Treatment for Endogenous Cushing's Syndrome
An Open Label Study to Assess the Safety and Efficacy of COR-003 (Levoketoconazole) in the Treatment of 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.
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
- 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
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.
Endogenous Cushing's Syndrome
Belgium, Bulgaria, Canada, Czech Republic, Denmark, France, Germany, Hungary, Israel, Italy, Netherlands, Poland, Serbia, Spain, Sweden, Switzerland, United Kingdom, United States, Georgia