Clinical Trial News

TYRA-300 is being developed to improve quality of life for individuals with skeletal dysplasia, including achondroplasia, caused by FGFR3 gene mutations. The drug, informed by FGFR crystal structures, is in Phase 2, with dosing starting Q1 2025.

Persistent Lyme Disease Symptoms (PLDS) are severe, affecting quality of life significantly, as validated by NIH-sponsored trials. Disagreements exist on causes and treatments, but physicians must address PLDS seriously, aiming for cure or symptom relief, acknowledging the significant impact on patients' lives.

High-risk essential thrombocythemia (ET) patients face limited treatment options. Hydroxyurea is first-line but some resist or can't tolerate it. Anagrelide is second-line but may increase disease transformation risk. Ropeginterferon alfa-2b, with a good safety profile, is being compared to anagrelide in the SURPASS-ET trial for ET patients resistant to or intolerant of hydroxyurea.

Study on 114 acute leukemia patients undergoing allo-HSCT found MRD+ status pre-transplant linked to worse survival outcomes. MRD+ group had higher relapse rates and significantly lower one-year OS and PFS. MRD+ is an independent risk factor for OS, suggesting need for better chemo regimens and GvHD management.

CDK 4/6 inhibitors, including abemaciclib, offer new treatments for ER+/HER2- advanced breast cancer. Abemaciclib differs in dosing and side effects but shows similar clinical benefits. Its major impact may be in adjuvant therapy, pending MONARCH-E trial results. Biomarker research could identify tumors most responsive to abemaciclib.

Psychedelics show promise in reducing anxiety and depression in cancer patients, with significant reductions noted in several trials. Benefits may extend 6-12 months post-treatment, offering a new option beyond traditional antidepressants. However, studies are limited by small sizes and methodological issues, including increased blood pressure and heart rate.

Landiolol hydrochloride effectively reduced heart rate in post-operative supraventricular tachycardia patients after pulmonary resection, with 56% converting to normal rhythm. Recurrence was high (68%), but no side effects were observed, confirming its safety and efficacy.

Switching to an advanced hybrid closed loop (AHCL) system significantly improved glycemic control in children and adolescents with type 1 diabetes, increasing time in the 70-140 mg/dL range and reducing average glucose levels, especially at night, compared to previous SAP-LGS/PLGS systems.

Topotecan showed significant activity in SCLC, especially in patients sensitive to prior chemotherapy, with manageable toxicity. Response rates were 6.4% in refractory and 37.8% in sensitive groups. Median survival was 5.4 months overall, 4.7 for refractory, and 6.9 for sensitive patients. Toxicity was mainly hematologic.

The PLEASE study is a double-blind, randomized trial comparing long-term antibiotic treatments (doxycycline or clarithromycin with hydroxychloroquine) to placebo in patients with borreliosis-attributed persistent symptoms. It aims to assess the efficacy of prolonged antibiotic therapy on health-related quality of life, fatigue, neuropsychological outcomes, and cost-effectiveness.