Clinical Trial News - 17Mar2025

The clinical trial landscape continues to evolve rapidly, with new developments in drug approvals, study designs, and regulatory frameworks. This update provides a snapshot of the latest advancements in clinical research as of March 17, 2025.

Breakthrough in Alzheimer's Treatment

Alzheimer’s Drug (Phase III): Early data indicate a 30% reduction in cognitive decline. This Phase III trial, conducted by NeuroPharma, demonstrated a significant slowing of disease progression in patients with early-stage Alzheimer's. This development could potentially change the landscape of Alzheimer's care and provide hope to millions of patients and their families.

Vaccine Trial Updates

Several new COVID-19 vaccine trials have commenced this month, focusing on next-generation vaccines designed to tackle emerging variants. Notably, BioVaxCorp launched a Phase II trial for its multivalent vaccine, which aims to provide broader protection against multiple strains of the virus.

The U.S. FDA placed BioNTech’s malaria vaccine study on clinical hold. The U.S. Food and Drug Administration has placed a clinical hold on BioNTech's early-to-mid stage trial of an experimental malaria vaccine, the drugmaker said in a filing. (Reuters)

HIV Treatment Advances

Gilead Sciences, Inc. (Nasdaq: GILD) has unveiled groundbreaking data and several oral presentations from its cutting-edge HIV treatment portfolio at the Conference on Retroviruses and Opportunistic Infections (CROI 2025). The findings underscore the company's innovative approach and swiftly progressing pipeline aimed at expanding treatment options and improving outcomes for individuals living with HIV, while also striving towards a cure. (Gilead)

Advances in Cancer Immunotherapy

In the realm of cancer research, a novel immunotherapy approach has shown remarkable efficacy in treating metastatic melanoma. The study, led by OncoImmuno Inc., reported a 70% response rate in patients who had previously exhausted other treatment options. These findings have sparked optimism for the future of cancer immunotherapy.

Regulatory Changes and New Guidelines

The FDA has issued final guidance on decentralized clinical trials (DCTs), addressing key issues such as data variability and investigator oversight, aiming to make the process more patient-centric and efficient. These changes are expected to facilitate greater participation and streamline trial operations, ultimately accelerating the development of new therapies. ( FDA Guidance)

The EMA has adopted the E6(R3) Good Clinical Practice guidelines, effective July 23, 2025, aligning with international standards for clinical trials. (EMA Recommendations)

Expansion of the Middle East and North Africa Clinical Trial Market

The "Middle East and North Africa Clinical Trial Infrastructure Market Report 2025" indicates that recent regulatory reforms have facilitated more expedient approvals, while the growth of public-private partnerships has enhanced research capabilities.

This report presents a comprehensive analysis of the clinical trial infrastructure markets within the Middle East and North Africa (MENA) region. It offers an in-depth examination of market dynamics and provides a thorough understanding of the MENA clinical trial landscape, categorized by study status, phase, type, results, and sponsor type. (Research and Markets)

Figure 1. Middle Eastern and North African Clinical Trial Infrastructure Market

Source Links

Topic	Link
FDA Guidelines for DCTs	https://www.fda.gov/regulatory-information/search-fda-guidance-documents/conducting-clinical-trials-decentralized-elements
EMA Recommendation Paper on DCTs	https://health.ec.europa.eu/system/files/2023-03/mp_decentralised-elements_clinical-trials_rec_en.pdf
Gilead HIV Treatment News Release 12Mar2025	https://www.gilead.com/news/news-details/2025/gilead-presents-new-hiv-treatment-and-cure-research-data-at-croi-2025-including-an-investigational-long-acting-twice-yearly-therapy-option

Conclusion

The clinical trial landscape continues to evolve rapidly, with each month bringing new and exciting developments. March 2025 has proven to be a significant period for advancements in various medical fields, and these breakthroughs underscore the importance of ongoing research and innovation in improving patient outcomes. Stay tuned for more updates as we continue to track the progress of these and other critical trials.

ClinicalTrials.gov Analyses - 13Mar2025

Top 25 Conditions Being Studied in Clinical Trials

Industry-Sponsored Clinical Trials with a start date after 01Jan2025

Clinical Trial News - 10Mar2025

FDA News Release – FDA Roundup: March 11, 2025

11 March 2025

The FDA shared updates in its March 11, 2025, roundup. Key points include:

Data Integrity Concerns: A General Correspondence Letter was issued to Mid-Link Technology Testing Co., Ltd., which has submitted falsified or copied study data. The FDA has rejected all studies from this facility until it resolves these issues, urging manufacturers to independently verify third-party testing results.

