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data-nosnippet>Spanish</a> <a class="dropdown-item" href="https://clinicaltrials.ucsf.edu/about/vietnamese" data-nosnippet>Vietnamese</a> </div></li></ul></div> </nav><nav class="breadcrumb-and-search-bar navbar navbar-expand navbar-light d-print d-flex justify-content-between"> <div><ol class="breadcrumb navbar-text my-0 pl-1" itemscope="itemscope" itemtype="https://schema.org/BreadcrumbList"><li itemprop="itemListElement" itemscope itemtype="https://schema.org/ListItem"> <a href="https://clinicaltrials.ucsf.edu/" itemscope="itemscope" itemprop="item" itemtype="https://schema.org/Thing" id="navbar--breadcrumb--link-1"> Home <meta itemprop="name" content="UCSF Clinical Trials"> </a> <meta itemprop="position" content="1"></li><li class="active">Down Syndrome Studies</li></ol></div> <div><div class="d-none d-md-block navbar-text"><form method="GET" action="https://clinicaltrials.ucsf.edu/" class="form-inline my-1" role="search"> <label class="sr-only" 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>Down Syndrome</strong> clinical trials at UCSF </h1><p class="h3 mb-5 text-muted" id="condition-header--clinical-trial-counts"><span data-nosnippet> 7 in progress, 2 open to eligible people </span></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"> Down syndrome happens when someone has an extra chromosome. UCSF is looking at the health of people with Down syndrome after they get treated for acute leukemia. UCSF is also checking bone marrow and blood in children with tough-to-treat leukemia. </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/NCT04726241" class="stretched-link">Pediatric Acute Leukemia (PedAL) Screening Trial - A Study to Test Bone Marrow and Blood in Children With Leukemia That Has Come Back After Treatment or Is Difficult to Treat - A Leukemia & Lymphoma Society and Children's Oncology Group Study</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 up to 22 years </p><div class="trials-list--trial-summary"><p>This study aims to use clinical and biological characteristics of acute leukemias to screen for patient eligibility for available pediatric leukemia sub-trials. Testing bone marrow and blood from patients with leukemia that has come back after treatment or is difficult to treat may provide information about the patient's leukemia that is important when deciding how to best treat it, and may help doctors find better ways to diagnose and treat leukemia in children, adolescents, and young adults.</p></div><p itemprop="location" itemscope itemtype="https://schema.org/Place" class="text-right text-muted small"><meta itemprop="name" content="UCSF Benioff Children's Hospital Oakland"> <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">Oakland</span>, <span itemprop="addressRegion">California</span><meta itemprop="postalCode" content="94609"><meta itemprop="addressCountry" content="United States"> </strong> and other locations </span></p></div></div></li><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/NCT05702645" class="stretched-link">Learn More About the Health of Persons With Down Syndrome After Treatment for Acute Leukemia</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 6-39 </p><div class="trials-list--trial-summary"><p>This study attempts to learn more about the health of persons with Down syndrome after treatment for acute leukemia. Children with Down syndrome are at increased risk for side effects during treatment for acute leukemia, but it is unclear of their risk for long-term effects of cancer treatment. By learning more about the factors that may contribute to chronic health conditions and long-term effects after treatment for leukemia in persons with Down syndrome, clinical practice guidelines for survivorship care can be developed to help improve their quality-of-life.</p></div><p itemprop="location" itemscope itemtype="https://schema.org/Place" class="text-right text-muted small"><meta itemprop="name" content="UCSF Benioff Children's Hospital Oakland"> <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">Oakland</span>, <span itemprop="addressRegion">California</span><meta itemprop="postalCode" content="94609"><meta itemprop="addressCountry" content="United States"> </strong> and other locations </span></p></div></div></li><li class="trials-list--trial card border trials-list--trial-not-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/NCT04546399" class="stretched-link">Blinatumomab Alone to Blinatumomab With Nivolumab in Patients Diagnosed With First Relapse B-Cell Acute Lymphoblastic Leukemia (B-ALL)</a> </h2><div