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Clinical Trial News

NMDA Receptor Research Reveals Novel Mechanism for Brain Stabilization and Potential Depression Treatments

  • Researchers at Tel Aviv University have discovered that NMDA receptors (NMDARs) play a crucial role in maintaining brain stability, or homeostasis, beyond their known function in learning and memory.
  • The study suggests that ketamine's rapid antidepressant effects may stem from its ability to reset the brain's baseline activity by blocking NMDARs, offering insights into treating brain instability-related conditions.
  • Experiments on neurons, mice, and computational models demonstrated that NMDARs control baseline activity in brain networks, influencing signal transmission and reducing background noise.
  • These findings pave the way for developing targeted therapies for depression, Alzheimer’s, and epilepsy by focusing on stabilizing brain activity through NMDAR modulation.

Tovorafenib Demonstrates Improved Quality of Life in Pediatric Low-Grade Glioma

  • Tovorafenib (Ojemda) shows promise in improving health-related quality of life (HRQOL) in pediatric patients with BRAF-altered relapsed/refractory low-grade glioma.
  • The phase 2 FIREFLY-1 trial exploratory analysis indicates stable or improved HRQOL, particularly in anxiety and cognition domains, during the first year of treatment.
  • The findings complement previously reported efficacy data, suggesting that tovorafenib not only provides clinical benefit but also enhances patients’ quality of life.
  • Long-term HRQOL analysis was limited, underscoring the need for future studies to assess the sustained impact of tovorafenib on overall well-being.

A Study to Evaluate Tovorafenib in Pediatric and Young Adult Participants With Relapsed or Progressive Low-Grade Glioma and Advance Solid Tumors

NCT04775485RecruitingPhase 2
Day One Biopharmaceuticals, Inc.
Posted 4/22/2021

Mirdametinib Shows Deep Responses in NF1-Associated Plexiform Neurofibromas

  • Mirdametinib demonstrates deep responses, defined as over 50% reduction in tumor volume, in patients with NF1-associated plexiform neurofibromas (NF1-PN).
  • Exploratory analysis found no specific baseline characteristics, such as age or sex, associated with a higher likelihood of achieving a deep response with mirdametinib.
  • Longer treatment duration with mirdametinib appeared to correlate with deeper responses, suggesting the importance of extended therapy for optimal outcomes.
  • The FDA granted priority review to mirdametinib for NF1-associated plexiform neurofibromas, with a decision expected by February 28, 2025.

MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas

NCT03962543Active, Not RecruitingPhase 2
SpringWorks Therapeutics, Inc.
Posted 9/29/2019

Trastuzumab Duocarmazine Demonstrates Efficacy in HER2-Positive Metastatic Breast Cancer

• Trastuzumab duocarmazine (T-Duo) significantly improved progression-free survival in patients with advanced HER2-positive metastatic breast cancer compared to physician's choice of treatment. • The TULIP trial reported a median progression-free survival of 7 months in the T-Duo group versus 5 months in the physician's choice group (HR = 0.64, P = .002). • While overall survival showed a positive trend with T-Duo (20 vs. 16 months), the difference was not statistically significant (P = .153). • Ocular toxicity, including keratitis and conjunctivitis, was a notable side effect, leading to treatment discontinuation in a significant portion of patients.

FDA Updates: Approvals and Designations in Leukemia, Prostate Cancer, and Melanoma

• Capivasertib plus abiraterone and ADT shows improved radiographic progression-free survival in PTEN-deficient metastatic hormone-sensitive prostate cancer. • LBS-007 receives FDA fast track designation for acute myeloid leukemia treatment based on promising early response data from a phase 1/2 trial. • BLA submitted for RP1 with nivolumab for advanced melanoma patients who progressed on prior PD-1 inhibitor therapy. • Revumenib receives FDA approval for relapsed/refractory KMT2A-rearranged acute leukemia based on phase 1/2 AUGMENT-101 trial data.

