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        <title>Experimental Biology and Medicine | Clinical Trials section | New and Recent Articles</title>
        <link>https://www.ebm-journal.org/journals/experimental-biology-and-medicine/sections/clinical-trials</link>
        <description>RSS Feed for Clinical Trials section in the Experimental Biology and Medicine journal | New and Recent Articles</description>
        <language>en-us</language>
        <generator>Frontiers Feed Generator,version:1</generator>
        <pubDate>2026-04-10T08:20:01.203+00:00</pubDate>
        <ttl>60</ttl>
        <item>
        <guid isPermaLink="true">https://www.ebm-journal.org/articles/10.3389/ebm.2025.10806</guid>
        <link>https://www.ebm-journal.org/articles/10.3389/ebm.2025.10806</link>
        <title><![CDATA[Clinical data comparison for FDA-approved gene therapies in sickle cell disease]]></title>
        <pubdate>2025-12-08T00:00:00Z</pubdate>
        <category>Original Research</category>
        <author>Alexis Leonard</author><author>Julie Kanter</author>
        <description><![CDATA[Sickle cell disease (SCD) is a severe inherited hemoglobinopathy with limited curative treatment options. In December 2023, the U.S. FDA approved two autologous gene therapies, lovo-cel (bluebird bio) and exa-cel (Vertex/CRISPR Therapeutics), offering potentially transformative outcomes. We performed a comparative analysis of these therapies based on published clinical trial design, patient eligibility, manufacturing requirements, and reported efficacy and safety outcomes. Overall, participants treated with lovo-cel had more severe baseline disease, reflected by a higher median rate of vaso-occlusive events (VOEs), despite the use of a more stringent VOE definition. Mobilization of hematopoietic stem cells (HSCs) with single-agent plerixafor proved challenging in both trials, with most participants requiring multiple mobilization and apheresis cycles. A greater proportion of exa-cel participants required three or more apheresis procedures, driven by higher CD34+ cell dose targets needed to compensate for CRISPR-associated HSC loss. Both therapies demonstrated greater than 90% resolution of severe VOEs, with near-complete resolution in pediatric participants. A small subset of participants experienced VOEs post-treatment, including events occurring beyond the primary efficacy assessment period. Notably, no recurrent strokes were reported among lovo-cel treated participants with a history of overt stroke. Both therapies provide durable, clinically meaningful benefit and represent a major advancement in SCD management. However, differences in trial populations, cell collection logistics, and manufacturing have important implications for real-world applications. Continued long-term follow-up and the establishment of standardized post-treatment registries will be critical to fully assess durability, monitor late effects, and inform patient selection.]]></description>
      </item><item>
        <guid isPermaLink="true">https://www.ebm-journal.org/articles/10.3389/ebm.2025.10624</guid>
        <link>https://www.ebm-journal.org/articles/10.3389/ebm.2025.10624</link>
        <title><![CDATA[Dependence of mitochondrial dysfunction in peripheral blood mononuclear cells on cervicocephalic atherosclerotic burden in acute ischemic stroke]]></title>
        <pubdate>2025-07-01T00:00:00Z</pubdate>
        <category>Original Research</category>
        <author>Xiaoxi Zhao</author><author>Yi Yang</author><author>Xiangying Du</author><author>Luguang Li</author><author>Chengbei Hou</author><author>Yanning Cai</author><author>Xin Ma</author>
        <description><![CDATA[As an inflammatory disease, atherosclerosis is associated with acute ischemic stroke (AIS), but its early identification and intervention efficacy remain suboptimal. A new research direction may be to explore peripheral atherosclerotic biomarkers from the perspective of mitochondrial dysfunction, which can induce inflammatory cell activation. Moreover, the degree of overall cervicocephalic atherosclerosis (namely, atherosclerotic burden) is more closely related to AIS prognosis than local atherosclerotic lesions. Therefore, this study investigated the relationship between mitochondrial dysfunction in peripheral blood mononuclear cells (PBMCs), including monocytes and lymphocytes, and overall cervicocephalic atherosclerotic burden and AIS outcome. Patients with AIS and cervicocephalic atherosclerosis were enrolled and followed up for 90 days. The reactive oxygen species (ROS) and the mitochondrial deoxyribonucleic acid copy number (mtDNA-CN) in PBMCs were measured respectively through a fluorescence probe and a droplet digital polymerase chain reaction to evaluate mitochondrial function. The overall intracranial and cervical atherosclerotic burden (ICAB) was quantified by summing up the atherosclerosis degree points in each arterial segment as assessed by computed tomography angiography. A modified Rankin Scale (mRS) score >2 was considered a 90-day unfavorable functional outcome. Five (4.9%) of the 103 