In society, the importance of health research, like the value of privacy, is immeasurable. It has the ability to give helpful data on disease trends and risk factors, treatment outcomes or public health interventions, functional abilities, care patterns, and health-care costs and utilization. The various research approaches provide complementary insights. Clinical trials can provide valuable information about the efficacy and side effects of medical interventions by limiting the variables that could affect the study’s results, but feedback from real-world clinical experience is also critical for comparing and improving the use of drugs, vaccines, medical devices, and diagnostics. For example, FDA approval of a drug for a specific indication is based on a series of controlled clinical trials, often with a few hundred to a few thousand patients, but after approval, it may be used by millions of people in a variety of settings. As a result, tracking clinical experience with the drug is critical for identifying relatively rare adverse effects and determining effectiveness in different populations or under different conditions. In order to produce best practices guidelines and assure high-quality patient care, it is also necessary to document and assess clinical practice experience. The development of Herceptin as a breast cancer treatment is a prime example of the advantages of using biological samples and patient records in research. Advances in health information technology are enabling a shift in health research that could allow previously impossible studies to be conducted, resulting in new insights into health and disease. Recently, clinical trials in different therapy areas can have an enormous impact on human health and longevity.

KX-826 (pyrilutamide) for the treatment of female androgenetic alopecia

Kintor Pharmaceutical Limited, a clinical-stage biotechnology company, announced that it had completed the enrollment of 160 patients for its phase II clinical trial of KX-826 (pyrilutamide) in China for the treatment of female androgenetic alopecia (AGA) on March 4, 2022, only four months after its initial launch. KX-826 is an androgen receptor (AR) antagonist with the potential to be a first-in-class topical treatment for AGA and acne vulgaris. Kintor Pharma announced on September 8, 2021, that the primary endpoint of the phase II clinical trial of KX-826 on adult male patients was met, with results showing good efficacy and safety profile.

The CIBIL clinical trial in kidney transplant patients

Cibiltech, a French MedTech company that develops artificial intelligence-based products for predictive medicine, has launched the CIBIL clinical trial to assess the performance of iBox AI as a prognostic measure of organ survival in kidney transplant patients.

The CIBIL trial, which stands for “Clinical Impact of the iBox as an Early Intervention tooL,” is a prospective randomized controlled trial being conducted internationally with 450 patients to assess the clinical and health economic benefits of using the iBox to predict allograft survival in kidney transplanted patients’ follow-up (NCT05112315).

‘GEN-001’ with Keytruda in biliary tract cancer patients

Genome and Company enters clinical trial collaboration with MSD to evaluate the safety and efficacy of its immuno-oncology microbiome therapeutic ‘GEN-001’ in combination with anti-PD-1 therapy Keytruda (pembrolizumab) in biliary tract cancer patients in a phase 2 trial.

Lactococcus lactis (L. lactis), a single live bacterial strain isolated from a healthy human, is the basis of GEN-001, an orally administered immuno-oncology microbiome therapeutic candidate. Biliary tract cancer is one of the cancers with a poor prognosis after diagnosis, limited treatment options, and a five-year survival rate of only 5 to 15%. According to the findings published in MDPI Cancers 2021, a SCI academic journal, immune cell infiltrations around cancer cells were seen in 70% of biliary tract cancer patients, confirming the relationship between immune cells and biliary tract cancer cells.

‘GEN-001’ is an oral microbiome therapeutic candidate that has been developed to have immune-modulating properties, potentially resulting in a collaboration with immune checkpoint inhibitors. ‘GEN-001’ is a single-strain bacteria (Lactococcus lactis, L.lactis) isolated from the gut of healthy human volunteers and shown to activate dendritic cells, macrophages, and T cells. In preclinical studies, ‘GEN-001’ demonstrated an optimal safety margin and synergistic effects when combined with immune checkpoint inhibitors, suppressing the growth of both immune checkpoint inhibitor sensitive and resistant tumor models.

Efanesoctocog alfa (BIVV001) in severe hemophilia A patients

Sanofi and Swedish Orphan Biovitrum AB (publ) (Sobi) announced positive topline results from the pivotal XTEND-1 phase 3 study evaluating the safety, efficacy, and pharmacokinetics of efanesoctocog alfa (BIVV001) in previously treated patients with severe hemophilia A.

The study met its primary endpoint, demonstrating a clinically meaningful reduction in bleeds in people with severe hemophilia A who received weekly prophylaxis with efanesoctocog alfa for 52 weeks. The annualized bleeding rate (ABR) was 0 on average, with a median ABR of 0.71.

The key secondary endpoint was also met, demonstrating that once-weekly efanesoctocog alfa was superior to prior prophylactic factor VIII replacement therapy in terms of ABR reduction, with a statistically significant reduction in ABR based on intrapatient comparison. Efanesoctocog alfa was well tolerated, and no factor VIII inhibitor development was observed. Headache, arthralgia, falling, and back pain were the most common treatment-emergent adverse events (>5% of participants overall).

Hemophilia A is a rare genetic disorder in which a person’s blood clotting ability is impaired due to a lack of factor VIII. Hemophilia A affects about one in every 5,000 male births each year, and it occurs much less frequently in females. Bleeding episodes in people with hemophilia can cause pain, irreversible joint damage, and life-threatening hemorrhages.

Pfizer Inc. announced the launch of EPIC-PEDS (Evaluation of Protease Inhibition for COVID-19 in Pediatric Patients), a phase 2/3 study to assess the safety, pharmacokinetics, and efficacy of Pfizer’s Paxlovid (nirmatrelvir [PF-07321332] tablets and ritonavir tablets) in non-hospitalized, symptomatic, pediatric participants with a confirmed diagnosis. Paxlovid is a SARS-CoV-2 main protease (Mpro) inhibitor therapy (also known as SARS-CoV-2 3CL protease inhibitor). Paxlovid is typically given twice daily for five days at a dose of 300 mg (two 150 mg tablets) of nirmatrelvir and one 100 mg tablet of ritonavir.

Paxlovid in pediatric participants for COVID-19

Paxlovid works intracellularly to inhibit viral replication by binding to the highly conserved Mpro of the SARS-CoV-2 virus. Nirmatrelvir has consistently demonstrated in vitro antiviral activity against previous and current variants of concern (i.e., Alpha, Beta, Delta, Gamma, Lambda, Mu, and Omicron). Paxlovid is typically given twice daily for five days at a dose of 300 mg (two 150 mg tablets) of nirmatrelvir and one 100 mg tablet of ritonavir.

The phase 2/3 trial is a multi-center, open-label, single-arm study involving approximately 140 pediatric participants under the age of 18. Cohort 1 includes participants aged 6 to 17 weighing at least 40 kg [88 lbs], while Cohort 2 includes participants aged 6 to 17 weighing more than 20 kg [44 lbs] but less than 40 kg [88 lbs].

Participants in Cohort 1 will be given Paxlovid (nirmatrelvir/ritonavir 300 mg/100 mg) orally twice daily for five days (10 total doses), which is the currently authorized dosing for pediatric patients 12 years and older weighing at least 40kg.

Paxlovid (nirmatrelvir/ritonavir 150 mg/100 mg) will be administered orally twice daily for five days to participants in Cohort 2. (10 doses total).

Pfizer is also developing an age-appropriate formulation for three additional planned cohorts of children younger than six years old, and the trial will be expanded to include these younger age groups once data from Cohorts 1 and 2 and the new formulation are available.