Clinical Trial News

A $3 million NIH-funded study led by Marzieh Salehi, MD, MS, at UT Health San Antonio investigates the antidiabetic and anti-obesity effects of semaglutide in people with spinal cord injury, aiming to impact treatment guidelines and understand underlying diabetes mechanisms in this population.

Emapalumab improves stem cell transplant outcomes for children with hemophagocytic lymphohistiocytosis (HLH), reducing mixed chimerism and enhancing intervention-free survival, according to a study published in the journal Blood.

Clinical trial finds adding anti-clotting drugs to stroke care ineffective; medications did not improve patients’ outcomes, as indicated in The New England Journal of Medicine.

Axsome Therapeutics announces FDA acceptance of NDA resubmission for AXS-07, a novel oral medicine for acute migraine treatment, with a PDUFA goal date of January 31, 2025.

A Big Ten Cancer Research Consortium study on plinabulin combined with nivolumab and ipilimumab for recurrent small cell lung cancer was published in Lung Cancer Journal. The study, BTCRC-LUN17-127, found the combination tolerable at 30 mg/m², though median progression-free survival was 1.6 months, below the target of 3.5 months. Some patients showed prolonged response.

REGENXBIO's RGX-121 shows sustained efficacy in reducing heparan sulfate levels in CSF, potentially allowing patients to remain off ERT, and is well-tolerated. The company plans to submit a BLA for accelerated approval using CSF D2S6 as a surrogate end point.

A clinical trial at Marshall University and Marshall Health Network evaluates the safety and efficacy of oral lofexidine, a non-opioid medication, for newborns with neonatal opioid withdrawal syndrome (NOWS) due to prenatal opioid exposure. The study, sponsored by USWM, LLC through a grant from NIDA, aims to provide a non-opioid treatment option and is ongoing with enrollment targeted to be completed by the end of 2024.

A Phase Ib/II Randomized Clinical Trial of Oleclumab with or without Durvalumab plus Chemotherapy in Patients with Metastatic Pancreatic Ductal Adenocarcinoma.

Developed an AI model to predict long-course sepsis patients for clinical trial enrollment, enhancing patient homogeneity. Utilized conformal prediction to assess model output uncertainty, aiding informed decision-making. Model performance validated internally and externally, with a web-based calculator aiding clinician understanding and feedback, potentially improving clinical decision-making.

In August 2024, the FDA approved Adaptimmune Therapeutics’ afami-cel for synovial sarcoma, modified REMS for CAR-T therapies, lifted a hold on 4D Molecular Therapeutics’ Fabry disease gene therapy, provided guidance for IN8bio’s INB-100 in AML, and cleared Biosyngen’s BRG01 for a pivotal trial in EBV-positive nasopharyngeal carcinoma.