Clinical trial NCT06567197: Multiple Sclerosis Treatment With Autologous Hematopoietic Stem Cell Transplantation in the Netherlands
Official title: Multiple Sclerosis Treatment With Autologous Hematopoietic Stem Cell Transplantation (MS-ACT): A Long-term Prospective Observational Study in the Netherlands
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Multiple Sclerosis, Relapsing-Remitting
Lead sponsor: Amsterdam UMC, location VUmc
Sponsor class: OTHER
Collaborators: name: St. Antonius Hospital
First posted: 2024-08-22
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 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.

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.


Eligibility criteria: 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

Primary outcomes:
measure: Treatment efficacy
timeFrame: 2 years
Secondary outcomes:
measure: Annual relapse rate
timeFrame: 2 years
measure: Time to first relapse
timeFrame: 2 years
measure: Confirmed disability progression (CDP)
timeFrame: 2 years
measure: Confirmed disability improvement (CDI)
timeFrame: 2 years
measure: Progression independent of relapse activity (PIRA)
timeFrame: 2 years
measure: Changes on the multiple sclerosis functional composite (MSFC)
timeFrame: 2 years
measure: Brain/spinal cord MRI
timeFrame: 2 years
measure: Optical coherence tomography (OCT)
timeFrame: 2 years
measure: Side-effects and toxicity
timeFrame: 2 years
measure: Multiple Sclerosis Impact Scale-29 (MSIS-29)
timeFrame: 2 years
measure: EuroQoL 5D (EQ-5D-5L)
timeFrame: 2 years
measure: Modified Fatigue Impact Scale (MFIS-5)
timeFrame: 2 years
measure: Hospital Anxiety and Depression Scale (HADS)
timeFrame: 2 years
measure: iMTA Medical Cost Questionnaire (iMCQ)
timeFrame: 2 years
measure: iMTA Productivity Cost Questionnaire (iPCQ)
timeFrame: 2 years
measure: Treatment Satisfaction Questionnaire for Medication (TSQM)
timeFrame: 2 years
measure: Biomarkers
timeFrame: 2 years
measure: Proteomics
timeFrame: 2 years
measure: Immune phenotyping
timeFrame: 2 years
measure: Genetic analysis
timeFrame: 2 years
Other outcomes:
Locations:
city: Amsterdam
country: Netherlands
facility: Amsterdam UMC
city: Nieuwegein
country: Netherlands
facility: St. Antonius Hospital
Study officials:
affiliation: Amsterdam UMC
name: J. Killestein, MSc, PhD
role: PRINCIPAL_INVESTIGATOR
References: