ATLG, Autologous Hematopoietic Stem Cell Transplant, Multiple Sclerosis
Conditions
Brief summary
This is a prospective, observational, biological, multicenter study to investigate IR profile, ATLG dynamics, main HSCT and disease outcomes in MS. This study will provide a preliminary descriptive evaluation of key study parameters, including ATLG pharmacokinetics and immune reconstitution trends, as well as an initial assessment of variability (e.g., dispersion of immunological biomarkers), to support the interpretation of results and the design of future studies. Since this is an observational study, no clinical decision will be made, and the interim analysis will not have an impact of the study conduction, and it will not be a stopping rule.
Interventions
DRUGATLG administration
Patients will receive standard conditioning regimen (BEAM or cyclophosphamide) and ATLG (total dose 30 mg/kg over 3 days: 10 mg/kg on days -3, -2 and -1).
Sponsors
Ciceri Fabio
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participant is willing and able to give informed consent for participation in the study. * Adult patients (age \>/= 18y) * Diagnosis of MS * Confirmed program of autologous HSCT according to standard EBMT indications (any conditioning regimen considered standard, BEAM or Cyclophosphamide) * ATLG (total dose 30 mg/kg over 3 days: 10 mg/kg on days -3, -2 and -1) in the conditioning regimen.
Exclusion criteria
* Subjects that did not accept to sign the informed consent. * Use of ATG. * Contraindications to HSCT procedures (including pregnancy and breast feeding, uncontrolled active infections)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Investigate immune status in correlation with ATLG | before mobilization, before conditioning, +1/3/6/12 months after HSCT. | Longitudinal IR monitoring (T/B/NK lymphocyte subpopulations including naïve- memory- stem/memory- regulatory subsets, cytokine profile, NFL/Macrophages) on PB |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Assess clinically relevant viral infections in correlation with viral specific IR | before mobilization, +1 month after HSCT within the first year after HSCT. | Assessment of pathogen-specific responses (CMV specific T cells by ELISpot) and occurrence of clinically relevant viral infections (CMV, EBV; ECIL 10 guidelines). |
| To assess HSCT and neurological outcomes | at 100 days and 1-year | Transplant-related mortality (TRM) |
| Assessment of Progression Free Survival (PFS) | at 1-year | Assessment of general HSCT outcomes (PFS) |
| Assessment of Overall survival (OS) | At 1 year | Assessment of general HSCT outcomes - OS |
| neurological status- Expanded Disability Status Scale (EDSS) | at 100 days, 6 months and 1-year | Expanded Disability Status Scale (EDSS) |
| neurological status - neurological progression | at 1 year | neurological status - neurological progression |
| neurological status -No Evidence of Disease Activity | At 1 year | No Evidence of Disease Activity (NEDA) |
Countries
Italy
Contacts
CONTACTRaffaella Greco
Outcome results
None listed