Multiple Sclerosis, Relapsing-RemittingBIO-MSNCT06195800
Identifying Immune Biomarkers of Disease and Disease Control in Autoimmune Neurological Disease Using Autologous Haematopoietic Stem Cell Transplantation
An observational study of Multiple Sclerosis, Relapsing-Remitting, run by Sheffield Teaching Hospitals NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
The underlying disease mechanisms which occur in patients with immune mediation neurological diseases, such as Multiple Sclerosis (MS), are incompletely understood. For such patients, autologous haematopoietic stem cell transplantation (aHSCT) has been increasingly used as a highly successful one-off treatment for… Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Multiple Sclerosis, Relapsing-Remitting
- How many people
- 15 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 9 Aug 2023to 30 Nov 2027
- Phase
- Observational
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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Outpatients who are attending the Sheffield (UK) transplant clinic for neurological disease.
Who may be able to join
- Diagnosis of a immune mediated neurological disease according to disease specific criteria (active treatment arm) or diagnosis of relapsing remitting multiple sclerosis (control arm).
- Treatment with autologous haematopoetic stem cell transplantation (active treatment arm) or high efficacy disease modifying treatment (control arm).
- Willing to provide biological samples for analysis and undergo clinical assessments for the duration of follow up.
- Able to understand English and provide informed consent.
Who cannot join
- 1. Inability to provide informed consent.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United Kingdom1 site, 1 recruiting
- Recruiting
Sheffield Teaching Hospitals NHS Foundation Trust
Sheffield, England, S10 2JF
Gavin Brittain, MBBS, MRCP, gavin.brittain@sheffield.ac.uk
Gavin Brittain, MBBS, MRCP
About this study
Official title: Identifying Immune Biomarkers of Disease and Disease Control in Autoimmune Neurological Disease Using Autologous Haematopoietic Stem Cell Transplantation: Biomarkers of aHSCT
The underlying disease mechanisms which occur in patients with immune mediation neurological diseases, such as Multiple Sclerosis (MS), are incompletely understood. For such patients, autologous haematopoietic stem cell transplantation (aHSCT) has been increasingly used as a highly successful one-off treatment for some patients. This treatment aims to delete the faulty immune system with a course of chemotherapy and then 'reboot' the immune system using a patients' own stem cells (a cell with the unique ability of being a building block to create many different cells in the body) to stop further damage. Over the last 20 years more than 1800 patients with MS have been treated in Europe with high levels of success. It may be more successful than disease modifying treatment but unfortunately, a small portion of people do not respond to this treatment optimally and continue to accumulate disability. There is a risk of side effects, restricted largely to the time of treatment, which necessitates the need to ensure appropriate patients are treated. Whilst aHSCT is a very effective therapy, it is still in its early phase of development, is not in widespread use, and there is incomplete knowledge regarding how it works and importantly, why it does not work in some patients, and how to monitor response to treatment.
Unfortunately, there is no way of detecting which patients will, and will not, benefit from the different treatments available or a way of monitoring the immune system to ensure further treatment is provided before irreversible damage occurs.
This study will investigate the immune system which is found in the fluid surrounding the brain and spinal cord, blood and stool of patients undergoing aHSCT and compare it to those receiving disease modifying treatment. This study will therefore further the understanding of biomarkers of aHSCT to develop an awareness of how it can be refined, may improve monitoring of patients following treatment and permit the development of markers which can predict potential treatment success or failure before patients are exposed to the risks.
- Conditions
- Multiple Sclerosis, Relapsing-Remitting
- Keywords
- multiple sclerosis, aHSCT, Autologous haematopoetic stem cell transplantation
- Medical subject headings
- Multiple Sclerosis, Relapsing-Remitting, Multiple Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Patients treated with aHSCT | Patients with aggressive disease treated with autologous haematopoetic stem cell transplantation. | |
| Patients treated with disease modifying treatment | Patients with aggressive disease treated with high efficacy disease modifying treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quantitatively and qualitatively characterise the immune profile of the stem cells, blood, cerebrospinal fluid (CSF) and the microbiome pre and post treatment.
Phenotype profiling of stem cells, cells in blood and CSF using multi-parameter flow cytometry.
Profile the gut microbiome using 16S rRNA sequencing and flow cytometric analysis.
Measured: 24 months
Perform single cell RNA sequencing (scRNA-Seq) on paired CSF and blood pre and post treatment.
Profiling of T cell receptor and B cell receptor repertoire diversity and clonality
Measured: 24 months
Secondary outcomes (7)
Characterisation of the regeneration of mucosal cell immunity and the reconstitution of pathogen specific immunity following aHSCT by scRNA-Seq on nasopharyngeal swabs and mucosal strips.
Assess immune response in treated patients post vaccination.
Measured: 24 months
Evaluate immunological disease response and the duration of response to aHSCT according to expanded disability status scale score (EDSS)
EDSS to be observed longitudinally following treatment.
Measured: 24 months
Evaluate immunological disease response and the duration of response to aHSCT according to low contrast visual acuity (LCLA)
LCLA to be observed longitudinally following treatment.
Measured: 24 months
Evaluate immunological disease response and the duration of response to aHSCT according to multiple sclerosis functional composite score (MSFC)
MSFC to be observed longitudinally following treatment.
Measured: 24 months
Evaluate immunological disease response and the duration of response to aHSCT according to short form 36 (SF-36)
SF-36 to be observed longitudinally following treatment.
Measured: 24 months
Evaluate immunological disease response and the duration of response to aHSCT according to symbol digit modality test (SDMT)
SDMT to be observed longitudinally following treatment.
Measured: 24 months
Evaluate immunological disease response and the duration of response to aHSCT according to Karnofsky performance status
Karnofsky performance status to be observed longitudinally following treatment.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jan 2024
- Study start9 Aug 2023
- Primary completion30 Nov 2027 (estimated)
- Study completion30 Nov 2027 (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.
- 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
- Case-control
- Time perspective
- Prospective
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sheffield Teaching Hospitals NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Collaborators
- Sheffield Hospitals Charity, Nottingham Trent University, University of Sheffield
- Responsible party
- Sponsor
- Investigators
- Gavin Brittain, MBBS, MRCP, Principal investigator, Sheffield Teaching Hospitals NHS Foundation Trust and The University of Sheffield
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06195800
- Sponsor's protocol ID
- STH22343
- Acronym
- BIO-MS
- Status last verified
- Oct 2026
- First submitted
- 13 Oct 2023
- Last updated
- 7 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided