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Clinical Trial News

Asciminib Demonstrates Durable Efficacy and Safety in Heavily Pretreated CML-CP Patients

  • Asciminib monotherapy shows sustained efficacy in chronic myeloid leukemia patients without T315I mutation after multiple prior tyrosine kinase inhibitors.
  • After roughly 4 years of follow-up, over 60% of patients achieved major molecular response (MMR), with continuous increase over time.
  • The treatment was generally well-tolerated, with most adverse events occurring early and managed through dose adjustments.
  • Long-term data support asciminib's role in heavily pretreated CML-CP, warranting further investigation in earlier lines of therapy.

MHRA Introduces New Measures to Streamline Clinical Trial Approvals in the UK

The Medicines and Healthcare products Regulatory Agency (MHRA) is set to implement a series of new measures aimed at making the UK a more attractive destination for clinical trials. These changes, the most significant in over 20 years, will streamline the approval process, enhance transparency, and support innovative trial designs, all while ensuring patient safety.

AI Technology Revolutionizes Rare Disease Diagnosis, Reducing Detection Time by 4.4 Years

  • AI-powered diagnostic tools like MendelScan are transforming rare disease detection in the UK, scanning over 800,000 patient records and identifying conditions an average of 4.4 years earlier than standard care.
  • The technology addresses a critical healthcare challenge where rare disease diagnosis typically takes 5.6 years, involves eight clinicians, and results in four misdiagnoses before accurate identification.
  • MendelScan's implementation across 50 NHS primary care practices demonstrates promising results, with 54% of identified cases deemed valuable for review and 36% proceeding to further analysis.

Darolutamide Approved in China for Metastatic Hormone-Sensitive Prostate Cancer

  • The Chinese NMPA has approved darolutamide (Nubeqa) plus docetaxel for metastatic hormone-sensitive prostate cancer (mHSPC).
  • The approval was based on the Phase III ARASENS trial, which showed a 32.5% reduction in mortality risk compared to ADT with docetaxel.
  • Darolutamide is an oral androgen receptor inhibitor developed by Bayer and Orion Corporation.
  • This approval provides a new treatment option for mHSPC patients in China, where prostate cancer cases have significantly increased.

Toripalimab, Lenvatinib, and GEMOX Combination Shows Promise in Advanced Intrahepatic Cholangiocarcinoma

  • A phase 2 trial demonstrated that toripalimab combined with lenvatinib and GEMOX chemotherapy achieved an 80% objective response rate (ORR) in patients with advanced intrahepatic cholangiocarcinoma (ICC).
  • The combination therapy resulted in a median overall survival (OS) of 22.5 months, suggesting a significant improvement in outcomes for this difficult-to-treat cancer.
  • The adverse events associated with the triple combination were manageable, with no treatment-related deaths, supporting the feasibility of this approach in clinical practice.
  • Patients with DDR-related gene mutations showed a better response to the triple combination therapy, suggesting DDR-related gene mutation may become a good predictor of the tumor response.

Two-cohort Study of Toripalimab(PD1)+Lenvatnib, or Gemox+Lenvatinib in Advanced Intrahepatic Cholangiocarcinoma

NCT04361331CompletedPhase 2
Shanghai Zhongshan Hospital
Posted 3/6/2020

Toripalimab Plus Lenvatinib and Gemcitabine-based Chemotherapy in 1L Treatment of Advanced ICC: a Phase III Study

NCT05342194RecruitingPhase 3
Shanghai Junshi Bioscience Co., Ltd.
Posted 6/19/2023

Bispecific CD20/CD19 CAR T-Cell Therapy Shows Promising Results in Mantle Cell Lymphoma

  • LV20.19 CAR, a novel bispecific CAR T-cell therapy targeting both CD19 and CD20 B-cell antigens, achieved a 100% overall response rate in patients with relapsed/refractory mantle cell lymphoma.
  • The phase 1/2 trial demonstrated a 92% complete response rate at day 90, with only one relapse observed among 14 patients after 22 months of follow-up.
  • The therapy showed a favorable safety profile with no grade 3/4 cytokine release syndrome and only 21% of patients experiencing neurotoxicity, suggesting potential advantages over existing CAR T-cell treatments.

