Methods for increasing mean corpuscular volume
An individual, compound technology for the treatment of hemoglobin deficiency
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Embodiment 1
[0054] Example 1: Increasing mean corpuscular volume (MCV) in human subjects with end-stage renal disease
[0055] The effect of compounds of the invention on MCV was determined in human subjects with end-stage renal disease as follows. Subjects had been on stable maintenance hemodialysis for at least four months prior to the start of the study. Also prior to the start of the study presented here, the subject had been receiving a stable standard of care dose of epoetin alfa for the treatment of anemia.
[0056] To examine the difference in MCV response in individuals treated with compounds of the present invention versus those treated with epoetin alfa (the current standard of care), all intravenous iron supplementation was stopped two weeks prior to treatment. Oral iron supplementation is permitted.
[0057] Subjects were administered Compound A (1.0 mg / kg, 1.5 mg / kg, or 2.0 mg / kg) or epoetin alfa three times per week (TIW) for six weeks. Baseline MCV levels (mean values ...
Embodiment 2
[0066] Example 2: Increasing mean corpuscular volume (MCV) in human subjects with chronic kidney disease
[0067] In another series of experiments, the effect of the compounds of the invention on MCV was determined in stage III and IV chronic kidney disease patients (estimated glomerular filtration rate eGFR<59 ml / min) as follows. Individuals with stage III and stage IV chronic kidney disease were administered placebo or Compound A (1.5 mg / kg or 2.0 mg / kg) two or three times per week for 4 weeks. MCV values were determined weekly.
[0068] Such as image 3 As shown, mean MCV increased over time in subjects administered Compound A at doses of 1.5 mg / kg or 2.0 mg / kg. In placebo-treated control individuals, there was no increase in mean MCV. Figure 4 The mean change from baseline MCV observed in treated and control individuals is shown. Such as Figure 4 As shown, the mean change (ie, increase) from baseline MCV in subjects administered Compound A was greater than 3 fL at 2...
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