Clinical trial NCT04872920: Observational Registry on Patients With Endogenous CS to Document Safety and Effectiveness of Ketoconazole.
Official title: Prospective, Multi-country, Observational Registry to Collect Clinical Information on Patients With Endogenous Cushing's Syndrome to Assess Drug Utilization Pattern and to Document the Safety and Effectiveness of Ketoconazole.
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Cushing Syndrome
Lead sponsor: HRA Pharma
Sponsor class: INDUSTRY
First posted: 2021-05-05
Enrollment: 200
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population will include all patients aged 12 years and above with endogenous CS from all causes exposed to ketoconazole and included by the participating centers in ERCUSYN. Patients with CS due to an adrenal carcinoma will be excluded from the study population since adrenal carcinoma is excluded from ERCUSYN registry.

Brief summary: This is a non-interventional, prospective, multinational, multicentre, observational study, in which data collected are those derived from routine clinical practice via an European registry on CS: ERCUSYN.

Detailed description: The aim is to further document the safety and efficacy of Ketoconazole HRA administered in routine clinical practice in patients with CS.

The study operates under real clinical practice conditions, i.e. there is no treatment protocol thus.

Eligibility criteria: Inclusion Criteria:

1. Male or female patients aged from 12 years or older with a diagnosis of CS
2. Patients who started ketoconazole therapy after study start
3. For patients previously treated with ketoconazole, patients must have taken their last dose at least 6 months before starting ketoconazole again in this study.
4. Written informed consent signed prior to registration of any patient data in HRA modules.

Exclusion Criteria:

1. Adrenal cortical carcinoma
2. Patients currently participating in any other trial (interventional or not) of an investigational medicine or participation in the past one month before start of ketoconazole
3. Patients who have at least one contraindication among those listed in section 4.3 of the ketoconazole SmPC

Primary outcomes:
measure: incidence of hepatotoxicity and QT prolongation to assess liver and cardiac tolerability profile of ketoconazole.
timeFrame: up to 5 years of follow up
Secondary outcomes:
measure: Number of patients with adverse events
timeFrame: up to 5 years of follow up
measure: Drug utilization pattern regarding safe utilization of ketoconazole according to HRA SmPC recommendations on liver function
timeFrame: up to 5 years of follow up
measure: Drug utilization pattern of ketoconazole according to HRA SmPC recommendations on liver function
timeFrame: up to 5 years of follow up
measure: Drug utilization pattern regarding safe use of ketoconazole according to HRA SmPC recommendations on liver function
timeFrame: up to 5 years of follow up
measure: Drug utilization pattern regarding safe utilization of ketoconazole according to HRA SmPC recommendations on Qt interval monitoring.
timeFrame: up to 5 years of follow up
measure: Drug utilization pattern of ketoconazole according to HRA SmPC recommendations on Qt interval monitoring.
timeFrame: up to 5 years of follow up
measure: Drug utilization pattern regarding safe use of ketoconazole according to HRA SmPC recommendations on Qt interval monitoring.
timeFrame: up to 5 years of follow up
measure: Effectiveness of ketoconazole
timeFrame: up to 5 years of follow up
measure: Impact on Quality of life, European Quality of Life questionnaire
timeFrame: up to 5 years of follow up
measure: Impact on Quality of life, Cushing Quality of Life questionnaire
timeFrame: up to 5 years of follow up
Other outcomes:
Locations:
city: Zagreb
country: Croatia
facility: University Hospital Zagreb
city: Grenoble
country: France
facility: Hôpital Universitaire Grenoble
city: Le Kremlin-Bicêtre
country: France
facility: Hôpital Bicêtre APHP
city: Marseille
country: France
facility: Hôpital de la Conception
city: Barcelona
country: Spain
facility: Institut de Recerca de la Santa Creu i Sant Pau
city: Gothenburg
country: Sweden
facility: Sahlgrenska University Hospital
Study officials:
References: