NCT00325104

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started May 2006

Shorter than P25 for phase_3

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 9, 2006

Completed
2 days until next milestone

First Submitted

Initial submission to the registry

May 11, 2006

Completed
1 day until next milestone

First Posted

Study publicly available on registry

May 12, 2006

Completed
11 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 31, 2007

Completed
Last Updated

July 2, 2017

Status Verified

March 31, 2007

First QC Date

May 11, 2006

Last Update Submit

June 30, 2017

Conditions

Keywords

Multiple Endocrine NeoplasiaGastrinomaProlacinomaMedullary Thyroid CancerInsulinomaMEN1MEN2AFamilial Primary Hyperparathytoidism

Interventions

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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
  • +3 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (1)

  • Rizzoli R, Green J 3rd, Marx SJ. Primary hyperparathyroidism in familial multiple endocrine neoplasia type I. Long-term follow-up of serum calcium levels after parathyroidectomy. Am J Med. 1985 Mar;78(3):467-74. doi: 10.1016/0002-9343(85)90340-7.

    PMID: 2858157BACKGROUND

MeSH Terms

Conditions

HypercalcemiaHyperparathyroidism 1Multiple Endocrine NeoplasiaGastrinomaCarcinoma, MedullaryInsulinomaMultiple Endocrine Neoplasia Type 2a

Condition Hierarchy (Ancestors)

Calcium Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesWater-Electrolyte ImbalanceEndocrine Gland NeoplasmsNeoplasms by SiteNeoplasmsNeoplasms, Multiple PrimaryNeoplastic Syndromes, HereditaryGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesEndocrine System DiseasesCarcinoma, Islet CellAdenocarcinomaCarcinomaNeoplasms, Glandular and EpithelialNeoplasms by Histologic TypePancreatic NeoplasmsDigestive System NeoplasmsDigestive System DiseasesPancreatic DiseasesCarcinoma, NeuroendocrineNeuroendocrine TumorsNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms, Ductal, Lobular, and MedullaryNeoplasms, Nerve TissueAdenoma, Islet CellAdenoma

Study Design

Study Type
interventional
Phase
phase 3
Purpose
TREATMENT
Sponsor Type
NIH

Study Record Dates

First Submitted

May 11, 2006

First Posted

May 12, 2006

Study Start

May 9, 2006

Study Completion

March 31, 2007

Last Updated

July 2, 2017

Record last verified: 2007-03-31

Locations