Treatment for Endogenous Cushing's Syndrome
The primary objectives of this study are to evaluate the efficacy of ascending doses of COR-003 in subjects with elevated levels of cortisol due to endogenous Cushing's Syndrome by assessment of reduction in Urinary Free Cortisol (UFC) concentrations and to identify the range of safe and effective doses of COR-003 that reduce mean UFC concentrations ≤ULN (upper limit of normal) of the assay at month 6 of the maintenance phase of dosing without a prior dose increase in that phase.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||An Open Label Study to Assess the Safety and Efficacy of COR--003 (2S, 4R-ketoconazole) in the Treatment of Endogenous Cushing's Syndrome|
- Reduction in urinary free cortisol in patients with endogenous Cushing's Syndrome. [ Time Frame: 6 months of maintenance phase therapy without a prior dose increase during that phase ] [ Designated as safety issue: No ]The response to COR-003 is defined as mean UFC concentration ≤ULN following 6 months of maintenance phase therapy without a prior dose increase during that phase.
|Study Start Date:||January 2014|
|Estimated Study Completion Date:||January 2016|
|Estimated Primary Completion Date:||January 2016 (Final data collection date for primary outcome measure)|
Male or female, ≥18 year of age, or of a minimal age as required by the local regulations with confirmed diagnosis of CS as defined according to the criteria in the guidelines for diagnosis of CS (Nieman 2008).
This will be a single period, open-label, dose titration study to assess efficacy, safety, tolerability, and PK of COR-003 in subjects with CS. The trial design will identify both the minimally effective and maximally tolerated doses in this CS population. Following an initial screening period, this study will be conducted in 2 treatment phases as follows:
- Dose titration phase: approximately 2 to 16 weeks to achieve an effective and tolerable maximum dose (the therapeutic dose)
- Maintenance phase: 6 months of treatment at the therapeutic dose without a prior dose increase following the establishment of the appropriate dose identified in the titration phase;
- Extended evaluation phase: 6 months of continued treatment after the maintenance phase (6 - 12 months); dose adjustments will be allowed as required for treatment
Efficacy will be assessed by measuring UFC concentrations at specified times as described in the clinical protocol.
Blood samples for the PK determination will be collected at the times indicated in the clinical protocol.
An independent Data Safety Monitoring Board (DSMB) will review the safety of the drug throughout the study. The constituents of the DSMB membership and a adjudication committee is specifically described in the clinical protocol.
Subjects completing the 6-month maintenance phase of the study will remain in the study for an additional 6 months for extended evaluations.
COR-003 will be provided under a compassionate use protocol for subjects who wish to continue treatment with COR-003.
|Contact: Theodore R Koziol, Ph.D.||email@example.com|