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Clinical Trial News

Applied Therapeutics Advances Govorestat for Galactosemia with FDA and EMA

  • Applied Therapeutics will hold a pre-NDA meeting with the FDA this summer to discuss a potential NDA submission for govorestat (AT-007) for treating classic Galactosemia.
  • The company plans to submit a Marketing Authorization Application (MAA) to the EMA in the fall of 2023 for govorestat to treat classic Galactosemia.
  • Govorestat has shown clinical benefits in children with Galactosemia, improving daily living activities, behavior, cognition, motor skills, and reducing plasma galactitol levels.

Safety and Efficacy of AK0529 in Respiratory Syncytial Virus-Infected Infant Patients

A Phase 2 study evaluates the safety and efficacy of AK0529, an oral RSV fusion protein inhibitor, in infants aged 1-24 months hospitalized with RSV infection. The study found AK0529 to be well tolerated, with a significant reduction in viral load and Wang Respiratory Score at a dosage of 2 mg/kg bid.

Viral Inhibition in Children for Treatment of RSV

NCT02654171CompletedPhase 2
Shanghai Ark Biopharmaceutical Co., Ltd.
Posted 5/27/2016

Samuraciclib Shows Promise in Advanced Breast Cancer Patients After CDK4/6 Inhibitor Failure

OncologyTargeted TherapyPrecision Medicine
  • Phase I clinical trials demonstrate samuraciclib, a selective CDK7 inhibitor, has an acceptable safety profile with manageable gastrointestinal side effects and shows clinical activity in various advanced solid tumors.
  • In HR+/HER2- breast cancer patients who progressed on CDK4/6 inhibitors, the combination of samuraciclib with fulvestrant achieved a clinical benefit rate of 36% and median progression-free survival of 3.7 months.
  • Exploratory analysis revealed patients without TP53 mutations had significantly longer progression-free survival (7.4 months vs 1.8 months), suggesting TP53 status may serve as a potential biomarker for treatment response.

Modular Study to Evaluate CT7001 Alone in Cancer Patients With Advanced Malignancies

NCT03363893CompletedPhase 1
Carrick Therapeutics Limited
Posted 11/14/2017

Update on Rapamycin Clinical Trial for PC Treatment

Palvella Therapeutics shared disappointing Phase 3 study results for QTORIN™ 3.9% rapamycin anhydrous gel, showing no significant treatment effect on PC patients' activity difficulty. Despite the setback, the trial provided valuable insights for future research, with ongoing support for PC treatment development.

Digital Transformation Reshapes Pharma Industry with $30B Investment Drive

  • The pharmaceutical industry is undergoing a major digital transformation accelerated by COVID-19, with companies expected to invest $30 billion in digital initiatives focusing on AI and Machine Learning technologies.
  • Digital solutions are addressing key industry challenges including data gaps in clinical trials, ineffective communication between teams, and growing patient demands for personalized care and faster access to treatments.
  • Advanced technologies like AI and ML are revolutionizing drug discovery, supply chain management, and patient care through improved data analysis, automated processes, and personalized treatment approaches.

Roche's Vabysmo Shows Sustained Vision Improvements with Extended Treatment Intervals in Retinal Vein Occlusion Studies

  • Vabysmo (faricimab) demonstrated maintained vision improvements and sustained retinal drying for up to 72 weeks in patients with retinal vein occlusion, using a personalized treat-and-extend dosing regimen.
  • If approved for retinal vein occlusion, Vabysmo would become the first treatment to show vision maintenance for more than a year in both branch and central RVO, potentially becoming the drug's third indication.
  • As the first bispecific antibody for the eye, Vabysmo uniquely targets two disease pathways by inhibiting both angiopoietin-2 and VEGF-A, with regulatory decisions expected in late 2023.

