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Clinical Trial News

Bluebird Bio Announces Strategic Split into Separate Gene Therapy and Oncology Companies

  • Bluebird Bio plans to divide into two independent companies, with the core business focusing on rare disease gene therapies and a new spin-off dedicated to oncology development.
  • Current CEO Nick Leschly will lead the new oncology company while maintaining an executive chair position at Bluebird, with Andrew Obenshain stepping up as CEO of the gene therapy focused entity.
  • The strategic separation aims to streamline operations and improve capital allocation, with the gene therapy unit focusing on beta-thalassemia, cerebral adrenoleukodystrophy, and sickle cell disease programs.

FDA Approves Scemblix (asciminib) for Chronic Myeloid Leukemia with Novel Mechanism

  • The FDA has approved Scemblix (asciminib) for Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML-CP) in adults after two or more tyrosine kinase inhibitors (TKIs).
  • Scemblix demonstrated a significantly higher major molecular response (MMR) rate of 25% compared to bosutinib's 13% at 24 weeks in the Phase III ASCEMBL trial.
  • Scemblix is also approved for Ph+ CML-CP patients with the T315I mutation and offers a novel mechanism of action as a STAMP inhibitor.
  • Discontinuation rates due to side effects were notably lower with Scemblix (7%) compared to bosutinib (25%) in the ASCEMBL trial.

A Study of Oral Asciminib Versus Other TKIs in Adult Patients With Newly Diagnosed Ph+ CML-CP

NCT04971226Active, Not RecruitingPhase 3
Novartis Pharmaceuticals
Posted 10/6/2021

A Phase I Study of Oral Asciminib (ABL001) in Patients With CML or Ph+ ALL

NCT02081378CompletedPhase 1
Novartis Pharmaceuticals
Posted 4/24/2014

Triplet Therapy of Durvalumab, Tremelimumab, and Paclitaxel Halted in Biliary Tract Cancer Trial Due to Safety Concerns

  • The IMMUNOBIL PRODIGE 57 trial, evaluating durvalumab and tremelimumab with or without paclitaxel in advanced biliary tract cancer, faced safety issues.
  • The combination arm with paclitaxel was stopped early due to dose-limiting toxicities, including anaphylactic reactions.
  • The trial will continue with only the durvalumab and tremelimumab combination arm to further assess its efficacy and safety.
  • This study highlights the importance of careful safety monitoring when combining immune checkpoint inhibitors with chemotherapy in biliary tract cancer.

Durvalumab + Tremelimumab ± Paclitaxel in Advanced BTC After Platinum Chemotherapy.

NCT03704480Active, Not RecruitingPhase 2
GERCOR - Multidisciplinary Oncology Cooperative Group
Posted 11/9/2018

Durvalumab or Placebo in Combination With Gemcitabine/Cisplatin in Patients With 1st Line Advanced Biliary Tract Cancer (TOPAZ-1)

NCT03875235Active, Not RecruitingPhase 3
AstraZeneca
Posted 4/16/2019

Pembrolizumab (MK-3475) Plus Gemcitabine/Cisplatin Versus Placebo Plus Gemcitabine/Cisplatin for First-Line Advanced and/or Unresectable Biliary Tract Carcinoma (BTC) (MK-3475-966/KEYNOTE-966)

NCT04003636CompletedPhase 3
Merck Sharp & Dohme LLC
Posted 9/24/2019

Phase II Clinical Trial of NIVO-IPI-TAXANE in Untreated Metastatic NSCLC

NCT03573947CompletedPhase 2
Jeffrey Clarke
Posted 10/2/2018

A Combination of Avelumab and Taxane (AVETAX) for Urothelial Cancer

NCT03575013Active, Not RecruitingPhase 1
Yousef Zakharia
Posted 10/29/2018

Gemcitabine Plus Cisplatin With or Without Bintrafusp Alfa (M7824) in Participants With 1L BTC

NCT04066491TerminatedPhase 2
EMD Serono Research & Development Institute, Inc.
Posted 9/20/2019

Role of Immunotherapy in Bladder Cancer

Immunotherapy, particularly checkpoint inhibitors, has become a significant treatment option for bladder cancer, with established roles in second-line therapy for metastatic urothelial cancers and as maintenance therapy post-platinum-based chemotherapy. It is also suitable for first-line treatment in PD-L1 high or platinum-ineligible patients and offers an alternative for high-risk BCG-refractory patients.

Biomarkers Identified for Camrelizumab-Apatinib Combination in Advanced Triple-Negative Breast Cancer

  • A phase II trial analysis reveals several potential biomarkers predicting response to camrelizumab plus apatinib in advanced triple-negative breast cancer, which previously showed a 43.3% objective response rate.
  • Higher baseline tumor-infiltrating lymphocytes, increased CD8+ T cells during treatment, and lower plasma levels of HGF and IL-8 were significantly associated with better clinical outcomes and treatment response.
  • Blood immune profiles showing higher baseline CD4+ T cells and B cell proportions, along with changes in plasma biomarkers like TIM-3 and CD152 during therapy, may help identify patients most likely to benefit from this combination therapy.

I-SPY COVID Trial: Adaptive Platform Design Accelerates COVID-19 Therapeutic Discovery

  • The I-SPY COVID trial utilized an adaptive platform design to rapidly evaluate potential treatments for severe COVID-19, addressing the urgent need for effective therapies.
  • This phase 2 trial design allowed for the parallel assessment of multiple therapeutic agents, expediting the identification of promising candidates for phase 3 testing.
  • The trial's adaptive nature and concurrent control arm facilitated adjustments to the evolving standard of care and emerging viral variants, enhancing the reliability of results.
  • I-SPY COVID demonstrated the feasibility of balancing pragmatism, safety, and discovery in clinical trials during a pandemic, providing valuable lessons for future research.

Innovative CAR T-cell and Oncolytic Virus Therapies Show Promise in Treating Aggressive Cancers

Elicera Therapeutics AB is advancing novel treatments for aggressive cancers, including a CAR T-cell therapy targeting CD20 for relapsed Non-Hodgkin's B-cell lymphoma and a new CAR T-cell therapy for glioblastoma. Additionally, their oncolytic virus, AdVince, is in clinical trials for neuroendocrine tumors, showcasing potential for both local tumor destruction and systemic immune response.

Study of Recombinant Adenovirus AdVince in Patients With Neuroendocrine Tumors; Safety and Efficacy

NCT02749331Active, Not RecruitingPhase 1
Uppsala University
Posted 3/1/2016

Rakuten Medical Acquires Critical IRDye 700DX Assets from LI-COR to Secure Photoimmunotherapy Supply Chain

Drug ApprovalOncology
  • Rakuten Medical acquired phthalocyanine dyes including IRDye 700DX from LI-COR Biosciences to ensure consistent supply for its Illuminox cancer therapy platform.
  • The acquisition supports ongoing ASP-1929 clinical trials across multiple oncology indications and strengthens the company's photoimmunotherapy development capabilities.
  • ASP-1929 has already received conditional marketing approval in Japan for head and neck cancer and is currently being studied in a global Phase 3 trial.
  • The strategic move follows recent clinical milestones including first patient enrollment in a Phase 1b/2 combination trial with anti-PD1 therapy at MD Anderson Cancer Center.

An Open-label Study Using ASP-1929 Photoimmunotherapy in Combination With Anti-PD1 Therapy in EGFR Expressing Advanced Solid Tumors

NCT04305795Active, Not RecruitingPhase 1
Rakuten Medical, Inc.
Posted 12/21/2020

COVID-19 Pandemic Reshapes Patient-Centric Clinical Trial Landscape in 2020

  • The COVID-19 pandemic catalyzed unprecedented changes in clinical trials, with remote monitoring evolving from an optional feature to an essential requirement for study continuation.
  • Clinical trial recruitment saw remarkable success during the pandemic, with studies like the RECOVERY Trial enrolling over 2,000 participants across 16 NHS sites in just three weeks.
  • While the pandemic accelerated certain patient-centric practices, the urgency of COVID-19 research sometimes compromised patient engagement, revealing areas for improvement in the clinical trial ecosystem.

Palforzia: A New Hope for Children with Peanut Allergy

Palforzia, a treatment for peanut allergy in children aged 1 to 17, has shown promising results in clinical trials, enabling some patients to tolerate small amounts of peanut protein with only mild symptoms. The treatment involves a phased approach under medical supervision and requires a prescription. Despite potential side effects, the European Medicines Agency has concluded that the benefits of Palforzia outweigh its risks.

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