Trial Outcomes & Findings for A Study to Determine the Effects of NPSP795 on the Calcium-sensing Receptor in Subjects With Autosomal Dominant Hypocalcemia as Measured by PTH Levels and Blood Calcium Concentrations (NCT NCT02204579)

NCT ID: NCT02204579

Last Updated: 2021-08-09

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 7 participants
• Primary outcome timeframe: From Day 1 up to safety follow-up assessment (upto Day 17 after discharge)
• Results posted on: 2021-08-09

Participant Flow

Participant milestones

Measure	NPSP795 NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.
Overall Study STARTED	7
Overall Study Participants Started in Pre-Amendment	2
Overall Study Participants Started in Post-Amendment	5
Overall Study Treated	5
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	NPSP795 NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.
Overall Study Screen failures	2

Baseline Characteristics

A Study to Determine the Effects of NPSP795 on the Calcium-sensing Receptor in Subjects With Autosomal Dominant Hypocalcemia as Measured by PTH Levels and Blood Calcium Concentrations

Baseline characteristics by cohort

Measure	NPSP795 n=5 Participants NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.
Age, Continuous	39.8 Years STANDARD_DEVIATION 15.2 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: From Day 1 up to safety follow-up assessment (upto Day 17 after discharge)

Population: Safety population included all participants who received at least 1 minute of study drug infusion (pre-amendment and post-amendment).

Outcome measures

Measure	NPSP795 n=5 Participants NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.	NPSP795 on Day 2 (15 mg/3.5 Hours)	NPSP795 on Day 3 (30 mg/3.5 Hours)	NPSP795 on Day 4 (30 mg/3.5 Hours)	NPSP795 on Day 4 (50 mg/3.5 Hours)
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) Participants with TEAEs	3 participants	—	—	—	—
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) Participants with TESAEs	1 participants	—	—	—	—

PRIMARY outcome

Timeframe: From Day 1 up to safety follow-up assessment (upto Day 17 after discharge)

Population: Safety population included all participants who received at least 1 minute of study drug infusion (both pre-amendment and post-amendment participants).

Outcome measures

Measure	NPSP795 n=5 Participants NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.	NPSP795 on Day 2 (15 mg/3.5 Hours)	NPSP795 on Day 3 (30 mg/3.5 Hours)	NPSP795 on Day 4 (30 mg/3.5 Hours)	NPSP795 on Day 4 (50 mg/3.5 Hours)
Number of Participants With Clinically Significant Vital Signs and Electrocardiogram (ECG) Abnormalities	0 participants	—	—	—	—

PRIMARY outcome

Timeframe: From Day 1 up to safety follow-up assessment (upto Day 17 after discharge)

Population: Safety population included all participants who received at least 1 minute of study drug infusion (pre-amendment and post-amendment).

Outcome measures

Measure	NPSP795 n=5 Participants NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.	NPSP795 on Day 2 (15 mg/3.5 Hours)	NPSP795 on Day 3 (30 mg/3.5 Hours)	NPSP795 on Day 4 (30 mg/3.5 Hours)	NPSP795 on Day 4 (50 mg/3.5 Hours)
Number of Participants With Potentially Clinically Important Laboratory Abnormalities	1 participants	—	—	—	—

PRIMARY outcome

Timeframe: From Day 1 up to safety follow-up assessment (upto Day 17 after discharge)

Population: Safety population included all participants who received at least 1 minute of study drug infusion (both pre-amendment and post-amendment participants).

