MedPath

Clinical Trial News

HIV Prevention Breakthroughs Face Access Barriers in Africa

• Lenacapavir, a twice-yearly injectable drug, showed 100% success in preventing HIV in young African women, but Gilead has not provided a timeline for affordable access. • Cabotegravir, another highly effective HIV prevention drug, remains largely unavailable in Africa despite successful trials there from 2017-2020. • Experts advocate for a new mandate requiring licensing, manufacturing, and distribution plans prior to conducting drug trials in low- and middle-income countries. • High HIV infection rates in Africa expedite and reduce the cost of drug trials, raising ethical concerns about access to the resulting therapies.

Kaida BioPharma Advances Novel Therapies for Gynecological Cancers, Launches Social Media Channels

  • Kaida BioPharma is focusing on developing innovative drugs for treatment-resistant gynecological cancers, addressing a critical unmet need.
  • The lead program, KAD101, is a novel biologic targeting the prolactin receptor, currently being developed for ovarian cancer with Phase 1 trials in 2025.
  • KAD101 has shown encouraging results in early clinical studies, demonstrating tumor reduction and a favorable safety profile as a daily injectable.
  • Kaida BioPharma has launched corporate social media channels to connect with investors, patients, and industry partners, enhancing its visibility.

Sharing Clinical Trial Results Boosts Engagement and Offers Secondary Health Benefits

  • Providing clinical trial participants with their medical test results significantly enhances their engagement and willingness to participate in future research efforts.
  • A study within the RECOVER initiative found that two-thirds of participants valued receiving test results, leading to informed discussions with their primary care providers.
  • Nearly three-quarters of participants shared results with their doctors, sometimes leading to new treatments, highlighting the potential for improved healthcare outcomes.
  • Researchers emphasize the need to proactively plan and budget for returning test results to participants, ensuring a stress-free and meaningful experience.

FDA Grants Fast Track Designation to Glycomine's GLM101 for PMM2-CDG Treatment

  • The FDA has granted Fast Track designation to Glycomine's GLM101, a mannose-1-phosphate replacement therapy, for treating phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG).
  • GLM101 is currently in a Phase 2 clinical study involving adult and adolescent patients, with plans to expand enrollment to pediatric patients aged two years and older.
  • Initial data from the Phase 2 study suggests promising clinical benefits with GLM101, showing good tolerability and only mild to moderate adverse events reported to date.
  • Fast Track designation will allow for more frequent interactions with the FDA, potentially accelerating the approval process for GLM101, addressing a significant unmet need.

24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG

NCT05549219Active, Not RecruitingPhase 2
Glycomine, Inc.
Posted 11/29/2022

Azitra's ATR-04 Receives FDA Fast Track Designation for EGFRi-Associated Skin Rash

  • Azitra's ATR-04, a topical therapy for moderate to severe skin rash induced by EGFR inhibitors, has received FDA Fast Track designation.
  • The Fast Track program aims to expedite the development and review of drugs addressing serious conditions with unmet medical needs, potentially accelerating ATR-04's approval.
  • ATR-04 is a live biotherapeutic product with a modified Staphylococcus epidermidis strain, designed to enhance safety and address skin immunity suppression.
  • Azitra plans to initiate a Phase I/II clinical trial by the end of 2024 to evaluate ATR-04's efficacy and safety in treating EGFR inhibitor-induced dermal toxicity.

Medtronic's Adaptive DBS Shows Promise in Parkinson's Disease Trial

  • Medtronic's ADAPT-PD trial evaluates adaptive deep brain stimulation (aDBS) using the Percept PC neurostimulator for Parkinson's disease, showing potential for personalized therapy.
  • Preliminary data indicates that aDBS can deliver targeted stimulation based on a patient’s brain state, potentially improving symptom control and reducing side effects compared to continuous DBS (cDBS).
  • The study, involving 68 participants, assesses aDBS efficacy in 'at-home' settings, focusing on increasing 'On' time without troublesome dyskinesias, a key measure of treatment success.
  • GlobalData analysis projects the neuromodulation device market to reach over $8.9 billion by 2027, with the DBS segment constituting $1.9 billion by 2030, highlighting significant growth potential.

Adaptive DBS Algorithm for Personalized Therapy in Parkinson's Disease

NCT04547712CompletedNot Applicable
MedtronicNeuro
Posted 12/14/2020

FDA Lifts Partial Clinical Hold on Vigil Neuroscience's VG-3927 Phase I Trial, Expands to Alzheimer's Patients

  • The FDA has lifted the partial clinical hold on Vigil Neuroscience's Phase I trial of VG-3927, a treatment for neurodegenerative diseases, following a complete response from the company.
  • Interim data from the trial indicated VG-3927's safety, tolerability, and potential for once-daily dosing, alongside a sustained decline in soluble TREM2 levels in cerebrospinal fluid.
  • The Phase I trial has expanded to include Alzheimer's disease patients, including those with TREM2 or other disease-related genetic variations, to assess biomarker response.
  • Vigil Neuroscience plans to release complete Phase I data, including results from the Alzheimer's patient cohort, in the first quarter of next year.

Insilico Medicine's AI-Designed Drug Shows Promise in Phase IIa IPF Trial

  • Insilico Medicine's ISM001-055 met its primary safety endpoint and secondary efficacy endpoints in a Phase IIa trial for idiopathic pulmonary fibrosis (IPF).
  • The AI-designed drug demonstrated a dose-dependent improvement in forced vital capacity (FVC), a key measure of lung function, in IPF patients.
  • The company plans to engage with regulatory authorities to design a Phase IIb study, exploring extended treatment durations and larger patient cohorts.
  • ISM001-055 targets TNIK, offering a novel approach to treating IPF by addressing both fibrosis and inflammation, potentially modifying the disease.

Study Evaluating INS018_055 Administered Orally to Subjects With Idiopathic Pulmonary Fibrosis (IPF)

NCT05938920Active, Not RecruitingPhase 2
InSilico Medicine Hong Kong Limited
Posted 6/19/2023

Study Evaluating INS018_055 Administered Orally to Subjects With Idiopathic Pulmonary Fibrosis

NCT05975983RecruitingPhase 2
InSilico Medicine Hong Kong Limited
Posted 2/8/2024

A4 Study Data Released to Accelerate Alzheimer's Research

  • The Anti-Amyloid Treatment in Asymptomatic Alzheimer’s (A4) Study data is now available for download, promoting open science in dementia research.
  • Despite solanezumab not meeting its primary endpoints, the A4 study's data sharing has already generated approximately 60 peer-reviewed research articles.
  • Researchers can access clinical data, biosamples, and neuroimaging from nearly 1,200 participants to explore disease biology and potential treatments.
  • The A4 study's impact and data will be discussed at the 2024 NIH Alzheimer’s Research Summit, fostering further insights into Alzheimer's.

Relutrigine Shows Promise in Reducing Seizures in SCN2A/SCN8A-DEE Phase 2 Trial

  • Relutrigine demonstrated a 46% reduction in motor seizures in pediatric patients with SCN2A and SCN8A developmental and epileptic encephalopathies (DEE).
  • A significant 30% of patients treated with relutrigine achieved seizure freedom, indicating a potential breakthrough in managing these rare disorders.
  • Patients experienced notable improvements in alertness, behavior, and communication, suggesting broader benefits beyond seizure control.
  • Praxis Precision's relutrigine, a sodium channel modulator, targets overactive sodium channels caused by SCN2A and SCN8A mutations.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.