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Clinical Trial News

EMA Finalizes Reflection Paper on Single-Arm Trials for Efficacy Assessment

  • The European Medicines Agency (EMA) has finalized a reflection paper addressing the use of single-arm trials for establishing drug efficacy, providing guidance for pharmaceutical companies.
  • The paper outlines considerations for when single-arm trials may be appropriate, particularly in rare diseases or situations with significant unmet medical needs.
  • It emphasizes the importance of robust data collection, clear endpoints, and rigorous statistical analysis to ensure the reliability of results from single-arm trials.
  • The EMA's guidance aims to harmonize the approach to evaluating single-arm trials, potentially accelerating drug development for conditions with limited treatment options.

Xofluza Significantly Reduces Influenza Transmission in Phase III Trial

  • Roche's Xofluza (baloxavir marboxil) significantly reduced influenza transmission within households in a Phase III trial, marking a potential advance in flu control.
  • The CENTERSTONE study met its primary endpoint, showing a single oral dose of Xofluza reduced the likelihood of household members contracting the virus.
  • The antiviral treatment was well-tolerated, with no new safety signals, suggesting a favorable risk-benefit profile for influenza prevention.
  • These results could improve health outcomes at both individual and community levels, especially for influenza pandemic preparedness.

Study to Assess the Efficacy of Baloxavir Marboxil Versus Placebo to Reduce Onward Transmission of Influenza A or B in Households

NCT03969212CompletedPhase 3
Hoffmann-La Roche
Posted 10/10/2019

Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Healthy Pediatric Participants From Birth to < 1 Year With Influenza-Like Symptoms

NCT03653364CompletedPhase 3
Hoffmann-La Roche
Posted 1/23/2019

Five Strategic Travel Solutions to Boost Clinical Trial Diversity and Patient Enrollment

  • Recent CISCRP report identifies travel to study clinics as the primary burden for clinical trial participants, highlighting a persistent barrier to patient enrollment and diversity.
  • Clinical trial sites' locations often exclude minority communities, who are disproportionately affected by certain diseases yet face significant transportation and accessibility challenges.
  • Implementing comprehensive travel support solutions, including coordination services, timely reimbursements, and specialized transport options, can significantly improve trial accessibility and diversity.

Rice and Baylor Receive $2.8 Million to Develop Cell Therapy for ARDS

  • Rice University and Baylor College of Medicine have been awarded $2.8 million by the NHLBI to develop a novel cell therapy for acute respiratory distress syndrome (ARDS).
  • The therapy uses engineered retinal pigment epithelial (RPE) cells to deliver anti-inflammatory cytokines directly to the lungs, reducing inflammation and lung damage.
  • This targeted approach aims to overcome the limitations of current cytokine therapies, such as poor biodistribution, toxicity, and immune complications, in ARDS patients.
  • The collaborative effort seeks to improve patient outcomes by creating cell therapy systems that enhance lung health and increase survival rates for individuals with ARDS.

CheckMate-9ER: Glycoproteomic Analysis Identifies Potential Biomarkers for Nivolumab Plus Cabozantinib in Advanced RCC

  • Post-hoc analysis of CheckMate-9ER reveals that higher degrees of fucosylation and sialylation in serum proteins are negative prognostic factors in advanced renal cell carcinoma (RCC).
  • Serum glycoproteins involved in complement cascade and lipid metabolism may predict response to nivolumab plus cabozantinib versus sunitinib.
  • High serum levels of CO3 glycopeptide potentially predict favorable response to nivolumab plus cabozantinib, while low levels of CFAH glycopeptide also indicate benefit.
  • These early exploratory findings suggest protein glycosylation as a potential area for further biomarker development in RCC to predict response to anti-PD-1-based therapy.

