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NCT Number: NCT06567197

Multiple Sclerosis Treatment With Autologous Hematopoietic Stem Cell Transplantation in the Netherlands

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.

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Key information

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Amsterdam UMC, Amsterdam, Netherlands

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. Treatment efficacy

    Time frame: 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 outcomes

  1. Annual relapse rate

    Time frame: 2 years

  2. Time to first relapse

    Time frame: 2 years

  3. Confirmed disability progression (CDP)

    Time frame: 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.

  4. Confirmed disability improvement (CDI)

    Time frame: 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.

  5. Progression independent of relapse activity (PIRA)

    Time frame: 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.

  6. Changes on the multiple sclerosis functional composite (MSFC)

    Time frame: 2 years

    MSFC consists of the timed 25-foot walk test, 9-hole peg test and standard digit modalities test (SDMT)

  7. Brain/spinal cord MRI

    Time frame: 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.

  8. Optical coherence tomography (OCT)

    Time frame: 2 years

    Developments of optic neuritis and longitudinal changes of the retinal nerve fiber layer will be assessed.

  9. Side-effects and toxicity

    Time frame: 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

  10. Multiple Sclerosis Impact Scale-29 (MSIS-29)

    Time frame: 2 years

    Patient reported outcome measures about the impact of MS on daily life

  11. EuroQoL 5D (EQ-5D-5L)

    Time frame: 2 years

    Patient reported outcome measures about quality of life

  12. Modified Fatigue Impact Scale (MFIS-5)

    Time frame: 2 years

    Patient reported outcome measures about fatigue

  13. Hospital Anxiety and Depression Scale (HADS)

    Time frame: 2 years

    Patient reported outcome measures about anxiety and depression

  14. iMTA Medical Cost Questionnaire (iMCQ)

    Time frame: 2 years

    Patient reported outcome measures about medical consumption

  15. iMTA Productivity Cost Questionnaire (iPCQ)

    Time frame: 2 years

    Patient reported outcome measures about productivity

  16. Treatment Satisfaction Questionnaire for Medication (TSQM)

    Time frame: 2 years

    Patient reported outcome measures about medication

  17. Biomarkers

    Time frame: 2 years

    Serum NfL and GFAP over time

  18. Proteomics

    Time frame: 2 years

    Olink discover panel

  19. Immune phenotyping

    Time frame: 2 years

    Characterization of innate and adaptive immune subsets with mass spectrometry imaging

  20. Genetic analysis

    Time frame: 2 years

    Immune gene profiling for gene signatures predictive for response to aHSCT

Study contacts

Contact information is provided by the study sponsor or research team.

Rick Heijnen, MSc

CONTACT

[email protected]

+31627228507

Sponsors and collaborators

Lead sponsor

Amsterdam UMC, location VUmc

Other

Collaborators

  • St. Antonius Hospital

Registry information

Official study title

Multiple Sclerosis Treatment With Autologous Hematopoietic Stem Cell Transplantation (MS-ACT): A Long-term Prospective Observational Study in the Netherlands

Acronym: MS-ACT

Important dates

Study start
2023
Primary completion
2028
Study completion
2028
First posted
Aug 22, 2024
Registry last updated
Aug 22, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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