Cushing Disease Clinical Trial
Official title:
A Proof of Concept, Open-label, Forced Titration, Multi-center Study to Assess the Safety/Tolerability and Efficacy of 10-weeks Treatment of LCI699 in Patients With Cushing's Disease
This exploratory study is a proof of concept study to determine whether LCI699 can safely reduce the level of urinary free cortisol in patients with Cushing's disease. In addition, this study evaluated the long term efficacy and safety of LCI699 including an additional 12 week of treatment followed by a 12 month long term optional extension. A second extension provided patients who were clinically benefitting from LCI699 an opportunity to continue to have access to the drug until LCI699 was commercially available and reimbursed or through the availability of a local access program.
The Primary objective of this study was to assess the effect of 10-week treatment osilodrostat on 24 hour urine free cortisol (UFC) in patients with Cushing's disease. The study consisted of a screening period of up to 60 days (to allow an adequate washout period for any medications that modified cortisol levels), a 10-14-day baseline period, a 10-week sequential dose escalation treatment period and a 14-day washout period followed by a Study Completion evaluation approximately 14 days after the last drug administration. Twelve patients were recruited and completed Part l of the study. Eligible patients were dosed at 2 mg b.i.d for the first two weeks, the dose could then be increased every two weeks as necessary (to doses of 5, 10, 20 and 50 mg b.i.d). If at anytime, the subject's UFC was < Upper Limit of Normal (ULN), dose escalation was halted and the subject remained on the current, efficacious dose through Week 10, with continued monitoring of UFC responses every 2 weeks to allow continued dose adjustments if necessary. If at any time the subject experienced side effects which were either intolerable or met dose adjustment criteria, the prescribed dose was adjusted. The primary endpoint (UFC ≤ ULN or ≥50% decrease at Day 70) was achieved by all patients. Subsequently, in order to confirm these observations, protocol was amended (Protocol amendment 4) and new patients were enrolled and investigated for a longer treatment period. Following Protocol amendment 4, the study design was modified to include patients in Part II of the study for evaluating the long-term efficacy and safety of osilodrostat treatment for 22 weeks. Nineteen patients (15 who were treated in the expansion cohort in Part ll and 4 who participated in Part l) with Cushing's disease were enrolled as part of the Expansion cohort in Part II of the study. The 12 patients who had entered the study in Part I, were allowed to re-enter the study as the Core proof of concept (PoC) Follow-up cohort. At Day 70 ± 2 days (Week 10), all patients (both patients entering for the first time and those reentering the study) entered the 12-week assessment period. At Day 154, patients completed the End of Treatment-Core visit. On Day 154 (Week 22), patients had the option to enter the 12-month extension phase (long-term extension 1). On Day 490, patients who continued in the study had the option to enter a second long term extension phase (extension-2) at the Investigator's discretion, provided they did not meet any of the study discontinuation criteria. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT02350153 -
Cushing´s Disease Epidemiology in Sweden
|
||
Not yet recruiting |
NCT03297892 -
Health Status of the Patients Followed for a Disease of Cushing in the Region Large-West of 1990 to 2015
|
N/A | |
Recruiting |
NCT05804669 -
A Study to Evaluate the Safety and PK of CRN04894 for the Treatment of Cushing's Syndrome
|
Phase 1/Phase 2 | |
Recruiting |
NCT03364803 -
Collecting Information About Treatment Results for Patients With Cushing's Syndrome
|
||
Recruiting |
NCT03774446 -
Multicenter Study of Seliciclib (R-roscovitine) for Cushing Disease
|
Phase 2 | |
Completed |
NCT03080181 -
Adipokine Profile in Patients With Cushing's Disease on Pasireotide Treatment
|
Phase 4 | |
Recruiting |
NCT03831958 -
Long-Term Follow-Up of Survivors of Pediatric Cushing Disease
|
||
Completed |
NCT04201444 -
Hair Cortisol and Cushing's Disease
|
N/A | |
Completed |
NCT02233335 -
The Factors Associated With the Recurrence in Patients With Cushing Disease
|
N/A | |
Recruiting |
NCT04374721 -
Clinical Study on Circadian Genes Dysregulation in Patients With Glucocorticoid Disorders
|
N/A | |
Recruiting |
NCT03974789 -
Discriminant Capacity and Thresholds of Salivary Cortisol in Chemiluminescence in the Diagnosis of Hypercorticisms
|
||
Completed |
NCT03621280 -
Open-label Treatment in Cushing's Syndrome
|
Phase 3 | |
Not yet recruiting |
NCT05971758 -
Fimepinostat, Combination HDAC and Pi3-kinase Inhibitor Tumor-Directed Therapy for Cushing Disease
|
Phase 2 | |
Recruiting |
NCT04486859 -
Postoperative Thrombosis Prevention in Patients With CD
|
N/A | |
Completed |
NCT00171951 -
Extension Study to Assess the Safety and Efficacy of Pasireotide in Participants With Cushing's Disease
|
Phase 2 | |
Recruiting |
NCT00001595 -
An Investigation of Pituitary Tumors and Related Hypothalmic Disorders
|