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Clinical Trial News

Microbot Medical Completes Trial for LIBERTY Surgical Robot, Eyes FDA Submission

  • Microbot Medical has concluded its ACCESS-PVI clinical trial for the LIBERTY endovascular surgical robot, with plans to submit a 510(k) application to the FDA by the end of 2024.
  • The LIBERTY system aims to improve endovascular procedures by reducing the need for bulky equipment and minimizing radiation exposure for physicians.
  • The company is accelerating its commercialization strategy, including hiring a seasoned healthcare executive, in anticipation of FDA clearance expected in Q2 2025.
  • Clinical data from the ACCESS-PVI trial will be presented at a medical conference in early 2025, showcasing the performance and safety of the LIBERTY system.

Health Canada Grants Priority Review to Biogen's Omaveloxolone for Friedreich's Ataxia

  • Health Canada has accepted Biogen's New Drug Submission for omaveloxolone under priority review for treating Friedreich's ataxia in patients aged 16 and older.
  • Omaveloxolone, a once-daily oral medication, could become the first disease-specific treatment option available for this rare and debilitating neuromuscular condition.
  • The submission is supported by data from the MOXIe Part 2 trial, which demonstrated significant improvements in mFARS scores compared to placebo at 48 weeks.
  • A regulatory decision from Health Canada regarding the approval of omaveloxolone is anticipated in early 2025, offering hope for Canadian patients with Friedreich's ataxia.

Sysmex Inostics' RAS-RAF-SEQ Assay Gains New York Approval for Clinical Trial Use

  • Sysmex Inostics' RAS-RAF-SEQ assay, a liquid biopsy test, has been approved by the New York State Department of Health for clinical trial use.
  • The assay targets key mutations in the RAS-RAF and PI3K signaling pathways, relevant in cancers like colorectal, pancreatic, and lung cancers.
  • RAS-RAF-SEQ utilizes Plasma-Safe-SeqS technology for ultrasensitive detection of low-level genomic mutations, enhancing therapy selection and monitoring.
  • This approval expands access to Sysmex Inostics' ultrasensitive assays for patients in New York, aiding in assessing novel cancer therapies.

Certepetide Shows Promise in Metastatic Pancreatic Cancer: Lisata Therapeutics Announces Encouraging Phase 2 Results

  • Lisata Therapeutics reported positive trends in overall survival for certepetide plus chemotherapy in metastatic pancreatic cancer patients.
  • The Phase 2 ASCEND trial's Cohort A showed a median overall survival of 12.68 months in the certepetide arm versus 9.72 months in the placebo arm.
  • Complete responses were observed in 6.2% of patients treated with certepetide, compared to none in the placebo group.
  • Lisata plans to advance certepetide to Phase 3 trials in early 2026, based on these results and expectations for Cohort B data.

A Study of LSTA1 When Added to Standard of Care Versus Standard of Care Alone in Patients With Advanced Solid Tumors

NCT05712356Active, Not RecruitingPhase 2
Lisata Therapeutics, Inc.
Posted 8/24/2023

The ASCEND Study: Gemcitabine and Nab-Paclitaxel With LSTA1 (Certepetide) or Placebo in Patients With Untreated Metastatic Pancreatic Ductal Adenocarcinoma

NCT05042128Active, Not RecruitingPhase 2
Australasian Gastro-Intestinal Trials Group
Posted 4/13/2022

NIH Funds Development of Decision Aid for Early Deep Brain Stimulation in Parkinson's Disease

  • A $1.5 million NIH grant supports the development of a patient-centered decision aid for early Deep Brain Stimulation (DBS) consideration in Parkinson's disease.
  • The decision aid aims to facilitate shared decision-making between patients, clinicians, and caregivers regarding DBS treatment.
  • The tool will address ethical concerns and individual preferences to determine the most appropriate timing for DBS intervention.
  • Researchers hope the decision aid will promote responsible use of neurotechnologies for various brain disorders beyond Parkinson's.

CMS Finalizes Coverage Pathway for FDA-Designated Breakthrough Devices

  • The Centers for Medicare & Medicaid Services (CMS) has issued a final procedural notice for a new Medicare coverage pathway.
  • This pathway aims to provide timely and predictable access to FDA-designated breakthrough devices.
  • The policy intends to expedite coverage decisions for innovative medical technologies.
  • Stakeholders are analyzing the policy's potential impact on patient access and innovation in the medical device industry.

Bispecific Antibodies and CAR T-cell Therapies Revolutionize Treatment of Relapsed/Refractory Lymphomas

  • Bispecific antibodies like glofitamab and epcoritamab have shown efficacy in diffuse large B-cell lymphoma (DLBCL) after two prior lines of treatment.
  • Epcoritamab and mosunetuzumab have received FDA approval for later-line treatment of follicular lymphoma, offering alternatives to withdrawn PI3K inhibitors.
  • CAR T-cell therapies and bispecific antibodies have improved outcomes compared to chemoimmunotherapy, though direct comparisons are lacking.
  • Bispecific antibodies offer an outpatient administration advantage over the inpatient requirement of CAR T-cell therapy, impacting treatment accessibility.

Vertex and Bluebird Bio Initiate Infusions of Sickle Cell Gene Therapies

  • Vertex Pharmaceuticals and CRISPR Therapeutics have begun infusions of their sickle cell gene therapy, Casgevy, marking a significant advancement in treatment.
  • Bluebird Bio has also started administering its sickle cell gene therapy, Lyfgenia, to patients after receiving FDA approval.
  • The initiation of these gene therapies represents a breakthrough, though the treatment process is lengthy and complex, potentially taking up to a year.
  • Analysts predict substantial revenue generation for both Casgevy and Lyfgenia, with peak sales for Casgevy potentially reaching $3.6 billion.

FDA Finalizes Guidance on Decentralized Clinical Trials, Clarifying Key Aspects

  • The FDA has issued final guidance on decentralized clinical trials (DCTs), providing clarity on various aspects including HCP task logs and inspection requirements.
  • The guidance addresses the management of data variability inherent in DCTs, offering strategies for maintaining data integrity and reliability.
  • This final guidance aims to facilitate the adoption of DCTs while ensuring patient safety and data quality, impacting pharmaceutical development.
  • The document provides recommendations for sponsors on how to operationalize DCTs, including considerations for remote monitoring and technology use.

Anti-Obesity Drugs Show Promise in Young Children, but Long-Term Effects Remain Unknown

• A recent study shows that liraglutide, a GLP-1 mimic, led to a BMI decrease in children aged 6-11, offering a potential treatment for early-onset obesity. • While trials indicate significant weight loss in adolescents using drugs like semaglutide and liraglutide, concerns persist regarding long-term impacts on growth and puberty. • Experts emphasize the need for diverse studies and prolonged monitoring to address ethical considerations and potential side effects in young children using GLP-1 mimics. • The use of BMI as a metric for measuring progress has disadvantages, and weight-related health problems should be considered.

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