Multiple Sclerosis, Relapsing-RemittingMS-ACTNCT06567197
Multiple Sclerosis Treatment With Autologous Hematopoietic Stem Cell Transplantation in the Netherlands
An observational study of Multiple Sclerosis, Relapsing-Remitting, run by Amsterdam UMC, location VUmc at 2 sites in Netherlands.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Multiple Sclerosis, Relapsing-Remitting
- How many people
- 24 participants
- Where
- 2 sitesNetherlands
- Study dates
- 25 Aug 2023to 1 Sep 2028
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- All patients approved for treatment with aHSCT in the Netherlands in accordance with the Dutch criteria for aHSCT treatment for RRMS
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Netherlands2 sites, 2 recruiting
- Recruiting
Amsterdam UMC
Amsterdam
Rick Heijnen, MSc, +31627228507, r.m.heijnen@amsterdamumc.nl
R.M. Heijnen, MSc
- Recruiting
St. Antonius Hospital
Nieuwegein
Rick Heijnen, MSc, +31627228507, r.m.heijnen@amsterdamumc.nl
E.L.J. Hoogervorst, MSc, PhD
About this study
Official title: Multiple Sclerosis Treatment With Autologous Hematopoietic Stem Cell Transplantation (MS-ACT): A Long-term Prospective Observational Study 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:
- Disease activity
- Safety and tolerability of aHSCT
- 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.
- Conditions
- Multiple Sclerosis, Relapsing-Remitting
- Medical subject headings
- Multiple Sclerosis, Relapsing-Remitting
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 2 years
Secondary outcomes (20)
Annual relapse rate
Measured: 2 years
Time to first relapse
Measured: 2 years
Confirmed disability progression (CDP)
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.
Measured: 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.
Measured: 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.
Measured: 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)
Measured: 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.
Measured: 2 years
Optical coherence tomography (OCT)
Developments of optic neuritis and longitudinal changes of the retinal nerve fiber layer will be assessed.
Measured: 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
Measured: 2 years
Multiple Sclerosis Impact Scale-29 (MSIS-29)
Patient reported outcome measures about the impact of MS on daily life
Measured: 2 years
EuroQoL 5D (EQ-5D-5L)
Patient reported outcome measures about quality of life
Measured: 2 years
Modified Fatigue Impact Scale (MFIS-5)
Patient reported outcome measures about fatigue
Measured: 2 years
Hospital Anxiety and Depression Scale (HADS)
Patient reported outcome measures about anxiety and depression
Measured: 2 years
iMTA Medical Cost Questionnaire (iMCQ)
Patient reported outcome measures about medical consumption
Measured: 2 years
iMTA Productivity Cost Questionnaire (iPCQ)
Patient reported outcome measures about productivity
Measured: 2 years
Treatment Satisfaction Questionnaire for Medication (TSQM)
Patient reported outcome measures about medication
Measured: 2 years
Biomarkers
Serum NfL and GFAP over time
Measured: 2 years
Proteomics
Olink discover panel
Measured: 2 years
Immune phenotyping
Characterization of innate and adaptive immune subsets with mass spectrometry imaging
Measured: 2 years
Genetic analysis
Immune gene profiling for gene signatures predictive for response to aHSCT
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Aug 2024
- Study start25 Aug 2023
- Primary completion1 Sep 2028 (estimated)
- Study completion1 Sep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple Sclerosis, Relapsing-RemittingStudies of the same condition, open and under way.
- More trials run by Amsterdam UMC, location VUmcThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 24 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Amsterdam UMC, location VUmc
- Sponsor type
- University, hospital or other
- Collaborators
- St. Antonius Hospital
- Responsible party
- Principal investigator, Joep Killestein, Amsterdam UMC, location VUmc
- Investigators
- J. Killestein, MSc, PhD, Principal investigator, Amsterdam UMC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06567197
- Sponsor's protocol ID
- NL83783.018.23
- Acronym
- MS-ACT
- Status last verified
- Aug 2024
- First submitted
- 25 Jun 2024
- Last updated
- 22 Aug 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multiple Sclerosis Treatment With Autologous Hematopoietic Stem Cell Transplantation (MS-ACT): A Long-term Prospective Observational Study in the Netherlands
- ClinicalTrials.gov ID
- NCT06567197
- Acronym / sponsor ID
- MS-ACT / NL83783.018.23
- Conditions
- Multiple Sclerosis, Relapsing-Remitting
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Amsterdam UMC, location VUmcUniversity, hospital or other
- Collaborators
- St. Antonius Hospital
- Study start
- 25 Aug 2023 (actual)
- Primary completion
- 1 Sep 2028 (estimated)
- Study completion
- 1 Sep 2028 (estimated)
- First posted
- 22 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- Disease activity
- Safety and tolerability of aHSCT
- 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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 5 Years
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (1 listed)
- All patients approved for treatment with aHSCT in the Netherlands in accordance with the Dutch criteria for aHSCT treatment for RRMS
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
20 secondary outcomes
Annual relapse rate
Time frame: 2 years
Time to first relapse
Time frame: 2 years
Confirmed disability progression (CDP)
Time frame: 2 years
Confirmed disability improvement (CDI)
Time frame: 2 years
Progression independent of relapse activity (PIRA)
Time frame: 2 years
Changes on the multiple sclerosis functional composite (MSFC)
Time frame: 2 years
Brain/spinal cord MRI
Time frame: 2 years
Optical coherence tomography (OCT)
Time frame: 2 years
Side-effects and toxicity
Time frame: 2 years
Multiple Sclerosis Impact Scale-29 (MSIS-29)
Time frame: 2 years
EuroQoL 5D (EQ-5D-5L)
Time frame: 2 years
Modified Fatigue Impact Scale (MFIS-5)
Time frame: 2 years
Hospital Anxiety and Depression Scale (HADS)
Time frame: 2 years
iMTA Medical Cost Questionnaire (iMCQ)
Time frame: 2 years
iMTA Productivity Cost Questionnaire (iPCQ)
Time frame: 2 years
Treatment Satisfaction Questionnaire for Medication (TSQM)
Time frame: 2 years
Biomarkers
Time frame: 2 years
Proteomics
Time frame: 2 years
Immune phenotyping
Time frame: 2 years
Genetic analysis
Time frame: 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Aug 2024.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
J. Killestein, MSc, PhD principal investigator
Amsterdam UMC
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Multiple Sclerosis, Relapsing-Remitting, run by Amsterdam UMC, location VUmc at 2 sites in Netherlands.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Rick Heijnen, MSc (+31627228507, r.m.heijnen@amsterdamumc.nl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment efficacy (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Aug 2024.