class="trials-list--trial-details"><p class="h6 text-danger"> <i class="fa fa-ban" aria-hidden="true"></i> <span class="sr-only">Sorry, </span> currently not accepting new patients, but might later </p><div class="trials-list--trial-summary"><p>This phase II trial studies the effect of nivolumab in combination with blinatumomab compared to blinatumomab alone in treating patients with B-cell acute lymphoblastic leukemia (B-ALL) that has come back (relapsed). Down syndrome patients with relapsed B-ALL are included in this study. Blinatumomab is an antibody, which is a protein that identifies and targets specific molecules in the body. Blinatumomab searches for and attaches itself to the cancer cell. Once attached, an immune response occurs which may kill the cancer cell. Nivolumab is a medicine that may boost a patient's immune system. Giving nivolumab in combination with blinatumomab may cause the cancer to stop growing for a period of time, and for some patients, it may lessen the symptoms, such as pain, that are caused by the cancer.</p></div><p itemprop="location" itemscope itemtype="https://schema.org/Place" class="text-right text-muted small"><meta itemprop="name" content="UCSF Medical Center-Mission Bay"> <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="94158"><meta itemprop="addressCountry" content="United States"> </strong> and other locations </span></p></div></div></li><li class="trials-list--trial card border trials-list--trial-not-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/NCT03914625" class="stretched-link">Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia</a> </h2><div class="trials-list--trial-details"><p class="h6 text-danger"> <i class="fa fa-ban" aria-hidden="true"></i> <span class="sr-only">Sorry, </span> in progress, not accepting new patients </p><div class="trials-list--trial-summary"><p>This phase III trial studies how well blinatumomab works in combination with chemotherapy in treating patients with newly diagnosed, standard risk B-lymphoblastic leukemia or B-lymphoblastic lymphoma with or without Down syndrome. Monoclonal antibodies, such as blinatumomab, may induce changes in the body's immune system and may interfere with the ability of cancer cells to grow and spread. Chemotherapy drugs, such as vincristine, dexamethasone, prednisone, prednisolone, pegaspargase, methotrexate, cytarabine, mercaptopurine, doxorubicin, cyclophosphamide, and thioguanine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Leucovorin decreases the toxic effects of methotrexate. Giving monoclonal antibody therapy with chemotherapy may kill more cancer cells. Giving blinatumomab and combination chemotherapy may work better than combination chemotherapy alone in treating patients with B-ALL. This trial also assigns patients into different chemotherapy treatment regimens based on risk (the chance of cancer returning after treatment). Treating patients with chemotherapy based on risk may help doctors decide which patients can best benefit from which chemotherapy treatment regimens.</p></div><p itemprop="location" itemscope itemtype="https://schema.org/Place" class="text-right text-muted small"><meta itemprop="name" content="UCSF Benioff Children's Hospital Oakland"> <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">Oakland</span>, <span itemprop="addressRegion">California</span><meta itemprop="postalCode" content="94609"><meta itemprop="addressCountry" content="United States"> </strong> and other locations </span></p></div></div></li><li class="trials-list--trial card border trials-list--trial-not-joinable trials-list--trial-open-to-age-under-18 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/NCT05146739" class="stretched-link">Highest Dose of Uproleselan in Combination With Fludarabine and Cytarabine for Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Mixed Phenotype Acute Leukemia Relapsed or Refractory and That Expresses E-selectin Ligand on the Cell Membrane</a> </h2><div class="trials-list--trial-details"><p class="h6 text-danger"> <i class="fa fa-ban" aria-hidden="true"></i> <span class="sr-only">Sorry, </span> in progress, not accepting new patients </p><div class="trials-list--trial-summary"><p>This phase I trial tests the safety, side effects, and best dose of uproleselan in combination with fludarabine and cytarabine in treating patients with acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that has come back (relapsed) or does not respond to treatment (refractory) and that expresses E-selectin ligand on the cell membrane. Uproleselan binds to E-selectin expressed on endothelial cells of the bone marrow and prevents their interaction with selectin-E ligand-expressing cancer cells. This may prevent leukemia cells from being sequestered in the bone marrow niche and escaping the effect of chemotherapy. Chemotherapy drugs, such as fludarabine and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving uproleselan in combination with fludarabine and cytarabine may enhance their activity.