Clinical Trial News

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.

Bill Gates expresses optimism in the M72 tuberculosis vaccine's clinical trial, hoping for 70% efficacy. The trial, costing $500 million and done in collaboration with GSK and Wellcome Trust, aims to be the first new TB vaccine in over a century.

The VALOR study investigates Lacosamide's efficacy in reducing seizures in patients with idiopathic generalized epilepsy, already on anti-epileptic drugs. Enrolling children and adults from age 4, with at least 2 seizures in the past 12 weeks, in 23 countries. Patients have a 50% chance of receiving placebo, with an option for Lacosamide in an open-label extension.

Aficamten, a cardiac myosin inhibitor, improved exercise tolerance in patients with hypertrophic obstructive cardiomyopathy in the SEQUIOA-HCM trial, enhancing peak oxygen uptake and cardiac function, as published in JAMA Cardiology.

Outlook Therapeutics completes enrollment for NORSE EIGHT trial, evaluating ONS-5010 in wet AMD patients. NORSE EIGHT, under an FDA SPA, is the final trial before resubmitting the BLA for ONS-5010. Topline results expected in Q4 CY2024, with BLA resubmission planned for Q1 CY2025.