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Clinical Trial News

Immunotherapy Improves Survival in Advanced Bladder Cancer Patients

A phase III clinical trial has demonstrated that the immunotherapy drug 'avelumab' significantly improves survival in patients with advanced bladder cancer, marking the first time an immune therapy has shown a survival advantage in this setting. The trial, led by Queen Mary University of London and Barts Cancer Centre, found a 31% reduction in the risk of death and extended median survival by over seven months.

FDA Approves New First-Line Treatment for Head and Neck Cancers

The FDA has approved a new first-line treatment for metastatic or recurrent head and neck cancer, using the immunotherapy drug pembrolizumab, either alone or with chemotherapy. This treatment, developed through research led by Yale's Dr. Barbara A. Burtness, significantly improves survival rates and is less toxic than previous treatments.

Increased Risk of Diabetic Ketoacidosis with Sotagliflozin in Type 1 Diabetes Patients

A study evaluating the incidence and risk factors for diabetic ketoacidosis (DKA) in adults with type 1 diabetes treated with sotagliflozin adjunctive to insulin found an increased incidence of DKA compared to insulin alone. The study, pooling data from two 52-week, randomized studies, observed higher DKA incidence rates with sotagliflozin, especially at higher doses. However, the implementation of a risk mitigation plan was associated with a lower incidence of DKA, suggesting that patient education and monitoring can manage this risk.

Efficacy, Safety, and Tolerability Study of Sotagliflozin as Adjunct Therapy in Adult Patients With Type 1 Diabetes Mellitus Who Have Inadequate Glycemic Control With Insulin Therapy

NCT02384941CompletedPhase 3
Lexicon Pharmaceuticals
Posted 3/1/2015

Efficacy, Safety, and Tolerability Study of Sotagliflozin as Adjunct Therapy in Adult Patients With Type 1 Diabetes Mellitus Who Have Inadequate Glycemic Control With Insulin Therapy

NCT02421510CompletedPhase 3
Lexicon Pharmaceuticals
Posted 5/1/2015

Genomic Medicine Evolution: Bridging the Gap Between Promise and Clinical Reality

  • Genomic medicine is advancing beyond initial human genome project expectations, with scientists now focusing on understanding the crucial 98% of non-protein-coding regions that regulate gene expression.
  • Long-read sequencing technology is emerging as a breakthrough tool for understanding complex genetic disorders, offering more comprehensive insights than traditional short-read methods.
  • Gene therapy using adeno-associated virus (AAV) vectors has achieved clinical success, notably in treating spinal muscular atrophy, marking a significant step toward personalized genomic medicine.

Crinetics Pharmaceuticals Announces Key Developments and Appointments

Crinetics Pharmaceuticals has announced positive Phase 2 trial results for atumelnant in treating congenital adrenal hyperplasia, participation in the 43rd Annual J.P. Morgan Healthcare Conference, and the appointment of Isabel Kalofonos as Chief Commercial Officer. Additionally, the company has granted stock options to new employees under Nasdaq Listing Rule 5635(c)(4).

Pre-filled Syringes Conference 2021 to Address Key Innovations in Injectable Drug Delivery

  • SMi Group's 13th annual Pre-filled Syringes Conference will convene industry leaders in London to discuss advances in biologics delivery, device design, and EU MDR implementation.
  • The two-day conference will feature expert presentations on novel drug products, parenteral packaging innovations, and lifecycle management strategies for combination products.
  • Early registration offers a £400 discount until September 30, 2020, with additional pre-conference workshops focusing on digital health and long-acting injectable developments.

Alzheimer Prevention Trials Webstudy Enrolls Over 30,000 Participants for Early Detection Research

  • The Alzheimer Prevention Trials (APT) Webstudy enrolled 30,650 participants, establishing a Trial-ready Cohort for Preclinical and Prodromal Alzheimer's disease (TRC-PAD).
  • Referrals from online registries proved to be the most effective recruitment method, accounting for nearly 70% of participants in the APT Webstudy.
  • Participants, primarily from across the US, undergo quarterly cognitive and functional assessments, with referrals for in-clinic testing and biomarker confirmation.
  • The APT Webstudy aims to identify individuals for potential enrollment in future Alzheimer's disease prevention trials, addressing a critical need for early intervention strategies.

RNA-Targeted Drug Discovery Summit to Address Key Challenges in Small Molecule Development

  • Leading biopharmaceutical executives and researchers gather at the 3rd RNA-Targeted Drug Discovery Summit to advance RNA-targeting small molecule therapeutics development and improve patient outcomes.
  • The summit aims to tackle critical challenges in RNA drug discovery, including RNA structure determination, target selection optimization, and translation into clinical development.
  • Industry leaders from Scripps Research Institute, Anima Biotech, and Arrakis Therapeutics will share insights on enhancing medicinal chemistry approaches and RNA biology understanding for robust therapeutic development.

Evolution of Pharmaceutical Industry: From Apothecaries to Modern Drug Development Giants

  • The pharmaceutical industry transformed from traditional apothecaries in the 19th century to scientific enterprises, with companies like Merck and Pfizer pioneering industrial medicine production.
  • Major breakthroughs including insulin treatment and penicillin development in the early 20th century marked the beginning of modern pharmaceutical research and mass production.
  • Recent innovations in immunotherapy, gene therapy, and COVID-19 vaccines showcase the industry's advancement, while challenges remain in antibiotic development and drug pricing.

Phase 3 Trial Shows Low-Dose Carbetocin Effective for Hyperphagia in Prader-Willi Syndrome

  • Levo Therapeutics' Phase 3 CARE-PWS trial found that the lower 3.2mg dose of intranasal carbetocin (LV-101) significantly reduced hyperphagia and anxiety symptoms in patients with Prader-Willi syndrome, while the higher 9.6mg dose did not meet primary endpoints.
  • The oxytocin-receptor agonist was generally well-tolerated, with mild to moderate side effects including headache, flushing, and diarrhea at the effective dose, offering potential relief for a condition with no currently approved treatments for its most challenging symptoms.
  • Over 98% of trial participants elected to continue receiving the treatment in the long-term follow-up phase, with maintained benefits or further improvements observed, demonstrating sustained clinical value for this rare neurodevelopmental disorder affecting approximately 1 in 16,000 births.

Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome

NCT03649477CompletedPhase 3
Levo Therapeutics, Inc.
Posted 11/20/2018

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