Clinical Trial News
No Clinically Relevant Differences in QOL and Neurocognitive Function with Lomustine/Temozolomide for Glioblastoma
A phase III trial found no significant differences in health-related quality of life or neurocognitive function between patients with newly diagnosed MGMT-methylated glioblastoma treated with lomustine/temozolomide versus temozolomide alone, despite a significant improvement in overall survival with the combination therapy.
MRD Negativity as a Surrogate Endpoint for PFS in Newly Diagnosed Multiple Myeloma
• Meta-analysis of six randomized trials with 3,283 NDMM patients supports MRD negativity as a surrogate endpoint for progression-free survival (PFS).
• Treatment effect on MRD negativity strongly correlates with the treatment effect on PFS, indicating its potential to predict clinical benefit.
• The study suggests MRD status can be used in clinical trials for NDMM, potentially accelerating drug development and approval processes.
• These findings align with FDA workshop guidelines, supporting MRD negativity as a Level 3 surrogate endpoint for PFS in NDMM.
Highlighted Clinical Trials:
Millennium Pharmaceuticals, Inc.
Posted 10/1/2007
Focused Ultrasound Successfully Opens Blood-Brain Barrier in ALS Patients: Groundbreaking Trial Results
• Sunnybrook researchers have demonstrated the safety of using focused ultrasound to temporarily open the blood-brain barrier in ALS patients, marking a world-first clinical achievement published in Nature Communications.
• The groundbreaking technique successfully targeted the motor cortex—a brain region critically affected by ALS—potentially creating a non-invasive pathway for delivering therapeutics directly to affected neural tissues.
• Following these promising Phase I results, researchers are preparing to advance to Phase II trials that will involve the delivery of actual ALS therapeutics through the temporarily opened blood-brain barrier.
Clinical Trial Retention Crisis: 30% Dropout Rate Costs Industry Billions and Delays Drug Development
• Global clinical trials face a critical challenge with 30% average dropout rates, while less than 5% of eligible participants initially enroll, significantly impacting drug development timelines and costs.
• Clinical trial complexity has increased over the past decade, with more frequent visits and data collection requirements, while rare disease focus has narrowed the eligible patient pool.
• Removing logistical barriers and providing comprehensive patient support services could significantly improve retention rates, according to Greenphire CEO Jim Murphy.
Doxycycline Prophylaxis Shows Promise in Preventing Bacterial STIs Among High-Risk Populations
• Doxycycline prophylaxis demonstrates potential in preventing bacterial sexually transmitted infections (STIs), particularly among gay, bisexual, and other men who have sex with men.
• Studies indicate that doxycycline prophylaxis can significantly reduce the incidence of syphilis and chlamydia, but further research is needed to confirm efficacy.
• Ongoing studies are exploring optimal doxycycline dosage, regimen, and formulation, as well as long-term safety and the potential for antimicrobial resistance.
• Experts emphasize the need for more robust data on community acceptability, behavioral risk compensation, and cost-effectiveness before broad recommendations can be made.
Minimal Residual Disease Status Validated as Surrogate Endpoint for Progression-Free Survival in Newly Diagnosed Multiple Myeloma
• Meta-analysis of six randomized studies confirms minimal residual disease (MRD) status as a surrogate endpoint for progression-free survival (PFS) in newly diagnosed multiple myeloma (NDMM).
• The treatment effect on MRD-negative response strongly correlated with the hazard ratio for PFS, demonstrating a high coefficient of determination of 0.97.
• Findings support the use of MRD status to accelerate drug development by providing a faster prediction of survival outcomes in NDMM patients.
• The study included 3283 patients and 2208 MRD samples, reinforcing the robustness of MRD as a surrogate marker for PFS.
RNA-Targeted Drug Discovery Summit to Explore Breakthrough Small Molecule Therapeutics
• Leading pharmaceutical companies and academic institutions unite at the 2nd RNA-Targeted Drug Discovery Summit to advance novel RNA-targeted small molecule therapeutics development.
• Industry pioneers from Pfizer, Novartis, Bristol-Myers Squibb, and other major players will address critical challenges in RNA targeting, including binding specificity and drug-like properties.
• The summit aims to transform previously "undruggable" RNA targets into viable therapeutic opportunities through innovative approaches and RNA-protein interaction studies.
Eptinezumab Shows Promise in Preventing Chronic Migraine
A phase 2b clinical trial demonstrates that eptinezumab, an intravenous anti-calcitonin gene-related peptide monoclonal antibody, is effective and well-tolerated for the preventive treatment of chronic migraine, paving the way for phase 3 trials.
FDA Approves Pexidartinib as First Treatment for Tenosynovial Giant Cell Tumour
• Daiichi Sankyo's pexidartinib (Turalio) becomes the first FDA-approved therapy for tenosynovial giant cell tumour (TGCT), offering a new treatment option beyond surgery.
• Phase III ENLIVEN trial demonstrated a 38% overall response rate with pexidartinib, including 15% complete responses and 23% partial responses, compared to zero response in placebo group.
• The approval includes a boxed warning for potential liver toxicity, requiring careful monitoring through a Risk Evaluation and Mitigation Strategy (REMS) program.
Glembatumumab Vedotin Fails to Meet Efficacy Endpoint in Recurrent Osteosarcoma Phase II Trial
• Glembatumumab vedotin (GV) was evaluated in a Phase II trial for patients aged 12-50 with relapsed or refractory osteosarcoma.
• The primary endpoint of disease control at 4 months was not met, with only one partial response and two cases of stable disease observed.
• The antibody-drug conjugate was generally well-tolerated, with rash being the most common grade III adverse event, and toxicities similar to previous studies.
• No correlation was found between glycoprotein non-metastatic B (gpNMB) expression and response to glembatumumab vedotin in this patient population.
Highlighted Clinical Trials:
National Cancer Institute (NCI)
Posted 2/16/2016