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3 Experts Resign from FDA Panel Over Alzheimer's Drug Approval

Three experts have resigned from an FDA advisory committee following the controversial approval of Aduhelm, an Alzheimer's drug, against the panel's recommendations. The resignations highlight concerns over the drug's efficacy, approval process, and potential impact on future drug evaluations.

Phase I/IIa Clinical Trial of RH5.1/AS01B Vaccine for Blood-Stage Malaria Shows Promising Safety and Immunogenicity

A first-in-human, open-label, non-randomized, multi-center, dose escalation Phase I/IIa clinical trial evaluated the safety, immunogenicity, and efficacy of the RH5.1/AS01B vaccine against blood-stage malaria. The study involved healthy, malaria-naive adults aged 18 to 45, demonstrating the vaccine's potential to reduce parasite multiplication rate and its safety profile across different doses.

A Challenge Study to Assess the Safety, Immunogenicity and Efficacy of a Malaria Vaccine Candidate

NCT02927145CompletedPhase 1
University of Oxford
Posted 10/17/2016

Laparoscopic Radical Hysterectomy vs. Open Surgery for Early-Stage Cervical Cancer

A recent study evaluates the oncologic outcomes of laparoscopic radical hysterectomy (LRH) compared to open surgery in early-stage cervical cancer patients with lesions less than 2 cm. The findings suggest that LRH may be associated with a higher risk of recurrence, despite offering benefits such as less intraoperative blood loss and faster recovery. The study highlights the need for further research to determine the most effective surgical approach for these patients.

FDA Approves Aduhelm: First Disease-Modifying Alzheimer's Treatment in Nearly Two Decades

Drug ApprovalMonoclonal AntibodyNeurology
  • The FDA granted accelerated approval to Biogen's aducanumab (Aduhelm) as the first disease-modifying Alzheimer's treatment in nearly two decades, despite divided expert opinions on its clinical efficacy.
  • Aduhelm, priced at $56,000 annually, works by reducing amyloid plaques in the brain and will require monthly intravenous infusions with comprehensive clinical and MRI monitoring for potential adverse effects.
  • As part of the accelerated approval, Biogen must conduct a Phase 4 confirmatory trial, while experts view this approval as a potential catalyst for developing more effective Alzheimer's treatments targeting multiple pathways.

Multiple Dose Study of Aducanumab (BIIB037) (Recombinant, Fully Human Anti-Aβ IgG1 mAb) in Participants With Prodromal or Mild Alzheimer's Disease

NCT01677572TerminatedPhase 1
Biogen
Posted 10/5/2012

221AD302 Phase 3 Study of Aducanumab (BIIB037) in Early Alzheimer's Disease

NCT02484547TerminatedPhase 3
Biogen
Posted 9/15/2015

A Study to Evaluate Safety and Tolerability of Aducanumab in Participants With Alzheimer's Disease Who Had Previously Participated in the Aducanumab Studies 221AD103, 221AD301, 221AD302 and 221AD205

NCT04241068TerminatedPhase 3
Biogen
Posted 3/2/2020

221AD301 Phase 3 Study of Aducanumab (BIIB037) in Early Alzheimer's Disease

NCT02477800TerminatedPhase 3
Biogen
Posted 8/13/2015

A Study of Aducanumab in Participants With Mild Cognitive Impairment Due to Alzheimer's Disease or With Mild Alzheimer's Disease Dementia to Evaluate the Safety of Continued Dosing in Participants With Asymptomatic Amyloid-Related Imaging Abnormalities

NCT03639987TerminatedPhase 2
Biogen
Posted 12/20/2018

FDA-Approved and Emerging Biomarkers Transform Immune Checkpoint Inhibitor Patient Selection

Precision Medicine
  • Three FDA-approved biomarkers—PD-L1, microsatellite instability/deficient mismatch repair (MSI/dMMR), and tumor mutational burden (TMB)—currently guide immune checkpoint inhibitor treatment decisions, though each has distinct limitations and variable predictive accuracy across cancer types.
  • Emerging gene signature biomarkers, including T cell-inflamed gene expression profiles and tumor immune dysfunction signatures, demonstrate superior predictive performance compared to single biomarkers and may better identify responders among immunologically cold tumors.
  • Combinational biomarker approaches, such as pairing gene expression profiles with TMB, show enhanced predictive value and could address the complex, multifactorial nature of immune checkpoint inhibitor response.
  • An integrated nucleic acid biomarker signature incorporating DNA and RNA markers from tumor microenvironment, neoantigenicity, and multipotency factors represents the future direction for more comprehensive and accurate patient selection.

Sotorasib Demonstrates Efficacy in KRAS G12C-Mutated Lung Cancer

  • Sotorasib (Lumakras) receives FDA approval as a targeted therapy for non-small cell lung cancer (NSCLC) patients with the KRAS G12C mutation after prior treatment.
  • A global phase 2 clinical trial showed sotorasib reduced tumor size in 82% of patients with KRAS G12C-mutated NSCLC, with 34% exhibiting a partial response.
  • The treatment led to an average progression-free survival of almost seven months and an overall survival of 12.5 months in patients with this specific mutation.
  • Researchers are now investigating sotorasib in combination with other experimental drugs to further improve responses and survival rates in these patients.

NGS-MRD Monitoring Predicts Relapse Risk in AML Patients Post-Transplant

  • NGS-MRD assay demonstrates prognostic value in AML patients undergoing allo-HSCT, both before and one month after transplantation, irrespective of mutation type.
  • Persistent mutations detected by NGS-MRD pre- and post-HSCT are significantly associated with increased post-transplant relapse and worse overall survival.
  • The optimal timing for NGS-MRD assessment varies with conditioning intensity: pre-HSCT for myeloablative conditioning (MAC) and post-HSCT for reduced-intensity conditioning (RIC).
  • Serial NGS-MRD monitoring post-transplantation offers a feasible approach to refine risk stratification and guide MRD-driven therapeutic decisions in AML.

First-in-Human Study of PF-06647020 (Cofetuzumab) in Patients with Advanced Solid Tumors

A phase I study evaluated the safety, tolerability, and pharmacokinetics of PF-06647020 in patients with advanced solid tumors. The study involved dose escalation and expansion to determine the maximum tolerated dose (MTD) and recommended phase II dose (RP2D). PF-06647020 was administered intravenously every 3 weeks or every 2 weeks, with the RP2D established at 2.8 mg/kg every 3 weeks. The study also assessed the drug's antitumor activity and immunogenicity, with a focus on patients with advanced ovarian cancer, NSCLC, and TNBC.

A Study Of PF-06647020 For Adult Patients With Advanced Solid Tumors

NCT02222922CompletedPhase 1
Pfizer
Posted 10/17/2014

Melanocortin Antagonists Emerge as Promising Treatment for Cancer Cachexia

  • Cancer cachexia, a devastating wasting syndrome affecting up to 40% of cancer patients, is gaining recognition as a critical therapeutic target beyond traditional tumor-focused treatments.
  • Novel melanocortin-4 receptor antagonist TCMCB07 demonstrates promising results in animal studies, successfully crossing the blood-brain barrier and preserving body mass without significant adverse effects.
  • Research indicates targeting the central melanocortin system could revolutionize cancer treatment approaches, with potential applications extending to other chronic conditions like kidney disease and COPD.

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