Clinical Trial News

Genomic advancements have led to precision oncology, focusing on targeted therapies based on genetic mutations. Traditional phase 1-3 trials are evolving to target biomarkers regardless of tumor type. New master protocols, including basket, umbrella, and platform trials, aim to improve efficiency and uniformity. Oncology nurses must stay informed to counsel patients effectively.

DirectRx, a specialty pharmacy, is now part of the exclusive U.S. network distributing Ohtuvayre (ensifentrine), a newly approved COPD therapy. Ohtuvayre, priced at $2,950 per month, is the first new COPD therapy in over two decades, offering both anti-inflammatory and airway-widening effects without the side effects of inhaled corticosteroids. It was approved based on Phase 3 trial data showing significant improvements in lung function and reduced exacerbation rates.

Post-approval changes are categorized into three classes: annual record reporting for minor modifications, CBE-30 supplements for moderate changes, and PAS for significant changes. The FDA introduced a document in July 2024 to clarify biosimilar post-approval modifications, emphasizing thorough studies and comparability protocols. Changes to dosage form or strength require PAS, including comparability and CAA data. Biosimilars can change route of administration, formulation, and introduce new indications without filing as new drugs, supported by the potential passage of the S-150 Bill.

Intravenous glibenclamide (CIRARA) combined with endovascular thrombectomy (EVT) significantly improves independent ambulation in patients with large hemispheric infarction (LHI), according to phase 3 CHARM trial data. Patients with lesion volumes under 125 mL showed an odds ratio of 4.1 for achieving modified Rankin Scale (mRS) scores of 0-3 (P < .01), and an overall OR of 18.5 for better functional outcomes (P = .03). Despite early termination, CIRARA demonstrates potential as a breakthrough treatment in stroke recovery.

Esther Biswas-Fiss, UD professor, received $2.3 million from NIH/NEI to study ABCA4 gene variants, aiming to understand inherited retinal diseases like Stargardt. Her interdisciplinary team uses AI and 3D modeling to classify genetic variants, potentially leading to personalized medicine and clinical trial eligibility.

The RDCA-DAP, funded by the FDA and run by C-Path, has received new CF datasets from the Cystic Fibrosis Therapeutics Development Network, coordinated by Seattle Children’s Research Institute. These datasets aim to accelerate CF research and therapy development, enhancing the RDCA-DAP’s role in combating rare diseases.

The FDA supports using the alpha-synuclein seed amplification assay (synSAA) to improve Parkinson’s and neurodegenerative disease clinical trials. Amprion’s SAAmplify-ɑSYN Biomarker Test, validated in 2023, is the only U.S. synSAA assay, aiding early Parkinson’s diagnosis. The FDA’s endorsement aims to accelerate treatment development and enhance trial efficiency.

Black women with breast cancer experience less neuropathy and fewer chemotherapy dose reductions with docetaxel, according to a study led by Dr. Bryan P. Schneider at Indiana University. This finding could improve outcomes for Black patients, historically underrepresented in breast cancer research.

Tami McGraw, severely allergic to red meat, found relief through Xolair, originally for asthma, which also reduced her food allergies. After years of off-label use, Xolair was approved for food allergies in 2023, offering protection against accidental exposure and transforming lives, though it's not a cure. The drug's approval followed a pivotal clinical trial, OUtMATCH, which showed significant improvement in patients allergic to multiple foods. Xolair's potential to treat multiple allergies simultaneously and enhance oral immunotherapy effectiveness is under study.

The European Commission approved BRAFTOVI® (encorafenib) in combination with MEKTOVI® (binimetinib) for advanced non-small cell lung cancer (NSCLC) with a BRAFV600E mutation, based on the Phase II PHAROS trial results showing a 75% objective response rate (ORR) in treatment-naïve patients and 46% in previously treated patients. The approval addresses limited targeted treatment options for BRAFV600E mutant NSCLC patients.