Multiple Sclerosis Treatment With Autologous Hematopoietic Stem Cell Transplantation in the Netherlands
- Conditions
- Multiple Sclerosis, Relapsing-Remitting
- Registration Number
- NCT06567197
- Lead Sponsor
- Amsterdam UMC, location VUmc
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 24
- All patients approved for treatment with aHSCT in the Netherlands in accordance with the Dutch criteria for aHSCT treatment for RRMS
- Contra-indications for treatment with aHSCT such as known hypersensitivity to the medication used for aHSCT
- Clinically relevant comorbidities preventing safe use of medication used for aHSCT
- Severe clinical depression
- Active addiction to drugs or alcohol
- Active infections such as but not limited to tuberculosis, cytomegalovirus, Epstein-Barr virus, herpes simplex, varicella zoster, viral hepatitis, toxoplasmosis, HIV or syphilis.
- Active malignancy or history of malignancy with the exception of local basal cell carcinoma or carcinoma in situ of the cervix
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Treatment efficacy 2 years 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.
- Secondary Outcome Measures
Name Time Method Time to first relapse 2 years EuroQoL 5D (EQ-5D-5L) 2 years Patient reported outcome measures about quality of life
Biomarkers 2 years Serum NfL and GFAP over time
Proteomics 2 years Olink discover panel
Immune phenotyping 2 years Characterization of innate and adaptive immune subsets with mass spectrometry imaging
Annual relapse rate 2 years Progression independent of relapse activity (PIRA) 2 years 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.
Side-effects and toxicity 2 years 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
Hospital Anxiety and Depression Scale (HADS) 2 years Patient reported outcome measures about anxiety and depression
Modified Fatigue Impact Scale (MFIS-5) 2 years Patient reported outcome measures about fatigue
iMTA Productivity Cost Questionnaire (iPCQ) 2 years Patient reported outcome measures about productivity
Treatment Satisfaction Questionnaire for Medication (TSQM) 2 years Patient reported outcome measures about medication
Confirmed disability progression (CDP) 2 years 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.
Confirmed disability improvement (CDI) 2 years 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.
Brain/spinal cord MRI 2 years 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.
Multiple Sclerosis Impact Scale-29 (MSIS-29) 2 years Patient reported outcome measures about the impact of MS on daily life
Changes on the multiple sclerosis functional composite (MSFC) 2 years MSFC consists of the timed 25-foot walk test, 9-hole peg test and standard digit modalities test (SDMT)
Optical coherence tomography (OCT) 2 years Developments of optic neuritis and longitudinal changes of the retinal nerve fiber layer will be assessed.
iMTA Medical Cost Questionnaire (iMCQ) 2 years Patient reported outcome measures about medical consumption
Genetic analysis 2 years Immune gene profiling for gene signatures predictive for response to aHSCT
Trial Locations
- Locations (2)
Amsterdam UMC
🇳🇱Amsterdam, Netherlands
St. Antonius Hospital
🇳🇱Nieuwegein, Netherlands