Fat-Dissolving Injections Warning: The FDA issued six warning letters to companies selling unapproved fat-dissolving injectable drug products, emphasizing the potential harm of such products.

Biosimilar Approval: The FDA approved Omlyclo (omalizumab-igec) as the first interchangeable biosimilar to Xolair for treating allergic, inflammatory, and autoimmune conditions. It works by blocking IgE, the antibody responsible for allergic reactions.

Mutated DNA Restored to Normal in Gene Therapy Advance

Researchers successfully restored a mutated gene to its normal state for the first time, marking a breakthrough in gene therapy. In a small study by Beam Therapeutics, nine patients with alpha-1 antitrypsin deficiency (AATD)—a genetic disorder causing liver and lung damage—received a single infusion of lipid nanoparticles carrying a CRISPR-based gene editor. This editor precisely corrected the mutation by replacing a single incorrect DNA base.

Patients receiving the highest dose produced sufficient levels of the functional protein to potentially halt further organ damage. This innovative technique represents a significant step toward developing precise, one-time treatments for genetic diseases. Long-term follow-up will confirm its safety and lasting benefits.

Base editing, which corrects misspellings in DNA, offers an alternative to existing forms of gene therapy, which either add or remove things from the genome.

Australian man survives 100 days with artificial heart in world-first success

10 March 2025

An Australian man with severe heart failure has made history as the first person to leave a hospital with a total artificial heart implant. The BiVACOR total artificial heart, designed by Dr. Daniel Timms, uses magnetic levitation technology to replicate the natural blood flow of a healthy heart. This groundbreaking device served as a bridge for the patient, allowing him to live over 100 days with the implant before successfully receiving a donor heart transplant in early March.

The achievement has been hailed as a major medical milestone, with researchers emphasizing its potential to transform heart failure treatment globally. The BiVACOR heart aims to provide a life-saving alternative for patients awaiting transplants and, in the future, could even eliminate the need for donor hearts altogether.

FDA News Release – FDA Roundup: March 7, 2025

07 March 2025

The FDA's March 7, 2025, roundup highlighted two significant updates:

Approval of Encelto (revakinagene taroretcel-lwey), an allogeneic cell-based gene therapy approved for treating idiopathic macular telangiectasia type 2 (MacTel), a rare progressive eye disease. Administered through a single surgical procedure, it works by expressing recombinant human ciliary neurotrophic factor to support the survival of macular photoreceptors.

Expanded Use of Neffy (epinephrine nasal spray): Now approved for children aged 4 and older, weighing 15-30 kg, for emergency treatment of anaphylaxis. Originally approved in 2024 for individuals weighing 30 kg or more. This is the first nasal spray approved for anaphylaxis, offering a non-injection alternative.

J&J to stop studies of depression drug due to low effectiveness

06 March 2025

Johnson & Johnson has decided to halt late-stage trials of aticaprant, an experimental drug for major depressive disorder, due to insufficient effectiveness. This drug targeted anhedonia—a condition that diminishes the ability to feel pleasure. Neumora Therapeutics' navacaprant, a similar drug, also recently failed to show effectiveness in trials.

J&J will explore other uses for aticaprant in areas of unmet need, while continuing to develop its neuroscience portfolio, which includes the approved nasal spray Spravato and other treatments in development. Their recent $14.6 billion acquisition of Intra-Cellular Therapies underscores their commitment to brain disease treatments.

These universities have the most retracted scientific articles

27 February 2025

A first-of-its-kind analysis by Nature reveals which institutions are retraction hotspots.

Clinical Trials Market Size is Estimated to be US$ 149.58 Billion by 2034

19 February 2025

The global clinical trials market is projected to grow significantly, reaching an estimated size of $149.58 billion by 2034, up from $83.75 billion in 2024. This growth, at a compound annual growth rate (CAGR) of 6.10% from 2025 to 2034, is driven by increased investments in pharmaceutical R&D, a surge in drug candidates, and advancements in medical technology.

Key highlights include:

• North America held the largest market share (59.3%) in 2024, while Asia Pacific is expected to grow at the fastest rate (CAGR of 7.16%).
• The oncology segment contributed the largest market share (38.4%) in 2024.
• Interventional studies dominated with a 77.96% market share in 2024.
• Phase III trials accounted for the largest share (49.1%) in 2024.
• The market's expansion reflects the growing demand for innovative treatments and the globalization of clinical trials to include diverse patient populations.