A Study to Evaluate the Long-Term Safety and Tolerability of Faricimab in Participants With Neovascular Age-Related Macular Degeneration

NCT04777201CompletedPhase 3
Hoffmann-La Roche
Posted 4/19/2021

A Study to Investigate Faricimab Treatment Response in Treatment-Naive, Underrepresented Patients With Diabetic Macular Edema

NCT05224102Active, Not RecruitingPhase 4
Genentech, Inc.
Posted 2/28/2022

A Study to Evaluate the Long-Term Safety and Tolerability of Faricimab in Participants With Diabetic Macular Edema

NCT04432831CompletedPhase 3
Hoffmann-La Roche
Posted 8/5/2020

A Study to Evaluate the Efficacy and Safety of Faricimab in Participants With Macular Edema Secondary to Branch Retinal Vein Occlusion

NCT04740905CompletedPhase 3
Hoffmann-La Roche
Posted 3/2/2021

A Study to Evaluate the Efficacy and Safety of Faricimab in Participants With Macular Edema Secondary to Central Retinal or Hemiretinal Vein Occlusion

NCT04740931CompletedPhase 3
Hoffmann-La Roche
Posted 3/2/2021

A Real-World Study to Gain Clinical Insights Into Roche Ophthalmology Products

NCT05476926Recruiting
Hoffmann-La Roche
Posted 11/21/2022

World-First Gene Therapy Trial Offers Hope for Young Boys with Duchenne Muscular Dystrophy

  • A global clinical trial is underway, evaluating gene replacement therapy for Duchenne Muscular Dystrophy (DMD) in boys under four years old.
  • The experimental treatment aims to replace the mutated gene responsible for DMD with a healthy version through a single-dose infusion.
  • Early results indicate potential improvements in muscle function and mobility in treated children, offering hope for slowing disease progression.
  • Researchers are optimistic that early intervention may prevent the severe weakness and disability typically associated with DMD, potentially extending life expectancy.

FDA Accepts NDA for Rivoceranib and Camrelizumab Combination in Unresectable Hepatocellular Carcinoma

  • The FDA has accepted the NDA for rivoceranib plus camrelizumab as a first-line treatment for unresectable hepatocellular carcinoma (HCC).
  • The NDA is supported by the Phase 3 CARES 310 study, which showed statistically significant improvements in overall survival (OS) and progression-free survival (PFS).
  • Patients treated with the combination therapy had a median OS of 22.1 months compared to 15.2 months with sorafenib, with a hazard ratio of 0.62 (p<0.0001).
  • The PDUFA target action date is set for May 16, 2024, marking a significant step toward a potential new treatment option for uHCC.

A Study to Evaluate SHR-1210 in Combination With Apatinib as First-Line Therapy in Patients With Advanced HCC

NCT03764293CompletedPhase 3
Jiangsu HengRui Medicine Co., Ltd.
Posted 6/10/2019

Psilocybin Shows Promise in Alleviating Cancer-Related Depression in Women

  • A new study suggests psilocybin, found in magic mushrooms, may offer rapid relief for cancer-related anxiety and depression in women, addressing the limitations of traditional therapies.
  • Researchers at the University of Texas highlight the potential of psilocybin-assisted psychotherapy to modulate brain activity and provide lasting benefits with fewer sessions.
  • Gynaecological cancer patients, particularly those with advanced disease, often face significant psychological challenges, including depression and anxiety, impacting their quality of life.
  • A forthcoming study will examine the effects of psilocybin on women with advanced cancer experiencing mental health issues, prioritizing safety and exploring its therapeutic potential.

MIND Diet Shows Limited Short-Term Cognitive Benefits in Clinical Trial

  • A clinical trial evaluating the MIND diet, designed to boost brain health, showed improvements in brain structure but similar gains in the control group.
  • The three-year study found that both the MIND diet group and the control group experienced improvements in cognitive function and weight loss.
  • Experts suggest the trial duration may have been too short to observe significant differences, and that increased awareness of healthy eating impacted both groups.
  • The MIND diet incorporates elements of the Mediterranean and DASH diets, emphasizing foods known to support brain health.

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