</p></div><p itemprop="location" itemscope itemtype="https://schema.org/Place" class="text-right text-muted small"><meta itemprop="name" content="UCSF Medical Center-Mission Bay"> <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="94158"><meta itemprop="addressCountry" content="United States"> </strong> and other locations </span></p></div></div></li><li class="trials-list--trial card border trials-list--trial-not-joinable trials-list--trial-open-to-age-under-18 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/NCT02521493" class="stretched-link">Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome</a> </h2><div class="trials-list--trial-details"><p class="h6 text-danger"> <i class="fa fa-ban" aria-hidden="true"></i> <span class="sr-only">Sorry, </span> in progress, not accepting new patients </p><div class="trials-list--trial-summary"><p>This phase III trial studies response-based chemotherapy in treating newly diagnosed acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Response-based chemotherapy separates patients into different risk groups and treats them according to how they respond to the first course of treatment (Induction I). Response-based treatment may be effective in treating acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome while reducing the side effects.</p></div><p itemprop="location" itemscope itemtype="https://schema.org/Place" class="text-right text-muted small"><meta itemprop="name" content="UCSF Benioff Children's Hospital Oakland"> <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">Oakland</span>, <span itemprop="addressRegion">California</span><meta itemprop="postalCode" content="94609"><meta itemprop="addressCountry" content="United States"> </strong> and other locations </span></p></div></div></li><li class="trials-list--trial card border trials-list--trial-not-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/NCT01190930" class="stretched-link">Risk-Adapted Chemotherapy in Treating Younger Patients With Newly Diagnosed Standard-Risk Acute Lymphoblastic Leukemia or Localized B-Lineage Lymphoblastic Lymphoma</a> </h2><div class="trials-list--trial-details"><p class="h6 text-danger"> <i class="fa fa-ban" aria-hidden="true"></i> <span class="sr-only">Sorry, </span> in progress, not accepting new patients </p><div class="trials-list--trial-summary"><p>This partially randomized phase III trial studies the side effects of different combinations of risk-adapted chemotherapy regimens and how well they work in treating younger patients with newly diagnosed standard-risk acute lymphoblastic leukemia or B-lineage lymphoblastic lymphoma that is found only in the tissue or organ where it began (localized). Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving more than one drug (combination chemotherapy), giving the drugs in different doses, and giving the drugs in different combinations may kill more cancer cells.</p></div><p itemprop="location" itemscope itemtype="https://schema.org/Place" class="text-right text-muted small"><meta itemprop="name" content="UCSF Benioff Children's Hospital Oakland"> <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">Oakland</span>, <span itemprop="addressRegion">California</span><meta itemprop="postalCode" content="94609"><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 Down Syndrome research studies include <span class="pi-name comma-list-item" itemprop="author" itemscope itemtype="https://schema.org/Person"><a href="https://profiles.ucsf.edu/jennifer.michlitsch" target="_blank" rel="noopener author" itemprop="url" class="condition--link-to-pi-profile"><span itemprop="name">Jennifer G. Michlitsch</span></a></span> <span class="pi-name comma-list-item" itemprop="author" itemscope itemtype="https://schema.org/Person"><a href="https://profiles.ucsf.edu/elliot.stieglitz" target="_blank" rel="noopener author" itemprop="url" class="condition--link-to-pi-profile"><span itemprop="name">Elliot Stieglitz</span></a></span>. </p><p class="text-muted small">Last updated: <time itemprop="dateModified" datetime="2024-11-05">November 5, 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=Down%20Syndrome" rel="nofollow">Down Syndrome 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>