Clinical Trial News
Blood-stage Malaria Vaccine Candidate RH5.1/Matrix-M Trial in Tanzania
A phase 1b trial of the blood-stage malaria vaccine candidate RH5.1/Matrix-M has been conducted in healthy Tanzanian adults and children, marking a significant step in malaria vaccine development.
FDA Approves Blinatumomab for CD19-Positive B-ALL Consolidation Phase
- The FDA has approved blinatumomab for treating CD19-positive, Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (B-ALL) in the consolidation phase.
- The approval is supported by the Phase 3 ECOG-ACRIN E1910 study, which showed superior overall survival with blinatumomab plus chemotherapy compared to chemotherapy alone.
- Another Phase 3 study, 20120215, demonstrated that blinatumomab led to a significantly higher 5-year overall survival rate compared to intensive chemotherapy in pediatric and young adult patients.
- Blinatumomab, a Bispecific T-cell Engager (BiTE®) therapy, is now a standard of care, offering a more effective treatment option than chemotherapy alone for B-ALL patients.
Highlighted Clinical Trials:
National Cancer Institute (NCI)
Posted 5/19/2014
National Cancer Institute (NCI)
Posted 12/17/2014
Lixte Biotechnology and Netherlands Cancer Institute Partner to Trial LB-100 in Metastatic Colon Cancer
- Lixte Biotechnology partners with the Netherlands Cancer Institute to evaluate LB-100 in metastatic colon cancer patients.
- The clinical trial will combine LB-100 with atezolizumab, a PD-L1 inhibitor, to enhance the immune response against cancer cells.
- Lixte will supply LB-100, while Roche will provide atezolizumab and fund the trial, with no clinical costs for Lixte.
- The trial includes a dose escalation phase followed by clinical activity and safety assessment in up to 37 patients over 24 months.
Deadline Approaching for Transitioning Clinical Trials from CTD to CTR
Sponsors and investigators are reminded of the approaching deadline of 30 January 2025 for transitioning clinical trials from the Clinical Trials Directive (CTD) to the Clinical Trials Regulation (CTR). Ongoing trials not concluded by this date must transition to avoid discontinuation. The process involves ensuring all substantial amendments are completed and documents harmonized or consolidated for multinational trials. Guidance documents and expedited review processes are available to facilitate the transition.
Xeltis' aXess Receives FDA IDE Approval for US Pivotal Trial in Hemodialysis Vascular Access
- Xeltis has received FDA approval for an Investigational Device Exemption (IDE) to initiate a US pivotal trial for its aXess vascular access conduit.
- aXess is designed to create a new, living vessel for hemodialysis, combining the benefits of arteriovenous fistulas and grafts, potentially reducing complications.
- The US trial follows positive 12-month data from a European first-in-human study and will run parallel to an ongoing EU pivotal trial.
- Xeltis' technology utilizes a regenerative approach, where the implant is gradually replaced by the patient's own tissue, offering a long-lasting vascular solution.
Highlighted Clinical Trials:
Xeltis
Posted 6/10/2021
FDA Panel Rejects MDMA Therapy for PTSD, Raising Concerns About Data and Safety
- An FDA advisory panel voted against approving MDMA-assisted therapy for PTSD, citing concerns about data credibility and research practices.
- Issues raised include potential bias due to unblinding in trials and strong prior beliefs about treatment benefits among participants and therapists.
- Concerns were also noted regarding a lack of diversity in trials, adverse effect data, and allegations of unethical conduct within the therapy model.
- Despite the setback, research into psychedelic therapies for mental health conditions continues, with the VA funding further studies.
UGN-102 Shows Durable Response in Non-Muscle Invasive Bladder Cancer Trial
- UroGen's UGN-102 demonstrated an 82.3% duration of response at 12 months in patients with low-grade, intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).
- The ENVISION trial results support UGN-102 as a potential non-surgical alternative to repeated surgeries for LG-IR-NMIBC, offering improved patient quality of life.
- A rolling New Drug Application (NDA) for UGN-102 has been submitted to the FDA, with completion expected in Q3 2024 and potential decision in Q1 2025.
- The ATLAS trial also showed a 55% reduction in recurrence, progression, or death with UGN-102 compared to transurethral resection of the bladder tumor (TURBT).
Highlighted Clinical Trials:
UroGen Pharma Ltd.
Posted 3/1/2022
Pfizer's DMD Gene Therapy Fails to Meet Primary Endpoint in Phase III Trial
- Pfizer's fordadistrogene movaparvovec gene therapy for Duchenne muscular dystrophy (DMD) did not meet the primary endpoint in the Phase III CIFFREO study.
- The study, involving patients aged 4-8, assessed motor function improvement using the North Star Ambulatory Assessment (NSAA) scale after one year.
- Key secondary endpoints, including 10-meter run/walk velocity and time to rise from the floor, also showed no significant difference compared to placebo.
- Pfizer plans to share more detailed results from the CIFFREO study at an upcoming medical meeting.
UroGen Pharma's UGN-102 Shows Promise in Phase 3 Trial for Bladder Cancer
- UroGen Pharma's UGN-102 met its primary endpoint in the Phase 3 ENVISION trial, demonstrating a high complete response rate at three months for low-grade non-muscle invasive bladder cancer.
- Many patients in the ENVISION trial maintained a complete response to UGN-102 at the 12-month mark, indicating durable efficacy.
- UroGen Pharma is advancing with a New Drug Application to the FDA for UGN-102, which could offer a significant alternative to current standard procedures.
- The potential market opportunity for UGN-102 is estimated to exceed $5 billion, highlighting the unmet need and potential impact of this novel treatment.
UroGen Pharma's UGN-102 Shows Promise in Bladder Cancer Treatment, Faces Market Cautiousness
- UroGen Pharma's UGN-102 demonstrated promising 12-month durability results in a Phase 3 ENVISION study, exceeding expectations for bladder cancer treatment.
- Goldman Sachs analyst Paul Choi maintains a Hold rating on UroGen Pharma, citing uncertainty regarding UGN-102's ability to replace the standard TURBT surgery.
- Despite positive clinical data and potential for priority review, caution is advised due to the entrenched nature of current surgical practices among urologists.
- Increased peak sales and market penetration projections for UGN-102 are noted, pending further regulatory information and assessment of UroGen Pharma's performance.