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Clinical Trial News

AstraZeneca's AZD1390 to Be Evaluated in GBM AGILE Trial for Glioblastoma

  • AstraZeneca's AZD1390 will be assessed in the Glioblastoma Adaptive Global Innovative Learning Environment (GBM AGILE) trial to treat newly diagnosed glioblastoma patients.
  • The global adaptive platform trial aims to accelerate the identification and confirmation of effective glioblastoma therapies through its unique design.
  • AZD1390, a brain-penetrant ATM kinase inhibitor, impedes ATM-dependent signaling and DNA repair, potentially enhancing glioblastoma treatment.
  • Recruitment for the AZD1390 arm of the trial is anticipated to commence in the second quarter of next year, with data potentially supporting regulatory submissions.

Stricter EU Regulations Hinder Medical Device Innovation, Threatening Competitiveness

  • New Medical Device Regulation (MDR) rules in Europe are creating significant hurdles for companies seeking approval for innovative medical technologies.
  • Cathvision, a Copenhagen-based company, faces challenges in obtaining approval for its heart rhythm disorder treatment technology under the MDR.
  • The stricter regulatory regime is raising concerns about Europe's competitiveness in the medical technology field, potentially stifling innovation.

Alzheimer's Disease: Cell and Gene Therapy Advances in 2024

  • Lexeo Therapeutics' LX1001 gene therapy shows dose-dependent APOE2 expression increase and reduced tau biomarkers in APOE4 homozygote Alzheimer's patients.
  • Lomecel-B cell therapy demonstrates safety and potential cognitive benefits in mild Alzheimer's patients, according to Phase 2a CLEAR MIND trial data.
  • NKGen Biotech advances SNK01 natural killer cell therapy to Phase 2 after promising Phase 1 results, including cognitive function improvements.
  • Regeneration Biomedical doses first patient in Phase 1 trial of RB-ADSC, an autologous stem cell therapy for Alzheimer's, showing no adverse events.

Safety of SNK01 in Subjects With Alzheimer's Disease (ASK-AD)

NCT04678453TerminatedPhase 1
NKGen Biotech, Inc.
Posted 1/6/2021

Gene Therapy for APOE4 Homozygote of Alzheimer's Disease

NCT03634007CompletedPhase 1
Lexeo Therapeutics
Posted 11/6/2019

Autologous Activated Adipose-derived Stem Cells (RB-ADSC) Injected Directly Into the Brain for Mild to Moderate Alzheimer's Disease

NCT05667649RecruitingPhase 1
Regeneration Biomedical, Inc.
Posted 8/14/2023

Lomecel-B Effects on Alzheimer's Disease

NCT05233774CompletedPhase 2
Longeveron Inc.
Posted 12/28/2021

FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

NCT05135091RecruitingPhase 1
Neurona Therapeutics
Posted 6/16/2022

AstraZeneca's AZD1390 to be Evaluated in GBM AGILE Trial for Glioblastoma

  • AstraZeneca's AZD1390 will be evaluated in the Glioblastoma Adaptive Global Innovative Learning Environment (GBM AGILE) trial, a global adaptive platform.
  • The trial, designed with response adaptive randomization and a Phase II/III structure, aims to accelerate the identification of effective glioblastoma therapies.
  • AZD1390 arm will focus on newly diagnosed glioblastoma patients, with recruitment anticipated to commence in the second quarter of next year.
  • GBM AGILE, operating under a master protocol, allows concurrent evaluation of multiple treatments against a common control arm.

FDA Approves BridgeBio's Attruby (acoramidis) for Transthyretin Amyloid Cardiomyopathy

• The FDA has approved BridgeBio Pharma's Attruby (acoramidis) for treating transthyretin amyloid cardiomyopathy (ATTR-CM) in adults, marking a significant advancement in cardiac care. • Attruby is the first approved therapy with a label indicating near-complete transthyretin (TTR) stabilization, aiming to reduce cardiovascular deaths and hospitalizations associated with ATTR-CM. • The approval was based on the Phase III ATTRibute-CM trial, which demonstrated a statistically significant impact on all-cause mortality, cardiovascular hospitalizations, and functional capacity. • BridgeBio plans to seek approvals globally, including in Europe, Japan, and Brazil, and will provide Attruby free for life to US trial participants as a gesture of gratitude.

Cenerimod Demonstrates Reduced Interferon Signatures in Phase 2b SLE Trial

  • Cenerimod 4 mg significantly reduced interferon (IFN)-associated protein and gene signature biomarkers in SLE patients after 6 months compared to placebo.
  • The 4 mg dose showed a more pronounced effect on IFN-1, IFN-γ, and plasma cell biomarkers compared to the 2 mg dose.
  • Cenerimod 4 mg prevented the transition from low to high IFN-1 status in patients with SLE, indicating a potential disease-modifying effect.
  • The findings support the rationale for using cenerimod 4 mg in Phase 3 clinical trials for moderate to severe SLE.

Efficacy and Safety of Four Doses of Cenerimod Compared to Placebo in Adult Subjects With Active Systemic Lupus Erythematosus

NCT03742037CompletedPhase 2
Idorsia Pharmaceuticals Ltd.
Posted 12/21/2018

Electronic Tool Streamlines Stroke Patient Screening for Clinical Trials

  • An electronic tool, the stroke trial tracker, has been developed to streamline the screening and management of stroke patients for clinical trial eligibility.
  • The implementation of the tool has reduced screening time by over 80%, freeing up nurses' time for recruitment activities and increasing patient involvement in trials.
  • The electronic system minimizes human error, enhances efficiency, prevents redundant patient approaches, and improves overall team communication and data accuracy.
  • A survey of UK stroke centers indicates a strong interest in adopting similar systems to save time and improve the accuracy of patient screening for research studies.

FDA Approves BridgeBio's Attruby (acoramidis) for ATTR-CM Treatment

• The FDA has approved BridgeBio Pharma's Attruby (acoramidis) for treating transthyretin amyloid cardiomyopathy (ATTR-CM) in adults, marking a significant advancement in cardiac care. • Attruby, an oral transthyretin (TTR) stabilizer, is the first approved product with a label specifying near-complete TTR stabilization, reducing cardiovascular deaths and hospitalizations. • The approval was based on the Phase III ATTRibute-CM trial, which demonstrated a win ratio of 1.8 and significant improvements in the six-minute walk test and NT-proBNP levels. • BridgeBio plans to pursue global approvals and provide Attruby free for life to US trial participants, with Bayer holding exclusive commercialization rights in Europe.

FDA Accepts GSK's Blenrep Combination Therapy for Relapsed/Refractory Multiple Myeloma

  • The FDA has accepted GSK's biologics license application for Blenrep in combination with BorDex and PomDex.
  • This combination therapy targets patients with relapsed or refractory multiple myeloma who have undergone at least one prior line of therapy.
  • The FDA's decision is expected by July 23, with the application based on positive results from the DREAMM-7 and DREAMM-8 phase III trials.
  • Multiple myeloma is the third most common type of blood cancer, with a need for new therapies due to resistance to available treatments.

Care Bundles Improve Enteral Nutrition Management in Stroke Patients: A Multi-Center Trial

  • A multi-center, stepped-wedge, cluster-based randomized controlled trial is underway to evaluate care bundles for enteral nutrition in stroke patients.
  • The primary hypothesis is that these care bundles will reduce feeding intolerance, a common and serious complication in stroke patients requiring nutritional support.
  • Secondary hypotheses include reduced complications, improved nutritional status, and better overall recovery for stroke patients receiving the intervention.
  • The trial involves eight hospitals in China, with a target sample size of 1224 patients, aiming to demonstrate the effectiveness of standardized enteral nutrition protocols.

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