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 range

18 Years

Sex

ALL

See this in plain English?

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

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

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