Clinical Research Focus. 9th Edition
Cromos Pharma Strengthens Its Operations in Türkiye, Kazakhstan and Serbia
Cromos Pharma, a US-based international CRO with a strong presence in Central and Eastern Europe, is strengthening its presence in Türkiye, Kazakhstan and Serbia by establishing local legal entities, opening full scale offices, and increasing the local staff presence. The company plans to use its office in Serbia as a regional hub that will oversee operations in the Balkan countries, including Croatia and Bulgaria.
Please read the full press release here.
GLP-1 Agonist and Insulin Glargine Were the Winners in GRADE Diabetes Trial
A 5,047-patient NIH-sponsored trial enrolled the participants who were already on metformin and randomized them to receive one of the 4 drugs: sitagliptin (DPP-4 inhibitor), liraglutide (GLP-1 agonist), glimepiride (Sulfonylurea) or insulin glargine U-100. After 4 years of follow up, insulin glargine and liraglutide achieved better glycemic control, albeit by a small margin. Additionally, patients on liraglutide lost the most weight. The trial did not include SGLT2 inhibitors which were not approved by the FDA at the launch of GRADE.
More than 37 million Americans have diabetes, and 90-95% of them have type 2 diabetes. Most of these people require more than one medication to control blood sugar levels over time.
Please read more about GRADE trial here.
AMX0035 (Relyvrio) Becomes the Third ALS Drug Approved in The US
Amylyx Pharmaceuticals’ AMX0035 (Relyvrio) was approved by the FDA on September 29, 2022. Relyvrio is a mix of two different medications, sodium phenylbutyrate (Buphenyl), and tauroursodeoxycholic acid.
The approval is based on the findings from CENTAUR, a multicenter phase 2 clinical trial and its open label extension. Please read the full article here.
ALS is the most common neurodegenerative disorder of midlife, affecting more than 120,000 people worldwide. When ALS progresses, people lose the ability to move, talk, and even breathe. As there is no cure, 80% of people affected by the disease die within two to five years of diagnosis.
Unfortunately, none of the available medications, including Relyvrio, have a curative potential and extend survival just by a few months. Clinical research into new approaches to treatment of this devastating disease is ongoing.
Biostatistics in Clinical Trials
There is a longstanding but false notion that biostatisticians are only helpful towards the latter part of a clinical study. The role that biostatistics plays in medical research spans the entire process: from study design to final data analysis.
Given the vast responsibility shouldered by biostatisticians in clinical trials, it is essential for organizations involved in medical research to understand the importance of their role.
Please read the full article here.
First HIV Patient Dosed with a Potentially Curative CRISPR-Based Gene Therapy
EBT-101, a CRISPR-based gene therapy, has been administered to the first patient in a Phase I/II trial. EBT-101 was developed through a collaboration between the Lewis Katz School of Medicine at Temple University, US, and Excision BioTherapeutics Inc.
The essential part for the efficient cure is the removal of HIV DNA from the cellular genome, as the virus hides in tissue reservoirs evading the immune system and escaping antiretroviral therapy.
“EBT-101 can potentially address long-standing unmet needs of individuals living with HIV/AIDS by removing viral DNA from their cells, thereby eradicating infection,” – commented Dr Kamel Khalili, the Laura H. Carnell Professor and Chair of the Department of Microbiology, Immunology, and Inflammation.
While significant advances have been made in controlling the disease and converting it into a chronic and manageable condition, it still has no cure, and 40 million people worldwide are suffering from its effects.
Please read more here.
Abbvie’s Skyrizi Demonstrated Impressive Long-Term Efficacy in Psoriasis/Psoriatic Arthritis
Risankizumab (Skyrizi) is a humanized monoclonal antibody targeting interleukin 23A. Pooled results from KEEPsAKE 1 and 2 trials showed that at 100 weeks half of the adult patients receiving Skyrizi achieved a 90% reduction in the Psoriasis Area and Severity Index (PASI 90) and an American College of Rheumatology 20 (ACR20) response. Additionally, 76 and 57 percent of patients, respectively, initially treated with SKYRIZI achieved resolution of dactylitis and enthesitis.
“It is critically important for physicians to have treatment options that demonstrate lasting efficacy, as we know people living with psoriatic arthritis discontinue therapies due to loss of efficacy or tolerability,” – stated Dr Doina Cosma-Roman, Vice President and Global Head, Clinical Development, Immunology, AbbVie.
Please read about the results of KEEPsAKE 1 and 2 trials here.
Draft Guidance for Protecting Children in Clinical Trials
The United States Food and Drug Administration (FDA) has published new draft guidelines named ‘Ethical Considerations for Clinical Investigations of Medical Products Involving Children’ to protect children in clinical trials.
This draft guidance is aimed at assisting industry, sponsors, and institutional review boards (IRBs) that consider enrolling children in clinical investigations of medical products. Scientific necessity of performing a clinical trial in children must be considered, as well as risk categories for procedures that do not offer a prospect of direct benefit to the child.
“The best way to provide children with safe and effective treatment options is by including them in clinical research and providing these additional safeguards to protect them during clinical trials,” – said Dionna Green, FDA Office of Pediatric Therapeutics Director.
Please read the full version of the guidance here.
Clinical Trials in Kazakhstan
There are many benefits to conducting clinical research in Kazakhstan, its strategic location being one of them. Kazakhstan is a transcontinental country located between Europe and Central Asia; its health demographics draw influence from both continents. This is one reason why more and more healthcare organizations are becoming interested in this nation as a venue for conducting their clinical studies.
Please read more about Clinical Trials in Kazakhstan here.