Multiple SclerosisOMSTNCT04674280
Observational Study in Multiple Sclerosis Patients Treated With Autologous Hematopoietic Stem Cell Transplantation
An observational study of Multiple Sclerosis, run by European Society for Blood and Marrow Transplantation.
Trial summary
Observational prospective , multi-center study Primary objective : To gain further homogenous evidence for clinical efficacy of aHSCT in patients undergoing aHSCT for MS as primary indication. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Multiple Sclerosis
- What is being tested?
- aHSCT
- Type of treatment
- Surgery or procedure
- How many people
- 50 participants
- Where
- No sites listed yet
- Study dates
- Jan 2021to Jan 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
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 wiht multiple sclerosis treated with autologous heamatopoietic stem cell transplantation
Who may be able to join
- Diagnosis of MS according to McDonald's criteria
- Availability of a detailed clinical history about MS, including progression of disability and relapse rate in the previous 2 years, treatments administered before aHSCT, and categorization of the sub-types of MS according to current EBMT guidelines
- Highly active relapsing remitting MS failing DMTs
- Progressive MS with active inflammatory component. In accordance with the EBMT guidelines, this category includes both secondary and primary progressive MS with active inflammatory component.
- Aggressive (malignant) MS not previously treated with a full course of DMT
- Patients aged 18 or over at time of first aHSCT
- Signed informed consent
Who cannot join
- Lack of one of the above criteria
- Progressive MS without active inflammatory component, as this indication is assigned 'GNR' according to EBMT guidelines, irrespective of whether it is secondary or primary progressive.
- Unwillingness or inability to comply with the requirements of this protocol including the presence of any condition (physical, mental, or social) that is likely to affect the patient be able to return for the scheduled follow-up visits.
- Patients with cognitive impairments, who are unable to provide written, informed consent prior to any testing under this protocol, including screening and baseline investigations that are considered part of routine patient care.
Trial locations and contacts
No sites are listed yet.
About this study
Observational prospective , multi-center study
Primary objective : To gain further homogenous evidence for clinical efficacy of aHSCT in patients undergoing aHSCT for MS as primary indication.
Read more
Secondary objectives:
- Safety, tolerability and toxicity of aHSCT in MS
- Quality of life and long-term disability after aHSCT
- MRI outcome after aHSCT
Primary endpoint : Time to failure to maintain a NEDA status
Secondary endpoints:
- Overall survival
- Transplant related mortality
- MRI Assessment including lesions
- Treatment-related complications . • Quality of life through the MS QL 54 standard assessment
- Improvement of disability
Inclusion criteria:
- Diagnosis of MS according to the 2010 revision McDonald's criteria
- Availability of a detailed clinical history about MS, including progression of disability and relapse rate in the previous 2 years, previous treatments administered
- Patients aged 18yrs or over at the time of the first aHSCT
Exclusion criteria:
- Lack of one of the above criteria
- Physical, mental, or social condition which could affect the patient from returning for follow-up visits
- Patients with cognitive impairments, who are unable to provide written, informed consent prior to any testing under this protocol, including screening and baseline investigations that are considered part of routine patient care.
Recruitment: 50 patients Recruitment period: 2 years starting from the inclusion of the 1st patient Follow-up duration: 2 years
Read the detailed description
Currently, a number of new prospective comparative trials have either started or are in preparation, and the EBMT guidelines recommend treatment on a clinical trial, where available. However, some MS patients receiving aHSCT will not be eligible for these trials, which are predominantly focussed on a tightly defined group of patients with highly active RRMS. In addition, access to such trials is not universal across EBMT and therefore there is a need for structured data collection.
The investigators propose here to conduct an observational cohort study that systematically gathers experience from MS patients, for whom an adequate set of data about their clinical history and their clinical situation at baseline is available; patients will be then be followed prospectively according to their local clinical center protocol. The expectation is that local clinical protocols will be in line with the current EBMT guidelines and recommendations. Therefore, the aim will be to prospectively and non-interventionally capture a complete dataset to safety, tolerability and efficacy outcomes for aHSCT in various sub-types of MS treated in accordance with the current EBMT guidelines and recommendations. Alongside ongoing clinical trials, the investigators hope this study will meaningfully inform the evidence-base and future guidelines and recommendations for good clinical practice.
1- Study design Multi-centre, non-interventional prospective study in patients diagnosed with Multiple Sclerosis, treated with aHSCT.
2 Aims and objectives of the study
- Primary objectives
- To evaluate the outcome of patients undergoing aHSCT for MS as primary indication.
- Secondary objectives
- To gain further knowledge on safety, tolerability and toxicity of aHSCT in MS
- To gain further knowledge on the quality of life and long-term disability of patients treated with aHSCT
- To gain further knowledge about the MRI outcome after aHSCT
- Study schedule The clinical assessment at enrolment and during follow-up will be carried out according to local policies, with the expectation that centres will be working in line with the EBMT Guidelines and Recommendations for MS and immune-mediated neurological diseases. However, based on common clinical practice in patients with MS on therapy, an MRI and one neurological assessment are usually carried out yearly. Baseline MRI should be carried out within 3 months before the start of the mobilization regimen, whilst neurological examination should be accomplished as baseline assessment no more than one month before the start of the treatment.
Enrolment time is 2 years from the registration of the first patient. Follow-up is anticipated for 24 months from individual start of treatment.
- Conditions
- Multiple Sclerosis
- Being tested
- aHSCT
- Medical subject headings
- Multiple Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
aHSCTProcedure or surgery
Autologous haematopoietic stem cell transplantation
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to NEDA-3 failure
NEDA-3 is defined as a composite endpoint including survival without relapses, disability progression and MRI activity (new or enlarging T2 lesions or Gd-enhancing lesions on MRI).
Measured: 4 years
Secondary outcomes (2)
Overall survival
Overall survival (OS), is defined as the interval between the date of aHSCT and either the last follow-up visit or the date of death from any cause.
Measured: 4 years
Transplant related mortality
Treatment related mortality (TRM) defined as any death following transplant that cannot be attributed to progression or relapse of the disease.
Measured: 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2020
- Study startJan 2021 (estimated)
- Primary completionJan 2025 (estimated)
- Study completionJan 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple SclerosisStudies of the same condition, open and under way.
- 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
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- European Society for Blood and Marrow Transplantation
- Sponsor type
- Research network
- Responsible party
- Sponsor
- Investigators
- Riccardo Saccardi, MD, Principal investigator, European Society for Blood and Marrow Transplantation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04674280
- Sponsor's protocol ID
- 8410-016
- Acronym
- OMST
- Status last verified
- Dec 2020
- First submitted
- 14 Dec 2020
- Last updated
- 21 Dec 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational Study in Multiple Sclerosis Patients Treated With Autologous Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT04674280
- Acronym / sponsor ID
- OMST / 8410-016
- Conditions
- Multiple Sclerosis
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- European Society for Blood and Marrow TransplantationResearch network
- Study start
- Jan 2021 (estimated)
- Primary completion
- Jan 2025 (estimated)
- Study completion
- Jan 2025 (estimated)
- First posted
- 19 Dec 2020 (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.
Observational prospective , multi-center study
Primary objective : To gain further homogenous evidence for clinical efficacy of aHSCT in patients undergoing aHSCT for MS as primary indication.
Secondary objectives:
- Safety, tolerability and toxicity of aHSCT in MS
- Quality of life and long-term disability after aHSCT
- MRI outcome after aHSCT
Primary endpoint : Time to failure to maintain a NEDA status
Secondary endpoints:
- Overall survival
- Transplant related mortality
- MRI Assessment including lesions
- Treatment-related complications . • Quality of life through the MS QL 54 standard assessment
- Improvement of disability
Inclusion criteria:
- Diagnosis of MS according to the 2010 revision McDonald's criteria
- Availability of a detailed clinical history about MS, including progression of disability and relapse rate in the previous 2 years, previous treatments administered
- Patients aged 18yrs or over at the time of the first aHSCT
Exclusion criteria:
- Lack of one of the above criteria
- Physical, mental, or social condition which could affect the patient from returning for follow-up visits
- Patients with cognitive impairments, who are unable to provide written, informed consent prior to any testing under this protocol, including screening and baseline investigations that are considered part of routine patient care.
