Cinacalcet to Treat Familial Primary Hyperparathyroidism

This study will evaluate the benefits and side effects of a new medication called Cinacalcet for treating patients with primary hyperparathyroidism associated with multiple endocrine neoplasia type 1 (MEN1) or type 2A (MEN2A). Patients with primary hyperparathyroidism have elevated levels of blood calcium caused by too much parathyroid hormone released by one or more parathyroid tumors. The parathyroids are small glands located in the neck. Most cases of primary hyperparathyroidism are due to a single overactive parathyroid gland, but in MEN1 and MEN2A, several glands are overgrown and overactive. Cinacalcet decreases the secretion of parathyroid hormone.

Patients 18 years of age and older with primary hyperparathyroidism and MEN1 or MEN2A and who are not candidates for parathyroid surgery may be eligible for this study.

Participants are admitted to the Clinical Center for 1 week blood and urine tests and imaging studies, and initiation of Cinacalcet treatment. They take the drug by mouth and have daily blood tests until the dosage required to achieve normal blood calcium levels is determined. Patients return to the hospital 2 weeks later for 1 week to evaluate the response to the drug and make any necessary adjustments. Treatment may continue for as long as 1 year with 1-week admissions every 3 months to monitor the benefits and side effects of Cinacalcet. Evaluations may include the following:

* Blood and urine analyses. * Measurement of gastric acid secretion. For this test, a soft plastic tube is inserted into the nose or mouth and then swallowed and then gently removed about an hour later. * Injections of secretin, calcium and arginine into a vein and collection of blood samples to measure the responding increase in levels of gastrin, calcitonin and insulin, respectively. These tests are used to diagnose and monitor hormone secretion from endocrine tumors and are used in this study to assess the response to Cinacalcet treatment. * Radioisotope test to evaluate tumors of the endocrine organs. A radioactive substance injected into a vein is taken up by the endocrine tissue and the concentrated radioactivity is measured. * Imaging tests, such as MRI and CT, to detect or follow growing tumors in the pituitary, neck, and abdomen. CT is a special type of x-ray machine that visualizes tissues, such as thyroid or parathyroid tumors. MRI uses a magnetic field and radio waves to obtain pictures of different tissues in the head, neck and abdomen. * DEXA scan to assess bone density. This test uses standard low-intensity x-rays.

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Key information

Conditions

Hypercalcemia Adenocarcinoma Adenoma Adenoma, Islet Cell Calcium Metabolism Disorders Carcinoma Carcinoma, Islet Cell Carcinoma, Medullary Carcinoma, Neuroendocrine Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Digestive System Neoplasms Endocrine Gland Neoplasms Endocrine System Diseases Familial Primary Hyperparathyroidism Gastrinoma Genetic Diseases, Inborn Hyperparathyroidism 1 Insulinoma Metabolic Diseases Multiple Endocrine Neoplasia Multiple Endocrine Neoplasia Type 2a Neoplasms Neoplasms by Histologic Type Neoplasms by Site Neoplasms, Ductal, Lobular, and Medullary Neoplasms, Germ Cell and Embryonal Neoplasms, Glandular and Epithelial Neoplasms, Multiple Primary Neoplasms, Nerve Tissue Neoplastic Syndromes, Hereditary Neuroectodermal Tumors Neuroendocrine Tumors Nutritional and Metabolic Diseases Pancreatic Diseases Pancreatic Neoplasms Water-Electrolyte Imbalance

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

About this study

This study will evaluate safety and efficacy of short- and long-term Cinacalcet in treatment of hypercalcemia of familial primary hyperparathyroidism. The study population will include patients with primary hyperparathyroidism caused by multiple endocrine neoplasia type 1 or type 2A. This is a prospective unblinded pilot study, where each patient will serve as his/her own control. It will include 6 one-week-long admissions, over a 1 year period. Main measurements will be tests of hyperparathyroidism such as parathyroid hormone and serum calcium as well as tests of nonparathyroid tumors. The latter tests may include hormones released by the tumors and images of the tumors.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

INCLUSION CRITERIA:

Criteria for inclusion

Patients with primary hyperparathyroidism associated with MEN1 or 2A syndrome and no indications for current surgical interventions.
For initial pilot study, 15-20 patients with MEN1 syndrome and biochemical evidence of hyperparathyroidism will be included.
For initial pilot study, 5-10 patients with MEN2A syndrome and biochemical evidence of hyperparathyroidism will be included. Each MEN2A case must have residual MTC tumor.
In case of ZES, good acidity control with proton pump inhibitors (PPI), documented by basal acid output (BAO) of less than 10 mEq/h.
Willingness and legal ability to give informed consent.
MEN1, like MEN2A has a normal gender and ethnic distribution. These will be maintained, within the limits of a small number of subjects studied.

Exclusion criteria

Criteria for exclusion or removal

Age less than 18 years
Pregnancy
Creatinine greater than 1.4
SGOT or SGPT greater than twice normal
Indications for current surgery:

Hyperparathyroidism

Albumin-adjusted serum calcium level higher than 3.0 mmol/L (12.0 mg/dL)
Kidney stones
Significant PTH-induced bone disease
Age below 50 is not considered as absolute indication for parathyroid surgery herein
Enteropancreatic neuroendocrine neoplasia

Single lesion meeting criteria for surgery
Need for debulking surgery for obstructive or other complications
Acute abdominal complications of any kind
Inability of control on PPI in case of ZES
Chemotherapy within last 6 months
Lactating females
Patients with contraindications for MRI study, including pacemakers, vascular clips, implants, foreign bodies etc., as per NIH-MRI Safety Guideline.

Treatment and study plan

Parathyroid hormone testing

Procedure

Serum calcium testing

Procedure

Sponsors and collaborators

Lead sponsor

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Nih

Registry information

Official study title

Cinacalcet Actions in Familial Primary Hyperparathyroidism

Important dates

Study start: 2006
Study completion: 2007
First posted: May 12, 2006
Registry last updated: Jul 2, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed