SciTech Development, Inc.’s Post

So, the basic pathway through clinical trials is phase 1 -> phase 2 -> phase 3. But that’s not the whole story. There are others… n-of-1 trials are a way of obtaining useful data from and for the treatment of individual patients Phase 0 are very small initial trials using a single, low dose of the test drug Phase 1/2 are combined phase 1 and 2, “first in man”, treating small numbers of patients with the target disease. They provide data on safety, drug metabolism, and also on efficacy against the target disease. Phase 2 trials may be split into small phase 2a and larger phase 2b trials. Only a single phase 3 trial may be needed for rare diseases where high patient numbers can be difficult to find. Phase 4, large scale (post-approval) trials are undertaken in order to investigate issues of concern or to target additional indications. So far, for phages, no phase 3 trials have been carried out. Earlier stage trials have been carried out. But pure phase 1 trials are largely meaningless (though still undertaken by some groups) since the input phage needs to replicate in order to produce the therapeutic dose. Without the target bacteria being present, phage cannot replicate so will not be able to generate the relevant dose. Some phase 1 work has used higher and repeated doses to try to obtain useful data, but this is not the same thing as phage replication in the body. In addition, this runs the risk of leading to the assumption that such dosing is needed, as with conventional antibiotics. But phase 1/2 can be “first in man” trials while allowing phages to replicate. They have been shown to be a viable approach for phage therapeutics. I know that for a fact. I ran the first one. See: Lillie EO et al. The n-of-1 clinical trial: the ultimate strategy for individualizing medicine? Per Med. 2011 Mar;8(2):161-173. doi: 10.2217/pme. https://lnkd.in/eEEjgGTk. Wright A et al (2009). A controlled clinical trial of a therapeutic bacteriophage preparation in chronic otitis due to antibiotic-resistant Pseudomonas aeruginosa; a preliminary report of efficacy. Clinical Otolaryngology 34: 349-357. #bacteriophage #phage #clinical trials #first_in_man #n-of-1 #phase_1/2 #phase_2a #phase_2b #phagetherapy #thephageadviser drh@thephageadviser.com

So, the basic pathway through clinical trials is phase 1 -> phase 2 -> phase 3. But that’s not the whole story. There are others… n-of-1 trials are a way of obtaining useful data from and for the treatment of individual patients Phase 0 are very small initial trials using a single, low dose of the test drug Phase 1/2 are combined phase 1 and 2, “first in man”, treating small numbers of patients with the target disease. They provide data on safety, drug metabolism, and also on efficacy against the target disease. Phase 2 trials may be split into small phase 2a and larger phase 2b trials. Only a single phase 3 trial may be needed for rare diseases where high patient numbers can be difficult to find. Phase 4, large scale (post-approval) trials are undertaken in order to investigate issues of concern or to target additional indications. So far, for phages, no phase 3 trials have been carried out. Earlier stage trials have been carried out. But pure phase 1 trials are largely meaningless (though still undertaken by some groups) since the input phage needs to replicate in order to produce the therapeutic dose. Without the target bacteria being present, phage cannot replicate so will not be able to generate the relevant dose. Some phase 1 work has used higher and repeated doses to try to obtain useful data, but this is not the same thing as phage replication in the body. In addition, this runs the risk of leading to the assumption that such dosing is needed, as with conventional antibiotics. But phase 1/2 can be “first in man” trials while allowing phages to replicate. They have been shown to be a viable approach for phage therapeutics. I know that for a fact. I ran the first one. See: Lillie EO et al. The n-of-1 clinical trial: the ultimate strategy for individualizing medicine? Per Med. 2011 Mar;8(2):161-173. doi: 10.2217/pme. https://lnkd.in/e_5-2FP3. Wright A et al (2009). A controlled clinical trial of a therapeutic bacteriophage preparation in chronic otitis due to antibiotic-resistant Pseudomonas aeruginosa; a preliminary report of efficacy. Clinical Otolaryngology 34: 349-357. #bacteriophage #phage #clinical trials #first_in_man #n-of-1 #phase_1/2 #phase_2a #phase_2b #phagetherapy #thephageadviser drh@thephageadviser.com

Atara is at the forefront of innovation for allogeneic #CAR #Tcell therapies as we advance novel therapeutic approaches. The FDA has cleared our Investigational New Drug (IND) application for ATA3219 as a potential treatment for systemic lupus erythematosus (#SLE) patients with #lupusnephritis. This is the second IND clearance for ATA3219, expanding the clinical development program beyond oncology into autoimmune disease.   ATA3219 combines the natural biology of unedited T cells with the promise of an allogeneic therapy as we aim to bring a CAR T-cell therapy option to more patients. Read more about this significant milestone for Atara: https://bit.ly/3UTAZK0

