Innovative Approaches for Hemophilia A and B With or Without Inhibitors by Janet Brewer, M.Ed
E
ver wonder just how many studies there are in hemophilia? A quick visit to clinicaltrials.gov with search criteria of “hemophilia and USA” yields 310 studies with status’ ranging from: •
Looking for participants (not yet recruiting to recruiting)
•
No longer looking for participants (active, not recruiting, completed, and terminated)
If you have time, it is a fascinating review. Especially as you start looking at which ones are open to women; not many but it is improving! We looked at a few with novel mechanisms of action.
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec In Hemophilia A with Active or Prior Inhibitors BioMarin is announcing the opening of their clinical studySponsor: BioMarin BMN270-205 or GENEr8-inh in adult male hemophilia A Pharmaceutical patients with active inhibitors and/or a history of hemophilia First Submitted: 12/10/2020 A with inhibitors to study the safety and efficacy of Roctavian (Valoctocogene roxaparvovec). Roctavian was FDA approved on Last Posted Update: 01/04/2023 June 29, 2023. It is an adeno-associated virus vector-based gene therapy for the treatment of adults with severe hemophilia A without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test. There are 5 United States study sites.
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https://clinicaltrials.gov/study/NCT04684940?locStr=United States&country=United States&cond=Hemophilia A With Inhibitors&term=gene therapy&intr=gene therapy&aggFilters=ages:adult,sex:m&rank=1
6 n LIFELINES for HEALTH n 2023 ISSUE
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