Multiple Sclerosis Treatment With Autologous Hematopoietic Stem Cell Transplantation in the Netherlands
NCT Number: NCT06567197
Acronym: MS-ACT
Status: RECRUITING
Study Type: OBSERVATIONAL
Conditions: Multiple Sclerosis, Relapsing-Remitting
Sponsor: Amsterdam UMC, location VUmc
Collaborators: St. Antonius Hospital
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 24
Study Design: Observational Model: |Time Perspective: p
Start Date: 2023-08-25
Completion Date: 2028-09-01
Locations: Amsterdam UMC, Amsterdam, Netherlands|St. Antonius Hospital, Nieuwegein, Netherlands

Summary:
The goal of this observational study is to study the long-term effects of autologous hematopoietic stem cell transplantation (aHSCT) in people with highly active relapsing-remitting multiple sclerosis. The study will evaluate the following items:

1. Disease activity
2. Safety and tolerability of aHSCT
3. Changes in the immune system

Participants will be subjected to frequent visits for five years after treatment with aHSCT. During these visits, clinical testing, evaluation by questionnaires, MRI scans and blood sampling will be performed.

Primary Outcome Measures: Treatment efficacy, The proportion of patients with ne evidence of disease activity-3 (NEDA-3) as defined by: no clinical relapse, no disability progression, no radiological disease activity., 2 years
Secondary Outcome Measures: Annual relapse rate, 2 years|Time to first relapse, 2 years|Confirmed disability progression (CDP), Measured by the Expanded Disability Status Scale (EDSS) CDP-EDSS is defined as an increase of one point in the EDSS score from baseline to month 24., 2 years|Confirmed disability improvement (CDI), Measured by the Expanded Disability Status Scale (EDSS) CDI-EDSS is defined as a decrease of one point in the EDSS score from baseline to month 24, with an absence of relapse at the point of assessment., 2 years|Progression independent of relapse activity (PIRA), Defined as an episode of CDP without relapse during the 90 days before EDSS increase and during the 6-month period between the EDSS increase and the confirmation of disability progression., 2 years|Changes on the multiple sclerosis functional composite (MSFC), MSFC consists of the timed 25-foot walk test, 9-hole peg test and standard digit modalities test (SDMT), 2 years|Brain/spinal cord MRI, Presence and number of contrast-enhancing lesions at baseline and the development of new or enlarging lesions between baseline and follow-up will be assessed., 2 years|Optical coherence tomography (OCT), Developments of optic neuritis and longitudinal changes of the retinal nerve fiber layer will be assessed., 2 years|Side-effects and toxicity, Frequency and type of (serious) adverse events such as infections, secondary auto-immunity, fertility problems, clinical relevant changes on physical examination and use of concomitant medications will be assessed, 2 years|Multiple Sclerosis Impact Scale-29 (MSIS-29), Patient reported outcome measures about the impact of MS on daily life, 2 years|EuroQoL 5D (EQ-5D-5L), Patient reported outcome measures about quality of life, 2 years|Modified Fatigue Impact Scale (MFIS-5), Patient reported outcome measures about fatigue, 2 years|Hospital Anxiety and Depression Scale (HADS), Patient reported outcome measures about anxiety and depression, 2 years|iMTA Medical Cost Questionnaire (iMCQ), Patient reported outcome measures about medical consumption, 2 years|iMTA Productivity Cost Questionnaire (iPCQ), Patient reported outcome measures about productivity, 2 years|Treatment Satisfaction Questionnaire for Medication (TSQM), Patient reported outcome measures about medication, 2 years|Biomarkers, Serum NfL and GFAP over time, 2 years|Proteomics, Olink discover panel, 2 years|Immune phenotyping, Characterization of innate and adaptive immune subsets with mass spectrometry imaging, 2 years|Genetic analysis, Immune gene profiling for gene signatures predictive for response to aHSCT, 2 years