SCLÉROSE EN PLAQUES : OMST (EBMT)- Greffe de cellules souches hématopoïétiques autologues – Inclusions ouvertes

Greffe de cellules souches hématopoïétiques autologues NCT04674280
Protocol Title:
Observational Study in Multiple Sclerosis patients treated with autologous hematopoietic stem cell Transplantation
Short name: OMST
Sponsor: EBMT/ADWP
Chief investigator: Riccardo Saccardi, Florence, Italy
Italie: Riccardo Saccardi (Florence), Giovanni L Mancardi (Genova)
France: Dominique Farge (Paris)
Sweden: Joachim Burman (Uppsala)
Switzerland: Roland Martin (Zürich)
United Kingdom: Paolo Muraro (London), Basile Sharrack (Sheffield)
Germany: Christoph Heesen (Hamburg)

Statistician: Maria Pia Sormani, Totino, Italy
Protocol Design: multi-centre, prospective, non-interventional study
Aim of the study: To gain further homogenous evidence for clinical efficacy of aHSCT in patients undergoing aHSCT for MS as primary indication
Inclusion criteria
Diagnosis of MS according to the 2010 revision McDonald’s criteria
Availability of a detailed clinical history about MS, including progression of disability and relapse rate in the previous 2 years, previous treatments administered
Patients aged 18yrs or over at the time of the first aHSCT
Exclusion criteria
Lack of one of the above criteria
Physical, mental, or social condition which could affect the patient from returning for follow-up visits
Patients with cognitive impairments, who are unable to provide written, informed consent prior to any testing under this protocol, including screening and baseline investigations that are considered part of routine patient care

Primary Endpoint: Time to failure to maintain a NEDA status

Secondary Endpoints
Overall survival
Transplant related mortality
MRI Assessment including lesions
Treatment-related complications.
Quality of life through the MS QL 54 standard assessment
Improvement of disability

Recruitment: 50 patients
Recruitment period: 2 years
Follow-up duration: 2 years