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Clinical Trial News

DirectRx to Distribute Novel COPD Therapy Ohtuvayre in the U.S.

• DirectRx, a specialty pharmacy, will distribute Ohtuvayre (ensifentrine), a new FDA-approved inhaled maintenance therapy for chronic obstructive pulmonary disease (COPD). • Ohtuvayre, developed by Verona Pharma, is priced at $2,950 per month and represents the first new COPD therapy mechanism in over two decades. • Clinical trials (ENHANCE-1 and ENHANCE-2) demonstrated that Ohtuvayre significantly improved lung function and reduced exacerbation rates in COPD patients. • Ohtuvayre's active component, ensifentrine, inhibits phosphodiesterase 3 and 4, reducing inflammation and widening airways, offering a novel approach to COPD management.

A Phase 3 Trial to Evaluate the Safety and Efficacy of Ensifentrine in Patients With COPD

NCT04542057CompletedPhase 3
Verona Pharma plc
Posted 9/22/2020

A Phase 3 Clinical Trial to Evaluate the Safety and Efficacy of Ensifentrine in Patients With COPD

NCT04535986CompletedPhase 3
Verona Pharma plc
Posted 9/29/2020

FDA Clarifies Post-Approval Changes and New Indications for Biosimilars

  • The FDA has clarified guidelines for post-approval changes to biosimilars, including manufacturing process modifications and quality control adjustments.
  • Biosimilars can now pursue new indications not included in the reference product's license without being classified as a new biological drug.
  • Changes to the route of administration and formulation are permissible for biosimilars, provided intellectual property rights are not infringed.
  • A proposed Senate bill (S-150) aims to prevent reference product manufacturers from securing patents on minor modifications, further supporting biosimilar development.

Glibenclamide Shows Promise in Improving Ambulation After Large Hemispheric Infarction

  • Post-hoc analysis of the Phase 3 CHARM trial indicates that intravenous glibenclamide (CIRARA) significantly improves independent ambulation in patients with large hemispheric infarction (LHI).
  • Patients with lesion volumes under 125 mL treated with CIRARA showed a significantly higher odds ratio for achieving favorable modified Rankin Scale (mRS) scores.
  • CIRARA, when combined with endovascular thrombectomy (EVT), demonstrated a substantial increase in the ability of LHI patients to walk independently at 12 months.
  • Remedy Pharmaceuticals is planning a confirmatory Phase 3 study of CIRARA in LHI, encouraged by the sustained effects and impact on patient ambulation.

Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous BIIB093 (Glibenclamide) for Severe Cerebral Edema Following Large Hemispheric Infarction

NCT02864953TerminatedPhase 3
Remedy Pharmaceuticals, Inc.
Posted 8/29/2018

AI-Powered Model Aims to Decode Genetic Variants in Stargardt Disease

  • Researchers are developing an AI model to classify genetic variants of the ABCA4 gene, crucial for understanding Stargardt disease.
  • The model integrates genetic, clinical, and structural data to predict the pathogenicity of previously unclassified variants.
  • This research aims to personalize medicine by identifying appropriate clinical trials and therapies for individuals with Stargardt disease.
  • The NIH-funded project addresses the challenge that nearly 40% of ABCA4 genetic variants are currently unclassified, hindering accurate diagnosis.

RDCA-DAP Boosts Cystic Fibrosis Research with New Data Sets

  • The RDCA-DAP has acquired new datasets from the Cystic Fibrosis Therapeutics Development Network to accelerate cystic fibrosis (CF) research and therapy development.
  • The data sharing initiative seeks to enhance understanding of CF progression, design new clinical outcome measures and biomarkers, and develop disease models.
  • By integrating data into the RDCA-DAP, researchers worldwide can access and leverage data, potentially leading to breakthroughs in CF care.
  • The RDCA-DAP is an initiative run by the Critical Path Institute (C-Path) and funded by the FDA to provide a centralized platform of patient data for rare diseases.

FDA Backs Alpha-Synuclein Assay for Parkinson's Trials, Aiding Drug Development

  • The FDA supports using the α-synuclein seed amplification assay (αSyn-SAA) biomarker in clinical trials for Parkinson's disease and related conditions, potentially accelerating drug development.
  • The decision follows collaboration between The Michael J. Fox Foundation and the Critical Path Institute, leveraging data from the Parkinson's Progression Markers Initiative.
  • αSyn-SAA demonstrates high accuracy in detecting Parkinson's early, even before symptoms, enabling better-targeted therapies and efficient clinical trials.
  • Amprion's SAAmplify-αSYN Biomarker Test is currently the only validated synSAA assay available in the U.S. for improving the diagnosis of Parkinson’s and other synucleinopathies.

Study of Two Digital Therapeutics for the Prevention of Episodic Migraine

NCT05853900CompletedNot Applicable
Click Therapeutics, Inc.
Posted 3/28/2023

Docetaxel Shows Promise in Reducing Neuropathy for Black Breast Cancer Patients

  • A clinical study reveals that docetaxel is associated with significantly less neuropathy in Black breast cancer patients compared to paclitaxel, potentially improving treatment outcomes.
  • The research addresses disparities in breast cancer treatment, as Black women are more likely to experience severe chemotherapy-induced peripheral neuropathy.
  • The study's design involved collaboration with Black patient advocates to ensure inclusivity and relevance, marking a significant step in addressing healthcare disparities.
  • Findings suggest docetaxel may be the preferred chemotherapy drug for Black women with breast cancer, leading to fewer dose reductions and improved quality of life.

Xolair Receives FDA Approval for Food Allergies After Two Decades

  • Xolair, initially approved for asthma in 2003, has now been FDA-approved for treating food allergies, offering relief to millions with severe allergic reactions.
  • The drug works by intercepting IgE molecules, key triggers of allergic reactions, and has shown significant promise in clinical trials, allowing patients to tolerate previously dangerous foods.
  • A major clinical trial, OUtMATCH, demonstrated that two-thirds of participants on Xolair could consume the equivalent of two and a half peanuts after 16 weeks of treatment.
  • While not a cure, Xolair provides a protective buffer against accidental allergen exposure and can be used in conjunction with oral immunotherapy to enhance safety and effectiveness.

European Commission Approves Encorafenib and Binimetinib Combination for BRAF V600E-Mutant NSCLC

  • The European Commission has approved encorafenib (BRAFTOVI) in combination with binimetinib (MEKTOVI) for treating adult patients with advanced non-small cell lung cancer (NSCLC) harboring a BRAFV600E mutation.
  • The approval is based on the Phase II PHAROS trial, which demonstrated a 75% objective response rate in treatment-naive patients and 46% in previously treated patients.
  • Updated results from the PHAROS trial showed that 64% of treatment-naive patients maintained a response for at least 12 months, with a median duration of response of 40 months.
  • The combination offers a new targeted therapy option for NSCLC patients with BRAFV600E mutations, addressing a significant unmet need in this population.

An Open-label Study of Encorafenib + Binimetinib in Patients With BRAFV600-mutant Non-small Cell Lung Cancer

NCT03915951Active, Not RecruitingPhase 2
Pfizer
Posted 6/4/2019

Zealand Pharma's Dapiglutide Shows Promising Weight Loss in Phase 1b Trial

  • Zealand Pharma's dapiglutide demonstrated up to 8.3% placebo-adjusted mean weight loss after 13 weekly doses in a Phase 1b trial.
  • The trial assessed dapiglutide at doses up to 13 mg, showing it was safe and well-tolerated, with gastrointestinal adverse events consistent with incretin-based therapies.
  • A Phase 2b trial in overweight and obese individuals is planned for the first half of 2025, following further evaluation of higher doses up to 26 mg in an ongoing trial.
  • Dapiglutide, a long-acting GLP-1/GLP-2 receptor dual agonist, is being developed for weight management and potential benefits in obesity-related comorbidities.

A Trial Assessing Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ZP7570

NCT06000891TerminatedPhase 1
Zealand Pharma
Posted 9/15/2023

A Multiple Ascending Dose Trial Assessing Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ZP7570

NCT04612517CompletedEarly Phase 1
Zealand Pharma
Posted 10/26/2020

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