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Clinical Trial News

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.

Small Clinical Trial Shows Drug Cocktail Reverses Biological Age by 2.5 Years

  • A small clinical trial of nine men aged 51-65 demonstrated that a combination of growth hormone and two diabetes medications reversed biological age by an average of 2.5 years as measured by epigenetic clocks.
  • The study, led by Gregory Fahy at Intervene Immune and analyzed by UCLA geneticist Steve Horvath, originally aimed to restore thymus gland tissue but unexpectedly showed broader anti-aging effects.
  • Participants showed rejuvenated immune systems with improved blood cell counts and thymus tissue regeneration, though researchers emphasize the preliminary nature of results from this small, uncontrolled study.
  • The biological age reversal effects accelerated after nine months of treatment and remained largely intact six months after the trial ended, suggesting potential durability of the intervention.

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.

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.

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.

Four-Drug Polypill Reduces Heart Attack and Stroke Risk by 34% in Landmark Trial

  • A daily polypill containing aspirin, atorvastatin, and two blood pressure medications reduced major cardiovascular events by 34% compared to lifestyle advice alone in a 5-year study of 6,841 participants.
  • The trial demonstrated that 35 people would need treatment to prevent one serious cardiovascular event, with the number dropping to 21 in patients with high adherence rates.
  • The low-cost combination pill showed particular effectiveness in preventing first cardiovascular events, reducing risk by approximately 40% in individuals without prior heart disease history.
  • Researchers suggest the polypill strategy could be especially valuable in developing countries where healthcare resources are limited and cardiovascular disease burden is high.

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.

Phase 3 Study of Pexidartinib for Pigmented Villonodular Synovitis (PVNS) or Giant Cell Tumor of the Tendon Sheath (GCT-TS)

NCT02371369CompletedPhase 3
Daiichi Sankyo
Posted 5/11/2015

Study to Evaluate Discontinuation and Re-Treatment in Participants With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib

NCT04526704CompletedPhase 4
Daiichi Sankyo
Posted 10/20/2020

A Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With TGCT

NCT04488822Active, Not RecruitingPhase 3
Daiichi Sankyo Co., Ltd.
Posted 9/25/2020

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.

Glembatumumab Vedotin in Treating Patients With Recurrent or Refractory Osteosarcoma

NCT02487979CompletedPhase 2
National Cancer Institute (NCI)
Posted 2/16/2016

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