patients with AIS were lost to follow-up. mtDNA-CN [adjusted β = −0.099, 95% confidence intervals (CIs) = −0.153 ∼ −0.044, p < 0.001] and ROS content (adjusted β = 1.275, 95%CI = 0.885 ∼ 1.665, p < 0.001) were correlated with ICAB. The risk of a 90-day unfavorable functional outcome increased with higher ROS content [adjusted odds ratio (OR) = 1.523, 95%CI = 1.172 ∼ 1.981, p = 0.002] and decreased with higher mtDNA-CN (adjusted OR = 0.911, 95%CI = 0.850 ∼ 0.976, p = 0.008). PBMC mitochondrial dysfunction was found to be independently associated with extensive and severe cervicocephalic atherosclerosis and a 90-day unfavorable functional outcome in patients with AIS, which may provide a novel approach to improving the early identification and risk stratification of cervicocephalic atherosclerosis, along with the prediction of the outcome of atherosclerotic AIS.]]></description>
      </item><item>
        <guid isPermaLink="true">https://www.ebm-journal.org/articles/10.3389/ebm.2025.10493</guid>
        <link>https://www.ebm-journal.org/articles/10.3389/ebm.2025.10493</link>
        <title><![CDATA[Pan-immune-inflammation value predicts survival in inflammatory breast cancer patients]]></title>
        <pubdate>2025-05-01T00:00:00Z</pubdate>
        <category>Original Research</category>
        <author>Yingjia Hu</author><author>Jian Li</author><author>Mingyu Wang</author><author>Xinyi Wang</author><author>Jiankang Li</author><author>Hongfei Ji</author><author>Xingjian Niu</author>
        <description><![CDATA[Inflammatory breast cancer (IBC) is a rare and aggressive breast cancer subtype with poor survival. Identifying novel biomarkers is needed to predict survival for this highly progressive form of breast cancer. In this retrospective study, we investigated pan-immune-inflammation value (PIV), a novel immune-inflammation-based biomarker which combined the peripheral blood parameters (lymphocytes, monocytes, neutrophils, and platelets) in a retrospective cohort of 143 IBC patients. Then we explored the difference of PIV levels in IBC and non-IBC cohorts and the relationship between PIV and clinical characteristics in IBC patients. The survival rates of disease-free survival (DFS) and overall survival (OS) in IBC patients were analyzed and univariate and multivariate statistics were used to evaluate the prognostic value. PIV had the most significantly predictive value in IBC patients compared with other peripheral blood parameters. The mean PIV value in IBC patients was significantly higher than non-IBC patients, and the significant difference between the IBC and non-IBC was also observed in subgroups with different clinical stages and pathologic types. Furthermore, PIV performed an extensive systemic immune prognostic factor on both DFS and OS in IBC patients, and PIV was identified an independent prognostic indicator for survival outcome in IBC patients in univariate and multivariate models. Our retrospective study demonstrated the prognostic value of PIV in IBC patients, suggesting the potential application of PIV in IBC treatment outcomes. PIV would also provide some insights into the mechanisms underlying the role of immune and inflammation in IBC development and progression.]]></description>
      </item><item>
        <guid isPermaLink="true">https://www.ebm-journal.org/articles/10.3389/ebm.2025.10447</guid>
        <link>https://www.ebm-journal.org/articles/10.3389/ebm.2025.10447</link>
        <title><![CDATA[Clinical characteristics and prognosis of ALL in children with CDKN2A/B gene deletion]]></title>
        <pubdate>2025-02-13T00:00:00Z</pubdate>
        <category>Original Research</category>
        <author>Yiyu Wang</author><author>Peijing Wu</author><author>Xiaoyan Mao</author><author>Nanjing Jiang</author><author>Yu Huang</author><author>Li Zhang</author><author>Li Liu</author><author>Xin Tian</author>
        <description><![CDATA[This study aimed to explore the correlation between the deletion of the CDKN2A/B gene and the prognosis of pediatric acute lymphoblastic leukemia (ALL) patients. A total of 310 pediatric patients who were diagnosed with acute lymphoblastic leukemia at our hospital from January 2020 to September 2023 were included in this study. Among them, 78 patients with CDKN2A/B deletion were included in the final analysis. Additionally, 78 ALL patients without CDKN2A/B deletion, who were diagnosed during the same period, were randomly selected for comparison. A statistical analysis was conducted to compare the clinical characteristics and prognosis between the CDKN2A/B deletion group and the non-deletion group in ALL patients. The results showed that pediatric ALL patients with CDKN2A/B deletion had higher white blood cell counts and a greater proportion of immature cells in peripheral blood at diagnosis. The age at diagnosis was older in the deletion group, with a greater proportion in the >10-year-old group. CDKN2A/B deletion occurred more frequently in pediatric patients with T-ALL than in pediatric patients with B-ALL. Patients with CDKN2A/B deletion were more likely to have positive BCR-ABL1 expression combined with IKZF1 deletion. The overall survival (OS) rate was 89.7%, and the event-free survival (EFS) rate was 83.3% in the CDKN2A/B deletion group, which was lower than the OS rate of 97.4% and EFS rate of 93.6% in the non-deletion group. These results suggest that CDKN2A/B deletion may be one of the factors affecting poor prognosis. It provides a new perspective for clinical treatment, risk stratification, and prognostic assessment in pediatric ALL patients.]]></description>
