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Clinical Trial News

Cysteamine Ophthalmic Solution Shows Promise in Japanese Cystinosis Patients

  • A Phase III study in Japan evaluated cysteamine hydrochloride 0.55% ophthalmic solution for treating corneal cystine crystals in cystinosis patients.
  • The study showed a decrease in corneal crystal density and improvement in photophobia, suggesting potential efficacy in Japanese patients.
  • The ophthalmic solution demonstrated an acceptable safety profile, with most adverse events being eye-related and manageable.
  • These findings support the use of cysteamine eye drops for managing ocular manifestations of cystinosis, a rare genetic disorder.

Peer Education Improves Medication Adherence and Cost-Effectiveness in Elderly Hypertensive Patients

  • A randomized clinical trial demonstrated that peer-led education significantly improved medication adherence among elderly patients with hypertension.
  • The study involved 74 participants and compared peer-led sessions to traditional nurse-led education, both based on Ministry of Health guidelines.
  • Cost-effectiveness analysis revealed that peer education was a more efficient method for enhancing medication adherence in this population.
  • The findings suggest peer education as a valuable strategy for hypertension management in elderly patients, offering both clinical and economic benefits.

TiCARos Unveils Switchable CAR-T Therapy Demonstrating Enhanced Safety and Efficacy in Solid Tumors

  • TiCARos' switchable CAR-T technology overcomes on-target off-tumor toxicity, a major limitation in existing CAR-T therapies, particularly for solid cancers.
  • The novel approach uses a chemically tagged cotinine adapter to indirectly target cancer cells, sparing normal tissues and reducing toxicity in animal studies.
  • A suicidal switch mechanism provides an added layer of safety, allowing for the removal of CAR-T cells if necessary, enhancing the therapeutic control.
  • Human T-cell trials confirmed the efficacy and safety of the switchable CAR-T system, suggesting its potential for treating both solid tumors and hematologic cancers.

Transferrin Receptor-Targeted ASOs Show Promise in Crossing the Blood-Brain Barrier

  • Researchers have developed antisense oligonucleotides (ASOs) conjugated with a transferrin receptor (TfR)-targeting antibody fragment for enhanced brain delivery.
  • Intravenous administration of these TfR-ASO conjugates in mice demonstrated effective ASO distribution throughout the brain.
  • The TfR-ASO conjugates led to significant target mRNA reduction in the brain, showcasing their potential for treating neurological disorders.
  • This targeted delivery approach may improve the efficacy and reduce systemic exposure of ASO therapeutics for CNS diseases.

Leclaza Downstages Lung Cancer in 55% of Patients in Preoperative Trial

  • A Korean study found that nine weeks of Leclaza treatment can lower the stage of inoperable lung cancer patients with EGFR mutations, making them eligible for surgery.
  • The NeoLazBAL phase 2 trial used lung lavage fluid biopsies to detect EGFR mutations and guide Leclaza treatment in suspected lung cancer patients.
  • Results showed a 55.8% partial remission rate with Leclaza, and surgical pathology confirmed lung adenocarcinoma in all patients who underwent resection.
  • Lung lavage fluid biopsy demonstrated 77.3% sensitivity for detecting EGFR mutations in stage I NSCLC, offering a less invasive diagnostic approach.

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

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

Gene Therapy for APOE4 Homozygote of Alzheimer's Disease

NCT03634007CompletedPhase 1
Lexeo Therapeutics
Posted 11/6/2019

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.

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