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Clinical Trial News

Belzupacap Sarotalocan Shows Promise in Early-Stage Bladder Cancer Trial

• Belzupacap sarotalocan (bel-sar) demonstrates encouraging clinical activity and a favorable safety profile in patients with non-muscle-invasive bladder cancer (NMIBC) in an ongoing phase 1 trial. • In low-grade NMIBC patients treated with light-activated bel-sar, 4 of 5 achieved a complete clinical response, showing no tumor cells detected post-treatment. • The trial observed immune activation in both treated and untreated tumors, suggesting a bladder urothelial field effect with a single low dose of bel-sar. • Aura Biosciences plans to expand the phase 1 trial to optimize dosing and treatment regimens, with potential for a phase 2 registration-supporting trial.

A Phase 1, Open-label Trial of Belzupacap Sarotalocan (AU-011) in Bladder Cancer

NCT05483868RecruitingPhase 1
Aura Biosciences
Posted 9/26/2022

Real-World Data Confirms High Effectiveness of RSV Vaccines in Older Adults

  • A real-world study found RSV vaccines offer approximately 80% protection against severe illness and hospitalization for adults aged 60 and older.
  • The study included data from multiple U.S. healthcare networks, representing 230 hospitals and 245 emergency departments nationwide.
  • The analysis showed the vaccine's benefit was particularly evident in adults aged 75 and older, the most vulnerable age group.
  • Findings highlight the potential for significant cost savings due to reduced RSV-related hospitalizations, estimated between $1.2 and $5 billion annually.

Evergreen Theragnostics Initiates Phase II Trial of 68Ga-EVG321 for Small Cell Lung Cancer in the EU

  • Evergreen Theragnostics has begun a Phase II trial in the EU to evaluate 68Ga-EVG321 in small cell lung cancer (SCLC) patients after EMA approval.
  • The CCK2-VIEW trial will assess 68Ga-EVG321 as a radioligand imaging agent to quantify radioactivity distribution for theragnostic applications.
  • 68Ga-EVG321 targets CCK2R, a protein highly expressed in SCLC, potentially enabling precise tumor-specific delivery and patient selection.
  • This trial supports Evergreen's pipeline of radioligand therapies and the development of a 68Ga/177Lu-EVG321 theragnostic pair for SCLC.

EMA Rejects Renewal of Translarna for Duchenne Muscular Dystrophy After Re-Examination

  • The European Medicines Agency (EMA) has upheld its decision against renewing the marketing authorization for Translarna (ataluren) for Duchenne muscular dystrophy.
  • The EMA's Committee for Medicinal Products for Human Use (CHMP) concluded that Translarna's effectiveness has not been confirmed in clinical trials and patient registry data.
  • The decision follows multiple re-examinations and consultations, including consideration of real-world data and input from patient and healthcare professional groups.
  • PTC Therapeutics, the drug's maker, expressed disappointment but will keep Translarna available while the European Commission makes its final decision.

Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

NCT02369731Active, Not Recruiting
PTC Therapeutics
Posted 4/30/2015

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

NCT03179631CompletedPhase 3
PTC Therapeutics
Posted 7/6/2017

CHMP Recommends Approval of Novo Nordisk's Concizumab for Hemophilia

  • The Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Novo Nordisk's concizumab for hemophilia treatment.
  • This positive recommendation contrasts with the situation in the US, where regulators requested additional data before approval.
  • Concizumab represents a novel approach to hemophilia management, potentially offering a new therapeutic option for patients.

Response Pharmaceuticals Initiates Phase II Trial of RDX-002 for Weight Rebound After GLP-1 Agonist Discontinuation

  • Response Pharmaceuticals has begun enrolling participants in a Phase II trial of RDX-002 to manage weight rebound following the cessation of GLP-1 agonist treatment.
  • RDX-002, a gut-specific iMTP inhibitor, aims to reduce post-prandial triglyceride levels and improve cardiometabolic risk factors in patients.
  • The double-blind study will assess the efficacy and tolerability of RDX-002 over 12 weeks, with data expected in the second half of 2025.
  • Previous trials have shown RDX-002 to lower triglyceride and LDL cholesterol levels, as well as contribute to weight reduction, with good tolerability.

Novo Nordisk's Alhemo Receives Positive CHMP Opinion for Hemophilia A and B Treatment

  • The European Medicines Agency's CHMP recommended Alhemo (concizumab) for approval as a once-daily subcutaneous prophylactic treatment for hemophilia A or B with inhibitors.
  • Alhemo, a monoclonal antibody, blocks tissue factor pathway inhibitor (TFPI) to promote blood clotting by ensuring thrombin production in hemophilia patients.
  • Phase 3 explorer7 study data showed an 86% reduction in treated spontaneous and traumatic bleeds with concizumab prophylaxis compared to no prophylaxis.
  • If approved by the European Commission, Alhemo will be available in a pre-filled pen, offering convenient subcutaneous administration.

Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors

NCT04083781Active, Not RecruitingPhase 3
Novo Nordisk A/S
Posted 10/21/2019

Risk Stratification and Population Health Key to Revolutionizing CKD Care Management

  • Advanced risk stratification models are enabling precise identification of high-risk CKD patients and connecting them with appropriate therapies, bridging the gap between diagnostics and treatment.
  • Population health strategies, particularly leveraging laboratory data as a safety net, are crucial for addressing CKD care disparities in marginalized communities and ensuring continuous patient monitoring.
  • Moving beyond the traditional EGFR-centered approach, new risk-prediction models can identify high-risk patients across all CKD stages, enabling early intervention with targeted therapies.

Advancing Early-Phase Immunotherapeutic Trials: Optimizing Design and Patient Selection

• Early-phase oncology trials are evolving to incorporate efficacy endpoints alongside safety, driven by novel targeted and immunotherapeutic agents. • Refining trial designs and patient selection is crucial to improve efficiency and maximize therapeutic intent in immunotherapeutic drug development. • Management of treatment-related adverse events from next-generation immunotherapies requires adaptive trial designs and specialized expertise. • Research aims to identify prognostic markers and implement pharmacodynamic markers to better assess novel immunotherapeutic agents.

Social Determinants Create Stark Disparities in Type 2 Diabetes Care and Outcomes

  • Patients with type 2 diabetes living in high social needs areas face double the emergency department visit rates compared to those in low-need neighborhoods, according to a comprehensive Vizient study analyzing Medicare claims data.
  • Early primary care intervention proves crucial, with patients seeing PCPs within 12 months of diagnosis showing significantly better outcomes and lower complication rates over a 5-year period.
  • Young Medicare patients in socially vulnerable areas experience severe diabetes complications at rates comparable to elderly patients, highlighting critical care access disparities.

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