MedPath

Clinical Trial News

Personalized ASO Treatment Fast-Tracked for Twins with Rare Batten Disease

  • Twins with a rare genetic variant of juvenile Batten disease received a personalized antisense oligonucleotide (ASO) treatment called Zebronkysen, developed through an 'N-of-2' study.
  • The treatment, targeting a unique mutation in the CLN3 gene, was rapidly developed and approved by the FDA in just over a year, marking a significant advancement in personalized medicine.
  • Early signs are positive, with hopes that Zebronkysen will stabilize neurodegeneration in the twins, and the study may serve as a proof-of-concept for similar personalized treatments for other genetic disorders.
  • The collaborative effort involved researchers, clinicians, and the ForeBatten Foundation, highlighting the potential of combining expertise to develop innovative therapies for rare diseases.

Novel Sarcoma Therapies Show Promise in Early-Phase Studies at ESMO 2024

  • Palbociclib plus retifanlimab demonstrates a 14.3% objective response rate in patients with advanced dedifferentiated liposarcoma, warranting further investigation.
  • Regorafenib maintenance therapy after doxorubicin-based chemotherapy significantly improves progression-free survival in patients with non-adipocytic soft tissue sarcomas.
  • Botensilimab plus balstilimab shows a 23% objective response rate across various refractory metastatic sarcomas, with a notable 39% in cutaneous or visceral angiosarcoma.

Testing the Addition of Cemiplimab to Palbociclib for the Treatment of Advanced Dedifferentiated Liposarcoma

NCT05694871Active, Not RecruitingPhase 2
Alliance for Clinical Trials in Oncology
Posted 6/7/2023

Study Comparing Efficacy of Doxorubicin With Trabectedin Followed by Trabectedin Versus Doxorubicine in Patients With Leiomyosarcoma

NCT02997358CompletedPhase 3
Gustave Roussy, Cancer Campus, Grand Paris
Posted 1/18/2017

Palbociclib and INCMGA00012 in People With Advanced Liposarcoma

NCT04438824Active, Not RecruitingPhase 2
Memorial Sloan Kettering Cancer Center
Posted 6/17/2020

Trial of Sunitinib and/or Nivolumab Plus Chemotherapy in Advanced Soft Tissue and Bone Sarcomas

NCT03277924CompletedPhase 1
Grupo Espanol de Investigacion en Sarcomas
Posted 5/31/2017

Fc-Engineered Anti-CTLA-4 Monoclonal Antibody in Advanced Cancer

NCT03860272Active, Not RecruitingPhase 1
Agenus Inc.
Posted 3/20/2019

Phase II Study of Regorafenib as Maintenance Therapy

NCT03793361CompletedPhase 2
Centre Oscar Lambret
Posted 5/15/2019

Duke Street Bio Receives EMA Approval for First-in-Human Trial of PARP1 Inhibitor DSB2455

  • Duke Street Bio has been granted EMA approval to begin a clinical trial for DSB2455, a highly-selective PARP1 inhibitor.
  • DSB2455 targets cancers with homologous recombination deficiencies, including BRCA mutations, potentially improving treatment outcomes.
  • Preclinical data suggests DSB2455 has a strong safety profile and compelling efficacy, offering a potential advancement in cancer treatment.
  • The trial aims to address the unmet medical need in patient populations where first-generation PARP inhibitors have limitations.

Tagrisso Approved for Unresectable Stage III EGFR-Mutated NSCLC

  • The FDA has approved Tagrisso (osimertinib) for adults with unresectable Stage III EGFR-mutated non-small cell lung cancer (NSCLC) after platinum-based chemoradiation therapy.
  • The approval was based on the LAURA Phase III trial, which showed Tagrisso significantly improved progression-free survival compared to placebo.
  • Tagrisso reduced the risk of disease progression or death by 84%, offering a new targeted therapy option for patients with these specific mutations.
  • This approval expands Tagrisso's use across all stages of EGFR-mutated NSCLC, addressing a critical unmet need for targeted treatments.

Durvalumab ± Tremelimumab in Combination With Platinum Based Chemotherapy in Untreated Extensive-Stage Small Cell Lung Cancer (CASPIAN)

NCT03043872Active, Not RecruitingPhase 3
AstraZeneca
Posted 3/27/2017

A Global Study to Assess the Effects of Osimertinib in Participants With EGFRm Stage IA2-IA3 NSCLC Following Complete Tumour Resection

NCT05120349Active, Not RecruitingPhase 3
AstraZeneca
Posted 2/21/2022

A Study of Amivantamab and Lazertinib Combination Therapy Versus Osimertinib in Locally Advanced or Metastatic Non-Small Cell Lung Cancer

NCT04487080Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 9/30/2020

Inventiva's Lanifibranor Phase III Trial NATiV3 Progresses in MASH/NASH Treatment

• Inventiva's NATiV3 trial for MASH/NASH treatment has enrolled over 85% of the targeted patients in the main cohort, with statistical powering expected to exceed 95% for both doses. • Blinded analyses of NATiV3 patients suggest positive biomarker evolution, aligning with Phase IIb NATIVE study results, and weight gain stabilizes after 24-36 weeks. • Last patient randomization in NATiV3 is anticipated in the first half of 2025, with topline results expected in the second half of 2026. • Inventiva secured €20.1 million through royalty certificates and is actively seeking additional financing to fund operations through Phase III topline results.

Vaccine Updates: FDA Approves Self-Administered FluMist, Updated Novavax COVID-19 Vaccine

• The FDA has approved FluMist, a nasal spray flu vaccine, for self- or caregiver-administration, making it the first flu vaccine with this capability, indicated for individuals aged 2 to 49 years. • Novavax's updated COVID-19 vaccine has received FDA emergency use authorization for individuals aged 12 years and older, set to be available in pharmacies and grocers across the United States. • GSK reported positive topline data from a phase 3 study for co-administration of its RSV vaccine (Arexvy) with its shingles vaccine (Shingrix) in adults 50 years and older. • The WHO has added the MVA-BN (Imvanex) vaccine to its prequalification list, marking the first mpox vaccine to receive this designation, indicated for adults and potentially for off-label use in younger populations.

EMA to Review Pfizer's Oxbryta Following Patient Deaths in Clinical Trials

  • The European Medicines Agency (EMA) is reviewing Pfizer's Oxbryta (voxelotor) after patient deaths occurred in two clinical trials.
  • One trial, involving patients at high stroke risk, showed a higher number of deaths in the Oxbryta treatment arm compared to placebo.
  • Another study on leg ulcers also reported a higher-than-anticipated number of deaths during the open-label portion of the trial.
  • Treatment with Oxbryta has been halted in both studies while the EMA assesses whether the deaths are linked to the drug.

ClinicalTrials.EU Platform Launches to Streamline Patient Recruitment for European Clinical Trials

  • ClinicalTrials.EU, a patient-centric platform, has officially launched across Europe to address challenges in clinical trial patient recruitment.
  • The platform aims to democratize patient access to clinical trials by providing comprehensive information in seven European languages.
  • By using AI pre-screening, ClinicalTrials.EU connects research centers with patients actively seeking therapies, especially for rare diseases.
  • The European clinical trials market is projected to reach USD 9.6 billion by 2029, with ClinicalTrials.EU poised to accelerate therapy development.

MesoPher Immunotherapy Shows Promise in Resected Pancreatic Cancer

  • A phase 2 trial of MesoPher, a dendritic cell-based immunotherapy, demonstrated a 64% 2-year recurrence-free survival rate in patients with resected pancreatic cancer.
  • The study also reported an 83% 2-year overall survival rate, suggesting a clinically meaningful immune response against the tumor.
  • MesoPher has received orphan drug designation from both the FDA and EMA for pancreatic cancer, potentially expediting its development and regulatory review.
  • A randomized phase 2/3 trial is being planned to further evaluate the efficacy of MesoPher in resected pancreatic cancer.

TC BioPharm Expands ACHIEVE AML Trial with New Site at Guys and St. Thomas Hospital

  • TC BioPharm has opened a new clinical trial site at Guys and St. Thomas Hospital in London to accelerate enrollment in the ACHIEVE study.
  • The ACHIEVE trial is a Phase II study evaluating TCB008, an allogeneic gamma-delta T cell therapy, for acute myeloid leukemia (AML) and MDS/AML patients.
  • Dr. Hugues de Lavallade, a hematologist with expertise in myeloid disorders, will serve as the Principal Investigator at the new site.
  • TC BioPharm anticipates data from the ACHIEVE clinical trial in the first half of 2025, with strong enrollment progress reported.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.