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Clinical Trial News

Global Research Community Applies COVID-19 Lessons to Combat African Mpox Emergency

• The World Health Organization has declared mpox outbreak in Africa a Public Health Emergency of International Concern, prompting urgent calls for enhanced treatment accessibility and research efforts.
• Researchers are leveraging COVID-19 pandemic experiences, including adaptive trial designs, real-time data collection, and streamlined regulatory processes, to accelerate mpox vaccine development.
• While infrastructure and expertise from COVID-19 research could benefit mpox trials, challenges remain in financing, logistics, and healthcare access in affected African regions.

Suzetrigine: Hope or Hype for New Non-Opioid Pain Relief?

  • The FDA is considering suzetrigine, a non-opioid analgesic from Vertex Pharmaceuticals, for priority review, potentially marking a new class of pain medication in over two decades.
  • Suzetrigine received 'Fast Track' and 'Breakthrough Therapy' designations for moderate-to-severe acute pain, but is currently studied only for diabetic peripheral neuropathy.
  • While hailed as a potential solution to the opioid crisis, questions remain about suzetrigine's efficacy compared to existing treatments, leading to skepticism about its true impact.
  • The FDA's eagerness to approve non-opioid alternatives may be driving the fast-track designation, raising concerns whether the drug's benefits justify the accelerated approval process.

Tacrolimus Eye Drops with Cyclodextrins Show Improved Safety and Compliance in Ocular Inflammatory Pathologies

  • A new formulation of 0.015% tacrolimus eye drops with hydroxypropyl-beta-cyclodextrin (HPβCD) was compared to a 0.03% tacrolimus formulation with ethanol (TE).
  • The HPβCD formulation (TCD) demonstrated comparable effectiveness to TE, with significant improvements in patient-reported burning sensations and ocular pain.
  • Patient adherence increased significantly with TCD, likely due to the removal of ethanol, an irritant excipient, leading to better tolerability and reduced ocular surface toxicity.
  • The study suggests that TCD is a safer and more compliant alternative for managing ocular inflammatory pathologies, with potential for wider adoption in hospital pharmacy departments.

Hydroxychloroquine Shows Moderate COVID-19 Prevention in Large Clinical Trial

  • A large, double-blind, placebo-controlled trial (COPCOV) found a 15% reduction in symptomatic COVID-19 with hydroxychloroquine compared to placebo.
  • The study also demonstrated a significant reduction in all-cause respiratory illness and fewer workdays lost due to illness among those taking hydroxychloroquine.
  • Hydroxychloroquine was well-tolerated and safe in the study, which included over 4,600 participants across 11 countries from 2020 to 2022.
  • Researchers emphasize that current immunity from vaccines and natural infections has reduced COVID-19 severity, making hydroxychloroquine unnecessary for prevention today.

Survodutide Shows Promise in Obesity and MASH with Significant Weight and Blood Pressure Reduction

• Survodutide, a novel dual GCGR/GLP-1R agonist, demonstrates significant weight reduction in overweight or obese patients, with nearly 40% achieving over 20% body weight reduction after 46 weeks. • Phase II trial results indicate survodutide substantially reduces blood pressure in patients with metabolic dysfunction-associated steatohepatitis (MASH), showing promise in this area. • The dual mechanism of action of survodutide offers benefits in lowering body weight and promoting blood pressure reduction without causing muscle loss, according to study findings. • Adverse events leading to discontinuation were mainly due to rapid dose escalation, suggesting a more gradual approach could mitigate this issue and improve tolerability.

A Study to Test Whether Different Doses of BI 456906 Help People With Overweight or Obesity to Lose Weight

NCT04667377CompletedPhase 2
Boehringer Ingelheim
Posted 3/8/2021

A Study to Test Safety and Efficacy of Survodutide (BI456906) in Adults With Non-alcoholic Steatohepatitis (NASH) and Fibrosis (F1-F3)

NCT04771273CompletedPhase 2
Boehringer Ingelheim
Posted 4/27/2021

Nivolumab and Nab-Paclitaxel Show Promise in Muscle-Invasive Bladder Cancer

  • A phase II trial (NURE-Combo) evaluated neoadjuvant nivolumab plus nab-paclitaxel followed by adjuvant nivolumab in muscle-invasive bladder cancer patients ineligible for cisplatin.
  • The study met its primary endpoint, with 32.3% of patients achieving a pathologic complete response (ypT0N0) after neoadjuvant treatment and radical cystectomy.
  • The combination demonstrated a manageable safety profile, with grade 3/4 treatment-related adverse events occurring in 25.8% of patients.
  • These findings suggest a potential non-cisplatin-based chemoimmunotherapy approach for muscle-invasive bladder cancer, warranting further investigation.

Ultrasound Elastography Variability Defined for Liver Disease Clinical Trials

  • A recent study published in Radiology identifies variability in ultrasound elastography measurements of liver tissue, crucial for interpreting disease progression in clinical trials.
  • The study, part of the FNIH's NIMBLE project, evaluated SWE and VCTE ultrasound technologies in patients with metabolic dysfunction-associated steatotic liver disease (MASLD).
  • Researchers found that differences of less than 30.7% in SWE and 35.6% in VCTE measurements may reflect technical variability rather than actual changes in liver stiffness.
  • These findings will help clinicians avoid unnecessary changes in patient management and guide future studies validating ultrasound technology for clinical trials.

Rilertinib Demonstrates Superior Survival in EGFR-Mutated NSCLC in Phase 3 Trial

  • Phase 3 trial reveals rilertinib significantly improves overall survival in patients with EGFR-mutated non-small cell lung cancer (NSCLC).
  • The study highlights rilertinib's potential as a novel treatment option for patients who have progressed on prior EGFR tyrosine kinase inhibitors.
  • Rilertinib showcases a manageable safety profile, offering a promising alternative with improved efficacy in advanced NSCLC.
  • These findings support rilertinib's role in addressing the unmet need for effective therapies in EGFR-mutated NSCLC, potentially changing treatment paradigms.

Tremfya (Guselkumab) Approved by FDA for Ulcerative Colitis Treatment

  • The FDA has approved Tremfya (guselkumab) for treating adults with moderately to severely active ulcerative colitis, marking a new treatment option.
  • Tremfya is the first and only approved fully-human, dual-acting monoclonal antibody that blocks IL-23 while also binding to CD64.
  • Clinical trials showed significant improvements in clinical remission and endoscopic remission compared to placebo, supporting the approval.
  • Tremfya is administered intravenously for induction, followed by subcutaneous injections for maintenance, offering flexible dosing regimens.

A Study of Guselkumab in Participants With Moderately to Severely Active Ulcerative Colitis

NCT04033445Active, Not RecruitingPhase 2
Janssen Research & Development, LLC
Posted 9/26/2019

Ascendis Pharma's Yorvipath Receives Orphan Drug Exclusivity in the U.S. for Hypoparathyroidism

  • Ascendis Pharma's Yorvipath (palopegteriparatide) has been granted Orphan Drug Exclusivity by the U.S. FDA for treating hypoparathyroidism in adults.
  • The exclusivity provides seven years of market protection in the U.S., preventing similar drugs from being marketed for the same indication.
  • Yorvipath is a prodrug of parathyroid hormone (PTH [1-34]) designed for once-daily administration, offering continuous PTH exposure over 24 hours.
  • Hypoparathyroidism, affecting 70,000-90,000 people in the U.S., is a rare endocrine disorder caused by insufficient parathyroid hormone levels.

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