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20. February 2023 : Clinical trials 23 : results to look forward!

20.02.2023 : The most interesting clinical trials coming to an end in 2023…

Recently, Nature Medicine reporters C. Arnold and P. Webster asked leading researchers to name the most interesting clinical trials coming to an end in 2023. 

Roger Albin, Professor of Neurology and co-director of the Movement Disorders Clinic at the University of Michigan Medical School, highlighted the Exenatide phase 3 clinical trial funded by the Michael J. Fox Foundation currently running at the University College of London. Exenatide is a medication used to treat Diabetes mellitus and is being repurposed as a potential disease modifying treatment for Parkinson’s disease. A previous double-blind placebo-controlled trial with 60 participants indicated that exenatide may be a “neuroprotective” drug. Triggering insulin-signaling pathways for neurodegenerative conditions has created a lot of buzz during the last couple of years, as such according to Prof. Albin “the community is looking for unequivocal results, whether positive or negative” with this study. 

Next, Dr. Rober L. Coleman Chief Scientific Officer at the U.S. Oncology Research, one of the largest networks dedicated to advancing high-quality evidence-based cancer care, emphasized the phase 3 MIRASOL trial in platinum-resistant ovarian cancer. This study from ImmunoGen Inc., a biotechnology pioneer in Antibody–Drug Conjugate (ADC) therapeutics is designed to compare the efficacy and safety of mirvetuximab soravtansine vs. investigator’s choice chemotherapy. This new ADC was given accelerated approval by the FDA, as such it needs a confirmatory trial to validate the overall safety and efficacy of the novel compound. 

Prof. Dr. Simone Spuler, head of the myology research group at the Experimental and Clinical Research Center in Berlin, Germany, underlined the importance of the GenPHSats-bASKet phase 1/2 study. This clinical trial will be fundamental to validate the safety of CRISP-Cas9 technology for the correction of genetic disorders. In this case, gene edited Primary Human Satellite cell derived muscle stem cells are being used to promote healthy muscle development in patients with Limb-Girdle Muscular Dystrophy (LGDM) – a group of rare progressive genetic disorders characterized by atrophy and weakness of the voluntary muscles of the hip and shoulders. Not only the safety of the technology is being evaluated, but also measurable clinical improvement. The regulatory agencies have an eye on the trial to make sure that a benefit is brought to the patient’s life, considering the high risk of using such novel biotechnology for the treatment of LGDM.

Important is also the study highlighted by Dr. Olaf Valverde Mordt, the clinical project leader in Human African Trypanosomiasis (HAT, sleeping sickness) at the Drugs and Neglected Diseases initiative (DNDi) – a not-for profit organization founded in 2003 for the discovery, development, and delivery of new treatments for neglected patients around the world. Currently, the only treatment available for stage II HAT is melarsoprol, an arsenic derivative that kills 5% of the patients – a very toxic drug. As such, the trial is evaluating whether fexinidazole is as effective in treating this devastating neuropsychiatric disorder, which is caused by trypanosome parasites transmitted by Tsetse flies’ stings. This disease affects millions of people in sub-Saharan African countries, and all-oral safe medicines are much needed.

by Catarina Carrão

If you want to help, DNDi donations are accepted and tax-deductible:

Original article: Arnold, C., Webster, P. 11 clinical trials that will shape medicine in 2023. Nat Med 28, 2444–2448 (2022).

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