Safety and Efficacy of PCI-32765 in Participants With Relapsed/Refractory Mantle Cell Lymphoma (MCL)

NCT01236391CompletedPhase 2
Pharmacyclics LLC.
Posted 2/1/2011

A Study Evaluating the Safety and Efficacy of Brexucabtagene Autoleucel (KTE-X19) in Adult Subjects With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia (ZUMA-3)

NCT02614066CompletedPhase 1
Kite, A Gilead Company
Posted 3/7/2016

Study of CAR-20/19-T Cells in Patients With Relapsed Refractory B Cell

NCT03019055CompletedPhase 1
Medical College of Wisconsin
Posted 10/16/2017

Study Evaluating the Safety and Efficacy of KTE-C19 in Adult Participants With Refractory Aggressive Non-Hodgkin Lymphoma

NCT02348216CompletedPhase 1
Kite, A Gilead Company
Posted 4/21/2015

Open Label Study to Evaluate the Safety and Efficacy of Lenalidomide With MOR00208 in Patients With R-R DLBCL

NCT02399085CompletedPhase 2
MorphoSys AG
Posted 3/29/2016

Ofatumumab Versus Rituximab Salvage Chemoimmunotherapy Followed by Autologous Stem Cell Transplant in Relapsed or Refractory Diffuse Large B Cell Lymphoma

NCT01014208CompletedPhase 3
GlaxoSmithKline
Posted 3/1/2010

Study of Effectiveness of Axicabtagene Ciloleucel Compared to Standard of Care Therapy in Patients With Relapsed/Refractory Diffuse Large B Cell Lymphoma

NCT03391466CompletedPhase 3
Kite, A Gilead Company
Posted 1/25/2018

Study of Efficacy and Safety of CTL019 in Adult DLBCL Patients

NCT02445248CompletedPhase 2
Novartis Pharmaceuticals
Posted 7/29/2015

CAR-20/19-T Cells in Patients With Relapsed Refractory B Cell Malignancies

NCT04186520RecruitingPhase 1
Medical College of Wisconsin
Posted 5/18/2020

A Study Comparing the Efficacy and Safety of Polatuzumab Vedotin With Rituximab-Cyclophosphamide, Doxorubicin, and Prednisone (R-CHP) Versus Rituximab-Cyclophosphamide, Doxorubicin, Vincristine, and Prednisone (R-CHOP) in Participants With Diffuse Large B-Cell Lymphoma

NCT03274492Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 11/16/2017

Study to Compare Axicabtagene Ciloleucel With Standard of Care Therapy as First-line Treatment in Participants With High-risk Large B-cell Lymphoma

NCT05605899RecruitingPhase 3
Kite, A Gilead Company
Posted 2/10/2023

A Study to Compare the Efficacy and Safety of JCAR017 to Standard of Care in Adult Subjects With High-risk, Transplant-eligible Relapsed or Refractory Aggressive B-cell Non-Hodgkin Lymphomas

NCT03575351CompletedPhase 3
Celgene
Posted 10/23/2018

Study to Evaluate the Efficacy and Safety of Loncastuximab Tesirine in Patients With Relapsed or Refractory Diffuse Large B-Cell Lymphoma

NCT03589469CompletedPhase 2
ADC Therapeutics S.A.
Posted 8/1/2018

Study to Evaluate Loncastuximab Tesirine With Rituximab Versus Immunochemotherapy in Participants With Relapsed or Refractory Diffuse Large B-Cell Lymphoma

NCT04384484Active, Not RecruitingPhase 3
ADC Therapeutics S.A.
Posted 9/16/2020

Safety and Efficacy of ALLO-501A Anti-CD19 Allogeneic CAR T Cells in Adults with Relapsed/Refractory Large B Cell Lymphoma, Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma (ALPHA2)

NCT04416984Active, Not RecruitingPhase 1
Allogene Therapeutics
Posted 5/21/2020

Tisagenlecleucel in Adult Patients With Aggressive B-cell Non-Hodgkin Lymphoma

NCT03570892Active, Not RecruitingPhase 3
Novartis Pharmaceuticals
Posted 5/7/2019

