Clinical Trial News

The Reagan Udall Foundation hosted a meeting on advancing PTSD treatments, highlighting the unmet need and discussing current clinical trials, including those for MDMA-assisted therapy. Participants emphasized the importance of representative clinical trials and the impact of trauma on lives, with some criticizing the FDA's handling of MDMA trials. Despite challenges, there is optimism for future advancements in PTSD treatment.

A study comparing traditional psychiatric care and a collaborative care model for depression and anxiety found both models significantly improved symptoms, with stronger results in collaborative care. Published in the Journal of Clinical Psychiatry, the study tracked 1,607 depression and 570 anxiety patients in collaborative care, and 8,773 depression and 2,365 anxiety patients in specialty psychiatry over 6 months. Collaborative care patients saw greater symptom reduction, with 9-point and 5.4-point decreases for depression and anxiety, respectively, compared to 5-point and 2.8-point decreases in specialty care. The collaborative care model, which includes regular symptom monitoring and personalized treatment, showed higher remission rates and addressed the clinician shortage.

NeurologyLive's Friday 5 highlights: 1) Sharon Cohen on mivelsiran for early-stage Alzheimer disease; 2) Aliza Alter on BHV-7000 for focal epilepsy; 3) Jim Cassidy on mirdametinib for neurofibromatosis type 1; 4) Crystal Proud on SMA research and treatments; 5) Michael Levy on ravulizumab for NMOSD.

CGTLive®’s Weekly Rewind highlights 5 gene and cell therapy advances: therapy for HLA-DRB1*15:01 positive progressive MS patients, newborn screening enhancements, GT201 IND clearance in China, 85% reduction in HS D2S6 levels in CSF, and data from the DEVOTE trial.

Cartesian Therapeutics' Descartes-08, an autologous BCMA-directed mRNA CAR-T therapy, showed deep remission in MG patients up to 12 months post-treatment at the ASGCT 27th Annual Meeting. The therapy, administered without lymphodepletion chemotherapy in an outpatient setting, demonstrated a strong safety profile with no cytokine release syndrome or neurotoxicity. Challenges included integrating different medical teams and COVID-19 affecting enrollment. Future plans include trials for systemic lupus and other autoimmune diseases, with an ongoing double-blind placebo-controlled trial in MG.

One year of adjuvant trastuzumab improved disease-free survival (DFS) over 9 weeks in ERBB2-positive breast cancer patients, with no significant difference in overall survival (OS). The 9-week regimen may be an option for patients unable to tolerate or afford 1-year treatment. DFS was shorter in the 9-week group (HR 1.36; 90% CI, 1.14-1.62; P = .004), while OS rates were similar at 5 and 10 years. Factors like docetaxel dose, positive axillary nodes, and disease stage affected DFS, but not OS. Four cardiac deaths occurred, three in the 9-week group. The study included 2176 women, randomized to 9 weeks or 1 year of trastuzumab, with chemotherapy cycles. The 9-week regimen offers benefits like reduced cardiac monitoring, fewer visits, and lower cost, but has limitations such as short follow-up and limited generalizability.

A study led by UCL researchers found that vutrisiran, a gene silencer drug, reduced hospitalisation and death risk by 28% over 3.5 years in ATTR cardiac amyloidosis patients, with a 33% reduction in non-tafamidis users. Vutrisiran, one of four promising treatments, binds to mRNA to reduce TTR protein production, potentially becoming the new standard of care.

D-VRd followed by ASCT, D-VRd consolidation, and DR maintenance improved MRD negativity rates in transplant-eligible myeloma patients compared to VRd induction, ASCT, VRd consolidation, and lenalidomide maintenance, according to PERSEUS trial data. D-VRd showed higher MRD-negativity rates at 10^-5 and 10^-6 thresholds, sustained MRD negativity, and improved PFS, suggesting it as a new standard of care.

Moshe Ornstein discusses IO/TKI doublet regimens in non-ccRCC, highlighting KEYNOTE-B61 trial (ORR 51%, median PFS 17.9 months) and cabozantinib/nivolumab trial (ORR 48%, median PFS 13 months). Both regimens supported by NCCN guidelines, emphasizing IO/TKI as standard care for non-ccRCC patients.

Sandra Christensen discusses the challenges of accessing obesity medications, noting insurance barriers and the need for comprehensive obesity management, including lifestyle changes and anticipatory guidance for side effects. She predicts increased insurance coverage due to clinical trial data and future FDA approvals of new medications.