Evaluation of Efficacy and Safety of Belantamab Mafodotin, Bortezomib and Dexamethasone Versus Daratumumab, Bortezomib and Dexamethasone in Participants With Relapsed/Refractory Multiple Myeloma

NCT04246047Active, Not RecruitingPhase 3
GlaxoSmithKline
Posted 5/7/2020

The Safety and Tolerability of LBS-007 in Patients With Relapsed or Resistant Acute Leukaemias

NCT05756322RecruitingPhase 1
Lin BioScience, Inc
Posted 7/20/2023

A Study of Revumenib in R/R Leukemias Including Those With an MLL/KMT2A Gene Rearrangement or NPM1 Mutation

NCT04065399RecruitingPhase 1
Syndax Pharmaceuticals
Posted 11/5/2019

Capivasertib+Abiraterone as Treatment for Patients With Metastatic Hormone-sensitive Prostate Cancer and PTEN Deficiency

NCT04493853Active, Not RecruitingPhase 3
AstraZeneca
Posted 7/13/2020

Adjuvant Therapy With an Alpha-lactalbumin Vaccine in Triple-Negative Breast Cancer

NCT04674306Active, Not RecruitingEarly Phase 1
George T. Budd
Posted 10/1/2021

Study of DS-1062a in Advanced or Metastatic Non-small Cell Lung Cancer With Actionable Genomic Alterations (TROPION-Lung05)

NCT04484142Active, Not RecruitingPhase 2
Daiichi Sankyo
Posted 3/30/2021

Belantamab Mafodotin Plus Pomalidomide and Dexamethasone (Pd) Versus Bortezomib Plus Pd in Relapsed/Refractory Multiple Myeloma

NCT04484623Active, Not RecruitingPhase 3
GlaxoSmithKline
Posted 10/1/2020

VO and Nivolumab vs Physician's Choice in Advanced Melanoma That Progressed on Anti-PD-1 & Anti-CTLA-4 Drugs (IGNYTE-3)

NCT06264180RecruitingPhase 3
Replimune Inc.
Posted 7/11/2024

Study of RP1 Monotherapy and RP1 in Combination With Nivolumab

NCT03767348RecruitingPhase 2
Replimune Inc.
Posted 9/20/2017

A Trial to Evaluate Efficacy and Safety of Ribociclib With Endocrine Therapy as Adjuvant Treatment in Patients With HR+/HER2- Early Breast Cancer

NCT03701334Active, Not RecruitingPhase 3
Novartis Pharmaceuticals
Posted 12/7/2018

First-in-human Study of DS-1062a for Advanced Solid Tumors (TROPION-PanTumor01)

NCT03401385Active, Not RecruitingPhase 1
Daiichi Sankyo Co., Ltd.
Posted 1/31/2018

Study Comparing Daratumumab, Lenalidomide, Bortezomib, and Dexamethasone (D-RVd) Versus Lenalidomide, Bortezomib, and Dexamethasone (RVd) in Subjects With Newly Diagnosed Multiple Myeloma

NCT02874742CompletedPhase 2
Janssen Research & Development, LLC
Posted 8/29/2016

Daratumumab, VELCADE (Bortezomib), Lenalidomide and Dexamethasone Compared to VELCADE, Lenalidomide and Dexamethasone in Subjects With Previously Untreated Multiple Myeloma

NCT03710603Active, Not RecruitingPhase 3
Stichting European Myeloma Network
Posted 12/14/2018

Study of DS-1062a Versus Docetaxel in Previously Treated Advanced or Metastatic Non-small Cell Lung Cancer With or Without Actionable Genomic Alterations (TROPION-LUNG01)

NCT04656652Active, Not RecruitingPhase 3
Daiichi Sankyo
Posted 12/21/2020

Darolutamide in Addition to ADT Versus ADT in Metastatic Hormone-sensitive Prostate Cancer

NCT04736199Active, Not RecruitingPhase 3
Bayer
Posted 2/23/2021

CellCentric Secures $60 Million to Advance Clinical Testing of Novel Cancer Therapy

• CellCentric has raised $60 million in funding to finalize clinical testing, aligning with FDA requirements before initiating Phase III trials. • The funding includes the conversion of a loan note from Pfizer into $25 million equity and an additional investment from BrightEdge, the American Cancer Society’s impact investment arm. • The company's research is based on the epigenetic research of Professor Azim Surani, focusing on epigenetic code beyond DNA to instruct cell fate.