A Study of Bortezomib, Lenalidomide and Dexamethasone (VRd) Followed by Cilta-cel, a CAR-T Therapy Directed Against BCMA Versus VRd Followed by Lenalidomide and Dexamethasone (Rd) Therapy in Participants With Newly Diagnosed Multiple Myeloma for Whom ASCT is Not Planned as Initial Therapy

NCT04923893Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 8/19/2021

A Study to Evaluate the Safety and Anti-cancer Activity of Loncastuximab Tesirine in Combination With Other Anti-cancer Agents in Participants With Relapsed or Refractory B-cell Non-Hodgkin Lymphoma (LOTIS-7)

NCT04970901RecruitingPhase 1
ADC Therapeutics S.A.
Posted 6/17/2022

Study Evaluating the Safety and Pharmacokinetics of JCAR017 in B-cell Non-Hodgkin Lymphoma (TRANSCEND-NHL-001)

NCT02631044CompletedPhase 1
Juno Therapeutics, a Subsidiary of Celgene
Posted 1/6/2016

Safety and Efficacy of ALLO-501 Anti-CD19 Allogeneic CAR T Cells in Adults With Relapsed/Refractory Large B Cell or Follicular Lymphoma

NCT03939026Active, Not RecruitingPhase 1
Allogene Therapeutics
Posted 5/1/2019

Novo Nordisk Reinstated to ABPI After Two-Year Suspension for Code Violations

• Novo Nordisk has rejoined the Association of the British Pharmaceutical Industry (ABPI) following a two-year suspension for serious code of practice violations related to covert promotion of its weight-loss drug Saxenda.
• The Danish pharmaceutical company demonstrated "significant and sustained improvements" in compliance processes during the suspension period, which included quarterly progress reports to the ABPI board.
• Despite the reinstatement, Novo Nordisk faced another code breach in 2022 for failing to disclose approximately £7.8 million in payments to healthcare professionals and organizations over a three-year period.

Revumenib Shows Promise in Phase 1 Trial for KMT2A-Rearranged and NPM1-Mutated Acute Leukemia

OncologyTargeted Therapy
  • Revumenib, a first-in-class menin inhibitor, demonstrated a 30% complete remission rate in heavily pretreated patients with KMT2A-rearranged or NPM1-mutated acute leukemia, with 78% achieving undetectable measurable residual disease.
  • The oral therapy works by disrupting the menin-KMT2A interaction, downregulating key leukemogenic genes and promoting differentiation of leukemic cells, addressing a critical unmet need for these poor-prognosis genetic subtypes.
  • While QT interval prolongation was the most common treatment-related adverse event (53%), the phase 1 trial established recommended phase 2 dosing with manageable safety profile, supporting further development of this targeted therapy.

Sapience Therapeutics' ST316 Receives FDA Clearance for Phase 1-2 Solid Tumor Trial

  • Sapience Therapeutics received FDA clearance for its IND application to begin a Phase 1-2 clinical trial of ST316 in patients with solid tumors.
  • ST316 is a first-in-class β-catenin antagonist peptide, targeting the Wnt signaling pathway, which is implicated in over 50% of solid tumors.
  • The Phase 1 portion will be a dose-escalation study, while Phase 2 will focus on cholangiocarcinoma, colorectal, triple-negative breast, and ovarian cancers.
  • Patient dosing is expected to commence in the first half of 2023, evaluating safety, clinical activity, pharmacokinetics, and pharmacodynamics of ST316.

Israeli Scientists Develop World's First mRNA Vaccine Against Antibiotic-Resistant Bacteria

  • Israeli researchers from Tel Aviv University and the Israel Institute for Biological Research have developed the world's first mRNA vaccine effective against deadly bacteria, achieving 100% protection in animal studies.
  • The breakthrough overcomes previous biological limitations by modifying bacterial protein secretion pathways and enhancing protein stability, enabling rapid vaccine development for antibiotic-resistant bacterial diseases.
  • In laboratory testing against plague-causing Yersinia pestis, all vaccinated mice survived infection while all unvaccinated animals died within a week.
  • The technology offers a potential solution to the growing threat of antibiotic-resistant bacteria and could enable rapid response to future bacterial pandemics.

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