistrial.com
  
• Email: astistrial@lumc.nl

ASTIMS Trial - Autologous Stem cell Transplantation International Multiple Sclerosis Trial

• High dose immunoablation (BEAM) and autologous stem cell transplantation versus Mitoxantrone in severe multiple sclerosis.
• The trial has officially open in may 2004 and continues to enrol patients.
• Study Chair: GL Mancardi
• Statistician: P Bruzzi
• Study Administration: University of Genova, L. Boni
• Web site: www.astims.org
• Information: GL Mancardi: glmancardi@neurologia.unige.it
• The ASTIMS trial as well as of the updated results on the follow up of the MS patients treated with HSCT registered in the EBMT database have been presented at the ECTRIMS meeting, in Madrid , September 2006. The BEAM/ATG based regimen adopted in the ASTIMS Trial appears among the safest.
• It is recommended that the centers which do not intend to join the trial but want to transplant MS patients, follow the same transplantation protocol of ASTIMS. Information requests should be addressed either to the Principle Investigator Prof. Gianluigi Mancardi, or to Dr. Riccardo Saccardi

ASTIC Trial- Autologous Stem cell Transplantation International Crohn's syndrome.

• All patients enrolled are mobilised with Cy and then randomised to either transplantation ("early" transplantation, conditioning: Cy + ATG, followed by autologous peripheral blood stem cell infusion) or to best available treatment for one year, with the option of transplantation at the end of this ("late" transplantation, same protocol as the "early").
• The trial has been approved by the EBMT Board and the accrual is expected to begin at the end of 2006.
• It is recommended that the centres which do not intend to join the trial but want to transplant Crohn's patients, follow the same transplantation protocol of ASTICS. Information requests should be addressed to the Principal Investigator: Prof Chris Hawkey, Nottingham
• Website: www.astic.eu
• Study Administration Officer: Hazel Brook, Nottingham

ASTIL Trial - Autologous Stem cell Transplantation International lupus (SLE)

• Patients randomised to transplantation with or without following maintenance therapy (proposed mofetil mycophenolate).
• Principal Investigators:Dominique Farge-Bancel (Paris)
• Information from: Dominique Farge-Bancel (Paris)
• Still in the planning phase.

A Phase I/II study for the treatment of severe cases of Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

• CIDP pilot trial is currently ongoing.
• Principal investigators: R. Hughes and M Kadmi (London, Guy's Hospital)
• Information also from: Majid.Kazmi@gstt.sthames.nhs.uk

Retrospective Analysis

Retrospective Analysis of HSCT for Autoimmune Diseases in Paediatric Patients

• This analysis has been promoted together with the Paediatric and Inborn Errors Working Parties
  
  
• More than 100 patients have been reported to the database
  
  
• This analysis is expected to be finalised in 2007.
  
  
• Principal Investigators: Nico Wulffraat (Utrecht) and Marco Rabusin (Trieste)
  
  
• For further information contact: N.Wulffraat@umcutrecht.nl and rabusin@burlo.trieste.it