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 ID: NCT02204579

Last Updated: 2021-08-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 7 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-07-18
• Study Completion Date: 2015-05-04

Brief 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.

Conditions

Autosomal Dominant Hypocalcemia (ADH)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

NPSP795

intravenous

Group Type: EXPERIMENTAL

NPSP795

Intervention Type: DRUG

Interventions

NPSP795

Intervention Type: DRUG

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 < 39 kg/m2

Exclusion Criteria

• Diseases or conditions that might compromise any major body system or interfere with the pharmacokinetics of NPSP795
• History of treatment with PTH 1-84 or 1-34 within the previous 6 months
• History of hypocalcemia requiring frequent IV calcium infusions
• History of hypocalcemic seizure within the past 3 months
• Blood 25-hydroxy vitamin D level < 25 ng/mL. If subjects have a blood 25-hydroxy vitamin D level < 25 ng/mL at the outpatient screening visit, they will be prescribed vitamin D replacement. Once the 25-hydroxy vitamin D level is > 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Institutes of Health (NIH)

NIH

Sponsor Role: collaborator

Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

National Institute of Health (NIH)

Bethesda, Maryland, United States

Countries

United States

Other Identifiers

CAL-C13-001

Identifier Type: -

Identifier Source: org_study_id