CompletedPhase 2

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

United States7 participantsStarted 2014-07-18

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)

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

Number of Participants With Clinically Significant Vital Signs and Electrocardiogram (ECG) Abnormalities

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

Number of Participants With Potentially Clinically Important Laboratory Abnormalities

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

Number of Participants With Clinically Significant Abnormalities Related to Physical Examination

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

Change From Baseline in Ionised Calcium

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

Change From Baseline in Serum Calcium

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.

Trial details

NCT IDNCT02204579

SponsorShire

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2015-05-04

Results submitted2016-10-27

View on ClinicalTrials.gov More Autosomal Dominant Hypocalcemia (ADH) trials