Clinical Trial: Mifepristone in Children With Refractory Cushing's Disease

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

Official Title: An Open-label Study of the Safety, Pharmacokinetics and Pharmacodynamics of Mifepristone in Children With Refractory Cushing's Disease

Brief Summary: Study objectives are to obtain safety, pharmacokinetic, and pharmacodynamic data on the effect of mifepristone on glucose metabolism, body weight and the growth-hormone-IGF in children with refractory Cushing's disease.

Detailed Summary: The study is being done to examine the effects of a medication called mifepristone in children with Cushing's disease. Since a child's body may absorb and use mifepristone in a different way than adults, it is important to have information about the amount of mifepristone to give children and what will happen to it. Mifepristone has been FDA approved for use only in adults with Cushing's syndrome, and it is important to learn if mifepristone improves the symptoms and signs of Cushing's disease in children. The study is limited to children with Cushing's syndrome due to a pituitary tumor (Cushing's disease) and will not enroll children with Cushing's syndrome due to other causes. The study will investigate how children's bodies absorb and process mifepristone, how it works in children and what effect it has on the use of sugar in the body, on the child's weight and on growth hormone. An important part of the study is to evaluate the side effects of mifepristone in children.
Sponsor: Corcept Therapeutics

Current Primary Outcome: Adverse events [ Time Frame: collected during the12 week study and 4 week follow-up period; up to 16 weeks total. ]

Patients who have received at least 1 dose of mifepristone will be included in the safety evaluations.


Original Primary Outcome: Adverse events [ Time Frame: collecte during the12 week study and 4 week follow-up period; up to 16 weeks total. ]

Patients who have received at least 1 dose of mifepristone will be included in the safety evaluations.


Current Secondary Outcome:

Original Secondary Outcome:

Information By: Corcept Therapeutics

Dates:
Date Received: August 8, 2013
Date Started: August 2013
Date Completion:
Last Updated: July 31, 2014
Last Verified: July 2014