Clinical Trials
772
43 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
1925
Active, not recruiting
27
3.5%
Completed
457
59.2%
Enrolling By Invitation
5
0.7%
No Longer Available
2
0.3%
Not yet recruiting
11
1.4%
Recruiting
112
14.5%
Suspended
4
0.5%
Temporarily Not Available
1
0.1%
Terminated
86
11.1%
Unknown
1
0.1%
Withdrawn
66
8.6%
No approval data available
- A new analysis published in JCO Global Oncology found that Imfinzi (durvalumab) significantly improves survival in limited-stage small cell lung cancer patients but is not cost-effective at current U.S. list prices. - The study showed Imfinzi extended median overall survival to 66.1 months compared to 57.8 months with standard care, but resulted in an incremental cost-effectiveness ratio of $383,069 per quality-adjusted life year. - Researchers from Sylvester Comprehensive Cancer Center highlighted the need to align clinical innovation with cost-effectiveness to ensure sustainable adoption in the U.S. healthcare system. - The analysis suggests that while Imfinzi represents clinical progress for a disease with historically poor outcomes, its high cost may limit patient access and worsen financial toxicity.
- Immorta Bio published preclinical data in the Journal of Translational Medicine showing SenoVax™ significantly reduced tumors in mouse models of lung, brain, pancreatic, and breast cancers. - The novel immunotherapy targets senescent cells to reprogram the tumor microenvironment, converting immunologically "cold" tumors to "hot" by boosting CD8+ T-cell infiltration. - The company has filed IND #30745 with the FDA for a first-in-human trial in advanced lung cancer and is completing GMP manufacturing requirements. - SenoVax™ demonstrated statistically significant tumor growth inhibition and prolonged survival in both orthotopic and syngeneic models as monotherapy and in combination treatments.
- UCLA and UC Davis will co-lead the PRISM Trial, a $16 million randomized controlled study evaluating artificial intelligence support for mammogram interpretation across seven academic medical centers. - The study represents the first large-scale randomized trial of AI in breast cancer screening in the United States, involving hundreds of thousands of mammograms across California, Florida, Massachusetts, Washington, and Wisconsin. - Researchers aim to determine whether AI tools help radiologists detect more cancers or simply increase false positives, addressing a critical evidence gap as AI-assisted mammograms become increasingly common in clinical practice. - The patient-centered trial will maintain routine screening procedures while randomly assigning mammograms to be interpreted either by radiologists alone or with FDA-cleared AI assistance from Transpara by ScreenPoint Medical.
- The HDAC inhibitors market across seven major markets is expected to surge significantly by 2034, driven by innovation in cancer therapies and expanding applications beyond traditional hematologic malignancies. - Several next-generation HDAC inhibitors are advancing through clinical trials, including Abexinostat in Phase III for renal cell carcinoma and Quisinostat in Phase II for uveal melanoma. - Recent market entrants include DUVYZAT for Duchenne muscular dystrophy and HIYASTA for T-cell lymphomas, demonstrating the therapeutic potential of HDAC inhibitors beyond oncology applications. - The market faces challenges from toxicity issues and competition from newer targeted therapies, but combination approaches with immune checkpoint inhibitors and precision oncology trends are creating new growth opportunities.
- Longeveron has licensed US Patent 12,168,028 B2 from the University of Miami, protecting unique GHRH-Receptor+ cardiomyogenic cells derived from induced pluripotent stem cells. - The new technology addresses a critical safety barrier in cardiac stem cell therapy by eliminating cells that cause malignant ventricular arrhythmias, a complication that has slowed clinical development. - This advancement expands Longeveron's stem cell therapy pipeline beyond their current programs in hypoplastic left heart syndrome, Alzheimer's disease, and pediatric dilated cardiomyopathy. - The technology has potential applications across a broad range of cardiovascular diseases in both adult and rare pediatric populations.
- The BIRD Foundation approved a fifth milestone payment of approximately $166,000 USD to support continued development of iTOL-102, a potential cure for Type 1 diabetes that would eliminate the need for lifelong immunosuppression. - The funding follows a successful Type B Pre-IND meeting with the FDA in February 2025, where the agency provided constructive guidance on the clinical development pathway for First-in-Human trials. - iTOL-102 combines Kadimastem's IsletRx stem cell-derived insulin-producing islet-like cells with iTolerance's iTOL-100 immunomodulatory platform, demonstrating functional insulin secretion and disease reversal in preclinical diabetic animal models. - The companies are currently preparing for safety toxicology studies and clinical trial submissions based on FDA guidance, with total BIRD Foundation support reaching nearly $882,352 USD to date.
- Florida becomes the first state to offer comprehensive newborn genetic screening through the Sunshine Genetics Pilot Program, providing free genetic sequencing to identify serious but treatable conditions before symptoms appear. - The initiative receives $7.5 million in state funding plus an additional $2 million from Florida State University, establishing the Institute for Pediatric Rare Diseases as the program's central hub. - The Sunshine Genetics Consortium unites Florida's top universities including FSU, UF, UM, USF, and FIU with hospitals and biotech companies to accelerate research and improve access to treatments for rare childhood diseases. - The program aims to end the "Diagnostic Odyssey" that families endure when seeking answers about rare diseases, with early diagnosis enabling proactive care and better outcomes for affected children.
- The FDA has authorized emergency compassionate use of ZyVersa Therapeutics' VAR 200 for a patient with ApoCII amyloidosis, an ultra-rare kidney disease with no effective treatment options. - VAR 200, currently in Phase 2a trials for diabetic kidney disease, targets lipid accumulation in kidneys and was selected based on evidence of lipotoxic glomerular injury in the patient's kidney biopsy. - The treatment will be administered at the University of Miami under Dr. Alessia Fornoni's care, following the protocol of the ongoing diabetic kidney disease clinical trial. - This compassionate use case represents a precision medicine approach to match patients with specific molecular signatures to targeted therapies for rare diseases.
- Adolore BioTherapeutics published preclinical data in Molecular Therapy demonstrating biosafety and efficacy of its rdHSV-CA8* gene therapy for chronic knee pain from osteoarthritis. - The gene therapy delivered via intra-articular injection showed profound analgesia lasting over 6 months without functional impairments or toxicity in mouse models. - The therapy works by activating Kv7 voltage-gated potassium channels to reduce nerve excitability, offering a potential non-opioid alternative for chronic pain treatment. - The company's lead program ADB-102 is fully funded by NIH/NINDS HEAL program with first-in-human studies expected to commence in 2026.
- The phase 2/3 Alliance A092104 trial evaluating olaparib plus temozolomide in advanced uterine leiomyosarcoma failed to meet its primary progression-free survival endpoint compared to standard care. - The combination showed a median PFS of 3.2 months versus 5.5 months with investigator's choice of pazopanib or trabectedin, leading to early trial closure for futility. - Despite promising early-phase data, the study highlights the challenge of demonstrating superiority over existing therapies in this rare cancer population. - The trial will be closed on October 1, 2025, with researchers emphasizing the need to better identify which patients might benefit from molecularly targeted approaches.
