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Clinical Trial News

Overview of Advanced Therapy Medicinal Products (ATMPs) in the European Union

This article provides a comprehensive overview of Advanced Therapy Medicinal Products (ATMPs) within the European Union, including those available via centralized marketing authorization or national Hospital exemption. It also forecasts innovative ATMPs in the process of EMA approval and those in phase III and IV clinical trials, highlighting the evolving landscape of personalized medicinal strategies.

A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

NCT01896102CompletedPhase 2
bluebird bio
Posted 8/21/2013

A Study to Compare the Efficacy and Safety of JCAR017 to Standard of Care in Adult Subjects With High-risk, Transplant-eligible Relapsed or Refractory Aggressive B-cell Non-Hodgkin Lymphomas

NCT03575351CompletedPhase 3
Celgene
Posted 10/23/2018

Safety and Efficacy of ATIR101 as Adjunctive Treatment to Blood Stem Cell Transplantation From a Haploidentical Family Donor Compared to Post-transplant Cyclophosphamide in Patients With Blood Cancer

NCT02999854TerminatedPhase 3
Kiadis Pharma
Posted 11/29/2017

Confirmatory Study of NeoCart in Knee Cartilage Repair

NCT01066702TerminatedPhase 3
Histogenics Corporation
Posted 5/1/2010

Scleroderma Treatment With Celution Processed Adipose Derived Regenerative Cells (STAR)

NCT02396238CompletedNot Applicable
Cytori Therapeutics
Posted 5/15/2015

MultiStem® Administration for Stroke Treatment and Enhanced Recovery Study

NCT03545607Unknown StatusPhase 3
Healios K.K.
Posted 7/28/2018

The Efficacy and Safety of REX-001 to Treat Ischemic Ulcers in Subjects With CLI Rutherford Category 5 and DM

NCT03174522TerminatedPhase 3
Ixaka Ltd
Posted 4/25/2017

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

NCT00999609Active, Not RecruitingPhase 3
Spark Therapeutics, Inc.
Posted 10/1/2012

Efficacy and Safety Study of bb2121 Versus Standard Regimens in Subjects With Relapsed and Refractory Multiple Myeloma (RRMM)

NCT03651128Active, Not RecruitingPhase 3
Celgene
Posted 4/16/2019

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation

NCT02652767CompletedPhase 3
GenSight Biologics
Posted 2/23/2016

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

NCT02906202CompletedPhase 3
bluebird bio
Posted 8/8/2016

Patient-Centric Websites and Plain Language Key to Boosting Clinical Trial Recruitment

  • Clinical trial recruitment faces persistent challenges due to low participation rates, with trials often failing before completion due to insufficient enrollment.
  • Companies are revolutionizing trial recruitment by developing patient-friendly websites with plain language information, improved accessibility, and enhanced search engine optimization.
  • Industry expert Thomas Wicks highlights how transparent communication and cross-company collaboration in trial referrals are creating more opportunities for patient participation.

AstraZeneca and Daiichi Sankyo Partner in $6.9B Deal for Novel HER2-Targeting Cancer Drug

• AstraZeneca commits $3.5 billion upfront and up to $5.55 billion in milestone payments to co-develop trastuzumab deruxtecan, a promising antibody-drug conjugate for HER2-expressing cancers.
• The investigational drug has received FDA Breakthrough Therapy Designation for HER2-positive advanced breast cancer and shows potential across multiple tumor types including gastric, lung, and colorectal cancers.
• The collaboration aims to accelerate global development with five pivotal trials already underway, targeting a first regulatory filing in advanced breast cancer by late 2019.

Feasibility Pilot Trial on First-Pass Intubation Success Rates

A single-centre, randomised controlled feasibility pilot trial aimed to determine the feasibility for a larger definitive trial on first-pass intubation success rates. The study revealed an overall first-pass success rate of 78%, suggesting that a larger trial assessing a robust endpoint of first-pass success is feasible. The trial compared video laryngoscopy (VL) and direct laryngoscopy (DL), highlighting the need for further research to assess the effectiveness and potential biases of these techniques.

Merck KGaA Partners with Iktos to Accelerate Drug Discovery Through AI-Powered Molecular Design

• Merck KGaA has established a new collaboration with AI technology firm Iktos, leveraging deep generative models to expedite the discovery and design of novel drug compounds.
• The partnership aims to address a critical challenge in drug development by rapidly identifying molecules that meet multiple drug-like criteria for clinical testing, potentially accelerating patient access to new treatments.
• This marks Merck's second major AI initiative following their recent agreement with Cyclica, demonstrating the company's strategic commitment to incorporating artificial intelligence in their R&D process.

BrainStorm Announces First Patient in Phase 2 Clinical Trial for Progressive MS

BrainStorm Cell Therapeutics Inc. has enrolled the first patient in its Phase 2 clinical trial for progressive Multiple Sclerosis (MS), utilizing its NurOwn® technology platform. This trial represents a significant step towards addressing the unmet medical needs in progressive MS, with top-line data expected by mid-2020.

Safety and Efficacy of Repeated Administration of NurOwn (MSC-NTF Cells) in Participants With Progressive MS

NCT03799718CompletedPhase 2
Brainstorm-Cell Therapeutics
Posted 3/13/2019

Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients

NCT03280056CompletedPhase 3
Brainstorm-Cell Therapeutics
Posted 8/28/2017

Innovations and Trials in Medical Technology

Recent advancements in medical technology include the launch of the CrossWise RF transseptal access system by Circa Scientific, completion of enrollment in a pivotal trial for Inquis Medical's Aventus thrombectomy system, a distribution agreement between Medtronic and Contego Medical, a $12 million Series A round for Relief Cardiovascular, CE mark approval for Johnson & Johnson MedTech's dual-energy catheter, a temporary pause on Varipulse PFA system cases by Johnson & Johnson MedTech, and the completion of a first-in-human study by AerWave for its lung denervation therapy.

Expanding Cancer Clinical Trial Eligibility to Include More Patients

The National Cancer Institute (NCI) and other organizations are working to broaden eligibility criteria for cancer clinical trials, aiming to include more patients and make trial results more applicable to the general population. This effort includes relaxing restrictions on age, HIV status, and other factors, while ensuring patient safety and the integrity of trial outcomes.

FDA Approves Resmetirom (Rezdiffra) for Treating MASH and Associated Fibrosis

The FDA has approved resmetirom, marketed as Rezdiffra, for the treatment of adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate to advanced liver fibrosis. This marks a significant advancement in addressing a condition that affects millions globally, particularly those with type 2 diabetes and obesity.

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