MedPath

Clinical Trial News

GC Biopharma and Novel Pharma Initiate Phase I Trial of GC1130A for Sanfilippo Syndrome Type A

  • GC Biopharma and Novel Pharma have dosed the first patient in a multinational Phase I clinical trial of GC1130A for Sanfilippo syndrome type A (MPS IIIA).
  • The Phase I trial will assess the safety, tolerability, and efficacy of GC1130A in children aged 2-6 years with MPS IIIA over a two-year period.
  • GC1130A is a first-in-class treatment using GC Biopharma’s platform to deliver concentrated recombinant protein via intracerebroventricular (ICV) injection.
  • The trial will be conducted across multiple sites in the US, Korea, and Japan, addressing a critical unmet need for this devastating genetic disorder.

ADELA Phase III Trial Explores Elacestrant and Everolimus Combination for Advanced Breast Cancer

  • The ADELA trial investigates elacestrant combined with everolimus for ER+/HER2- advanced breast cancer with ESR1 mutations, addressing endocrine therapy resistance.
  • Presented at SABCS 2024, the study aims to improve outcomes for patients who have progressed after standard first-line treatments.
  • The international, randomized, double-blind trial evaluates progression-free survival, overall survival, toxicity, and quality of life.
  • MEDSIR's involvement highlights its commitment to personalized oncology and overcoming tumor resistance, potentially leading to regulatory approval.

Phase 3 Trial of Elacestrant vs. Standard of Care for the Treatment of Patients With ER+/HER2- Advanced Breast Cancer

NCT03778931CompletedPhase 3
Stemline Therapeutics, Inc.
Posted 5/10/2019

Elacestrant versus Standard Endocrine Therapy in Women and Men with Node-positive, Estrogen Receptor-positive, HER2-negative, Early Breast Cancer with High Risk of Recurrence - A Global, Multicenter, Randomized, Open-label Phase 3 Study (ELEGANT)

2024-515445-42-00RecruitingPhase 3
Stemline Therapeutics Inc.
Posted 9/27/2024

A randomized phase 3, double-blind, placebo-controlled study of elacestrant plus everolimus versus elacestrant in patients with estrogen receptor-positive/human epidermal growth factor receptor 2-negative, ESR1-mutated, advanced breast cancer progressing to endocrine therapy and CDK4/6 inhibitors - The ADELA study-

2024-512926-27-00RecruitingPhase 3
Medica Scientia Innovation Research S.L., Medica Scientia Innovation Research S.L., Stemline Therapeutics Inc.
Posted 12/5/2024

ELACESTRANT in Women and Men With CDK4/6 Inhibitor-Naive Estrogen Receptor Positive, HER-2 Negative Metastatic Breast Cancer Study

NCT05596409Active, Not RecruitingPhase 2
Stemline Therapeutics, Inc.
Posted 5/19/2023

Open-Label Umbrella Study To Evaluate Safety And Efficacy Of Elacestrant In Various Combination In Participants With Metastatic Breast Cancer

NCT05563220RecruitingPhase 1
Stemline Therapeutics, Inc.
Posted 1/24/2023

Study of Abemaciclib and Elacestrant in Patients With Brain Metastasis Due to ER+/HER-2- Breast Cancer

NCT05386108RecruitingPhase 1
Stemline Therapeutics, Inc.
Posted 8/31/2022

Slope and LabConnect Launch Revolutionary API Integration to Transform Clinical Trial Sample Management

• Slope and LabConnect have introduced a groundbreaking bidirectional API integration combining Biospecimen360 platform with Laboratory Information Management System to enhance clinical trial data integrity.
• The integration eliminates common errors in sample management, streamlines operations, and provides complete control over sample metadata, kit inventory, and shipping processes.
• This innovative solution sets a new industry standard for real-time data exchange in clinical trials, offering mutual clients improved biospecimen lifecycle management capabilities.

EU Postpones Decision on BioNTech-Pfizer Vaccine Approval

  • The European Medicines Agency (EMA) has delayed its decision on approving the BioNTech-Pfizer COVID-19 vaccine, pushing the potential distribution of the vaccine into early next year.
  • The EMA will review the BioNTech-Pfizer vaccine on December 29 and then forward its opinion to member countries for final approval.
  • Evaluation of Moderna's vaccine has also been postponed until January 12 of the following year, further delaying the availability of vaccines in the EU.
  • German Health Minister Jens Spahn anticipates vaccinations to begin in Germany by January, with widespread availability expected by early summer.

Deep Brain Stimulation of Hippocampus Shows Promise for Treatment-Resistant Schizophrenia in New Trial

  • A crossover randomized controlled trial, DBS-HITS, is investigating deep brain stimulation (DBS) of the hippocampus for treatment-resistant schizophrenia.
  • The study involves six participants with chronic, treatment-resistant schizophrenia undergoing DBS surgery and subsequent stimulation parameter optimization.
  • Participants will be randomized to receive either high-frequency stimulation or sham stimulation for three months each, with blinded evaluations.
  • The primary outcome is the comparison of response rates in PANSS total, positive, and negative scores, with secondary outcomes including cognitive and neuroimaging changes.

Zanubrutinib Demonstrates Sustained Efficacy in Relapsed/Refractory CLL: ALPINE Trial Update

  • Zanubrutinib shows durable efficacy and a favorable safety profile in relapsed/refractory chronic lymphocytic leukemia (CLL) patients, according to long-term data from the ALPINE trial.
  • The final comparative analysis of the phase 3 ALPINE trial supports zanubrutinib as a valuable BTK inhibitor option for CLL, with promising outcomes.
  • Experts discussed managing zanubrutinib-associated toxicities and the evolving role of BTK inhibitors in CLL treatment during an OncLive On Air discussion.

A Study of Zanubrutinib (BGB-3111) Versus Ibrutinib in Participants With Relapsed/Refractory Chronic Lymphocytic Leukemia

NCT03734016CompletedPhase 3
BeiGene
Posted 11/1/2018

PMCPA Sanctions Moderna for Code Violations in COVID-19 Vaccine Promotion and Trial Recruitment

• The UK's Prescription Medicines Code of Practice Authority (PMCPA) found Moderna in breach of industry ethics for inappropriately promoting its Spikevax COVID-19 vaccine and using unapproved clinical trial recruitment materials.
• Moderna faces financial penalties and public disclosure requirements after being found to have "brought discredit upon and reduced confidence in the pharmaceutical industry" through its actions.
• The ABPI has updated its code of practice with new mandatory disclosure requirements and procedures, set to take effect in 2025, aiming to enhance transparency in healthcare professional relationships.

Advancements in Clinical Outcome Assessments and Biomarkers Pave the Way for CMT Treatment

  • New clinical outcome assessments (COAs) like the CMTNSv2 and CMT-FOM show promise but require validation in longitudinal studies for Charcot-Marie-Tooth disease (CMT).
  • MRI muscle fat fraction emerges as a responsive biomarker, yet its correlation with functional outcomes in CMT treatment trials remains uncertain.
  • Serum biomarkers such as neurofilament light reliably differentiate CMT patients but lack proven responsiveness to effective therapies.
  • Ongoing clinical trials are testing promising COAs and biomarkers to refine patient selection and enrollment strategies for CMT treatment development.

Alector's AL002 Fails to Meet Primary Endpoint in Phase 2 Alzheimer's Trial

  • Alector Therapeutics' AL002, an experimental antibody designed to activate microglia, failed to slow disease progression in a Phase 2 trial for early Alzheimer's disease.
  • The INVOKE-2 trial, involving 381 participants, did not demonstrate significant treatment effects on secondary clinical, functional endpoints, or Alzheimer's fluid biomarkers.
  • Following the negative results, Alector is discontinuing the long-term extension trial for AL002 and laying off 17% of its workforce to focus on partnered monoclonal antibody programs.
  • Alector plans to focus on its collaboration with GSK, including the Phase III INFRONT-3 trial evaluating latozinemab for frontotemporal dementia, with results expected in late 2025/early 2026.

Efficacy and Safety of GSK4527226 [AL101] in Participants With Early Alzheimer's Disease

NCT06079190Active, Not RecruitingPhase 2
GlaxoSmithKline
Posted 10/20/2023

A Phase 3 Study to Evaluate Efficacy and Safety of AL001 in Frontotemporal Dementia (INFRONT-3)

NCT04374136Active, Not RecruitingPhase 3
Alector Inc.
Posted 7/23/2020

A Long-term Extension Study to Evaluate Safety, Tolerability, and Efficacy of AL002 in Alzheimer's Disease

NCT05744401TerminatedPhase 2
Alector Inc.
Posted 1/4/2023

A Phase 2 Study to Evaluate Efficacy and Safety of AL002 in Participants With Early Alzheimer's Disease

NCT04592874Active, Not RecruitingPhase 2
Alector Inc.
Posted 1/22/2021

Simufilam 100 mg for Mild-to-Moderate Alzheimer's Disease

NCT04994483CompletedPhase 3
Cassava Sciences, Inc.
Posted 11/3/2021

Empyrean Therapeutics Acquires TLR-2 Antagonist EMS-312 from Eos Therapies to Advance Cancer Treatment

  • Empyrean Therapeutics acquired a novel TLR-2 antagonist molecule, EMS-312, from Eos Therapies to develop a new cancer drug.
  • EMS-312 is in the pre-IND phase and targets aggressive cancers like brain, pancreatic, and breast, with plans for in vivo validation.
  • The acquisition aims to leverage Empyrean's R&D infrastructure to expedite clinical trials and market launch of EMS-312.
  • EMS-312 exhibits excellent target affinity, potency, oral bioavailability, and brain uptake, making it a promising therapeutic candidate.

MedPath

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

© 2025 MedPath, Inc. All rights reserved.