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for="header-search--input">Search</label> <input id="header-search--input" name="q" type="text" class="form-control form-control-sm" placeholder="Search UCSF clinical trials" title="Search clinical trials at UCSF"> <input class="sr-only form-control-sm btn btn-sm btn-secondary" type="submit" value="Search trials"> </form></div></div> </nav></header><main id="content" class="mx-1 mx-md-0"> <div class="container-md"><div id="condition-header"><h1 class="mb-2"> <strong>Paroxysmal Nocturnal Hemoglobinuria</strong> clinical trials at UCSF </h1><p class="h3 mb-5 text-muted" id="condition-header--clinical-trial-counts"> 1 research study open to eligible people </p><div class="row"><p id="condition-header--clinical-trial-portfolio-description" class="trials-list-header--explanatory-paragraph col col-md-9 col-lg-8"> Paroxysmal nocturnal hemoglobinuria is a disease where red blood cells break apart too easily. UCSF is testing a new treatment that uses treosulfan and fludarabine before a blood or bone marrow transplant. These trials are looking for better ways to treat bone marrow failure diseases. </p></div></div><div class="row"> <div class="col col-md-9 col-lg-8 "> <div class="trials-list--filter-container card bg-light text-center mb-3 d-none d-print-none" data-nosnippet><div class="card-body p-1"><span class="trials-list--filter-main-heading d-none d-md-inline"> Showing <span class="trials-list--number-of-trials-shown" aria-live="assertive"></span> trials for </span><div class="btn-group btn-group-sm btn-group-toggle ml-3" role="group" aria-label="Filter trials by sex/gender" data-toggle="buttons"> <label class="btn btn-light mr-1 active"> <input checked type="radio" name="trials-list--filter-sex" data-class-wanted=""> <i class="fa fa-male d-none d-lg-inline" aria-hidden="true"></i><i class="fa fa-female d-none d-lg-inline" aria-hidden="true"></i> All </label> <label class="btn btn-light mr-1"> <input type="radio" name="trials-list--filter-sex" data-class-wanted="trials-list--trial-open-to-sex-female"> <i class="fa fa-female d-none d-lg-inline" aria-hidden="true"></i> Female </label> <label class="btn btn-light mr-1"> <input type="radio" name="trials-list--filter-sex" data-class-wanted="trials-list--trial-open-to-sex-male"> <i class="fa fa-male d-none d-lg-inline" aria-hidden="true"></i> Male </label> </div><div class="btn-group btn-group-sm btn-group-toggle ml-3" role="group" aria-label="Filter trials by age range" data-toggle="buttons"> <label class="btn btn-light active"><input checked type="radio" name="trials-list--filter-age" data-class-wanted="">All ages</label> <label class="btn btn-light"><input type="radio" name="trials-list--filter-age" data-class-wanted="trials-list--trial-open-to-age-under-18">Under 18</label> <label class="btn btn-light"><input type="radio" name="trials-list--filter-age" data-class-wanted="trials-list--trial-open-to-age-18-and-over">Over 18</label> </div></div></div> <ul class="list-unstyled" id="trials-list"><li class="trials-list--trial card border trials-list--trial-joinable trials-list--trial-open-to-age-under-18 trials-list--trial-open-to-age-18-and-over trials-list--trial-open-to-sex-female trials-list--trial-open-to-sex-male "><div class="card-body"><h2 class="h4 card-title"> <a href="https://clinicaltrials.ucsf.edu/trial/NCT04965597" class="stretched-link">Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)</a> </h2><div class="trials-list--trial-details"><p class="h6 "> <i class="fa fa-user d-none d-md-inline fa-small-icon" aria-hidden="true"></i> <i class="fa fa-user d-none d-md-inline" aria-hidden="true"></i> open to eligible people ages 1-49 </p><div class="trials-list--trial-summary"><p>This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with bone marrow failure diseases. Chemotherapy drugs, such as treosulfan, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Fludarabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. rATG is used to decrease the body's immune response and may improve bone marrow function and increase blood cell counts. Adding treosulfan to a conditioning regimen with fludarabine and rATG may result in patients having less severe complications after a blood or bone marrow transplant.</p></div><p itemprop="location" itemscope itemtype="https://schema.org/Place" class="text-right text-muted small"><meta itemprop="name" content="UCSF"> <i class="fa fa-map-marker fa-fw" aria-hidden="true"></i> <span itemprop="address" itemscope itemtype="https://schema.org/PostalAddress"> <strong> <span itemprop="addressLocality">San Francisco</span>, <span itemprop="addressRegion">California</span><meta itemprop="postalCode" content="94143"><meta itemprop="addressCountry" content="United States"> </strong> and other locations </span></p></div></div></li></ul><div itemscope itemtype="https://schema.org/WebPage"><p class="text-muted small comma-list"> Our lead scientists for Paroxysmal Nocturnal Hemoglobinuria research studies include <span class="pi-name comma-list-item" itemprop="author" itemscope itemtype="https://schema.org/Person"><a href="https://profiles.ucsf.edu/kristin.shimano" target="_blank" rel="noopener author" itemprop="url" class="condition--link-to-pi-profile"><span itemprop="name">Kristin Shimano, MD</span></a></span>. </p><p class="text-muted small">Last updated: <time itemprop="dateModified" datetime="2024-06-14">June 2024</time></p></div></div> <div class="col-md-3 col-lg-3 offset-lg-1"><aside class="d-print-none"><h3 class="h5 mt-5" role="complementary">Related Links</h3><ul><li><a href="https://www.ucsfhealth.org/find-a-doctor/" rel="nofollow">UCSF Health doctors</a></li><li><a href="https://www.researchmatch.org/" rel="nofollow">More studies at ResearchMatch</a></li><li><a href="https://www.ucsf.edu/search?search=Paroxysmal%20Nocturnal%20Hemoglobinuria" rel="nofollow">Paroxysmal Nocturnal Hemoglobinuria resources at UCSF</a></li></ul></aside></div> </div> </div> </main><footer class="mt-auto" role="contentinfo" data-nosnippet><div id="ucsf--footer--body"><div class="container" data-nosnippet><div class="row mb-1 d-print-none"><div class="col-12 text-center text-md-left"> We use cookies to operate our website, analyze performance, and improve your experience on our site. <a href="https://www.ucsf.edu/website-privacy-policy" target="_blank">Learn more.</a> </div></div><div class="row"><div class="col-md-6 text-center text-md-left"><p class="my-0"> © The Regents of the University of California </p></div><div class="col-md-6 text-center text-md-right pt-3 pt-md-0"> <a href="https://websites.ucsf.edu/digital-accessibility" target="_blank">Accessibility</a> <a href="https://www.ucsf.edu/website-privacy-policy" target="_blank">Privacy Policy</a> <a href="https://websites.ucsf.edu/website-terms-use" target="_blank">Terms of Use</a> </div></div></div></div></footer><script>(function(){var b=document.createElement('script');b.type='text/javascript';b.async=true;b.src='//siteimproveanalytics.com/js/siteanalyze_8343.js';var a=document.getElementsByTagName('script')[0];a.parentNode.insertBefore(b,a)})();</script></body></html>