Clinical trials in multiple sclerosis: potential future trial designs
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 comp...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
SAGE Publishing
2019-05-01
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| Series: | Therapeutic Advances in Neurological Disorders |
| Online Access: | https://doi.org/10.1177/1756286419847095 |
| _version_ | 1811203641501024256 |
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| author | Navid Manouchehri Yinan Zhang Amber Salter Rehana Z. Hussain Hans-Peter Hartung Bernhard Hemmer Ralf Linker Benjamin M. Segal Gary Cutter Olaf Stüve |
| author_facet | Navid Manouchehri Yinan Zhang Amber Salter Rehana Z. Hussain Hans-Peter Hartung Bernhard Hemmer Ralf Linker Benjamin M. Segal Gary Cutter Olaf Stüve |
| author_sort | Navid Manouchehri |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-04-12T02:58:39Z |
| format | Article |
| id | doaj.art-7d8a7e1b2ee545328d5244d0d77e1428 |
| institution | Directory Open Access Journal |
| issn | 1756-2864 |
| language | English |
| last_indexed | 2024-04-12T02:58:39Z |
| publishDate | 2019-05-01 |
| publisher | SAGE Publishing |
| record_format | Article |
| series | Therapeutic Advances in Neurological Disorders |
| spelling | doaj.art-7d8a7e1b2ee545328d5244d0d77e14282022-12-22T03:50:43ZengSAGE PublishingTherapeutic Advances in Neurological Disorders1756-28642019-05-011210.1177/1756286419847095Clinical trials in multiple sclerosis: potential future trial designsNavid ManouchehriYinan ZhangAmber SalterRehana Z. HussainHans-Peter HartungBernhard HemmerRalf LinkerBenjamin M. SegalGary CutterOlaf StüveClinical 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.https://doi.org/10.1177/1756286419847095 |
| spellingShingle | Navid Manouchehri Yinan Zhang Amber Salter Rehana Z. Hussain Hans-Peter Hartung Bernhard Hemmer Ralf Linker Benjamin M. Segal Gary Cutter Olaf Stüve Clinical trials in multiple sclerosis: potential future trial designs Therapeutic Advances in Neurological Disorders |
| title | Clinical trials in multiple sclerosis: potential future trial designs |
| title_full | Clinical trials in multiple sclerosis: potential future trial designs |
| title_fullStr | Clinical trials in multiple sclerosis: potential future trial designs |
| title_full_unstemmed | Clinical trials in multiple sclerosis: potential future trial designs |
| title_short | Clinical trials in multiple sclerosis: potential future trial designs |
| title_sort | clinical trials in multiple sclerosis potential future trial designs |
| url | https://doi.org/10.1177/1756286419847095 |
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