New weight loss drugs aim to save muscle

19 February 2025

About a dozen drugmakers are developing new weight-loss treatments aimed at preserving muscle, and industry analysts, clinical trial experts and doctors say they may also need to demonstrate additional health benefits beyond the numbers on a scale to get approved.

Manual vs AI-Assisted Prescreening for Trial Eligibility Using Large Language Models—A Randomized Clinical Trial

17 February 2025

Patient identification and screening are persistent challenges in clinical trial recruitment and often result in delayed and incomplete patient enrollment, exacerbating the high cost of cardiovascular outcomes trials, which can be in hundreds of millions of dollars. Traditional manual screening methods are labor-intensive and may contribute up to one-third of clinical trial costs. Structured electronic health record queries improve the efficiency of clinical trial screening but still require manual review of unstructured data. Large language models (LLMs) parse unstructured data, which enables a shift in how patients are screened and enrolled. We developed an LLM tool, the Retrieval Augmented Generation Enabled Clinical Trial Infrastructure for Inclusion Exclusion Review (RECTIFIER), and demonstrated retrospectively that it can assess patient eligibility for specific inclusion and exclusion criteria with sensitivity, specificity, and accuracy exceeding that of study staff.

Genome Engineering Market To Reach USD 30.9 Billion by 2032, Growing at CAGR 16.7% | Agilent Technologies, Bluebird Bio, Boehringer Ingelheim

12 February 2025

The shift towards personal medicine is changing healthcare, running the demand for genome engineering. By taking advantage of advanced equipment such as CRISPR, scientists can now edit genes with remarkable accuracy to develop cancer, rare diseases and adapted treatment for chronic conditions. The promise of more effective, patient-specific remedies has made this approach highly attractive to researchers, healthcare providers and pharmaceutical companies. This increased attention on accurate therapy not only improves patient results, but also improves innovation, promotes growth in the genome engineering market as the medical industry strives to deal with diseases on its genetic roots.

Genome engineering increases moral concerns, especially when it involves human germinal editing. The possibility of creating "designer infants" or inadvertently transforms future generations increases the widespread debate. In addition, strict rules limit certain research areas and slow down the speed of innovation. Public apprehensions around safety requirements and security and current obstacles for both researchers and companies’ misuse. These moral and legal complications prevent the entire capacity of the market, forcing stakeholders to navigate a highly regulated environment to balance innovation with moral ideas and gain public belief.

Clinical Trial News - 24Feb2025

22 February 2025

Identifying key randomised clinical trials that could transform clinical care and public health

Clinical trials have been pivotal to the improvements in public health and clinical care, leading to many millions of additional years of healthy life globally. Some examples of disease areas in which outcomes have improved include primary and secondary prevention of cardiovascular disease, breast cancer, some forms of leukaemia, and HIV treatment and prevention. In all of these disease domains, clinical practitioners have embraced the centrality of evidence from clinical trials and large-scale randomised clinical trial (RCT) collaborations have been implemented in partnership with patients, health systems, industry, and other partners.

21 February 2025

F.D.A. Firings Decimated Teams Reviewing A.I. and Food Safety

The Trump administration has implemented significant layoffs at the FDA, targeting recently hired employees in the food safety, medical devices, and tobacco products divisions. These cuts are part of a broader effort to reduce the federal workforce, impacting the FDA's ability to review the safety of food additives and medical devices. The layoffs have raised concerns about the agency's capacity to protect public health, especially given the FDA's reliance on newer staff with up-to-date technical skills.

 

21 February 2025

FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease

The FDA has approved Ctexli (chenodiol) as the first treatment for cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease. This approval provides a new therapeutic option for adults suffering from CTX, which can lead to neurological and systemic complications if left untreated.

21 February 2025

US FDA approves Mirum Pharma's genetic disorder drug

The U.S. Food and Drug Administration (FDA) has approved Mirum Pharmaceuticals' drug, Livmarli, for treating progressive familial intrahepatic cholestasis (PFIC), a rare genetic liver disorder. This approval allows the drug to be used for reducing severe itching in patients aged five years and older. The decision was based on positive results from a late-stage trial involving 93 patients.

13 February 2025

How Trump’s Medical Research Cuts Would Hit Colleges and Hospitals in Every State

Changes to a key funding formula will reduce research grants at hospitals and universities by billions — and may discourage future research.

The Trump administration's decision to cut funding for the National Institutes of Health (NIH) is causing significant concern in the scientific community. By capping indirect costs at 15%, the administration has effectively reduced the budget for essential administrative and facility expenses. This move has led to lawsuits and fears that major research institutions will struggle to continue their work, potentially stalling scientific progress and impacting the careers of young researchers.

