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Clinical Trial News

PARP Inhibitors Show Promise in Epithelial Ovarian and Breast Cancers with BRCA1/2 Mutations

  • PARP inhibitors have demonstrated efficacy in platinum-sensitive epithelial ovarian cancer, particularly in high-grade serous disease, and in breast cancer with BRCA1/2 mutations.
  • Olaparib maintenance therapy significantly improved progression-free survival in relapsed high-grade serous ovarian cancer, especially in patients with BRCA1/2 mutations.
  • Clinical trials are underway to evaluate PARP inhibitors like veliparib, rucaparib, and niraparib in various solid tumors, aiming to identify predictive biomarkers for patient selection.
  • Research suggests PARP inhibitors' mechanism involves multiple aspects of PARP1 biology, including BER inhibition, PARP1 trapping, defective BRCA1 recruitment, and NHEJ activation.

Olaparib Maintenance Monotherapy in Patients With BRCA Mutated Ovarian Cancer Following First Line Platinum Based Chemotherapy.

NCT01844986Active, Not RecruitingPhase 3
AstraZeneca
Posted 8/26/2013

A Phase III Trial of Niraparib Versus Physician's Choice in HER2 Negative, Germline BRCA Mutation-positive Breast Cancer Patients

NCT01905592TerminatedPhase 3
Tesaro, Inc.
Posted 2/25/2014

A Maintenance Study With Niraparib Versus Placebo in Patients With Platinum Sensitive Ovarian Cancer

NCT01847274CompletedPhase 3
Tesaro, Inc.
Posted 6/21/2013

Phase 3 Study of Rucaparib as Switch Maintenance After Platinum in Relapsed High Grade Serous or Endometrioid Ovarian Cancer (ARIEL3)

NCT01968213CompletedPhase 3
pharmaand GmbH
Posted 4/7/2014

Olaparib in gBRCA Mutated Pancreatic Cancer Whose Disease Has Not Progressed on First Line Platinum-Based Chemotherapy

NCT02184195CompletedPhase 3
AstraZeneca
Posted 12/16/2014

Olaparib as Adjuvant Treatment in Patients With Germline BRCA Mutated High Risk HER2 Negative Primary Breast Cancer

NCT02032823Active, Not RecruitingPhase 3
AstraZeneca
Posted 4/22/2014

Efficacy and Safety Study of Olaparib in Combination With Paclitaxel to Treat Advanced Gastric Cancer.

NCT01924533CompletedPhase 3
AstraZeneca
Posted 9/3/2013

Temozolomide With or Without Veliparib in Treating Patients With Newly Diagnosed Glioblastoma Multiforme

NCT02152982Active, Not RecruitingPhase 2
National Cancer Institute (NCI)
Posted 12/15/2014

A Phase 3 Randomized, Placebo-controlled Trial of Carboplatin and Paclitaxel With or Without Veliparib (ABT-888) in HER2-negative Metastatic or Locally Advanced Unresectable BRCA-associated Breast Cancer

NCT02163694CompletedPhase 3
AbbVie
Posted 4/8/2014

Study Comparing Veliparib Plus Carboplatin and Paclitaxel Versus Placebo Plus Carboplatin and Paclitaxel in Previously Untreated Advanced or Metastatic Squamous Non-Small Cell Lung Cancer

NCT02106546CompletedPhase 3
AbbVie
Posted 4/10/2014

A Study Evaluating Safety and Efficacy of the Addition of ABT-888 Plus Carboplatin Versus the Addition of Carboplatin to Standard Chemotherapy Versus Standard Chemotherapy in Subjects With Early Stage Triple Negative Breast Cancer

NCT02032277CompletedPhase 3
AbbVie
Posted 4/2/2014

Assessment of the Efficacy and Safety of Olaparib Monotherapy Versus Physicians Choice Chemotherapy in the Treatment of Metastatic Breast Cancer Patients With Germline BRCA1/2 Mutations.

NCT02000622Active, Not RecruitingPhase 3
AstraZeneca
Posted 3/27/2014

Tyra Biosciences Advances FGFR Inhibitor Pipeline with Multiple Clinical Trials

  • Tyra Biosciences is developing TYRA-300, a selective FGFR3 inhibitor, for bladder cancer and skeletal dysplasia, with Phase 2 trials for achondroplasia expected to begin in Q1 2025.
  • SURF201, a Phase 1 clinical study, is evaluating TYRA-200, an oral FGFR1/2/3 inhibitor, in patients with advanced cholangiocarcinoma and other solid tumors harboring FGFR2 alterations.
  • The FDA has cleared Tyra Biosciences' IND for TYRA-430, a FGFR4/3-biased inhibitor, allowing a Phase 1 study in advanced hepatocellular carcinoma and other solid tumors with FGF/FGFR pathway aberrations.