Recruitment: 50 patients Recruitment period: 2 years starting from the inclusion of the 1st patient Follow-up duration: 2 years
Detailed description
Currently, a number of new prospective comparative trials have either started or are in preparation, and the EBMT guidelines recommend treatment on a clinical trial, where available. However, some MS patients receiving aHSCT will not be eligible for these trials, which are predominantly focussed on a tightly defined group of patients with highly active RRMS. In addition, access to such trials is not universal across EBMT and therefore there is a need for structured data collection.
The investigators propose here to conduct an observational cohort study that systematically gathers experience from MS patients, for whom an adequate set of data about their clinical history and their clinical situation at baseline is available; patients will be then be followed prospectively according to their local clinical center protocol. The expectation is that local clinical protocols will be in line with the current EBMT guidelines and recommendations. Therefore, the aim will be to prospectively and non-interventionally capture a complete dataset to safety, tolerability and efficacy outcomes for aHSCT in various sub-types of MS treated in accordance with the current EBMT guidelines and recommendations. Alongside ongoing clinical trials, the investigators hope this study will meaningfully inform the evidence-base and future guidelines and recommendations for good clinical practice.
1- Study design Multi-centre, non-interventional prospective study in patients diagnosed with Multiple Sclerosis, treated with aHSCT.
2 Aims and objectives of the study
- Primary objectives
- To evaluate the outcome of patients undergoing aHSCT for MS as primary indication.
- Secondary objectives
- To gain further knowledge on safety, tolerability and toxicity of aHSCT in MS
- To gain further knowledge on the quality of life and long-term disability of patients treated with aHSCT
- To gain further knowledge about the MRI outcome after aHSCT
- Study schedule The clinical assessment at enrolment and during follow-up will be carried out according to local policies, with the expectation that centres will be working in line with the EBMT Guidelines and Recommendations for MS and immune-mediated neurological diseases. However, based on common clinical practice in patients with MS on therapy, an MRI and one neurological assessment are usually carried out yearly. Baseline MRI should be carried out within 3 months before the start of the mobilization regimen, whilst neurological examination should be accomplished as baseline assessment no more than one month before the start of the treatment.
Enrolment time is 2 years from the registration of the first patient. Follow-up is anticipated for 24 months from individual start of treatment.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients wiht multiple sclerosis treated with autologous heamatopoietic stem cell transplantation
Key inclusion criteria (7 listed)
- Diagnosis of MS according to McDonald's criteria
- Availability of a detailed clinical history about MS, including progression of disability and relapse rate in the previous 2 years, treatments administered before aHSCT, and categorization of the sub-types of MS according to current EBMT guidelines
- Patients aged 18 or over at time of first aHSCT
- Signed informed consent
Key exclusion criteria (4 listed)
- Lack of one of the above criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| aHSCTAutologous haematopoietic stem cell transplantation | Procedure or surgery | – |
Primary outcome
Time to NEDA-3 failure
Time frame: 4 years
NEDA-3 is defined as a composite endpoint including survival without relapses, disability progression and MRI activity (new or enlarging T2 lesions or Gd-enhancing lesions on MRI).
2 secondary outcomes
Overall survival
Time frame: 4 years
Transplant related mortality
Time frame: 4 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Dec 2020.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Riccardo Saccardi, MD principal investigator
European Society for Blood and Marrow Transplantation
Questions about this trial
What is this clinical trial studying?
An observational study of Multiple Sclerosis, run by European Society for Blood and Marrow Transplantation.
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 7 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 21 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact EBMT ADWP- OMST study coordinator (+33.1.70.64.24.16, manuela.badoglio@upmc.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to NEDA-3 failure (time frame: 4 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 21 Dec 2020.