 

19 February 2025

Artificial intelligence for modelling infectious disease epidemics

Infectious disease threats to individual and public health are numerous, varied and frequently unexpected. Artificial intelligence (AI) and related technologies, which are already supporting human decision making in economics, medicine and social science, have the potential to transform the scope and power of infectious disease epidemiology. Here we consider the application to infectious disease modelling of AI systems that combine machine learning, computational statistics, information retrieval and data science. We first outline how recent advances in AI can accelerate breakthroughs in answering key epidemiological questions and we discuss specific AI methods that can be applied to routinely collected infectious disease surveillance data. Second, we elaborate on the social context of AI for infectious disease epidemiology, including issues such as explainability, safety, accountability and ethics. Finally, we summarize some limitations of AI applications in this field and provide recommendations for how infectious disease epidemiology can harness most effectively current and future developments in AI.

20 January 2025

Young people are crucial to research — how can they be paid fairly?

Young people are playing a crucial role in research, but compensating them fairly presents ethical and practical challenges. Kids and teens, aged 12 to 24, are increasingly involved as advisers and co-researchers, not just participants. This shift, supported by major funders, aims to empower youth through participatory action research, where they identify research questions and analyze data themselves. Their involvement has led to significant contributions, such as policy recommendations for climate adaptation and blocking child marriages in Bangladesh.

 

19 February 2025

Vaccine targeting pancreatic cancer shows promise in new study of clinical trial

A clinical trial at Memorial Sloan Kettering Cancer Center is showing promise for a new mRNA vaccine targeting pancreatic cancer. The trial, led by Dr. Vinod Balachandran, combines standard treatments with a personalized vaccine designed from the patient's tumor. This approach aims to teach the immune system to recognize and attack cancer cells, inspired by the immune responses seen in long-term pancreatic cancer survivors. Early results from the phase 1 trial, which included 16 patients, suggest that the vaccine could potentially extend survival times for patients with this typically deadly cancer.

 

18 February 2025

Can large, simple trials bring drug developers back to common diseases?

Martin Landray, a physician and epidemiologist at Oxford University, argues that large, simple clinical trials can make drug development for common diseases more affordable and efficient. He highlights the success of the UK's RECOVERY trial during the COVID-19 pandemic, which tested multiple treatments on a modest budget. Landray's non-profit, Protas, aims to replicate this model for other common diseases, reducing the cost and complexity of phase III trials. This approach could encourage drug developers to invest more in treatments for widespread health issues like heart disease and stroke.

 

1.6 Million Clinical Research Records With PII and Patient Medical Info Exposed in Data Breach

A data breach at DM Clinical Research exposed over 1.6 million records containing sensitive personal and medical information. The unprotected database included names, dates of birth, contact details, vaccination statuses, and other health-related data. Discovered by cybersecurity researcher Jeremiah Fowler, the breach was quickly reported and secured. However, the incident raises significant concerns about data privacy and the protection of patient information.

18 February 2025

2024's top 10 clinical trial flops

Fierce Biotech's report on the top 10 clinical trial flops of 2024 highlights some significant setbacks in drug development. Notable failures include AbbVie's emraclidine for schizophrenia, which flunked phase 2 trials, and GSK's HSV vaccine, which failed in phase 2, ceding the race to Moderna. These high-profile flops underscore the inherent risks in clinical-stage drug development, particularly in challenging therapeutic areas like Alzheimer's disease, schizophrenia, and herpes simplex virus.

  

30 January 2025

FDA Approves Novel Non-Opioid Treatment for Moderate to Severe Acute Pain

The FDA has approved Journavx (suzetrigine), a new non-opioid medication for treating moderate to severe acute pain in adults. This first-in-class drug targets pain-signaling pathways involving sodium channels in the peripheral nervous system, reducing pain before it reaches the brain. The approval is based on successful trials showing significant pain reduction compared to placebo. This new option aims to provide effective pain relief while mitigating the risks associated with opioid use.

Introducing JOURNAVX™ (suzetrigine) for Moderate-to-Severe Acute Pain

 

31 January 2025

European Medicines Agency Clinical Trials Regulation becomes fully applicable

The Clinical Trials Regulation (CTR) is now fully applicable across the European Union (EU) as of January 31, 2025. This regulation replaces the previous Clinical Trials Directive and aims to streamline the submission, assessment, and oversight of clinical trials through the Clinical Trials Information System (CTIS). All ongoing trials must now comply with the CTR, and any that haven't transitioned may face corrective measures. The CTR is designed to enhance transparency and make Europe a more attractive location for clinical research.