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Emapalumab Enhances Intervention-Free Survival in Pediatric HLH Patients Undergoing Stem Cell Transplant

  • A new study shows that emapalumab improves outcomes for children with hemophagocytic lymphohistiocytosis (HLH) undergoing stem cell transplantation.
  • Emapalumab treatment was associated with a lower incidence of mixed chimerism and severe mixed chimerism post-transplant.
  • The study found that 73% of children treated with emapalumab achieved intervention-free survival, compared to 43% without the treatment.
  • Infants, who are at higher risk, showed even more significant improvement in intervention-free survival with emapalumab treatment.

MOST Trial: Anti-Clotting Drugs Fail to Improve Outcomes in Stroke Patients

  • The MOST trial investigated the efficacy of adding argatroban or eptifibatide to intravenous thrombolysis for stroke patients to prevent further clots.
  • Results indicated that neither argatroban nor eptifibatide improved patient outcomes and had a less than 1% probability of being helpful.
  • The study, conducted across 57 U.S. sites, found that these drugs were linked to greater incidences of disability and mortality within three months post-treatment.
  • Researchers emphasize that negative trials like MOST are crucial for informing future research and developing new anticoagulant treatments for stroke.

FDA Accepts Axsome's NDA Resubmission for AXS-07 in Acute Migraine Treatment

  • The FDA has accepted Axsome Therapeutics' NDA resubmission for AXS-07, a novel oral treatment for acute migraine, with a PDUFA date set for January 31, 2025.
  • AXS-07 combines MoSEIC meloxicam and rizatriptan to provide rapid and consistent migraine relief, potentially addressing the unmet needs of patients dissatisfied with current treatments.
  • Migraine affects an estimated 39 million Americans and is a leading cause of neurological disability, highlighting the importance of new therapeutic options like AXS-07.
  • AXS-07 is protected by over 200 patents extending to at least 2038, underscoring its potential long-term market exclusivity if approved.

Plinabulin Combination Shows Tolerability in Recurrent Small Cell Lung Cancer Trial

  • A Phase I/II trial evaluated plinabulin with nivolumab and ipilimumab in recurrent small cell lung cancer (SCLC) patients, focusing on safety and efficacy.
  • The combination was found to be tolerable, with a recommended Phase 2 dose of plinabulin at 30 mg/m², though common adverse events included vomiting and nausea.
  • The trial did not meet its primary endpoint of a median progression-free survival of 3.5 months, but some patients exhibited durable responses to the treatment.
  • The rate of grade 3 or higher immune-related adverse events was lower than anticipated, suggesting a potentially manageable safety profile for the combination therapy.

A Phase I/II Study of Nivolumab, Ipilimumab and Plinabulin in Patients With Recurrent Small Cell Lung Cancer

NCT03575793CompletedPhase 1
Salma Sabbour
Posted 9/6/2018

RGX-121 Gene Therapy Shows Sustained Efficacy in Hunter Syndrome Trial

  • RGX-121 gene therapy demonstrates an 85% median reduction in cerebrospinal fluid levels of heparan sulfate D2S6, a key biomarker in Hunter syndrome.
  • The CAMPSIITE trial data supports RGX-121 as a potential first gene therapy for Hunter syndrome, with sustained effects observed for up to two years.
  • REGENXBIO plans to initiate a rolling biologics license application (BLA) using the accelerated approval pathway in Q3 2024 based on these results.
  • A significant number of patients remained free from enzyme replacement therapy (ERT) post-treatment, suggesting systemic activity of RGX-121.

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

NCT03566043Active, Not RecruitingPhase 2
REGENXBIO Inc.
Posted 9/27/2018

Lofexidine Trial Launched for Neonatal Opioid Withdrawal Syndrome

  • Marshall University is conducting a clinical trial to assess oral lofexidine, a non-opioid medication, for treating Neonatal Opioid Withdrawal Syndrome (NOWS).
  • The Phase 2 trial aims to evaluate the safety and pharmacokinetics of lofexidine in newborns exposed to opioids prenatally.
  • Unlike opioid-based treatments, lofexidine does not produce euphoria or addiction potential, offering a potentially safer alternative.
  • The study will monitor NOWS symptoms using the Modified Finnegan Neonatal Abstinence Scoring Tool (mFNAST) and assess treatment duration.

Pharmacokinetic and Safety Study of Oral Lofexidine in Neonates Experiencing Opioid Withdrawal Due to Intrauterine Exposure to Opioids

NCT06047834RecruitingPhase 2
USWM, LLC (dba US WorldMeds)
Posted 10/2/2023

Oleclumab Plus Chemotherapy Shows Promise in Metastatic Pancreatic Cancer

  • A Phase Ib/II trial evaluated oleclumab, with or without durvalumab, combined with chemotherapy in metastatic pancreatic ductal adenocarcinoma (mPDAC).
  • The combination of oleclumab plus chemotherapy demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS).
  • The study suggests oleclumab could offer a new treatment option for mPDAC, addressing a critical unmet need in this aggressive cancer.
  • Further research is warranted to confirm these findings and explore the full potential of oleclumab in pancreatic cancer treatment.

AI Model Predicts Sepsis Trajectories to Enhance Clinical Trial Enrollment

  • An AI model was developed to predict sepsis patient trajectories (rapid death, persistent illness, or recovery) using data from MIMIC-IV and eICU-CRD databases.
  • The gradient boosting machine (GBM) model demonstrated the best performance, achieving an AUROC of 0.807 for predicting persistent illness in internal validation.
  • Conformal prediction was used to estimate model uncertainty, reducing prediction errors by incorporating confidence measures and enabling human review of uncertain outcomes.
  • A web-based calculator was created to assist clinicians in understanding the AI model's outputs and facilitate its integration into clinical decision-making.

FDA Roundup: TCR T-Cell Therapy Approved, CAR-T REMS Modified, and Clinical Holds Lifted

  • The FDA granted accelerated approval to Adaptimmune's afami-cel (Tecelra) for unresectable or metastatic synovial sarcoma in patients who have received prior chemotherapy.
  • Risk Evaluation and Mitigation Strategies (REMS) for autologous CAR-T immunotherapies were modified by the FDA to reduce the burden on healthcare systems.
  • A clinical hold on 4D Molecular Therapeutics' Fabry disease gene therapy 4D-310 trial was lifted, with plans to resume enrollment before the end of 2024.
  • Biosyngen's CAR-T therapy BRG01 has been cleared by the FDA for a pivotal phase 2 clinical trial in patients with Epstein-Barr virus (EBV)-positive relapsed/metastatic nasopharyngeal carcinoma.

Expanded/Activated Gamma Delta T-cell Infusion Following Hematopoietic Stem Cell Transplantation and Post-transplant Cyclophosphamide

NCT03533816RecruitingPhase 1
University of Kansas Medical Center
Posted 1/31/2020

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease

NCT04519749Active, Not RecruitingPhase 1
4D Molecular Therapeutics
Posted 9/1/2020

Bill Gates Optimistic About Progress in M72 Tuberculosis Vaccine Clinical Trial

  • Bill Gates expressed optimism about the M72 tuberculosis vaccine clinical trial, anticipating a 70% efficacy in reducing the disease.
  • The ongoing Phase 3 trial, costing $500 million, is a collaboration between GSK, the Wellcome Trust, and the Gates MRI.
  • The M72/AS01E vaccine aims to prevent pulmonary TB in adolescents and adults, potentially marking the first new TB vaccine in over a century.
  • Current TB treatments require a nine-month regimen, and multidrug-resistant TB remains a significant public health challenge, spurring the need for new drugs and vaccines.
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