Mifepristone in Children With Refractory Cushing's Disease
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT01925092 |
Recruitment Status
:
Withdrawn
(Lack of enrollment)
First Posted
: August 19, 2013
Last Update Posted
: August 4, 2014
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Cushing's Disease | Drug: mifepristone | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 0 participants |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open-label Study of the Safety, Pharmacokinetics and Pharmacodynamics of Mifepristone in Children With Refractory Cushing's Disease |
Study Start Date : | August 2013 |
Estimated Primary Completion Date : | December 2016 |

Arm | Intervention/treatment |
---|---|
Experimental: mifepristone
Daily doses of mifepristone over 84 days.
|
Drug: mifepristone
tablets
Other Name: Korlym
|
- 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.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 6 Years to 17 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Males and females 6-17 years at informed consent
- Active Cushing's disease as demonstrated by the following:
- 24 hour Urinary Free Cortisol greater than the upper limit of normal for age on two urine collections during screening and
- midnight serum cortisol >4.4 mcg/dL (mean of two determinations on a single day at 2330 and 2400 during screening)
- Previous trans-sphenoidal surgery (TSS) for ACTH secreting pituitary tumor at least 3 months prior to screening
- Increased body weight defined by BMI Z-score of 1.5 or above
- Able to provide consent/assent
- Able to swallow study drug tablets (not crushed or split)
- Willing to use non-hormonal method of contraception in patients of reproductive potential
- Primary health care provider in home location
Exclusion Criteria:
- Hypercortisolism not due to Cushing's disease.
- Type 1 diabetes mellitus
- HbA1c ≥9.5% at Screening
- Body weight <25 kg
- Use of certain medications that are CYP3A substrates with narrow therapeutic ranges, such as simvastatin, lovastatin, cyclosporine, dihydroergotamine, ergotamine, fentanyl, pimozide, quinidine, sirolimus, and tacrolimus during the 4 weeks prior to starting study drug. Use of these medications is also prohibited until 2 weeks after end of dosing.
- Use of certain medications that are strong CYP3A inhibitors such as itraconazole, nefazodone, ritonavir, nelfinavir, indinavir, atazanavir, amprenavir, fosamprenavir, boceprevir, clarithromycin, conivaptan, lopinavir, mibefradil, posaconazole, saquinavir, telaprevir, telithromycin, and voriconazole during the 2 weeks prior to starting study drug. Use of these medications is also prohibited until 2 weeks after end of dosing. Grapefruit and grapefruit juice, as well as grapefruit-related fruits and their juice (e.g. Seville oranges, pomelos), are prohibited during this time frame.
- Use of certain medications that are strong inducers of CYP3A such as rifampin, rifabutin, rifapentine, phenobarbital, phenytoin, carbamazepine, St. John's wort during the 2 weeks prior to starting study drug. Use of these medications is also prohibited until 2 weeks after end of dosing.
- Use of medications used to treat hypercortisolism from the duration indicated below prior to Day 1. Use of the medications is also prohibited until after the end of study 4 week follow up visit.
- steroidogenesis inhibitors such as ketoconazole, metyrapone: 4 weeks
- cabergoline, bromocriptine, somatostatin analogs such as octreotide, lanreotide, pasireotide long acting formulations: 8 weeks (immediate release formulations: 2 weeks)
- mitotane: 8 weeks
- Use of systemic glucocorticoid medications beginning 1 month prior to screening or anticipated use of these medications except for the treatment of adrenal insufficiency. Use of glucocorticoid medications is prohibited during the study until after the end of study 4 week study visit.
- Inflammatory, rheumatological, proliferative or other disorder(s) that would be anticipated to worsen with glucocorticoid blockade (e.g. inflammatory bowel disease, rheumatoid arthritis, psoriasis, etc.).
- Uncontrolled hypo- or hyperthyroidism.
- Uncorrected hypokalemia (<3.5 mEq/L). The screening period may be used to correct hypokalemia prior to starting study drug. Use of potassium and/or mineralocorticoid antagonists is permitted during the study.
- QTc ≥450 msec on Screening electrocardiogram
- Unexplained vaginal bleeding in females and/or any history of endometrial pathology.
- Positive pregnancy test in females.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01925092
United States, Maryland | |
National Institute of Child Health and Human Development (NICHD) | |
Bethesda, Maryland, United States, 20892-1103 |
Additional Information:
Responsible Party: | Corcept Therapeutics |
ClinicalTrials.gov Identifier: | NCT01925092 History of Changes |
Other Study ID Numbers: |
13-CH-0170 02811-12 ( Other Identifier: NICHD ) |
First Posted: | August 19, 2013 Key Record Dates |
Last Update Posted: | August 4, 2014 |
Last Verified: | July 2014 |
Keywords provided by Corcept Therapeutics:
Cushing's disease Mifepristone Cushing's syndrome |
Pharmacokinetic-pharmacodynamic Child/pediatric population Safety-efficacy |
Additional relevant MeSH terms:
Pituitary ACTH Hypersecretion ACTH-Secreting Pituitary Adenoma Hyperpituitarism Pituitary Diseases Hypothalamic Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Endocrine System Diseases Adenoma Neoplasms, Glandular and Epithelial Neoplasms by Histologic Type Neoplasms Pituitary Neoplasms Endocrine Gland Neoplasms |
Neoplasms by Site Mifepristone Abortifacient Agents, Steroidal Abortifacient Agents Reproductive Control Agents Physiological Effects of Drugs Contraceptives, Oral, Synthetic Contraceptives, Oral Contraceptive Agents, Female Contraceptive Agents Contraceptives, Postcoital, Synthetic Contraceptives, Postcoital Hormone Antagonists Hormones, Hormone Substitutes, and Hormone Antagonists Luteolytic Agents |