To perform long-term pre-specified follow-up of patients undergoing aHSCT for refractory RRMS.
ID
Source
Brief title
Condition
- Autoimmune disorders
- Demyelinating disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Patients will be subjected to frequent visits with clinical, radiological and
biomarker follow-up until five years after aHSCT (primary phase and extension
phase). During these visits, clinical testing, evaluation by questionnaires,
MRI scans and blood sampling will be performed.
The main study parameters are: 1. no evidence of disease activity (meaning no
MS relapses, no disability progression and no MS MRI activity, i.e. NEDA3)
Secondary outcome
Secundary study parameters are safety and tolerability of aHSCT, biomarkers and
longitudinal analysis of immune reconstitution.
Background summary
In December 2022, autologous hematopoietic stem cell transplantation (aHSCT)
was approved in the Netherlands for reimbursement for patients with treatment
refractory relapsing remitting multiple sclerosis (RRMS). The Dutch Healthcare
institute (*Zorginstituut Nederland: ZIN*) requested research of the outcomes
of aHSCT for this indication.
Study objective
To perform long-term pre-specified follow-up of patients undergoing aHSCT for
refractory RRMS.
Study design
This is a multicenter, prospective, long-term observational study.
Study burden and risks
Patients will be subjected to frequent visits after aHSCT. Follow-up after
aHSCT in this study is more comprehensive than it would be in standard care
(longer MRI protocols, more extensive laboratory measures and biobanking, more
frequent clinical assessments).
De Boelelaan 1118
amsterdam 1081 HZ
NL
De Boelelaan 1118
amsterdam 1081 HZ
NL
Listed location countries
Age
Inclusion criteria
All patients who undergo aHSCT for the treatment of RRMS in the Netherlands are
eligible to participate in this observational study.
Exclusion criteria
The only exclusion criteria for this observational study is unwillingness to
participate or to sign informed consent.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL83783.018.23 |