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Clinical Trial News

Palliative Care in Advanced Dementia: Key Challenges and Best Practices

  • Advanced dementia is a life-limiting condition with complex care needs, requiring early integration of palliative care to manage symptoms and enhance quality of life.
  • People with advanced dementia experience significant symptom burden including pain, breathing difficulties, and neuropsychiatric symptoms, yet often have limited access to specialized palliative care services.
  • Nursing homes are the most common place of death for people with advanced dementia, highlighting the need to strengthen palliative care capabilities in long-term care facilities.

Fedratinib: A Comprehensive Review of Pharmacology, Efficacy, and Safety in Myelofibrosis Treatment

  • Fedratinib is an effective treatment for myelofibrosis (MF), improving splenomegaly and symptom burden in both JAK-inhibitor-naïve and ruxolitinib-pretreated patients.
  • The drug's mechanism involves selective JAK2 inhibition and off-target effects on FLT3 and BRD4, potentially offering benefits beyond JAK2 inhibition alone.
  • Clinical trials like JAKARTA and JAKARTA2 have demonstrated fedratinib's efficacy in reducing spleen volume and improving symptoms, with manageable safety profiles.
  • Ongoing trials (FREEDOM and FREEDOM2) aim to evaluate long-term safety and efficacy, further solidifying fedratinib's role in MF treatment.

Tofersen Shows Promise in Phase 1/2 Trial for Inherited Form of ALS

Rare Disease
  • Biogen's experimental drug tofersen demonstrated safety and successfully lowered levels of disease-causing SOD1 protein in patients with a rare inherited form of ALS in a phase 1/2 clinical trial.
  • The antisense oligonucleotide treatment reduced SOD1 protein concentrations by up to 33% in the high-dose group, with some indications it may slow disease progression, prompting advancement to phase 3 trials.
  • While targeting only approximately 2% of ALS patients with SOD1 mutations, researchers believe this approach could establish a platform for treating other forms of ALS and neurodegenerative conditions through protein regulation.

Novartis Agrees to $678M Settlement Over Doctor Kickback Scheme in US

  • Novartis will pay $678 million to resolve allegations of using speaker programs and lavish entertainment to incentivize doctors to prescribe their cardiovascular and diabetes medications.
  • The company faces an additional $51 million settlement for illegally covering Medicare copayments for patients prescribed Gilenya and Afinitor.
  • As part of the settlement, Novartis commits to new corporate integrity measures through 2025, including limited use of paid external physicians and a shift toward digital engagement.

Butantan-DV Dengue Vaccine Shows Promising Safety and Immunogenicity in Phase 2 Trial

A phase 2 clinical trial of the Butantan-DV, a tetravalent live-attenuated dengue vaccine, demonstrated its safety and immunogenicity in both dengue-naive and dengue-exposed individuals. The study, conducted in São Paulo, Brazil, found that the vaccine was well-tolerated and induced robust immune responses against all four dengue virus serotypes.

Phase II Trial to Evaluate Safety and Immunogenicity of a Dengue 1,2,3,4 (Attenuated) Vaccine

NCT01696422Unknown StatusPhase 2
Butantan Institute
Posted 10/1/2013

FDA Rejects Intercept's NASH Drug Obeticholic Acid, Raising Industry-Wide Concerns

  • Intercept Pharmaceuticals faces major setback as FDA issues Complete Response Letter for obeticholic acid in NASH treatment, causing company shares to drop over 40%.
  • FDA cites uncertainty in drug's benefit-risk profile and requests additional safety and efficacy data from the ongoing REGENERATE study before considering approval.
  • CEO Mark Pruzanski criticizes FDA's evolving expectations for NASH treatments, expressing concerns about challenges in bringing innovative therapies to patients with high unmet needs.

Efficacy and Safety of Zihua Wenfei Granules in Treating Postinfectious Cough

A randomized, double-blind, placebo-controlled trial evaluates the efficacy and safety of Zihua Wenfei granules (ZHWFG) for treating postinfectious cough, specifically targeting the wind-cold invading lungs syndrome in traditional Chinese medicine. The study aims to assess the cough resolution and relief rates, alongside monitoring adverse events, across multiple centers in China.

Positive Phase 3 Data and New Glaucoma Treatments Highlight Advancements in Ophthalmology

  • A recent clinical trial achieved its primary visual acuity endpoints in both the US and EU/UK, demonstrating statistically significant near vision improvements over 8 hours.
  • Long-term analysis of a glaucoma treatment shows clinically and statistically significant reductions in intraocular pressure up to 36 months postoperatively.
  • Oxular initiated a Phase 2 trial in October 2024 for OXU-001, a therapeutic candidate targeting diabetic macular edema.

Sequana Medical's alfapump MOSAIC Study Results Published in Liver Transplantation

The results of the MOSAIC study, focusing on the utility, safety, and efficacy of the alfapump in managing recurrent and refractory ascites in liver disease patients, have been published in Liver Transplantation. The study highlights the alfapump's potential to improve quality of life and reduce the need for large-volume paracentesis in cirrhosis patients not suitable for TIPS.

Alfapump® System in the Treatment of Refractory or Recurrent Ascites (POSEIDON Study)

NCT03973866Active, Not RecruitingNot Applicable
Sequana Medical N.V.
Posted 9/16/2019

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