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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06195800
Other study ID # STH22343
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date August 9, 2023
Est. completion date August 9, 2026

Study information

Verified date January 2024
Source Sheffield Teaching Hospitals NHS Foundation Trust
Contact Gavin Brittain, MBBS, MRCP
Phone +44114 271 1900
Email gavin.brittain@sheffield.ac.uk
Is FDA regulated No
Health authority
Study type Observational

Clinical 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 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.


Recruitment information / eligibility

Status Recruiting
Enrollment 15
Est. completion date August 9, 2026
Est. primary completion date August 9, 2026
Accepts healthy volunteers No
Gender All
Age group 16 Years and older
Eligibility Inclusion Criteria: 1. Diagnosis of a immune mediated neurological disease according to disease specific criteria (active treatment arm) or diagnosis of relapsing remitting multiple sclerosis (control arm). 2. Treatment with autologous haematopoetic stem cell transplantation (active treatment arm) or high efficacy disease modifying treatment (control arm). 3. Willing to provide biological samples for analysis and undergo clinical assessments for the duration of follow up. 4. Able to understand English and provide informed consent. Exclusion Criteria: 1. Inability to provide informed consent.

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
United Kingdom Sheffield Teaching Hospitals NHS Foundation Trust Sheffield England

Sponsors (4)

Lead Sponsor Collaborator
Sheffield Teaching Hospitals NHS Foundation Trust Nottingham Trent University, Sheffield Hospitals Charity, University of Sheffield

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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.
24 months
Primary 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 24 months
Secondary 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. 24 months
Secondary 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. 24 months
Secondary 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. 24 months
Secondary 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. 24 months
Secondary 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. 24 months
Secondary 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. 24 months
Secondary 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. 24 months
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