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Clinical Trial News

Ruxolitinib Cream Prescriptions for Vitiligo Issued in Greater Bay Area, China; NDA Accepted by NMPA

  • China Medical System Holdings (CMS) announces the first prescriptions of ruxolitinib cream for vitiligo in the Greater Bay Area.
  • The prescriptions were issued at designated hospitals following approval via the "Hong Kong and Macau Medicine and Equipment Connect" policy.
  • The National Medical Products Administration of China (NMPA) has accepted the New Drug Application (NDA) for ruxolitinib cream for vitiligo.
  • Real-world studies in China showed positive efficacy and safety, aligning with global pivotal clinical study outcomes for vitiligo treatment.

Cardiol Therapeutics Completes Enrollment in Phase II Acute Myocarditis Trial

  • Cardiol Therapeutics has achieved full enrollment for its Phase II clinical trial investigating a novel therapy for acute myocarditis.
  • The trial assesses the safety and efficacy of CardiolRx, a pharmaceutically produced cannabidiol formulation, in reducing myocardial inflammation.
  • Completion of enrollment marks a significant milestone in the development of a potential new treatment option for this inflammatory heart condition.
  • The study's results could provide critical insights into the therapeutic potential of cannabidiol in cardiovascular diseases.

Optimizing Neoadjuvant Immunotherapy Strategies in Colorectal Cancer

  • Neoadjuvant immunotherapy shows promise in treating dMMR/MSI-H colorectal cancer, leading to high pathological response rates and potential organ preservation.
  • Research explores biomarkers like TMB and PD-L1 to predict immunotherapy response, aiming to personalize treatment and improve outcomes.
  • Combination therapies and novel agents like botensilimab are being investigated to enhance efficacy in both dMMR/MSI-H and pMMR/MSS colorectal cancers.
  • Ongoing trials are evaluating the role of neoadjuvant immunotherapy in locally advanced rectal cancer, with a focus on balancing efficacy and toxicity.

Low-Intensity Therapy Shows Positive Outcomes for Pediatric Leukemia Subtypes

  • St. Jude Children's Research Hospital trials show genomics and early treatment response can guide risk classification in B-cell acute lymphoblastic leukemia (B-ALL).
  • Patients with ETV6::RUNX1 and high-hyperdiploid B-ALL achieved positive outcomes with low-intensity chemotherapy, reducing side effects like thrombosis and pancreatitis.
  • The study highlights the potential for personalized treatment approaches based on individual patient characteristics, improving event-free survival rates.
  • Researchers emphasize the importance of minimizing treatment intensity to reduce long-term health issues in pediatric cancer patients.

Synergia Medical Implants First Patients with Novel Neurostimulator for Epilepsy

  • Synergia Medical has implanted its NAO.VNS device in first-in-human study participants with drug-resistant epilepsy, testing a novel neurostimulation technology.
  • The AURORA trial, conducted across sites in Belgium and Germany, will evaluate the safety and efficacy of Synergia's vagus nerve stimulation therapy.
  • Synergia's device uses optical fibers instead of metal wires, potentially allowing patients to undergo MRI scans, which are typically restricted with conventional devices.
  • The global neuromodulation device market is projected to reach nearly $11 billion by 2033, with vagus nerve stimulation contributing significantly to this growth.

Low-Intensity rTMS Shows Promise in Enhancing Myelin Repair in Multiple Sclerosis Models

  • Low-intensity repetitive transcranial magnetic stimulation (LI-rTMS) boosts myelin repair capacity of oligodendrocytes in lab models of multiple sclerosis.
  • LI-rTMS increases myelin production by both new and mature oligodendrocytes, particularly in the corpus callosum, enhancing brain communication pathways.
  • The treatment improves myelin efficiency by increasing the length and thickness of myelin sections, suggesting potential for restoring nerve function.
  • A Phase 2 clinical trial is underway to assess the effectiveness of LI-rTMS in promoting myelin repair and improving clinical outcomes in MS patients.

Cassava Sciences' Simufilam Maintains Safety Profile in Phase 3 Alzheimer's Trials

  • An independent Data and Safety Monitoring Board (DSMB) has endorsed the continuation of Cassava Sciences' Phase 3 trials for simufilam in Alzheimer's disease.
  • The DSMB's recommendation is based on an interim safety review, with no modifications suggested for the ongoing clinical trials.
  • Top-line results from the first Phase 3 trial, involving 804 patients over 52 weeks, are anticipated by the end of 2024.
  • Simufilam, a small molecule drug, targets filamin A and has shown no association with treatment-emergent ARIA in interim MRI data.

Simufilam 100 mg for Mild-to-Moderate Alzheimer's Disease

NCT04994483CompletedPhase 3
Cassava Sciences, Inc.
Posted 11/3/2021

Simufilam 50 mg or 100 mg for Mild-to-Moderate Alzheimer's Disease

NCT05026177TerminatedPhase 3
Cassava Sciences, Inc.
Posted 11/18/2021

Simufilam (PTI-125), 100 mg, for Mild-to-moderate Alzheimer's Disease Patients

NCT04388254CompletedPhase 2
Cassava Sciences, Inc.
Posted 3/24/2020

Psychedelic Therapies Approach Europe Amidst Regulatory and Access Challenges

• Psychedelic therapies, including psilocybin and MDMA, are gaining traction in Europe for treating mental health conditions like depression and addiction. • Clinical trials are underway across Europe, with Compass Pathways leading the way in testing psilocybin for treatment-resistant depression. • Regulatory hurdles, highlighted by the FDA's rejection of MDMA-assisted therapy for PTSD in the US, pose challenges to European approval processes. • Experts emphasize the need for rigorous clinical trials and strategies to ensure patient access and integration into standard healthcare practices.

Enlivex's Allocetra Receives Green Light for Phase II Knee Osteoarthritis Trial

  • Enlivex Therapeutics has been authorized by the Danish Medicines Agency to advance Allocetra into Phase II trials for moderate to severe knee osteoarthritis.
  • The decision follows positive safety data from the Phase I run-in stage, where Allocetra demonstrated a favorable safety profile in the initial twelve patients.
  • The Phase II trial will assess both the safety and efficacy of Allocetra injections compared to placebo, measuring joint pain and function over 12 months.
  • Allocetra represents a novel, off-the-shelf cell therapy designed to reprogram macrophages, potentially offering a new approach to treating osteoarthritis.

Leronlimab Shows Promise in Reversing Liver Fibrosis in Preclinical MASH Study

• CytoDyn's leronlimab demonstrated statistically significant fibrosis reversal in a preclinical MASH study compared to the control, with a p-value of less than 0.01. • The study suggests a dose-dependent anti-fibrotic activity of leronlimab, with the 700 mg dose showing better results than the 350 mg dose. • Leronlimab monotherapy at 700 mg exhibited better anti-fibrotic activity compared to Resmetirom, the only currently approved therapy for MASH, although the p-value was 0.057.

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