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  • ||||||||||  Natesto (testosterone) / Acerus, Medac, Aytu BioPharma
    Enrollment change, Trial termination:  Evaluation of Testosterone Nasal Gel in Hypogonadal Boys (clinicaltrials.gov) -  Oct 26, 2023   
    P1,  N=8, Terminated, 
    N=20 --> 8 | Unknown status --> Terminated; Lack of patients and commercial reasons
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    Treatment of CMML in German stem cell transplantation centers  (Hall F) -  Sep 30, 2023 - Abstract #DGHO2023DGHO_620;    
    Yet, the exact modalities of HCT are handled differently. Further studies are warranted to identify which HCT regimens are associated with most favorable outcomes.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    Review, Journal:  Haematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease. (Pubmed Central) -  Sep 28, 2023   
    Since then, refinements in transplant technique, donor selection, conditioning regimens, and graft engineering have widened the option of HSCT to most patients with CGD. This review charts the progress made in HSCT for CGD.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    Journal:  Is Treosulfan-Based Conditioning Attractive as a Reduced-Intensity Conditioning Regimen in Korea? (Pubmed Central) -  Sep 16, 2023   
    The final model described an effect of the body surface area (BSA) on CL, V1, V2, and Q. The final model resulted in a modified dose recommendation for children and advises treosulfan doses of 10 This study suggests that Treo/Flu conditioning may be an alternative to Bu/Flu regimen in elderly patients with high-risk who are not suitable for standard conditioning.
  • ||||||||||  oNKord (GCT NK cells) / Glycostem, Medac, Korea Kolmar
    Journal:  Early TRAIL-engagement elicits potent multimodal targeting of melanoma by CD34 progenitor cell-derived NK cells. (Pubmed Central) -  Jul 10, 2023   
    Umbilical cord blood (UCB) CD34 progenitor cell-derived natural killer (NK) cells exert efficient cytotoxicity against various melanoma cell lines...Strikingly, combinatorial receptor blocking led to more pronounced inhibition of cytotoxicity (up to 95%) than individual receptor blocking, especially in combination with TRAIL-blocking, suggesting synergistic cytotoxic NK cell activity via engagement of multiple receptors which was also confirmed in a spheroid model. Importantly, lack of NK cell-related gene signature in metastatic melanomas correlates with poor survival highlighting the clinical significance of NK cell therapies as a promising treatment for high-risk melanoma patients.
  • ||||||||||  Jivadco (trastuzumab duocarmazine) / Byondis, Medac
    Trial completion, Enrollment change, Combination therapy, Metastases:  Phase I Study of SYD985 With Niraparib in Patients With Solid Tumors (clinicaltrials.gov) -  Jul 5, 2023   
    P1,  N=32, Completed, 
    Therefore, our real-world multicenter study suggests that FT14 is associated with better outcomes in primary refractory/relapsed AML. Active, not recruiting --> Completed | N=120 --> 32
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    Journal:  Efficacy and Safety of High-Dose Chemotherapy with Treosulfan and Melphalan in Multiple Myeloma. (Pubmed Central) -  Jun 22, 2023   
    However, female patients had a significantly higher AUC (p = 0.007) and peak value (p = 0.001), and the higher values were associated with longer hospitalizations. (4) Treatment consolidation with TreoMel HDCT demonstrated a promising efficacy and safety profile in our cohort of MM patients and deserves further investigation in prospective studies.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus, Jakafi (ruxolitinib) / Novartis, Incyte
    Journal:  Splenic irradiation prior to allogeneic transplant conditioning in myelofibrosis: A pilot experience. (Pubmed Central) -  Jun 18, 2023   
    In a small cohort of mostly ruxolitinib pre-treated patients, SI and treosulfan-based conditioning appeared a safe and effective tool to reduce spleen dimensions and ameliorate symptoms. Future prospective studies with adequate sample size are warranted to further investigate the usefulness and safety of this approach in MF.
  • ||||||||||  Jivadco (trastuzumab duocarmazine) / Byondis, Medac
    Trial completion date, Combination therapy, Metastases:  Phase I Study of SYD985 With Niraparib in Patients With Solid Tumors (clinicaltrials.gov) -  May 16, 2023   
    P1,  N=120, Active, not recruiting, 
    Notably, full control of inflammatory activity with emapalumab and anakinra may help to obtain a successful HSCT and reduce the risk of rejection. Trial completion date: Dec 2023 --> Jun 2023
  • ||||||||||  Natesto (testosterone) / Acerus, Medac, Aytu BioPharma
    Journal:  UTP11 deficiency suppresses cancer development via nucleolar stress and ferroptosis. (Pubmed Central) -  May 12, 2023   
    In this study, we found that UTP11 plays an important role in the biosynthesis of 18S ribosomal RNAs (rRNA) by binding to the pre-rRNA processing factor, MPP10...On the other hand, UTP11 deficiency represses the expression of SLC7A11 by promoting the decay of NRF2 mRNA, resulting in reduced levels of glutathione (GSH) and enhanced ferroptosis. Altogether, our study uncovers a critical role for UTP11 in maintaining cancer cell survival and growth, as depleting UTP11 leads to p53-dependent cancer cell growth arrest and p53-independent ferroptosis.
  • ||||||||||  Natesto (testosterone) / Acerus, Medac, Aytu BioPharma
    Trial completion date, Trial primary completion date:  The Effects of Natesto For Treatment Of Hypogonadism (clinicaltrials.gov) -  May 11, 2023   
    P1,  N=40, Not yet recruiting, 
    Altogether, our study uncovers a critical role for UTP11 in maintaining cancer cell survival and growth, as depleting UTP11 leads to p53-dependent cancer cell growth arrest and p53-independent ferroptosis. Trial completion date: Dec 2023 --> Dec 2024 | Trial primary completion date: Dec 2023 --> Dec 2024
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    Enrollment open:  Clinical Trial Investigating the Chemotherapeutic Compound Treosulfan (Trecondi (clinicaltrials.gov) -  May 8, 2023   
    P2,  N=120, Recruiting, 
    Trial completion date: Dec 2023 --> Dec 2024 | Trial primary completion date: Dec 2023 --> Dec 2024 Not yet recruiting --> Recruiting