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Clinical Trial News

Lecanemab Slows Alzheimer's Cognitive Decline in Phase 3 Trial

  • Lecanemab, an Alzheimer's drug developed by Biogen and Eisai, demonstrated a 27% reduction in cognitive decline compared to placebo in a Phase 3 trial.
  • The trial involved nearly 1,800 participants with mild cognitive impairment or mild Alzheimer's disease, showing promising results in early stages.
  • Lecanemab targets and removes toxic beta-amyloid proteins in the brain, offering a potential disease-modifying approach.
  • Regulatory applications are underway in the US, Europe, and Japan, marking a potentially significant advancement in Alzheimer's treatment.

Photocure and Karl Storz Launch Advanced Blue Light Cystoscopy System in US Market

Oncology
  • Photocure ASA announces the commercial availability of Karl Storz's New Blue Light equipment powered by Saphira technology in the United States for bladder cancer detection.
  • The advanced system shows strong market demand with orders outpacing previous systems, signaling potential for blue light cystoscopy to become standard of care for bladder cancer visualization.
  • Bladder cancer affects 1.72 million patients globally with high recurrence rates up to 78% over five years, making improved detection technology critically important for patient outcomes.

Lecanemab PK/PD Analysis Shows Effective Amyloid Reduction in Early Alzheimer's

• Lecanemab, a monoclonal antibody, demonstrates well-characterized pharmacokinetics in early Alzheimer's disease patients, reducing amyloid plaques. • PK/PD models reveal that bi-weekly 10 mg/kg dosing of lecanemab leads to faster and greater amyloid reduction compared to monthly dosing. • Simulations suggest slow amyloid re-accumulation post-treatment, with a recovery half-life of approximately 4 years. • The study supports using PK/PD models to optimize lecanemab dosing in future clinical trials for early Alzheimer's disease.

Initial TNF-α Levels Predict Infliximab Response in Pediatric Crohn's Disease

  • A recent study reveals that initial levels of TNF-α can predict the treatment response to Infliximab (IFX) in pediatric Crohn's Disease (CD).
  • Higher initial TNF-α concentrations correlate with lower IFX trough concentrations, potentially leading to suboptimal therapeutic effects.
  • A TNF-α cutoff value of 27.6 pg/mL was identified, above which higher IFX doses may be needed to maintain therapeutic concentrations.
  • Measuring initial cytokine levels could help personalize IFX dosing strategies, improving treatment outcomes for pediatric CD patients.

Genetically Modified Herpes Virus Shows Promise Against Advanced Cancers in Early Trial

  • A genetically engineered herpes simplex virus called RP2 demonstrated effectiveness in 25% of patients with advanced cancers who had exhausted other treatment options.
  • The virus works through a dual mechanism, directly destroying cancer cells from within while activating the immune system to target tumors.
  • One patient with salivary gland cancer achieved complete tumor disappearance and remains cancer-free 15 months after treatment.
  • The phase I trial showed generally mild side effects, with researchers planning larger studies to confirm the therapy's potential.

Real-World Study Shows Axicabtagene Ciloleucel Outperforms Tisagenlecleucel in Relapsed/Refractory DLBCL

CAR-TReal World Evidence
  • A large French real-world study of 809 patients with relapsed/refractory DLBCL found axicabtagene ciloleucel (axi-cel) demonstrated superior efficacy compared to tisagenlecleucel (tisa-cel) with significantly higher response rates and improved survival outcomes.
  • After propensity score matching of 418 patients, axi-cel achieved 80.4% overall response rate and 60.3% complete response rate versus 66.0% and 42.1% respectively for tisa-cel, with one-year progression-free survival of 46.6% versus 33.2%.
  • The superior efficacy of axi-cel came with increased toxicity, including significantly higher rates of severe immune cell-associated neurotoxicity syndrome (13.9% vs 2.9%) and prolonged hematological toxicity.
  • This comprehensive analysis from the DESCAR-T registry provides the first real-world evidence of overall survival advantage for axi-cel over tisa-cel in third-line or later treatment of DLBCL.

French Register Of Patients With Hemopathy Eligible For CAR-T Cell Treatment (DESCAR-T)

NCT04328298Recruiting
The Lymphoma Academic Research Organisation
Posted 12/19/2019

Experts Call for Reform in Experimental Treatment Access: Balancing Patient Rights with Sustainable Development

• Leading healthcare experts advocate for a pre-approval access platform to streamline experimental treatment availability while maintaining safety oversight and clinical research integrity.
• Industry veterans highlight the critical need for reimbursement mechanisms to incentivize pharmaceutical companies' participation in pre-approval access programs across the United States.
• The debate around Right-to-Try laws has created an opportunity to reshape drug development systems, with experts proposing enhanced Phase 2 trials supplemented by real-world data.

Adeno-associated Virus Gene Therapy for Hemophilia: A New Therapeutic Paradigm

Adeno-associated virus (AAV) gene therapy is emerging as a promising treatment for hemophilia A and B, offering the potential for long-term factor expression and reduced bleeding episodes. This article reviews the clinical development of AAV gene therapy, its efficacy, safety considerations, and the challenges of pre-existing immunity to AAV vectors.

Gene Therapy for Haemophilia A.

NCT03001830Active, Not RecruitingPhase 1
University College, London
Posted 6/14/2017

Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A

NCT03432520Active, Not Recruiting
Spark Therapeutics, Inc.
Posted 8/14/2018

A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A

NCT03061201CompletedPhase 2
Pfizer
Posted 6/21/2017

A Factor IX Gene Therapy Study (FIX-GT)

NCT03369444TerminatedPhase 1
University College, London
Posted 12/5/2017

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

NCT03569891CompletedPhase 3
CSL Behring
Posted 6/27/2018

A Gene Transfer Study for Hemophilia A

NCT03003533CompletedPhase 1
Spark Therapeutics, Inc.
Posted 1/26/2017

Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

NCT03307980Active, Not RecruitingPhase 2
Pfizer
Posted 6/22/2017

Gene Therapy Study in Severe Haemophilia A Patients (270-201)

NCT02576795CompletedPhase 1
BioMarin Pharmaceutical
Posted 9/28/2015

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

NCT03370913CompletedPhase 3
BioMarin Pharmaceutical
Posted 12/19/2017

A Gene Therapy Study for Hemophilia B

NCT02484092CompletedPhase 2
Pfizer
Posted 11/18/2015

A Study of BAX 888 in Male Adults With Severe Hemophilia A

NCT03370172CompletedPhase 1
Baxalta now part of Shire
Posted 3/31/2018

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"

NCT03588299Active, Not RecruitingPhase 1
Bayer
Posted 11/7/2018

Dose Confirmation Trial of AAV5-hFIXco-Padua

NCT03489291CompletedPhase 2
CSL Behring
Posted 7/24/2018

Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B

NCT02396342CompletedPhase 1
CSL Behring
Posted 6/10/2015

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

NCT03392974CompletedPhase 3
BioMarin Pharmaceutical
Posted 3/14/2018

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