ATLG Levels in Autologous HSCT for Multiple Sclerosis
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorCiceri Fabio
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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)
Disqualifiers
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)
Trial population
The study will involve 20 MS patients, treated with autologous HSCT as standard-of-care according to guidelines and consensus recommendations. Autologous HSCT is considered a standard-of-care intervention in the following selected MS patients: RRMS who have failed to respond to one or more high-efficacy DMTs or progressive MS with superimposed inflammatory activity, or aggressive forms of MS where rapid neurological decline occurs early in the disease course. 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).
Trial design
Design model
Case-only
Time perspective
Prospective
Treatments tested in this trial
ATLG administration
DrugPatients 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).
Treatment groups
20 Participants
are divided into 1 treatment groupGroup A: Multiple Sclerosis patients treated with autologous HSCT as standard of care1 intervention
Trial outcomes
Primary outcomes
1
Investigate immune status in correlation with ATLG
Longitudinal IR monitoring (T/B/NK lymphocyte subpopulations including naïve- memory- stem/memory- regulatory subsets, cytokine profile, NFL/Macrophages) on PB
Time frame
before mobilization, before conditioning, +1/3/6/12 months after HSCT.2
Investigate immune status in correlation with ATLG
Assessment of ATLG levels \& kinetics
Time frame
before and 30' after the end of ATLG dose, day0, day1, weekly for 4 weeks.Secondary outcomes
1
Assess clinically relevant viral infections in correlation with viral specific IR
Assessment of pathogen-specific responses (CMV specific T cells by ELISpot) and occurrence of clinically relevant viral infections (CMV, EBV; ECIL 10 guidelines).
Time frame
before mobilization, +1 month after HSCT within the first year after HSCT.2
To assess HSCT and neurological outcomes
Transplant-related mortality (TRM)
Time frame
at 100 days and 1-year3
Assessment of Progression Free Survival (PFS)
Assessment of general HSCT outcomes (PFS)
Time frame
at 1-year4
Assessment of Overall survival (OS)
Assessment of general HSCT outcomes - OS
Time frame
At 1 yearSponsors and contacts
Click on the lead sponsor to view all of their trials.
Ciceri Fabio
Lead sponsor
IRCCS San Raffaele
Sponsor institution
Source ClinicalTrials.gov