Safety and Anti-Tumor Activity of TYRA-200 in Advanced Cholangiocarcinoma With Activating FGFR2 Gene Alterations

NCT06160752RecruitingPhase 1
Tyra Biosciences, Inc
Posted 11/22/2023

NIH-Sponsored Trials Validate Severity of Persistent Lyme Disease Symptoms

  • Four NIH-sponsored, double-blind, randomized, placebo-controlled trials validate the severity of Persistent Lyme Disease Symptoms (PLDS).
  • PLDS result in a quality of life comparable to other serious chronic illnesses, impacting fatigue, pain, role function, psychopathology, and cognition.
  • The trials refute claims that PLDS are merely "aches and pains of daily living" or symptoms unrelated to Lyme disease.
  • Findings suggest physicians should address PLDS with the same vigor as other chronic illnesses, focusing on cure or symptom amelioration.

Real-World Evidence Shows Positive Impact on HTA Drug Approvals, Analysis Reveals

• Analysis of 1,840 Health Technology Assessment decisions reveals that submissions including real-world evidence achieved 77% approval rate compared to 67% without RWE.
• Case studies from Australia, Scotland, and France demonstrate how real-world evidence helped secure positive reimbursement decisions for drugs like Yervoy, Zaltrap, and Myozyme.
• Despite proven benefits, real-world evidence was only utilized in 6% of HTA evaluations, suggesting significant untapped potential for both pharmaceutical companies and assessment agencies.

Beyond the Pill: Pharmaceutical Industry Shifts Focus to Holistic Patient Solutions

  • Healthcare systems' financial constraints and rising patient expectations are forcing pharmaceutical companies to expand beyond traditional drug development to provide comprehensive healthcare solutions.
  • Innovation in pharmaceutical industry now encompasses smart packaging, diagnostic tools, and digital health solutions to improve medication adherence and patient outcomes.
  • Companies must redefine 'unmet needs' to include broader healthcare challenges, focusing on value demonstration and improved patient outcomes rather than just pharmacological interventions.

Navigating the $35 Billion Biosimilar Market: New Marketing Paradigms for Success

  • The biosimilar market is projected to reach $35 billion by 2020, presenting significant opportunities within the $169 billion biologics sector for companies that can effectively differentiate their products.
  • Marketing biosimilars presents a unique challenge: manufacturers must first establish similarity to gain physician trust, then differentiate their products through innovative delivery systems and value-added services.
  • With over 700 biosimilars currently in development, success requires extensive market education, strategic differentiation, and substantial investment in patient-centric solutions beyond competitive parity.

UCLA Gene Therapy Achieves 100% Cure Rate for Bubble Baby Disease in Clinical Trials

FDA Approval
  • UCLA researchers led by Dr. Donald Kohn have successfully cured all 18 children with ADA-deficient SCID using a novel stem cell gene therapy approach over two clinical trials since 2009.
  • The breakthrough treatment uses patients' own genetically modified blood stem cells to restore immune function, eliminating the need for lifelong enzyme injections or risky bone marrow transplants.
  • All treated patients, ranging from 3 months to 4 years old, developed fully functioning immune systems without side effects and can now live normal lives.
  • The team is seeking FDA approval for the therapy and plans to extend the approach to treat sickle cell disease in clinical trials beginning in 2015.

Pre-Transplant MRD Positivity Linked to Poorer Survival in Acute Leukemia Patients

  • A study of 114 acute leukemia patients undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) reveals that pre-transplant minimal residual disease (MRD) positivity is associated with worse outcomes.
  • Patients with MRD+ status before transplantation had significantly lower one-year overall survival (OS) and progression-free survival (PFS) rates compared to MRD- patients.
  • Multivariate analysis identified MRD positivity as an independent prognostic factor for overall survival, highlighting the need for improved pre-transplant MRD management strategies.
  • The research suggests focusing on more effective chemo regimens with targeted agents to achieve MRD- status before transplantation and better management of GvHD prophylaxis.

Topotecan Shows Significant Activity in Second-Line Treatment of Small-Cell Lung Cancer

  • Topotecan demonstrates notable efficacy in treating small-cell lung cancer (SCLC) patients who have previously undergone chemotherapy.
  • The study reveals a higher response rate among patients sensitive to prior chemotherapy, with an overall response rate of 37.8%.
  • Hematologic toxicity, particularly neutropenia, was the primary adverse effect, but it was generally manageable and short-lived.
  • The findings suggest that topotecan could be a valuable component in future combination chemotherapy regimens for SCLC.

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