Multiple Sclerosis, Relapsing-Remitting
Conditions
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.
Interventions
None listed
Sponsors
St. Antonius Hospital
Amsterdam UMC, location VUmc
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* All patients approved for treatment with aHSCT in the Netherlands in accordance with the Dutch criteria for aHSCT treatment for RRMS
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| Annual relapse rate | 2 years | — |
| Time to first relapse | 2 years | — |
| 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. |
| 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. |
| 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. |
| 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 |
| Multiple Sclerosis Impact Scale-29 (MSIS-29) | 2 years | Patient reported outcome measures about the impact of MS on daily life |
| 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. |
| Modified Fatigue Impact Scale (MFIS-5) | 2 years | Patient reported outcome measures about fatigue |
| Hospital Anxiety and Depression Scale (HADS) | 2 years | Patient reported outcome measures about anxiety and depression |
| iMTA Medical Cost Questionnaire (iMCQ) | 2 years | Patient reported outcome measures about medical consumption |
| 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 |
| 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 |
| Genetic analysis | 2 years | Immune gene profiling for gene signatures predictive for response to aHSCT |
| EuroQoL 5D (EQ-5D-5L) | 2 years | Patient reported outcome measures about quality of life |
Countries
Netherlands
Contacts
Primary ContactRick Heijnen, MSc
Outcome results
None listed