Safety, Tolerability, Pharmacodynamics and Pharmacokinetics, and Efficacy of CLTX-305 in Autosomal Dominant Hypocalcemia Type 1 (ADH1)

• Status: Not yet recruiting
• Trial end date: 2021-09-30

Summary

Background:

The Calcium-Sensing Receptor (CaSR) detects the amount of calcium in the blood and urine. Parathyroid hormone (PTH) helps keep blood calcium levels normal. When PTH and calcium blood levels are low, this is called hypoparathyroidism. People with changes in the CaSR have a type of hypoparathyroidism called ADH1. ADH1 is treated with calcium supplements and vitamin D. But these do not always work, and can cause problems like kidney stones. Researchers want to see if the drug CLTX-305 is a better treatment option.

Objective:

To see if CLTX-305 is safe and works in people with ADH1.

Eligibility:

People ages 16 and older with ADH1

Design:

Participants will be screened with:

Medical history

Physical exam

Kidney ultrasound: Participants will lie on a table while a wand is moved over their back.

Bone density test: Participants will lie on a table while an X-ray machine moves around them.

Blood, urine, and heart tests

The study is split into 3 periods. Participants may take part in some or all periods.

In Periods 1 and 2, participants will take CLTX-305 by mouth once or twice daily for up to 5 days. Participants will stay at the NIH for 7 days and 6 nights.

In Period 3, participants will take CLTX-305 at home for 24 weeks. They will have 3 inpatient visits that last 1 2 days each.

Screening tests will be repeated during the study. Blood and urine will be collected often.

Participants dose of calcium and vitamin D may be changed. They may be asked to stop taking other medicines or change the dose.

• Phase: Phase 2

Details

• Lead Sponsor: National Institute of Dental and Craniofacial Research (NIDCR)