Trial Outcomes & Findings for Patient Research Cohort: Rapidly Evolving Multiple Sclerosis (NCT NCT01044576)

Patient Research Cohort: Rapidly Evolving Multiple Sclerosis

The primary goal of the observational research cohort is to facilitate patient access to clinical trials testing new therapeutic interventions or appropriate management for rapidly evolving multiple sclerosis (MS). This was devised as a single, combined primary outcome measure. The primary outcome is the proportion of research cohort subjects either referred into a clinical trial or offered treatment with an appropriate second-line therapy. (approved Protocol Version 4.1 - September 13th, 2011). The statistical assumption based on data from similar research cohorts stipulated that 50% of recruited patients will consent to proceed to further clinical trials or access new therapies.

The outcome reports the Number of participants whose data was used in any approved research. Examples of utilisation of data include imaging data analysis for MS-related research performed on the study participants' dataset and analysis of correlation of clinical phenotype with imaging data. Given the exploratory nature, no specific quantitative assumptions are made on the secondary outcome. Examples of studies utilising the anonymised cohort data: (1) Rapidly evolving multiple sclerosis: MRI findings predict clinical progression and disease phenotype (Dr Jean Lee, Dr A Waldmann, Dr R Newbould, ICL). (2) A study to characterize the novel TSPO PET radioligand \[18F\]PBR111 as an in vivo marker of microglial activation in Multiple Sclerosis (Dr A Colasanti, Imanova Ltd and GlaxoSmithKline). Conditions for participants to meet this outcome measure are: (A) completion of study visits (B) maintained (non-revoked) consent and (C) utilisation of the anonymised data in any further study.

The outcome consists of the Number of participants whose data was used in biomarkers development studies. Biomarkers studies include analysis of blood immunological studies performed on the study participants' dataset and analysis of correlation of clinical phenotype with immunological data, examples given below. Conditions for participants to meet this outcome measure are: (A) completion of study visits (B) maintained (non-revoked) consent and (C) utilisation of the anonymised immunological and clinical data in any biomarkers study. Given the exploratory nature, no quantitative assumptions are made on the outcome. Examples of studies of biomarkers: 1) Functional relevance of haematopoietic stem cell mobilisation following therapeutic alpha 4-integrin blockade in multiple sclerosis (Dr MMattoscio, ICL). 2)The relationship between T cell responses and disease progression in demyelinating disorders of the central nervous system (Prof D Altmann, ICL).

The outcome consists of the Number of participants whose data was used in studies aimed at the development of markers of clinical prognosis. Those are studies involving statistical analysis and models that may enable prognostic predictions from clinical phenotype, imaging and immunological data in any combination, with examples indicated below. Conditions for participants to meet this outcome measure are: (A) completion of study visits (B) maintained (non-revoked) consent and (C) utilisation of the anonymised imaging immunological and clinical data in any prognostic development research. Given their exploratory nature, no specific quantitative assumptions are made on the secondary outcome. Example of studies of prognostic markers: 1) Worse Physical Disability is associated with High Blood frequency of CD8+CD57+(ILT2+PD-1+) T-cells in MS Patients with Older Appearing Brains (Dr S Jacobs, Prof R Nicholas and Prof J Cole, Imperial College London and KCL).