Manouchehri, Navid and Zhang, Yinan and Salter, Amber and Hussain, Rehana Z. and Hartung, Hans-Peter and Hemmer, Bernhard and Linker, Ralf and Segal, Benjamin M. and Cutter, Gary and Stüve, Olaf (2019) Clinical trials in multiple sclerosis: potential future trial designs. THERAPEUTIC ADVANCES IN NEUROLOGICAL DISORDERS, 12. ISSN 1756-2856, 1756-2864
Full text not available from this repository. (Request a copy)Abstract
Clinical trials of new treatments in multiple sclerosis (MS) currently require large sample sizes and long durations in order to yield reliable results. The differential responses of an already heterogeneous population of MS patients to individual disease-modifying therapies (DMTs) will further complicate future trials. MS trials with smaller samples and faster outcomes are conceivable through the substitution of current clinical and MRI outcomes with objectively measureable genomic and proteomic biomarkers. Currently, biomarkers that could be utilized for diagnosis and monitoring of MS disease activity are in the early validation phase. The power of single biomarkers or multiple correlated biomarkers to predict prognosis and response to treatment could initially be compared with currently accepted methods. These prospectively validated disease biomarkers could then be used to subcategorize the spectrum of MS patients into a finite number of endophenotypes with demonstrable different molecular pathogeneses and DMT response profiles. Newly developed DMT could potentially be assessed within specific endophenotypes and compared with pharmacogenomically relevant active comparator DMT. This approach may increase the efficiency of MS trials through homogenization of patient population and minimization of nonresponders in study groups, providing the potential for the development of targeted therapies.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | ENVIRONMENTAL RISK-FACTORS; DOUBLE-BLIND; BIOMARKERS; SUSCEPTIBILITY; MULTICENTER; DIAGNOSIS; UMBRELLA; PROGRESS; SEARCH; SCREEN; Multiple Sclerosis; Endophenotypes; Clinical trial; Trial design; Disease Modifying therapy; Pharmacology |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Lehrstuhl für Neurologie |
| Depositing User: | Petra Gürster |
| Date Deposited: | 30 Mar 2020 10:28 |
| Last Modified: | 30 Mar 2020 10:28 |
| URI: | https://pred.uni-regensburg.de/id/eprint/27038 |
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