Placebo-controlled study of the safety and efficacy of osilodrostat in patients with Cushing's Disease

A Phase III, multi-center, randomized, double-blind, 48 week study with an initial 12 week placebo-controlled period to evaluate the safety and efficacy of osilodrostat in patients with Cushing?s disease - LINC-4

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2014-004092-23-ES

Enrollment

Registered

2016-03-18

Start date

2016-04-29

Completion date

Unknown

Last updated

2022-04-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cushing's disease MedDRA version: 18.1 Level: LLT Classification code 10011651 Term: Cushing's disease System Organ Class: 100000004860

Interventions

Product Name: osilodrostat 1mg Product Code: LCI699 Pharmaceutical Form: Tablet INN or Proposed INN: osilodrostat Current Sponsor code: LCI699 Concentration unit: mg milligram(s) Concentration type: e

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Male or female patients aged 18 - 75 years Confirmed CD that is persistent or recurrent For patients with a history of pituitary irradiation: at least 2 years since stereotactic radiosurgery (SRS), and 3 years since conventional (fractionated) radiation Able to communicate well with the investigator, to understand and comply with the requirements of the study Washout of any current drug therapy for CD Additional inclusion criteria as per full protocol may apply. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 55 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 14

Exclusion criteria

Exclusion criteria: - Use of other investigational drugs at the time of enrollment - Pseudo-Cushing?s syndrome - History of malignancy of any organ system - Risk factors for QTc prolongation or Torsade de Pointes - Pregnant or nursing (lactating) women - Women of child-bearing potential, unless they are using highly effective methods of contraception Additional exclusion criteria as per full protocol may apply.

Design outcomes

Primary

Measure	Time frame
Primary end point(s): The proportion of randomized patients with a complete response, i.e. mUFC ?;Timepoint(s) of evaluation of this end point: Week 12;Main Objective: To demonstrate the superiority of osilodrostat compared to placebo in achieving a complete response (mUFC ? ULN) at Week 12;Secondary Objective: To assess the complete response rate after 24 weeks of open-label osilodrostat treatment To assess the overall response rate at Week 12, 36, and 48 To assess the change in mUFC To compare the time-to-first control of UFC during period 1 between treatment arms To assess the time-to-escape To assess cardiovascular and metabolic-related parameters associated with CD To assess the change over time in Health Related Quality of Life To assess the change from baseline in physical features of CD To assess the change from baseline in bone mineral density To evaluate pharmacokinetic exposure of osilodrostat To determine the safety and tolerability of osilodrostat Other objectives may apply	—

Measure

Time frame

Primary end point(s): The proportion of randomized patients with a complete response, i.e. mUFC ?;Timepoint(s) of evaluation of this end point: Week 12;Main Objective: To demonstrate the superiority of osilodrostat compared to placebo in achieving a complete response (mUFC ? ULN) at Week 12;Secondary Objective: To assess the complete response rate after 24 weeks of open-label osilodrostat treatment To assess the overall response rate at Week 12, 36, and 48 To assess the change in mUFC To compare the time-to-first control of UFC during period 1 between treatment arms To assess the time-to-escape To assess cardiovascular and metabolic-related parameters associated with CD To assess the change over time in Health Related Quality of Life To assess the change from baseline in physical features of CD To assess the change from baseline in bone mineral density To evaluate pharmacokinetic exposure of osilodrostat To determine the safety and tolerability of osilodrostat Other objectives may apply

—

Secondary

Measure	Time frame
Secondary end point(s): To assess the complete response rate after 24 weeks of open-label osilodrostat treatment;Timepoint(s) of evaluation of this end point: Week 36	—

Countries

Belgium, Brazil, Denmark, Greece, Poland, Portugal, Russian Federation, Spain, Switzerland, Thailand, Turkey, United States

Contacts

Public ContactDepartamento Médico (ICRO)

Novartis Farmacéutica, S.A.

eecc.novartis@novartis.com+34900353036

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026