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| Name | Class |
|---|---|
| St. Antonius Hospital | OTHER |
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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:
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.
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| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Annual relapse rate | 2 years | |
| Time to first relapse | 2 years | |
| Confirmed disability progression (CDP) |
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Inclusion Criteria:
Exclusion Criteria:
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Patients with highly active relapsing-remitting multiple sclerosis are eligible for treatment with aHSCT in the Netherlands if they meet the criteria as defined by the Dutch National Health Care Institute.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Rick Heijnen, MSc | Contact | +31627228507 | r.m.heijnen@amsterdamumc.nl |
| Name | Affiliation | Role |
|---|---|---|
| J. Killestein, MSc, PhD | Amsterdam UMC | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Amsterdam UMC | Recruiting | Amsterdam | Netherlands |
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| ID | Term |
|---|---|
| D020529 | Multiple Sclerosis, Relapsing-Remitting |
| ID | Term |
|---|---|
| D009103 | Multiple Sclerosis |
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
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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 |
| St. Antonius Hospital | Recruiting | Nieuwegein | Netherlands |
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| D003711 | Demyelinating Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |