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Clinical Trial News

NO-CUT Trial: Non-Operative Management Shows Promise in Rectal Cancer

  • The NO-CUT trial reveals that non-operative management (NOM) following total neoadjuvant therapy (TNT) maintains distant relapse-free survival in pMMR locally advanced rectal cancer patients.
  • A clinical complete response (cCR) was observed in 26% of patients, allowing them to proceed with NOM, with 85% achieving organ preservation.
  • Circulating tumor DNA (ctDNA) analysis post-TNT predicts clinical response and distant relapse-free survival, potentially refining patient selection for NOM.
  • The 3-year distant relapse-free survival rate was 85.8% for ctDNA-negative patients versus 64.0% for ctDNA-positive patients (HR, 3.23; P = .035).

Aramchol Shows Significant Anti-Fibrotic Effects in MASH Patients, Phase 3 Trial Results Published in Hepatology

• Aramchol 300mg BID demonstrates a significant anti-fibrotic effect in patients with metabolic dysfunction-associated steatohepatitis (MASH), as confirmed by multiple objective measurements. • The Phase 3 trial (ARCON) utilized NASH CRN, paired ranked reading, and Artificial Intelligence (AI) quantitative digital analysis to validate the data. • Digital pathology images analysis (DIA) provides continuous histological fibrosis scores, quantifying anti-fibrotic effects with greater sensitivity than conventional pathology. • Galmed believes Aramchol's unique mechanism of action positions it as a potent anti-fibrotic compound for combination therapy in MASH treatment.

Semaglutide Shows Promise in Reducing Opioid Overdose Risk, Study Suggests

  • A recent study indicates that semaglutide, a drug primarily used for diabetes and weight management, may significantly reduce the risk of opioid overdose.
  • The research suggests a potential link between semaglutide's effects on appetite regulation and decreased cravings for opioids.
  • Further investigation is warranted to fully understand the mechanisms behind this association and to explore semaglutide's role in addressing the opioid crisis.

Histotripsy Shows Promise in Non-Invasive Liver Tumor Treatment

• A clinical trial of histotripsy for liver tumors met its goals for technical success and safety, supporting early clinical adoption. • The non-invasive procedure uses focused ultrasound to mechanically break down tumors, offering an alternative to traditional treatments. • Histotripsy achieved a 95% technical success rate in the trial, with a complication rate within reported ranges for other local techniques. • Researchers are optimistic about histotripsy's potential to improve treatment for primary and metastatic liver tumors, with ongoing trials exploring its use in kidney tumors.

The HistoSonics System for Treatment of Primary and Metastatic Liver Tumors Using Histotripsy

NCT04572633Active, Not RecruitingNot Applicable
HistoSonics, Inc.
Posted 1/27/2021

Acasti Completes Enrollment in Phase 3 STRIVE-ON Trial of GTX-104 for aSAH

  • Acasti Pharma has completed patient enrollment in its Phase 3 STRIVE-ON trial evaluating GTX-104 for aneurysmal subarachnoid hemorrhage (aSAH).
  • The STRIVE-ON trial is comparing intravenous GTX-104 to oral nimodipine in 100 patients hospitalized with aSAH across 25 U.S. hospitals.
  • GTX-104, an injectable formulation of nimodipine, aims to address challenges associated with oral administration and improve patient outcomes.
  • Acasti anticipates STRIVE-ON data readout in early 2025 and plans to submit an NDA to the FDA in the first half of 2025.

Safety and Tolerability of GTX-104 Compared With Oral Nimodipine in Patients With aSAH

NCT05995405Active, Not RecruitingPhase 3
Acasti Pharma Inc.
Posted 10/20/2023

Rosiglitazone and Trametinib Combination Shows Promise in Aggressive Bladder Cancer

  • A study in mice found that rosiglitazone and trametinib synergistically induce tumor cell death in muscle-invasive bladder cancer.
  • The drug combination shifted aggressive tumor cells to a more benign molecular subtype, improving the prognosis.
  • Treatment with both drugs led to a 91% reduction in tumor size, with many cases showing undetectable residual tumors.
  • Researchers are optimistic about initiating clinical trials to assess the efficacy of this combination therapy in humans.

AyuVis' AVR-48 Receives FDA Fast Track for Preventing Bronchopulmonary Dysplasia

  • AyuVis Research Inc. received FDA Fast Track designation for AVR-48, a drug aimed at preventing bronchopulmonary dysplasia (BPD) in preterm infants.
  • AVR-48 targets the long-term inflammation and damage from oxygen treatments that lead to BPD, a major cause of mortality and complications in premature babies.
  • The Fast Track designation will expedite the review process, potentially bringing AVR-48 to patients sooner and improving outcomes for infants at risk of BPD.
  • AyuVis, incubated at The University of North Texas Health Science Center, highlights the role of innovation labs in nurturing healthcare startups.

Inebilizumab Shows Promise in Myasthenia Gravis, Phase 3 Data Suggests

  • Phase 3 MINT trial data indicates inebilizumab significantly improves Myasthenia Gravis Activities of Daily Living (MG-ADL) scores compared to placebo.
  • The study included patients with both anti-acetylcholine receptor (AChR) and muscle-specific tyrosine kinase (MuSK) antibody-positive generalized myasthenia gravis (gMG).
  • Amgen is planning regulatory submissions for inebilizumab as a potential new treatment option for generalized myasthenia gravis (gMG).
  • Ongoing trials are exploring new therapeutic approaches, including bispecific nanoantibodies and complement inhibitors, to address unmet needs in gMG treatment.

A Study of Inebilizumab Efficacy and Safety in IgG4- Related Disease

NCT04540497Active, Not RecruitingPhase 3
Amgen
Posted 12/4/2020

Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy

NCT04811716CompletedPhase 2
Regeneron Pharmaceuticals
Posted 7/29/2021

A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis

NCT06298552Active, Not RecruitingPhase 3
argenx
Posted 4/16/2024

Evaluating the Long-Term Safety and Tolerability of Efgartigimod PH20 SC Administered Subcutaneously in Patients With Generalized Myasthenia Gravis

NCT04818671CompletedPhase 3
argenx
Posted 4/26/2021

Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis

NCT05556096Active, Not RecruitingPhase 3
Alexion Pharmaceuticals, Inc.
Posted 11/21/2022

A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis

NCT05070858Active, Not RecruitingPhase 3
Regeneron Pharmaceuticals
Posted 12/14/2021

Evaluating the Pharmacodynamic Noninferiority of Efgartigimod PH20 SC Administered Subcutaneously as Compared to Efgartigimod Administered Intravenously in Patients With Generalized Myasthenia Gravis

NCT04735432CompletedPhase 3
argenx
Posted 2/5/2021

Myasthenia Gravis Inebilizumab Trial

NCT04524273Active, Not RecruitingPhase 3
Amgen
Posted 10/15/2020

An Efficacy and Safety Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness

NCT03669588CompletedPhase 3
argenx
Posted 8/22/2018

Fever Prevention Post-Stroke Reduces Fever Burden, But Fails to Improve Functional Outcomes

• A randomized clinical trial assessed the effectiveness of automated temperature management in preventing fever in acute vascular brain injury patients. • The study found that temperature modulation significantly reduced daily mean fever burden compared to standard care. • Despite reduced fever burden, there was no significant difference in functional outcomes at 3 months between the two groups. • The trial suggests that while fever prevention is achievable, it may not translate to improved patient recovery after stroke.

Impact of Fever Prevention in Brain Injured Patients

NCT02996266TerminatedNot Applicable
C. R. Bard
Posted 1/1/2017

Advances in Immunotherapy and Precision Medicine Transform Endometrial Cancer Treatment

• Recent FDA approvals of pembrolizumab, durvalumab, and dostarlimab combined with chemotherapy have significantly improved outcomes for advanced endometrial cancer. • Ongoing trials are exploring PARP inhibitors, CDK 4/6 inhibitors, and antibody-drug conjugates to further refine treatment strategies for endometrial cancer. • Molecular testing is increasingly crucial for personalizing treatment, with MMR status guiding immunotherapy eligibility and TCGA categories predicting outcomes. • Despite progress, the rising incidence of endometrial cancer and the need for better early detection strategies remain critical challenges.

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