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Clinical Trial News

UNITY Biotechnology to Highlight Foselutoclax (UBX1325) Data at Ophthalmology Day

  • UNITY Biotechnology will host an Ophthalmology Day on October 15, 2024, focusing on its ASPIRE study.
  • The ASPIRE study evaluates foselutoclax (UBX1325) against aflibercept for diabetic macular edema (DME).
  • Twenty-four-week safety and efficacy data from the ASPIRE study are expected in Q1 2025.
  • Key opinion leaders will discuss UBX1325's potential in addressing unmet needs in DME.

Univo IRB Expands Services with Canadian Research Ethics Board Review

  • Univo IRB has launched Canadian Research Ethics Board (REB) review services to support clinical trials across North America, ensuring high ethical and regulatory standards.
  • The new service aims to streamline the REB approval process for researchers and sponsors, facilitating more efficient clinical trials in compliance with Canadian guidelines.
  • Univo IRB's Canadian REB includes experts in ethics, law, and science, guided by the Tri-Council Policy Statement for ethical research involving humans.
  • This expansion reaffirms Univo IRB's commitment to advancing research responsibly, unifying clinical research with patient protection, education, and advocacy.

Silexion's LODER Shows Promise in Non-Resectable Pancreatic Cancer

  • Silexion Therapeutics' LODER demonstrated a 56% objective response rate in patients with non-resectable pancreatic cancer bearing KRAS G12D/V mutations.
  • The objective response rate increased to 67% in patients whose tumors became resectable following treatment with LODER.
  • Patients treated with LODER plus chemotherapy showed a 9.3-month improvement in overall survival compared to chemotherapy alone.
  • Silexion is advancing SIL-204, a next-generation therapy targeting a broader range of KRAS mutations with enhanced stability.

Zevra Therapeutics' KP1077 Shows Promise in Phase 2 Trial for Idiopathic Hypersomnia

  • Zevra Therapeutics' KP1077 (serdexmethylphenidate) demonstrated clinically meaningful improvements in key symptoms of idiopathic hypersomnia (IH) in a Phase 2 trial.
  • The Phase 2 trial was a placebo-controlled, double-blind, randomized withdrawal study evaluating the safety and efficacy of KP1077 in IH patients.
  • Top-line data from the trial will inform the design of a Phase 3 study, marking a significant step in developing a potential new treatment for IH.
  • KP1077 has been granted Orphan Drug Designation by both the FDA and the European Commission for the treatment of idiopathic hypersomnia.

Placebo-Controlled, Double-Blind, Study to Determine the Safety and Efficacy of SDX in Patients With IH

NCT05668754CompletedPhase 2
Zevra Therapeutics
Posted 12/28/2022

Wave Life Sciences' WVE-N531 Shows Promise in Duchenne Muscular Dystrophy Trial

  • Wave Life Sciences announced positive interim data from its FORWARD-53 trial of WVE-N531 in Duchenne Muscular Dystrophy (DMD) patients amenable to exon 53 skipping.
  • The trial showed a mean muscle content-adjusted dystrophin expression of 9.0% and improved muscle health biomarkers with bi-weekly 10 mg/kg dosing.
  • WVE-N531 demonstrated a favorable safety profile and a 61-day tissue half-life, supporting potential monthly dosing.
  • Wave Life Sciences plans to discuss accelerated approval pathways with regulators, with complete 48-week data expected in Q1 2025.

