Phase 2
N=7
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
Autosomal Dominant Hypocalcemia (ADH)
Bottom Line
View on ClinicalTrials.gov: NCT02204579 ↗Enrolled (actual)
7
Serious AEs
20.0%
Results posted
Dec 2016
Primary outcome: Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) — 3; 1 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- NPSP795 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Shire
- Primary completion
- May 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) |
3; 1 | — |
| PRIMARY Number of Participants With Clinically Significant Vital Signs and Electrocardiogram (ECG) Abnormalities |
— | — |
| PRIMARY Number of Participants With Potentially Clinically Important Laboratory Abnormalities |
1 | — |
| PRIMARY Number of Participants With Clinically Significant Abnormalities Related to Physical Examination |
1 | — |
| PRIMARY Change From Baseline in Ionised Calcium |
0.972; 0.950; 0.986; 0.933; 1.025; -0.016 | — |
| PRIMARY Change From Baseline in Serum Calcium |
1.914; 1.790; 1.852; 1.677; 1.980; -0.114 | — |
| PRIMARY Change From Baseline in Urinary Calcium |
3.302; 3.912; 2.936; 2.537; 2.065; -0.590 | — |
| PRIMARY Change From Baseline in Serum Parathyroid Hormone (PTH) |
7.32; 9.14; 9.58; 12.80; 7.40; NA | — |
| SECONDARY Area Under the Plasma Concentration Versus Time Curve (AUC[0-t]) of NPSP795 |
309.512; 795.766; 1561.746; 1657.057; 2251.818 | — |
| SECONDARY Area Under the Concentration Time Curve Extrapolated to Infinity (AUC0-infinity) of NPSP795 |
2.672; 5.572; 5.437; 7.648; 4.521 | — |
| SECONDARY Maximum Observed Drug Concentration (Cmax) of NPSP795 in Plasma |
301.900; 745.335; 1467.102; 1515.086; 2141.500 | — |
| SECONDARY Elimination Half-life (t1/2) of NPSP795 in Plasma |
3.013; 3.913; 4.213; 3.839; 4.000 | — |
| SECONDARY Change From Baseline in Fractional Excretion of Calcium (FECa) |
0.030; 0.031; 0.027; 0.030; 0.026; 0.002 | — |
Summary
This is an open-label study looking at the effects of NPSP795 (a selective calcium receptor antagonist) on activating mutations of the Calcium-sensing receptor in patients with Autosomal Dominant Hypocalcemia. Patients with ADH have low blood calcium levels and an inappropriately increased renal calcium excretion, decreased renal phosphate excretion, and hyperphosphatemia. PTH and blood calcium levels will be tested during and after the IV infusion of NPSP795. Concentrations of NPSP795 and length of time of IV infusion will vary depending on measured levels of ionized calcium.
Eligibility Criteria
Inclusion Criteria
- Subjects with a heterozygous activating mutation of the CaSR gene (ADH); if not previously confirmed, genetic testing will be performed at the screening visit
- At least 18 years of age
- Body mass index (BMI) ≥ 18.5 to 25 ng/mL, the subject will be eligible to continue on to the treatment phase of the study
- Estimated glomerular filtration rate (GFR) < 25 mL/minute, and/or abnormal hepatic, hematologic, and/or clotting function
- 12 lead resting electrocardiogram (ECG) with clinically significant abnormalities
- Concomitant medications with the potential to interfere with NPSP795 metabolism
- History of thyroid or parathyroid surgery
Data sourced from ClinicalTrials.gov (NCT02204579). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.