5 Clinical Trials for Hypoparathyroidism
The purpose of this study is to evaluate the long-term safety and tolerability of MBX 2109 in patients with hypoparathyroidism who completed the 12-week treatment period in the Phase 2 study, MBX-2H1002.
This is a prospective three year natural history study of adults with hypoparathyroidism. The goal is to monitor patients with hypoparathyroidism to define end-organ damage in the context of the disease. Funding Source- FDA OOPD
Background: Parathyroid glands in the neck make a hormone that keeps blood calcium levels stable. Sometimes these glands are damaged or removed during neck surgery. This can lead to a condition called postsurgical hypoparathyroidism (PSH). People with PSH have low levels of calcium in their blood. Calcium and vitamin D pills can help them keep their blood calcium levels steady. But this can increase calcium in the urine and result in kidney problems. New treatments for PSH are needed. Objective: To test a drug (encaleret) in people with PSH. Eligibility: People aged 18 or older who have PSH. Design: Participants will be in the study for 6 months. They will have a screening visit and a treatment visit. Screening will take up to 2 days. Participants will have a physical exam. They will have blood and urine tests and tests of their heart function. They will have an ultrasound of their kidneys; they will lie on a table for 15 to 30 minutes while a wand is moved over their back. Treatment will require participants to stay in the clinic for 7 days and 6 nights. They will take the study drug (encaleret) by mouth twice a day for 5 days. They will have a small, flexible tube inserted into a vein; this will remain in place during the visit. Blood samples will be taken through the tube 4 to 9 times each day. Participants urine will be collected. Participants will have follow-up blood tests 1 week after leaving the clinic. They will have 3 follow-up phone calls.
Options for treatment of severe, refractory hypocalcemia are limited for the thousands of patients in the United States who suffer from hypoparathyroidism. Parathyroid allotransplantation is an emerging treatment that provides hope for these individuals. Currently, this therapy has only been successfully provided by a few centers in the world. In the UAB PATH trial, we propose to become one of the few centers worldwide to successfully achieve parathyroid allotransplantation in transplant-naïve patients.
This main aim of this study is to check the level of knowledge and assess attitudes and behaviors of both participants and prescribing physicians regarding the risks and safe use of NATPARA. The survey will be done via internet, telephone, or paper and participants will be able to choose the method that is preferred. No study medicines will be provided to patients in this study.