      </item><item>
        <guid isPermaLink="true">https://www.ebm-journal.org/articles/10.3389/ebm.2024.10137</guid>
        <link>https://www.ebm-journal.org/articles/10.3389/ebm.2024.10137</link>
        <title><![CDATA[Establishment and validation of a 5-factor diagnostic model for obstructive and non-obstructive azoospermia based on routine clinical parameters]]></title>
        <pubdate>2024-04-09T00:00:00Z</pubdate>
        <category>Original Research</category>
        <author>Xiaoyu Zhu</author><author>Yin Liu</author><author>Ying Huang</author><author>Hongxia Tan</author><author>Meifang He</author><author>Dong Wang</author>
        <description><![CDATA[Azoospermia is a serious leading male-factor cause of infertility in couples of childbearing age. The two main azoospermia types, obstructive (OA) and non-obstructive (NOA) azoospermia, differ in their treatment approaches. Therefore, their clinical diagnosis is extremely important, requiring an accurate, efficient, and easy-to-use diagnostic model. This retrospective observational study included 707 patients with azoospermia treated between 2017 and 2021, 498 with OA, and 209 with NOA. Hematological and seminal plasma parameters, hormone levels, and testicular volume were used in logistic regression analysis to evaluate and compare their diagnostic performance, results showed that the optimal diagnostic model is constructed by five variables including semen volume, semen pH, seminal plasma neutral α-glucosidase activity, follicle-stimulating hormone in the serum, and testicular volume, compared with follicle-stimulating hormone-based and testicular volume-based models. The 5-factor diagnostic model had an accuracy of 90.4%, sensitivity of 96.4%, positive predictive value of 90.6%, negative predictive value of 89.8%, and area under the curve of 0.931, all higher than in the other two models. However, its specificity (76.1%) was slightly lower than in the other models. Meantime, the internal 5-fold cross-validation results indicated that the 5-factor diagnostic model had a good clinical application value. This study established an accurate, efficient, and relatively accessible 5-factor diagnostic model for OA and NOA, providing a reference for clinical decision-making when selecting an appropriate treatment.]]></description>
      </item><item>
        <guid isPermaLink="true">https://www.ebm-journal.org/articles/10.3389/ebm.2024.10035</guid>
        <link>https://www.ebm-journal.org/articles/10.3389/ebm.2024.10035</link>
        <title><![CDATA[Clinical and genetic characteristics of Chinese patients with Shwachman Diamond syndrome: a literature review of Chinese publication]]></title>
        <pubdate>2024-04-08T00:00:00Z</pubdate>
        <category>Original Research</category>
        <author>Lijun Wang</author><author>Youpeng Jin</author><author>Yuan Chen</author><author>Ping Zhao</author><author>Xiaohong Shang</author><author>Haiyan Liu</author><author>Lifeng Sun</author>
        <description><![CDATA[Shwachman Diamond syndrome (SDS) is a rare autosomal recessive genetic disorder and due to its complex and varied clinical manifestations, diagnosis is often delayed. The purpose of this study was to investigate the clinical manifestations and genetic characteristics of SDS in Chinese patients, in order to increase pediatricians’ awareness of SDS and to allow early diagnosis. We conducted a search to identify patients presenting SBDS gene pathogenic variant in two Chinese academic databases. We analyzed and summarized the epidemiology, clinical features, gene pathogenic variants, and key points in the diagnosis and treatment of SDS. We reviewed the clinical data of 39 children with SDS from previously published articles. The interval from the onset of the first symptoms to diagnosis was very long for most of our patients. The age of presentation ranged from 1 day to 10 years (median: 3 months). However, the age of diagnosis was significantly delayed, ranging from 1 month to 14 years (median: 14 months). Hematological abnormalities were the most common presentation, 89.7% (35/39) at the beginning and 94.9% (37/39) at diagnosis of SDS. Diarrhea was the second most common clinical abnormality at the time of diagnosis. 59% (23/39) of patients had a typical history of persistent chronic diarrhea. Furthermore, hepatic enlargement or elevation of transaminase occurred in 15 cases (38.5%). 56.4% patients (22/39) had a short stature, and 17.9% (7/39) patients showed developmental delay. Additionally, twenty patients had compound heterozygous pathogenic variants of c.258 + 2T > C and c.183_ 184TA > CT. Children with SDS in China had high incidence rates of chronic diarrhea, cytopenia, short stature, and liver damage. Furthermore, SBDS c.258 + 2T > C and c.183_ 184TA > CT were the most common pathogenic variants in patients with SDS. The diagnosis of SDS can be delayed if the clinical phenotype is not recognized by the health care provider.]]></description>