A Study of Nivolumab Combined With Cabozantinib Compared to Sunitinib in Previously Untreated Advanced or Metastatic Renal Cell Carcinoma

NCT03141177Active, Not RecruitingPhase 3
Bristol-Myers Squibb
Posted 7/23/2017

Immix Biopharma's NXC-201 Shows Promise in AL Amyloidosis Treatment

  • Immix Biopharma's stock hits a 52-week low amidst market challenges, reflecting investor concerns about the company's financial performance.
  • NXC-201, a CAR-T therapy, demonstrates a 92% overall response rate in an ex-U.S. study for AL Amyloidosis, leading to expansion of U.S. Phase 1b/2 trial.
  • The European Commission grants orphan drug designation to NXC-201 for multiple myeloma, supporting its development for this indication.

Kiromic BioPharma Expands Deltacel-01 Trial for NSCLC to University of Arizona Cancer Center

  • Kiromic BioPharma has activated the University of Arizona Cancer Center as the fifth clinical trial site for its Deltacel-01 Phase 1 trial.
  • The trial evaluates Deltacel™ (KB-GDT-01), an allogeneic gamma delta T-cell therapy, in patients with stage 4 metastatic non-small cell lung cancer (NSCLC).
  • The primary objective is to assess safety, with secondary endpoints including objective response and overall survival in patients who have failed standard therapies.
  • Patient enrollment at the University of Arizona Cancer Center is expected to commence in the coming weeks, expanding the trial's reach.

Safety Study for a Gamma Delta T Cell Product Used with Low Dose Radiotherapy in Patients with Locally Advanced or Metastatic NSCLC or Solid Tumors with Bone Metastases

NCT06069570RecruitingPhase 1
Kiromic BioPharma Inc.
Posted 11/7/2023

Beacon Therapeutics' AGTC-501 Shows Sustained Efficacy in XLRP Phase 2 Trial

  • Beacon Therapeutics announced positive 24-month data from the Phase 2 SKYLINE trial of AGTC-501 for X-linked retinitis pigmentosa (XLRP).
  • The high-dose cohort of AGTC-501 demonstrated a 57% response rate in retinal sensitivity improvement, the trial's primary endpoint.
  • AGTC-501 was generally safe and well-tolerated, with no clinically significant safety events associated with the treatment.
  • Pivotal Phase 2/3 VISTA and open-label Phase 2 DAWN trials are ongoing, supporting further development of AGTC-501 for XLRP.

Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

NCT03316560Active, Not RecruitingPhase 1
Beacon Therapeutics
Posted 4/16/2018

A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

NCT04850118RecruitingPhase 2
Beacon Therapeutics
Posted 3/14/2024

A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)

NCT06275620Enrolling by InvitationPhase 2
Beacon Therapeutics
Posted 11/14/2023

ACCESS-GC Trial Integrates Genetic Counselors into Primary Care in British Columbia

  • The ACCESS-GC trial aims to integrate genetic counselors into primary care settings in British Columbia to address healthcare inequities.
  • Led by Dr. Jehannine (J9) Austin, the trial seeks to provide personalized genetic care directly within communities, enhancing precision medicine.
  • The trial will assess the effectiveness of this integrated approach for conditions with a genetic component, such as diabetes and psychiatric disorders.
  • Funded by Genome BC, the initiative aims to improve patient outcomes and evaluate the economic benefits of embedding genetic counselors in primary care.

Narmafotinib Receives FDA Fast Track Designation for Advanced Pancreatic Cancer

  • The FDA has granted Fast Track Designation to Amplia Therapeutics' narmafotinib for advanced pancreatic cancer, expediting its development and review.
  • Narmafotinib, a FAK inhibitor, may offer an advantage over existing therapies, potentially qualifying it for Accelerated Approval and Priority Review.
  • The ACCENT trial, evaluating narmafotinib with gemcitabine and Abraxane, is ongoing in Australia, South Korea, and planned for the US, showing promising early results.
  • Fast Track Designation allows Amplia Therapeutics to work more closely with the FDA, accelerating the clinical program for this devastating disease.

ACCENT: AMP945 in Combination with Nab-paclitaxel and Gemcitabine for Treatment of Pancreatic Cancer

NCT05355298Active, Not RecruitingPhase 1
Amplia Therapeutics Limited
Posted 5/31/2022

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