Cerevance Secures Additional Funding to Advance CNS Therapies, Including Parkinson's Disease Treatment

• Cerevance has augmented its Series B-1 funding with an additional $47 million to propel its clinical pipeline of central nervous system (CNS) therapies. • The funding will expedite the Phase 3 clinical trial of CVN424, a novel non-dopamine therapy for Parkinson's disease, representing a potential breakthrough in treatment. • Cerevance is also utilizing its NETSseq platform and machine learning to identify new therapeutic targets for Alzheimer's disease, supported by a collaboration with Merck. • The company's extensive brain tissue sample collection is fueling the development of innovative treatments for frontotemporal dementia and other CNS disorders.

BIOS Health's Neural Digital Therapy Platform to Power NIH-Funded Study in Worldwide Clinics

  • BIOS Health's Neural Digital Therapy platform will be used in a new NIH-funded study across eight worldwide clinics, marking a significant step in personalized medicine.
  • The platform leverages AI to analyze neural data in real-time, aiming to improve the quality of life for patients with conditions like hypertension and diabetes.
  • BIOS Health is expanding its global presence with a new research and clinical trials center in California, following its AI and neuroscience site in Canada.
  • The company's technology identifies neural biomarkers to link nerve activity to health conditions, potentially revolutionizing treatment approaches.

CRISPR Therapeutics Faces Market Challenges Despite Casgevy Approval

• CRISPR Therapeutics' stock has declined 47% since March, despite FDA and EU approval of Casgevy for sickle cell disease and transfusion-dependent beta thalassemia. • Casgevy's initial launch has been slower than expected due to complex administration and the need for a conditioning regimen, impacting revenue. • ARK Investment Management shows bullish sentiment by increasing its stake in CRISPR Therapeutics, viewing gene editing as a valuable long-term investment. • CRISPR Therapeutics is advancing CTX112, an experimental blood-cancer treatment, with promising Phase 1 results showing tumor shrinkage in lymphoma patients.

Effect of Tumor Treating Fields (TTFields, 150 kHz) as Front-Line Treatment of Locally-advanced Pancreatic Adenocarcinoma Concomitant With Gemcitabine and Nab-paclitaxel (PANOVA-3)

NCT03377491CompletedPhase 3
NovoCure Ltd.
Posted 2/10/2018

A Study of CM24 in Combination with Nivolumab in Adults with Advanced Solid Tumors

NCT04731467CompletedPhase 1
Famewave Ltd.
Posted 3/19/2021

Randomized, Double-Blind, Placebo-Controlled Trial of REL-1017 as Adjunctive Treatment for MDD

NCT06011577TerminatedPhase 3
Relmada Therapeutics, Inc.
Posted 8/17/2023

Anavex Life Sciences Awaits Topline Results from Rett Syndrome Trial

  • Anavex Life Sciences is developing Anavex 2-73, a small molecule targeting the sigma-1 receptor, for neurodegenerative and neurodevelopmental disorders.
  • The EXCELLENCE trial, a phase II/III study of Anavex 2-73 in pediatric Rett syndrome patients, is expected to release topline data in the coming months.
  • Anavex 2-73 has demonstrated statistically significant cognitive improvement in a phase 2b/3 study for early Alzheimer's disease.
  • The company's pipeline also includes ANAVEX 3-71, targeting SIGMAR1 and muscarinic receptors, currently in phase I trials for Frontotemporal Dementia, Schizophrenia, and Alzheimer's.

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