Clinical Trials
328
40 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
1841
Active, not recruiting
28
8.5%
Completed
182
55.5%
Enrolling By Invitation
1
0.3%
Not yet recruiting
11
3.4%
Recruiting
45
13.7%
Terminated
29
8.8%
Unknown
20
6.1%
Withdrawn
12
3.7%
No approval data available
- Andelyn Biosciences has partnered with Queen's University to develop and manufacture AAV9-GM2, a gene therapy candidate for GM2 gangliosidoses including Tay-Sachs and Sandhoff diseases. - The collaboration leverages Andelyn's regulatory-proven AAV Curator® Platform to execute a multi-phase program advancing AAV9-GM2 toward patient dosing. - GM2 gangliosidosis is a rare, inherited lysosomal storage disorder that progressively destroys nerve cells in the brain and spinal cord, causing severe neurological decline. - Andelyn brings more than 20 years of experience, having produced cGMP material for over 500 clinical batches and 85 global clinical trials.
- A new JAMA study analyzing 20 years of FDA cancer drug approvals reveals increasing reliance on surrogate endpoints and single-arm trials, including outside the accelerated approval pathway. - Researchers found that many recent regular approvals are based on weak surrogate endpoints rather than using the accelerated approval pathway, which requires confirmatory trials. - The study highlights that premature regular approval removes the safety net of confirmatory trial requirements and the ability to withdraw drugs that fail to demonstrate clinical benefit. - Lead author Brian Shkabari and senior author Bishal Gyawali emphasize that the accelerated approval pathway is being underutilized despite providing a balance between speed and evidence.
- The CHALLENGE trial, published in the New England Journal of Medicine, showed that a structured exercise program reduced disease recurrence or death by 28% in stage II/III colon cancer survivors over a median eight-year follow-up. - Patients in the exercise group had a 37% lower risk of death from any cause, with 90% overall survival at eight years compared to 83% in the control group receiving standard education alone. - A separate study found that patients with the most pro-inflammatory diets faced an 87% higher risk of death compared to those with the least inflammatory diets, suggesting nutrition and exercise interact to influence outcomes. - Researchers plan to analyze blood samples to identify biological mechanisms, including immune surveillance, chronic inflammation, and metabolic pathways that may explain exercise's anti-cancer effects.
- Australia has implemented tumour-agnostic immunotherapy reimbursement, broadening drug availability based on molecular markers rather than tumour origin. - The policy aims to improve equity of access but carries significant risks shared among government, industry, clinicians, and patients. - Authors Stevens and Karikios argue that a national health learning system is essential to convert this bold decision into usable evidence. - Rigorous evaluation of patient outcomes, quality use of medicines, equity of access, and toxicities is needed to balance speed, access, and certainty.
- 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.
- Researchers at Kingston Health Sciences Centre Research Institute have launched the first Health Canada-approved Phase 2 clinical trial examining psilocybin microdosing for generalized anxiety disorder. - The study will test 2-3 milligram daily doses of psilocybin in up to 60 adults over eight weeks, aiming to provide anxiety relief without hallucinogenic side effects. - Generalized anxiety disorder rates have more than doubled from 2.6% to 5.5% among Canadians aged 15 and older between 2012-2022, highlighting the need for new treatment approaches. - The trial represents a shift from traditional high-dose psychedelic studies that require medical supervision to home-based microdosing protocols.
- A comprehensive analysis of 50 recent Phase III cancer trials published in top journals found significant deficiencies in adherence to common-sense oncology principles for transparent and patient-centered research. - Only approximately one-third of the evaluated trials included quality-of-life outcomes, despite their critical importance to patients and clinical decision-making. - The study revealed that many trials failed to adequately justify their control arms or endpoints, and few reported chronic toxicity, cost, or patient-reported outcomes. - Researchers emphasize the urgent need for more rigorous, transparent cancer research that goes beyond statistical significance to address what truly matters to patients and clinicians.
- France's National Agency for Medicines and Health Products Safety (ANSM) has approved compassionate access for Agenus' botensilimab plus balstilimab combination in patients with refractory microsatellite-stable metastatic colorectal cancer. - The treatment is fully reimbursed by France's Assurance Maladie for eligible patients meeting specific criteria, including MSS status and no active liver metastases. - Clinical data from Phase 1b trials demonstrate a 20% objective response rate and median overall survival of 20.9 months in heavily pretreated MSS colorectal cancer patients. - The global Phase 3 BATTMAN trial is set to launch in November 2025, enrolling 834 patients to evaluate the combination against best supportive care.
- A groundbreaking 17-year international clinical trial involving 889 colon cancer patients shows that structured exercise programs reduce cancer recurrence risk by 28% and death risk by 37%. - The CO.21 Challenge study, published in the New England Journal of Medicine, is the first clinical trial designed specifically to test whether exercise can improve cancer survival outcomes. - Researchers emphasize that exercise should now be considered a standard treatment for colon cancer rather than just a quality-of-life intervention, requiring integration into healthcare systems. - The study involved patients with Stage 3 or high-risk Stage 2 colon cancer who completed surgery and chemotherapy, with participants following a 3-year structured exercise program of 2.5 hours weekly moderate-intensity activity.
- myeloMATCH, a collaborative effort by leading cancer research organizations, launches clinical trials to advance precision medicine for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). - The program offers advanced biomarker testing to identify genomic and molecular features, matching patients with targeted treatments in a tiered approach across their treatment journey. - Three treatment trials are now open, focusing on initial induction, residual disease, and consolidation therapy, with the goal of improving outcomes and developing new therapies for myeloid malignancies. - The initiative aims to enroll 5,000 patients, tracking their progress and clonal evolution to refine treatments and address unmet needs in AML and MDS management.
