4 results
This is a so-called *lead-in study* in which patients receive their standard treatment and no study treatment. The researchers will collect dataabout the patients* bleeding episodes, FVIII containing treatments used to prevent bleeding episodes and…
Part 1PrimaryTo assess dose limiting toxicities (DLTs) of EDR to select an RP3D for the combination to be used in Part 2 of this study.Secondary• To evaluate the overall safety profile of EDR to select an RP3D for the combination to be used in Part…
To test if tamoxifen treatment, compared to placebo, reduces the progression of the disease in 6.5-12 years old ambulant DMD patients by at least 50% (using the MFM D1 subscore as primary clinical endpoint in group A patients).To test if tamoxifen…
Primary:• To compare the efficacy of elranatamab vs lenalidomide Secondary:- To compare the efficacy of elranatamab vs lenalidomide- To determine the safety and tolerability of elranatamab- To evaluate the PK of elranatamab- To evaluate the…