Outcome measures

Measure	NPSP795 n=5 Participants NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.	NPSP795 on Day 2 (15 mg/3.5 Hours)	NPSP795 on Day 3 (30 mg/3.5 Hours)	NPSP795 on Day 4 (30 mg/3.5 Hours)	NPSP795 on Day 4 (50 mg/3.5 Hours)
Number of Participants With Clinically Significant Abnormalities Related to Physical Examination	1 participants	—	—	—	—

PRIMARY outcome

Timeframe: 10 Minute (min) Infusion Time: within 5 min pre-dose; & post-dose 15, 30, 45, 60, 75, 90, 105 min, and 2, 2.5, 3, 3.5, & 4 hour (hr) 3.5 hr Infusion Time: within 5 min pre-dose; & post-dose 15, 30, 45, 60, 75, 90, 105 min, & 2, 2.5, 3, 3.5, 4, 5, and 8 hr

Population: Post-amendment pharmacodynamic (PD) analysis population included all participants who received at least 5 minutes of the study drug infusion and had at least one PD measurement on at least 1 day of infusion. Here, n=number of participants analysed for specified category at the specified time points in each arm respectively.

Outcome measures

Measure	NPSP795 n=5 Participants NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.	NPSP795 on Day 2 (15 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 3 (30 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 4 (30 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 4 (50 mg/3.5 Hours) n=5 Participants
Change From Baseline in Ionised Calcium Baseline	0.972 millimole/liter (mmol/L) Standard Deviation 0.058	0.950 millimole/liter (mmol/L) Standard Deviation 0.054	0.986 millimole/liter (mmol/L) Standard Deviation 0.045	0.933 millimole/liter (mmol/L) Standard Deviation 0.055	1.025 millimole/liter (mmol/L) Standard Deviation 0.078
Change From Baseline in Ionised Calcium Change at 4 Hours Post dose	-0.016 millimole/liter (mmol/L) Standard Deviation 0.027	0.010 millimole/liter (mmol/L) Standard Deviation 0.026	-0.014 millimole/liter (mmol/L) Standard Deviation 0.023	-0.030 millimole/liter (mmol/L) Standard Deviation 0.046	-0.015 millimole/liter (mmol/L) Standard Deviation 0.035
Change From Baseline in Ionised Calcium Change at 8 Hours Post dose	NA millimole/liter (mmol/L) Standard Deviation NA Data was calculated for 5 mg/10 minutes infusion until hour 4 only.	-0.034 millimole/liter (mmol/L) Standard Deviation 0.044	-0.040 millimole/liter (mmol/L) Standard Deviation 0.060	-0.033 millimole/liter (mmol/L) Standard Deviation 0.046	-0.060 millimole/liter (mmol/L) Standard Deviation 0.028

PRIMARY outcome

Timeframe: 10 Minutes (min) Infusion Time: within 5 min pre-dose; & post-dose 1, 2, 3, 4, 8 12 hours (hr). 3.5 hr Infusion Time: within 5 min pre-dose; & post-dose 1, 2, 3, 4, 8 12 hr.

Population: Post-amendment PD analysis population included all participants who received at least 5 minutes of the study drug infusion and had at least one PD measurement on at least 1 day of infusion. Here, n=number of participants analysed for specified category at the specified time points in each arm respectively.

Outcome measures

Measure	NPSP795 n=5 Participants NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.	NPSP795 on Day 2 (15 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 3 (30 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 4 (30 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 4 (50 mg/3.5 Hours) n=5 Participants
Change From Baseline in Serum Calcium Baseline	1.914 millimole/liter (mmol/L) Standard Deviation 0.157	1.790 millimole/liter (mmol/L) Standard Deviation 0.125	1.852 millimole/liter (mmol/L) Standard Deviation 0.132	1.677 millimole/liter (mmol/L) Standard Deviation 0.135	1.980 millimole/liter (mmol/L) Standard Deviation 0.184
Change From Baseline in Serum Calcium Change at 12 Hours Postdose	-0.114 millimole/liter (mmol/L) Standard Deviation 0.141	-0.042 millimole/liter (mmol/L) Standard Deviation 0.054	-0.106 millimole/liter (mmol/L) Standard Deviation 0.079	-0.040 millimole/liter (mmol/L) Standard Deviation 0.053	-0.270 millimole/liter (mmol/L) Standard Deviation 0.057

PRIMARY outcome

Timeframe: 10 Minutes (min) Infusion Time: within 5 min pre-dose; & post-dose 1, 2, 3, 4, 8 12 hours (hr). 3.5 hr Infusion Time: within 5 min pre-dose; & post-dose 1, 2, 3, 4, 8 12 hr.

Population: PD analysis population included all participants who received at least 5 minutes of the study drug infusion and had at least one PD measurement on at least 1 day of infusion. Here, n=number of participants analysed for specified category at the specified time points in each arm respectively.

Outcome measures

Measure	NPSP795 n=5 Participants NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.	NPSP795 on Day 2 (15 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 3 (30 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 4 (30 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 4 (50 mg/3.5 Hours) n=5 Participants
Change From Baseline in Urinary Calcium Baseline	3.302 millimole/liter (mmol/L) Standard Deviation 2.283	3.912 millimole/liter (mmol/L) Standard Deviation 3.299	2.936 millimole/liter (mmol/L) Standard Deviation 1.750	2.537 millimole/liter (mmol/L) Standard Deviation 1.968	2.065 millimole/liter (mmol/L) Standard Deviation 1.916
Change From Baseline in Urinary Calcium Change at 12 Hours Postdose	-0.590 millimole/liter (mmol/L) Standard Deviation 0.861	-2.162 millimole/liter (mmol/L) Standard Deviation 2.027	-1.188 millimole/liter (mmol/L) Standard Deviation 1.634	-1.190 millimole/liter (mmol/L) Standard Deviation 1.460	-0.755 millimole/liter (mmol/L) Standard Deviation 1.082

PRIMARY outcome

Timeframe: 10 Minutes (min) Infusion Time: within 5 min pre-dose; & post-dose 5, 10, 15, 30 min, & 1, 1.5, 2, 2.5, 3, 3.5, 4, 8 hours (hr). 3.5 hr Infusion Time: within 5 min pre-dose; & post-dose 15, 30 min, & 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 5.5 hr

Population: Post amendment PD analysis population included all participants who received at least 5 minutes of the study drug infusion and had at least one PD measurement on at least 1 day of infusion. Here, n=number of participants analysed for specified category at the specified time points in each arm respectively.

Outcome measures

Measure	NPSP795 n=5 Participants NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.	NPSP795 on Day 2 (15 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 3 (30 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 4 (30 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 4 (50 mg/3.5 Hours) n=5 Participants
Change From Baseline in Serum Parathyroid Hormone (PTH) Baseline	7.32 nanogram/liter (ng/L) Standard Deviation 2.93	9.14 nanogram/liter (ng/L) Standard Deviation 3.29	9.58 nanogram/liter (ng/L) Standard Deviation 5.32	12.80 nanogram/liter (ng/L) Standard Deviation 8.68	7.40 nanogram/liter (ng/L) Standard Deviation 3.54
Change From Baseline in Serum Parathyroid Hormone (PTH) Change at 5.5 Hours Postdose	NA nanogram/liter (ng/L) Standard Deviation NA Data for 10 minutes infusion was not calculated at 5.5-hours.	0.84 nanogram/liter (ng/L) Standard Deviation 0.86	1.92 nanogram/liter (ng/L) Standard Deviation 1.53	0.13 nanogram/liter (ng/L) Standard Deviation 1.50	1.75 nanogram/liter (ng/L) Standard Deviation 0.64
Change From Baseline in Serum Parathyroid Hormone (PTH) Change at 8 Hours Postdose	0.60 nanogram/liter (ng/L) Standard Deviation 1.56	NA nanogram/liter (ng/L) Standard Deviation NA Data for 3.5-hours infusions was calculated until hour 5.5 only.	NA nanogram/liter (ng/L) Standard Deviation NA Data for 3.5-hours infusions was calculated until hour 5.5 only.	NA nanogram/liter (ng/L) Standard Deviation NA Data for 3.5-hours infusions was calculated until hour 5.5 only.	NA nanogram/liter (ng/L) Standard Deviation NA Data for 3.5-hours infusions was calculated until hour 5.5 only.

SECONDARY outcome

Timeframe: 10 Minutes (min) Infusion Time: within 5 min pre-dose; & post-dose 5, 10, 15, 30 min, & 1, 1.5, 2, 2.5, 3, 3.5, 4, 8 hours (hr). 3.5 hr Infusion Time: within 5 min pre-dose; & post-dose 15, 30 min, & 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 5.5 hr

Population: Post amendment pharmacokinetic (PK) analysis population included all participants who received at least 5 minutes of the study drug infusion and had at least one PK measurement on at least 1 day of infusion.

Outcome measures

Measure	NPSP795 n=5 Participants NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.	NPSP795 on Day 2 (15 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 3 (30 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 4 (30 mg/3.5 Hours) n=3 Participants	NPSP795 on Day 4 (50 mg/3.5 Hours) n=2 Participants
Area Under the Plasma Concentration Versus Time Curve (AUC[0-t]) of NPSP795	309.512 nanogram*hour per millilitre (ng·h/mL) Standard Deviation 122.601	795.766 nanogram*hour per millilitre (ng·h/mL) Standard Deviation 294.345	1561.746 nanogram*hour per millilitre (ng·h/mL) Standard Deviation 546.479	1657.057 nanogram*hour per millilitre (ng·h/mL) Standard Deviation 650.156	2251.818 nanogram*hour per millilitre (ng·h/mL) Standard Deviation 568.782

SECONDARY outcome

Timeframe: 10 Minutes (min) Infusion Time: within 5 min pre-dose; & post-dose 5, 10, 15, 30 min, & 1, 1.5, 2, 2.5, 3, 3.5, 4, 8 hours (hr). 3.5 hr Infusion Time: within 5 min pre-dose; & post-dose 15, 30 min, & 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 5.5 hr

Population: Post amendment PK analysis population included all participants who received at least 5 minutes of the study drug infusion and had at least one PK measurement on at least 1 day of infusion.

Outcome measures

Measure	NPSP795 n=5 Participants NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.	NPSP795 on Day 2 (15 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 3 (30 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 4 (30 mg/3.5 Hours) n=3 Participants	NPSP795 on Day 4 (50 mg/3.5 Hours) n=2 Participants
Area Under the Concentration Time Curve Extrapolated to Infinity (AUC0-infinity) of NPSP795	2.672 nanogram*hour per milliliter(ng·h/mL) Standard Deviation 1.775	5.572 nanogram*hour per milliliter(ng·h/mL) Standard Deviation 2.724	5.437 nanogram*hour per milliliter(ng·h/mL) Standard Deviation 2.587	7.648 nanogram*hour per milliliter(ng·h/mL) Standard Deviation 3.607	4.521 nanogram*hour per milliliter(ng·h/mL) Standard Deviation 2.991

SECONDARY outcome

Timeframe: 10 Minutes (min) Infusion Time: within 5 min pre-dose; & post-dose 5, 10, 15, 30 min, & 1, 1.5, 2, 2.5, 3, 3.5, 4, 8 hours (hr). 3.5 hr Infusion Time: within 5 min pre-dose; & post-dose 15, 30 min, & 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 5.5 hr

Population: Post amendment PK analysis population included all randomized participants who received at least 5 minutes of the study drug infusion and had at least one PK measurement on at least one day of infusion.

Outcome measures

Measure	NPSP795 n=5 Participants NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.	NPSP795 on Day 2 (15 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 3 (30 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 4 (30 mg/3.5 Hours) n=3 Participants	NPSP795 on Day 4 (50 mg/3.5 Hours) n=2 Participants
Maximum Observed Drug Concentration (Cmax) of NPSP795 in Plasma	301.900 nanogram/milliliter (ng/mL) Standard Deviation 120.664	745.335 nanogram/milliliter (ng/mL) Standard Deviation 254.205	1467.102 nanogram/milliliter (ng/mL) Standard Deviation 475.343	1515.086 nanogram/milliliter (ng/mL) Standard Deviation 548.056	2141.500 nanogram/milliliter (ng/mL) Standard Deviation 475.706

SECONDARY outcome

Timeframe: 10 Minutes (min) Infusion Time: within 5 min pre-dose; & post-dose 5, 10, 15, 30 min, & 1, 1.5, 2, 2.5, 3, 3.5, 4, 8 hours (hr). 3.5 hr Infusion Time: within 5 min pre-dose; & post-dose 15, 30 min, & 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 5.5 hr

Population: Post amendment PK analysis population included all participants who received at least 5 minutes of the study drug infusion and had at least one PK measurement on at least 1 day of infusion.

Outcome measures

Measure	NPSP795 n=5 Participants NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.	NPSP795 on Day 2 (15 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 3 (30 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 4 (30 mg/3.5 Hours) n=3 Participants	NPSP795 on Day 4 (50 mg/3.5 Hours) n=2 Participants
Elimination Half-life (t1/2) of NPSP795 in Plasma	3.013 hour Standard Deviation 0.612	3.913 hour Standard Deviation 0.222	4.213 hour Standard Deviation 0.449	3.839 hour Standard Deviation 0.294	4.000 hour Standard Deviation 0.000

SECONDARY outcome

Timeframe: 10 Minutes (min) Infusion Time: within 5 min pre-dose; & post-dose 1, 2, 3, 4, 8, 12 hours (hr). 3.5 hr Infusion Time: within 5 min pre-dose; & post-dose 1, 2, 3, 4, 8, 12 hr.

Population: Post-amendment PD analysis population included all participants who received at least 5 minutes of the study drug infusion and had at least one PD measurement on at least 1 day of infusion. Here n=number of participants analysed for specified category at the specified time points in each arm respectively.

Outcome measures

Measure	NPSP795 n=5 Participants NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.	NPSP795 on Day 2 (15 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 3 (30 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 4 (30 mg/3.5 Hours) n=5 Participants	NPSP795 on Day 4 (50 mg/3.5 Hours) n=5 Participants
Change From Baseline in Fractional Excretion of Calcium (FECa) FECa: Baseline	0.030 Fraction of excretion Standard Deviation 0.023	0.031 Fraction of excretion Standard Deviation 0.017	0.027 Fraction of excretion Standard Deviation 0.014	0.030 Fraction of excretion Standard Deviation 0.011	0.026 Fraction of excretion Standard Deviation 0.032
Change From Baseline in Fractional Excretion of Calcium (FECa) FECa: Change at 12 Hours Postdose	0.002 Fraction of excretion Standard Deviation 0.005	0.002 Fraction of excretion Standard Deviation 0.012	0.001 Fraction of excretion Standard Deviation 0.011	-0.009 Fraction of excretion Standard Deviation 0.018	0.002 Fraction of excretion Standard Deviation 0.007

Adverse Events

NPSP795

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Measure

NPSP795 n=5 participants at risk NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.

Hepatobiliary disorders Cholelithiasis

20.0% 1/5 • Number of events 1 • From the sign of informed consent form (Day -93) up to safety follow-up assessment (upto Day 17 after discharge)

Other adverse events

Measure	NPSP795 n=5 participants at risk NPSP795 administered Intravenous (IV) infusion for 10 min on Day 1 if certain criteria for ionized calcium and/or blood parathyroid hormone (PTH) were met, an infusion was administered for either 10 min or 3.5 hours on Days 2 \& 3, according to a predefined dose escalation scheme. NPSP795 was administered as a 3.5-hour infusion on Day 4, or the participant underwent discharge procedures.
Hepatobiliary disorders Cholelithiasis	20.0% 1/5 • Number of events 1 • From the sign of informed consent form (Day -93) up to safety follow-up assessment (upto Day 17 after discharge)
Immune system disorders Hypersensitivity reaction	20.0% 1/5 • Number of events 2 • From the sign of informed consent form (Day -93) up to safety follow-up assessment (upto Day 17 after discharge)
Reproductive system and breast disorders Nipple pain	20.0% 1/5 • Number of events 1 • From the sign of informed consent form (Day -93) up to safety follow-up assessment (upto Day 17 after discharge)

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Email: ClinicalTransparency@shire.com

Results disclosure agreements

• Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
• Publication restrictions are in place

Restriction type: OTHER