      </item><item>
        <guid isPermaLink="true">https://www.ebm-journal.org/articles/10.3389/ebm.2024.10108</guid>
        <link>https://www.ebm-journal.org/articles/10.3389/ebm.2024.10108</link>
        <title><![CDATA[Prognostic implications of cGAS and STING gene expression in acute myeloid leukemia]]></title>
        <pubdate>2024-02-29T00:00:00Z</pubdate>
        <category>Original Research</category>
        <author>Qiuling Chen</author><author>Yan Hong</author><author>WeiFeng Chen</author><author>Feng Lin</author><author>Jiawei Zeng</author><author>Yueting Huang</author><author>Li Zhang</author><author>Jingwei Yao</author><author>Bing Xu</author>
        <description><![CDATA[Acute myeloid leukemia (AML) is one of the most threatening hematological malignances. cGAS-STING pathway plays an important role in tumor immunity and development. However, the prognostic role of cGAS-STING pathway in AML remains unknown. Firstly, The expression of cGAS and STING was analyzed by bioinformatics analysis. Subsequently, Bone marrow samples were collected from 120 AML patients and 15 healthy individuals in an independent cohort. The cGAS and STING expression was significantly elevated in AML patients compared with healthy controls. Patients with high cGAS and STING expression had a higher NRAS/KRAS mutation rate and lower complete remission (CR) rate. High cGAS and STING expression was significantly associated with lower overall survival (OS) and disease-free survival (DFS). Our findings revealed that the expression levels of cGAS and STING in AML are elevated. High expression of cGAS and STING correlated with worse OS and DFS and may be a useful biomarker for inferior prognosis in AML patients.]]></description>
      </item><item>
        <guid isPermaLink="true">https://www.ebm-journal.org/articles/10.3389/ebm.2024.10021</guid>
        <link>https://www.ebm-journal.org/articles/10.3389/ebm.2024.10021</link>
        <title><![CDATA[A phase 1 trial of human telomerase reverse transcriptase (hTERT) vaccination combined with therapeutic strategies to control immune-suppressor mechanisms]]></title>
        <pubdate>2024-01-31T00:00:00Z</pubdate>
        <category>Original Research</category>
        <author>Nahid Zareian</author><author>Oleg Eremin</author><author>Hardev Pandha</author><author>Richard Baird</author><author>Vineet Kwatra</author><author>Gabriel Funingana</author><author>Chandan Verma</author><author>Desmond Choy</author><author>Steven Hargreaves</author><author>Pejvak Moghimi</author><author>Adrian Shepherd</author><author>Dileep N. Lobo</author><author>Jennifer Eremin</author><author>Farzin Farzaneh</author><author>Shahram Kordasti</author><author>James Spicer</author>
        <description><![CDATA[The presence of inhibitory immune cells and difficulty in generating activated effector T cells remain obstacles to development of effective cancer vaccines. We designed a vaccine regimen combining human telomerase reverse transcriptase (hTERT) peptides with concomitant therapies targeting regulatory T cells (Tregs) and cyclooxygenase-2 (COX2)-mediated immunosuppression. This Phase 1 trial combined an hTERT-derived 7-peptide library, selected to ensure presentation by both HLA class-I and class-II in 90% of patients, with oral low-dose cyclophosphamide (to modulate Tregs) and the COX2 inhibitor celecoxib. Adjuvants were Montanide and topical TLR-7 agonist, to optimise antigen presentation. The primary objective was determination of the safety and tolerability of this combination therapy, with anti-cancer activity, immune response and detection of antigen-specific T cells as additional endpoints. Twenty-nine patients with advanced solid tumours were treated. All were multiply-pretreated, and the majority had either colorectal or prostate cancer. The most common adverse events were injection-site reactions, fatigue and nausea. Median progression-free survival was 9 weeks, with no complete or partial responses, but 24% remained progression-free for ≥6 months. Immunophenotyping showed post-vaccination expansion of CD4+ and CD8+ T cells with effector phenotypes. The in vitro re-challenge of T cells with hTERT peptides, TCR sequencing, and TCR similarity index analysis demonstrated the expansion following vaccination of oligoclonal T cells with specificity for hTERT. However, a population of exhausted PD-1+ cytotoxic T cells was also expanded in vaccinated patients. This vaccine combination regimen was safe and associated with antigen-specific immunological responses. Clinical activity could be improved in future by combination with anti-PD1 checkpoint inhibition to address the emergence of an exhausted T cell population.]]></description>
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