MedPath

Clinical Trial News

Cartesian Therapeutics' Descartes-08 Shows Promise in Myasthenia Gravis Phase 2b Trial

  • Cartesian Therapeutics' Descartes-08 met the primary endpoint in a Phase 2b trial for myasthenia gravis, demonstrating statistically significant improvement in MGC scores.
  • 71% of patients treated with Descartes-08 had clinically meaningful improvement in MGC score at Month 3, compared to 25% for placebo, highlighting its potential as a novel therapy.
  • The mRNA CAR-T therapy showed a favorable safety profile, supporting outpatient administration without the need for lymphodepleting chemotherapy, a significant advantage over traditional CAR-T therapies.
  • Updated Phase 2a results showed durable responses up to one year after retreatment, reinforcing Descartes-08's potential for long-term management of myasthenia gravis.

One-Year Trastuzumab Improves Disease-Free Survival in ERBB2-Positive Breast Cancer

  • A secondary analysis of the SOLD trial indicates that one year of adjuvant trastuzumab improves disease-free survival (DFS) compared to a 9-week regimen in patients with ERBB2-positive breast cancer.
  • The 5-year DFS rate was 90.7% in the 1-year trastuzumab group versus 87.7% in the 9-week group, highlighting a statistically significant benefit for the longer treatment duration.
  • The study suggests the 9-week regimen may be an option for patients who cannot tolerate or afford the one-year treatment, though overall survival rates were similar between both groups.
  • Longer follow-up revealed that factors such as positive axillary nodes, age, and disease stage were independently associated with the risk of death, but not the treatment duration.
NCT00593697CompletedPhase 3
Finnish Breast Cancer Group
Posted 1/1/2008

Vutrisiran Shows Promise in Reducing Hospitalization and Deaths in ATTR Cardiac Amyloidosis

• A new study reveals that vutrisiran, a gene-silencing drug, significantly reduces the risk of death and cardiovascular events in patients with transthyretin (ATTR) cardiac amyloidosis. • The HELIOS-B clinical trial demonstrated a 28% reduction in the risk of death and recurring cardiovascular events over three and a half years with vutrisiran treatment. • Vutrisiran, which blocks the production of the disease-causing TTR protein, improved the quality of life and showed benefits in disease progression markers among treated patients. • The findings suggest vutrisiran could become a new standard of care for ATTR cardiac amyloidosis, offering a significant advancement in treating this progressive and often fatal disease.

Daratumumab-Based Regimens Deepen Responses and Improve MRD Negativity in Multiple Myeloma

  • Daratumumab plus lenalidomide maintenance significantly increased MRD-negative conversion rates compared to lenalidomide alone in transplant-eligible newly diagnosed multiple myeloma patients.
  • The addition of daratumumab to VRd improved MRD negativity rates in transplant-ineligible or deferred newly diagnosed multiple myeloma patients.
  • D-VRd followed by D-R maintenance resulted in higher rates of overall MRD-negativity in revised high-risk multiple myeloma subgroups.
NCT04934475Active, Not RecruitingPhase 3
Intergroupe Francophone du Myelome
Posted 12/8/2021

IO/TKI Doublets Emerge as Standard of Care in Non-Clear Cell Renal Cell Carcinoma

• Phase 2 trials show that IO/TKI combinations, like lenvatinib plus pembrolizumab, demonstrate efficacy in non-clear cell renal cell carcinoma (non-ccRCC) with manageable safety profiles. • KEYNOTE-B61 trial reported a 51% objective response rate (ORR) in non-ccRCC patients treated with lenvatinib and pembrolizumab, supporting NCCN guideline recommendations. • Cabozantinib plus nivolumab also shows promise, with a 48% ORR in a phase 2 trial, establishing IO/TKI doublets as effective first-line options for non-ccRCC. • Treatment decisions should consider patient preference and individual data interpretation, according to Dr. Moshe Ornstein from Cleveland Clinic.

Access and Cost Complicate Obesity Medication Prescriptions

• Access to obesity medications is hindered by cost and insurance coverage, creating disparities in treatment. • Many insurance providers now require a diabetes diagnosis for GLP-1 receptor agonist prescriptions, limiting access for some patients. • Comprehensive obesity management includes lifestyle changes, medication, and bariatric surgery as adjuncts. • Continued use of obesity medications is often necessary for long-term weight management due to the body's natural resistance to weight loss.

Vor Biopharma Announces Positive Data and New CD45 ADC Program, Analysts See Significant Upside

• Vor Biopharma reported updated Phase 1/2 VBP101 study results showing promising relapse-free survival data in AML/MDS patients, exceeding historical benchmarks. • Initial PK data for VCAR33ALLO demonstrated in vivo expansion at the lowest dose, with dose escalation planned, indicating potential efficacy. • The company is developing a new CD45 ADC program (VADC45) for conditioning regimens, offering a fast path to commercial development and partnership opportunities. • Analysts maintain an Outperform rating with a price target of US$11.00, citing significant upside potential from trem-cel and CD45-based depletion approaches.

Denileukin Diftitox Added to NCCN Guidelines for Cutaneous T-Cell Lymphoma

  • Denileukin diftitox-cxdl (Lymphir) has been included in the NCCN Clinical Practice Guidelines in Oncology as a category 2A recommendation for CTCL treatment.
  • The inclusion is based on data from the pivotal phase 3 Study 302, which demonstrated an objective response rate of 36.2% in patients treated with denileukin diftitox.
  • Denileukin diftitox targets the IL-2 receptor and is the only CTCL therapy with this mechanism, offering a novel approach to managing the disease.
  • The NCCN's recognition may broaden access to denileukin diftitox, particularly for patients eligible for Medicare and Medicaid coverage.

Medtronic's Inceptiv Spinal Cord Stimulator Receives FDA Approval for Chronic Pain Relief

  • Medtronic's Inceptiv, a spinal cord stimulator, has gained FDA approval for managing chronic pain by automatically adjusting stimulation based on biological signals.
  • The device is implanted and works by sensing signals and blocking pain signals before they reach the brain, offering a potential alternative to traditional pain management.
  • Dr. Mellisa Murphy and patient Jan Hamilton highlight the device's potential to improve the quality of life for individuals struggling with persistent pain.
  • Inceptiv represents a significant advancement in neuromodulation therapy, providing personalized and responsive pain relief for chronic pain sufferers.

Text Messaging Shows Promise for Lung Cancer Screening Eligibility Assessment, But Equity Gaps Persist

  • A new study reveals 90% of respondents use text messaging regularly, with 83% willing to report smoking status via text for lung cancer screening eligibility assessment.
  • Middle-aged, college-educated, and higher-income individuals showed greater willingness to participate in text-based screening, highlighting potential disparities in reach.
  • Researchers recommend a multimodal approach to ensure equitable identification of lung cancer screening candidates across all sociodemographic groups.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.