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT03406780CompletedPhase 2
Capricor Inc.
Posted 4/4/2018

Open-label Study of WVE-N531 in Patients with Duchenne Muscular Dystrophy (FORWARD-53)

NCT04906460Active, Not RecruitingPhase 1
Wave Life Sciences Ltd.
Posted 9/28/2021

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

NCT05429372Active, Not RecruitingPhase 2
Pfizer
Posted 8/8/2022

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

NCT04281485Active, Not RecruitingPhase 3
Pfizer
Posted 11/5/2020

The Effect of RNS60 on ALS Biomarkers

NCT03456882CompletedPhase 2
Mario Negri Institute for Pharmacological Research
Posted 5/30/2017

A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT05126758Active, Not RecruitingPhase 3
Capricor Inc.
Posted 6/22/2022

UCB and Biogen's Dapirolizumab Pegol Shows Promise in Phase 3 Lupus Trial

  • UCB and Biogen's dapirolizumab pegol met the primary endpoint in a Phase 3 trial for moderate-to-severe systemic lupus erythematosus (SLE).
  • The drug demonstrated significant clinical improvement in disease activity compared to placebo when added to standard of care.
  • A second Phase 3 trial, PHOENYCS FLY, is planned to further evaluate the efficacy and safety of dapirolizumab pegol in SLE patients.
  • Dapirolizumab pegol targets the CD40L pathway, offering a novel approach to reduce B cell activation and autoantibody production in SLE.

A Study to Evaluate the Efficacy and Safety of Dapirolizumab Pegol in Study Participants With Moderately to Severely Active Systemic Lupus Erythematosus

NCT04294667CompletedPhase 3
UCB Biopharma SRL
Posted 8/12/2020

RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus

NCT06121297RecruitingPhase 1
Cabaletta Bio
Posted 2/16/2024

United Therapeutics to Present Tyvaso DPI Data at CHEST 2024

  • United Therapeutics will present data from the BREEZE open-label extension study of Tyvaso DPI, detailing long-term outcomes in pulmonary arterial hypertension patients.
  • The company is sponsoring a symposium on Tyvaso DPI, focusing on clinical pearls and drug-device characteristics, at the CHEST 2024 Annual Meeting.
  • Additional presentations will cover patient-reported outcomes, healthcare resource utilization, and the impact of inhaled treprostinil in pulmonary hypertension.
  • United Therapeutics also sponsors the Women in Chest Medicine Annual Luncheon and the Advanced Practice Providers (APPs) in Chest Medicine Forum.

MRE and TE Show Limited Accuracy in Detecting Advanced Liver Fibrosis in Pediatric MASLD Patients

  • A new study at Children's Hospital Los Angeles reveals that magnetic resonance elastography (MRE) and transient elastography (TE) demonstrate insufficient accuracy in detecting high-grade liver fibrosis in pediatric MASLD patients.
  • Research findings show MRE performed marginally better than TE with AUROC values of 0.817 versus 0.750, though the difference was not statistically significant (P=0.4785).
  • The study, involving 77 pediatric patients with biopsy-proven MASLD, highlights the urgent need for more reliable non-invasive monitoring tools as MASLD affects up to 38% of children with obesity.

DETERMINE Trial Aims to Improve Targeted Therapy for Rare Cancers Through Genetic Understanding

  • The DETERMINE trial expands treatment options for patients with rare cancers beyond standard chemotherapy, leveraging increased knowledge of cancer genetics.
  • DETERMINE Translational aims to understand how genetic changes causing cancer are activated by conditions like mutations, inflammation, or epigenetics.
  • Researchers hope to identify ways to prevent certain cancers by interfering with triggering conditions or blocking secondary processes.
  • The combined results of DETERMINE and DETERMINE Translational could provide new treatment options and improve patient selection for effective treatments.

Technology-Assisted Health Coaching Fails to Achieve Significant Weight Loss in Primary Care Trial

  • A cluster-randomized controlled trial evaluated a technology-assisted health coaching intervention (GEM) for weight management in primary care settings.
  • The GEM intervention, involving health coaching and a software tool, did not result in clinically significant weight loss compared to enhanced usual care.
  • The study found no statistically significant differences in secondary outcomes, including weight management program attendance and dietary changes.
  • Exploratory analyses suggested a potential increase in moderate to vigorous physical activity in the GEM arm, warranting further investigation.

The GEM (Goals for Eating and Moving) Study

NCT03006328CompletedNot Applicable
NYU Langone Health
Posted 11/20/2017

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