Clinical Trial News
Clinical Trial Sponsors Still Failing to Report Results Despite Regulations
- A recent investigation reveals that many clinical trial sponsors continue to neglect the mandatory reporting of their results in the federal database, ClinicalTrials.gov.
- Despite a 2007 law and a 2017 "final rule" by the NIH and FDA intended to enforce compliance, many trial sponsors still fail to disclose their findings.
- The lack of enforcement by federal officials allows this non-compliance to persist, hindering doctors and patients from accessing crucial information on treatment safety and efficacy.
Cytokinetics Unveils Vision 2025 and 2020 Corporate Milestones
Cytokinetics, Inc. has announced its Vision 2025, outlining key imperatives for the next five years, including achieving regulatory approvals for at least two drugs, building commercial capabilities, and expanding its development pipeline. The company also detailed its expected 2020 milestones, focusing on advancing cardiac and skeletal muscle programs, with significant clinical trial results anticipated.
MRD Monitoring Predicts Relapse Risk Post-Allo-HSCT in AML Patients
- Measurable residual disease (MRD) after induction chemotherapy is a strong predictor of relapse and shorter survival in acute myeloid leukemia (AML) patients.
- A Spanish AML12 trial evaluated risk-adapted treatment based on AML genetics and MRD presence after consolidation chemotherapy.
- The AML12 trial's results indicated that MRD monitoring can effectively predict relapse risk following allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Blood-Brain Barrier Breakthrough: Novel Delivery System Shows Promise for Brain Disease Treatment
- A new molecular Trojan horse delivery system successfully transports therapeutic proteins and genes across the blood-brain barrier, potentially revolutionizing treatment for neurological diseases.
- The HIRMAb-IDUA fusion protein (valanafusp alpha) has shown positive results in human clinical trials for MPSI, demonstrating both safety and efficacy in treating this rare brain disorder.
- Trojan horse liposomes enable targeted delivery of gene therapies to the brain with lower doses than viral methods, while avoiding safety concerns associated with viral vectors.
Record Attendance at San Antonio Breast Cancer Symposium 2024
The San Antonio Breast Cancer Symposium (SABCS) 2024 concluded with record attendance, featuring groundbreaking clinical trial studies and a strong sense of community among participants. Highlights and updates from the event are available online, with plans already underway for SABCS 2025.
PARP Inhibitors in Frontline Ovarian Cancer Treatment: A Review of Clinical Trials
- Frontline PARP inhibitor maintenance shows greatest benefit in patients with BRCA-associated ovarian cancers, followed by those with homologous recombination deficient (HRD) tumors.
- Key factors in deciding on frontline PARP inhibitor therapy include FDA indication, dosing preference, toxicity profiles, patient-specific risk-benefit assessment, and cost considerations.
- Ongoing trials are exploring PARP inhibitor combinations with anti-angiogenics, immunotherapies, and agents targeting PARP inhibitor resistance to further improve outcomes.
- The optimal timing of PARP inhibitor use should also be considered, balancing potential benefits in the frontline setting against the possibility of PARP-inhibitor resistance when used later.
Highlighted Clinical Trials:
AstraZeneca
Posted 6/8/2017
pharmaand GmbH
Posted 5/14/2018
Tesaro, Inc.
Posted 10/11/2018
Merck Sharp & Dohme LLC
Posted 12/18/2018
Tesaro, Inc.
Posted 12/12/2017
AstraZeneca
Posted 1/4/2019
MRD Negativity as a Surrogate Endpoint for PFS in Multiple Myeloma
- Achieving minimal residual disease (MRD) negativity is increasingly recognized as a crucial endpoint in multiple myeloma treatment, indicating deeper remission and improved outcomes.
- Meta-analysis of six randomized clinical trials suggests that MRD status could serve as a surrogate endpoint for progression-free survival (PFS) in newly diagnosed multiple myeloma (NDMM).
- The FDA is considering MRD negativity as a potential surrogate endpoint for accelerated drug approval, which could expedite the development of more effective therapies.
- Studies show that patients achieving MRD-negative complete response (CR) experience superior survival outcomes compared to those with MRD-positive CR, highlighting the importance of MRD monitoring.
Exploring Anti PD-L1 (Atezolizumab) as an Immune Primer and Concurrent Treatment with Chemoradiotherapy for Locally Advanced Cervical Cancer
A clinical trial, NRG Oncology GY017, investigates the use of Anti PD-L1 (atezolizumab) as an immune primer and concurrently with extended field chemoradiotherapy for node-positive locally advanced cervical cancer. This study aims to address the unmet need for therapeutic options in patients with para-aortic lymph node metastases, focusing on the synergistic effects of immunotherapy and radiation. The trial explores the optimal sequencing of atezolizumab and chemoradiation to enhance immune activation and improve clinical outcomes.
Highlighted Clinical Trials:
Study of Pembrolizumab (MK-3475) in Participants With Advanced Solid Tumors (MK-3475-028/KEYNOTE-28)
Merck Sharp & Dohme LLC
Posted 2/17/2014
Bristol-Myers Squibb
Posted 10/13/2015
FDA Unveils Historic Drug Import Plan from Canada to Combat High US Prices
- The Trump administration has proposed a groundbreaking plan allowing states, wholesalers, and pharmacies to import certain prescription drugs from Canada to reduce US drug prices.
- The FDA's new draft guidance enables drug manufacturers to import FDA-approved medications manufactured abroad, though biologics, controlled substances, and certain injectable drugs are excluded.
- Implementation faces significant challenges, including Canada's concerns about potential domestic shortages and former FBI director Louis Freeh's warnings about enforcement capabilities.
Biogen's Alzheimer's Drug in Spotlight Following Sarepta's Vyondys 53 Approval
The unexpected FDA approval of Sarepta Therapeutics' Vyondys 53 for Duchenne muscular dystrophy has sparked discussions about the potential flexibility the FDA might show towards Biogen's Alzheimer's drug, aducanumab. This comes amidst skepticism over the efficacy and safety of both drugs, with Biogen's application based on a controversial Phase 3 study.