Cushing's DiseasePhase 3NCT01925092
Mifepristone in Children With Refractory Cushing's Disease
A Phase 3 study testing mifepristone in people with Cushing's Disease, run by Corcept Therapeutics at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Cushing's Disease
- What is being tested?
- mifepristone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2013to Dec 2016
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Show all 10Show fewer
- 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
Who cannot join
- 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.
Show all 18Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institute of Child Health and Human Development (NICHD)
Bethesda, Maryland, 20892-1103
About this study
Official title: An Open-label Study of the Safety, Pharmacokinetics and Pharmacodynamics of Mifepristone in Children With Refractory Cushing's Disease
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.
Read the detailed description
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.
- Conditions
- Cushing's Disease
- Being tested
- mifepristone
- Keywords
- Cushing's disease, Mifepristone, Cushing's syndrome, Pharmacokinetic-pharmacodynamic, Child/pediatric population, Safety-efficacy
- Medical subject headings
- Pituitary ACTH Hypersecretion, Cushing Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mifepristoneDrug
Also called Korlym
tablets
| Study group | Role | What they receive |
|---|---|---|
| mifepristone | Experimental | Daily doses of mifepristone over 84 days. Receives: Drug: mifepristone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events
Patients who have received at least 1 dose of mifepristone will be included in the safety evaluations.
Measured: collected during the12 week study and 4 week follow-up period; up to 16 weeks total.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2013 (estimated)
- Study startAug 2013
- Primary completionDec 2016 (estimated)
Why it stopped: Lack of enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cushing's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Corcept Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01925092
- Sponsor's protocol ID
- 13-CH-0170
- Other IDs
- 02811-12
- Status last verified
- Jul 2014
- First submitted
- 8 Aug 2013
- Last updated
- 4 Aug 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 Aug 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Study of the Safety, Pharmacokinetics and Pharmacodynamics of Mifepristone in Children With Refractory Cushing's Disease
- ClinicalTrials.gov ID
- NCT01925092
- Acronym / sponsor ID
- 13-CH-0170
- Conditions
- Cushing's Disease
- Keywords
- Cushing's disease, Mifepristone, Cushing's syndrome, Pharmacokinetic-pharmacodynamic, Child/pediatric population, Safety-efficacy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of enrollment
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Corcept TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Study start
- Aug 2013
- Primary completion
- Dec 2016 (estimated)
- First posted
- 19 Aug 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| mifepristone | Experimental | Drug: mifepristoneDaily doses of mifepristone over 84 days. |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- 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
Key exclusion criteria (18 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| mifepristoneAlso: Korlymtablets | Drug | mifepristone |
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.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Aug 2014.
- An Open-Label Study of The Safety, Pharmacokinetics and Pharmacodynamics of Mifepristone in Children With Refractory Cushing's DiseaseLink provided by the sponsor.
Related trials for Cushing's Disease
RecruitingObservational study
Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena)
Cushing s Syndrome, Cushing Disease
Active, not recruitingPhase 2
Extension Study to Evaluate the Safety of Long-Term Use of Relacorilant in Patients With Cushing Syndrome
Cushing Syndrome
RecruitingPhase 1/2
Diagnostic Value of CXCR4-targeted PET/CT in ACTH-dependent and Independent Cushing's Syndrome
Cushing s Syndrome
RecruitingObservational study
Long-Term Follow-Up of Survivors of Pediatric Cushing Disease
Cushing Disease
RecruitingPhase 4
Osilodrostat in Patients With Hypertension Caused by Hypercortisolaemia Due to Cushing's Syndrome
Hypertension, Hypercortisolemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing mifepristone in people with Cushing's Disease, run by Corcept Therapeutics at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 4 Aug 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events (time frame: collected during the12 week study and 4 week follow-up period; up to 16 weeks total.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Aug 2014.