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. 2023 Sep 28;389(13):1245-1247.
doi: 10.1056/NEJMc2302708.

Efficacy and Safety of Encaleret in Autosomal Dominant Hypocalcemia Type 1

Rachel I Gafni  1 , Iris R Hartley  1 , Kelly L Roszko  1 , Edward F Nemeth  2 , Karen A Pozo  3 , Ed Lombardi  4 , Ananth V Sridhar  4 , Mary S Roberts  4 , Jonathan C Fox  4 , Michael T Collins  5
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Efficacy and Safety of Encaleret in Autosomal Dominant Hypocalcemia Type 1

Rachel I Gafni et al. N Engl J Med. .
No abstract available

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Figures

Figure 1.
Figure 1.. Effect of Encaleret on Mineral Homeostasis in 13 Participants with Autosomal Dominant Hypocalcemia Type 1.
The study included a 5-day inpatient dose-escalation period (period 1), a 5-day inpatient dose-adjustment period (period 2), and a 24-week outpatient dose-maintenance period (period 3). The line graph on the left side of each panel shows mean laboratory values and 95% confidence intervals (I bars) throughout periods 2 and 3. Period 2 included serial sampling. Period 3 included a combination of serial sampling (at weeks 8, 16, and 24) and outpatient laboratory measurements; results shown at serial sampling visits were obtained before the morning dose of encaleret, and the timing of samples outside these visits was variable. Time points shown for period 2 in the line graphs (including throughout day 5) are provided in the Supplementary Appendix. The bar graph on the right side of each panel shows laboratory values at baseline; period 2, day 5; and period 3, week 24. With encaleret treatment, mean levels of albumin-corrected blood calcium, intact parathyroid hormone (PTH), and blood magnesium increased from baseline and were within the normal range at the later time points. The 24-hour urinary calcium excretion and blood phosphorus levels decreased with encaleret treatment. The values shown at period 2, day 5, and period 3, week 24, are means and 95% confidence intervals of serial measurements over a 24-hour period for blood variables. In both the line and bar graphs, normal ranges are indicated by shading. In Panel B, PTH values from one participant were excluded from the analysis owing to the variable presence of heterophile antibodies that interfered with the intact PTH assay. In Panel C, the baseline value was obtained at screening during standard care.
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References

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    1. Roberts MS, Gafni RI, Brillante B, et al. Treatment of autosomal dominant hypocalcemia type 1 with the calcilytic NPSP795 (SHP635). J Bone Miner Res 2019; 34: 1609–18. - PMC - PubMed

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