🖇Example: In a clinical trial testing a new cancer drug, the initial phases might involve a double-blind setup where neither the patients nor the doctors know who is receiving the drug and who is receiving a placebo. If the drug shows significant benefits, the trial might move to an open-label phase where all participants are informed about and given the actual drug to further evaluate its effectiveness and monitor any long-term side effects. Recently, the Gilead Science run PURPOSE 1 study of which DTHF was a part of, has started moving into an open-label phase given the exceptional preliminary effectiveness results of Lenacapivir. 📍Why It Matters: The open-label phase is important for understanding how a treatment performs in a real-world setting without the constraints of blinding. It provides valuable information on the drug's safety profile, adherence, and long-term benefits or risks, contributing to the overall assessment of its clinical utility. Related Terms: 🔍Double-Blind Study 🔍Randomized Controlled Trial (RCT) 🔍Clinical Trial Phases 🔍Placebo-Controlled Study 🔍Bias Reduction #research #research101 #researchbuzzwords #researchjargon #jargon #terminilogy #openlabel #clinicaltrials #lenacapivir

Endevica Bio Doses First Patient in Phase 1 Clinical Trial of TCMCB07 for Cachexia Endevica Bio has commenced the first patient dosing in a Phase 1 clinical trial for their peptide-based drug candidate, TCMCB07, aimed at treating cachexia. Cachexia is a severe condition characterized by significant weight loss and muscle wasting, often linked with cancer and other chronic diseases. The Phase 1 trial will evaluate the safety and tolerability of TCMCB07 in healthy volunteers. Key objectives include assessing the drug’s safety profile and understanding its pharmacokinetics and pharmacodynamics. TCMCB07 is a novel peptide therapeutic designed to target the underlying mechanisms of cachexia. The successful outcome of this trial could represent a significant advancement in the treatment of cachexia, offering new therapeutic options for patients affected by this debilitating condition.

In this engaging session, our panel of Parexel experts will discuss critical challenges and potential solutions in developing drugs for Acute Myeloid Leukemia (AML), with a special focus on relapsed and refractory cases. Key topics include: - Current obstacles in AML drug development - Regulatory considerations for novel therapies - Strategies for improving clinical trial design - Emerging trends and innovative approaches Whether you're a researcher, clinician, or industry professional, this webinar will provide you with actionable knowledge to advance AML drug development and improve patient outcomes. Don't miss this opportunity to gain valuable insights from both clinical and regulatory perspectives. Register now to be part of this important conversation! Registration: https://lnkd.in/e_A__gM4 #Oncology #Webinar #ParexelBiotech

Vorasidenib has been approved by the FDA for use in the treatment of adults or paediatric patients older than 12 with grade 2 #astrocytoma or #oligodendroglioma who have a susceptible IDH1 or IDH2 mutation. The decision was made based on the results of the #INDIGO trial, which found #vorasidenib to significantly improve progression-free survival in patients. The therapeutic is the first to be approved for this specific subset of patients with astrocytoma or oligodendroglioma. #cancer #clinicaltrials #cancertrials https://lnkd.in/gyTa43qR

There are some very good recommendations from FDA on eligibility criteria for oncology clinical trials: 1. Lab values - They recommend to consider only the lab values important for the drug and the ones which are less important to be more relaxed so more patients could qualify for the study. 2. Washout and concomitant medications - The new guideline recommends that patients are not excluded unnecessary because of concomitant medications. This is particularly relevant for older population which tends to be taking more medications. 3. Performance status - The recommendation is to consider the performance status of the patients with the medical condition rather than aiming for fit patients only. #clinicaltrials #oncology #FDA

Glioblastoma Awareness Day: Join us in making a difference! July 17 marks Glioblastoma Awareness Day, a time to reflect on the global impact of this brain cancer. At Soterius, we understand the profound effects on individuals and communities worldwide. Glioblastoma affects a large number of people, challenging healthcare systems and demanding innovative solutions. As a leader in Pharmacovigilance and Drug Safety, we manage pharmacovigilance not only for clinical trials but also in post-marketing phases, ensuring the safety and effectiveness of medications throughout their lifecycle. Together, let's continue our journey toward a healthier future. #glioblastoma #cancer #oncology #braincancer #brain #glioblastomaawareness #health #healthcare #pv #pharmacovigilance #drugsafety #gbmawareness #17July #GBMDay #braintumour

In this engaging session, our panel of Parexel experts will discuss critical challenges and potential solutions in developing drugs for Acute Myeloid Leukemia (AML), with a special focus on relapsed and refractory cases. Key topics include: - Current obstacles in AML drug development - Regulatory considerations for novel therapies - Strategies for improving clinical trial design - Emerging trends and innovative approaches Whether you're a researcher, clinician, or industry professional, this webinar will provide you with actionable knowledge to advance AML drug development and improve patient outcomes. Don't miss this opportunity to gain valuable insights from both clinical and regulatory perspectives. Register now to be part of this important conversation! Registration: https://lnkd.in/e7AVRnRw #Oncology #Webinar #ParexelBiotech