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NCT01331239 · ClinicalTrials.gov registry record · Phase 2

Safety and Efficacy of LCI699 in Cushing's Disease Patients

A Phase 2 study of Cushing Disease and Cushing's Disease, sponsored by Novartis Pharmaceuticals.

Completed
Registry status
Phase 2
Development phase
31
Enrollment target
10
Study locations

NCT01331239: Completed Phase 2 study of Cushing Disease and Cushing's Disease, sponsored by Novartis Pharmaceuticals.

NCT01331239 is a Phase 2 study of Cushing Disease and Cushing's Disease that has completed, run by Novartis Pharmaceuticals. The registered enrollment target is 31 participants, below the 389-participant average among 8 other Cushing Disease trials with a reported enrollment target (92% lower). The trial reports 10 study locations across 6 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT01331239, a Phase 2 study of Cushing Disease and Cushing's Disease, has completed, sponsored by Novartis Pharmaceuticals.

COMPLETED
Registry status
Phase 2
Development phase
31 participants
Enrollment target
10
Study locations

Study Summary

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.

Primary Outcome

A patient was considered to be a responder if his/her mean UFC level from the three 24-hour urine samples collected at Week 10 was ≤ Upper Limit of Normal (ULN), as defined by the local laboratories, or represented a ≥50% decrease from baseline. Patients who discontinued for a disease or treatment related reason (e.g. death, adverse event, clinical disease progression etc.), or whose mean Week 10 24-hour UFC levels were higher than the normal limit and experienced \<50% decrease in UFC were clas

Conditions Studied

Cushing Disease Cushing's Disease

Interventions

  • DRUG LCI699

Study Locations (10)

Other

  • Novartis Investigative Site - Le Kremlin-Bicêtre
  • Novartis Investigative Site - Paris
  • Novartis Investigative Site - Ancona
  • Novartis Investigative Site - Naples
  • Novartis Investigative Site - Chiba

Illinois

  • Northwestern University Endo, Metabolism and Molecular - Chicago

Massachusetts

  • Massachusetts General Hospital Neuroendocrine Unit - Boston

Ohio

  • Cleveland Clinic Foundation - Cleveland

Oregon

  • Oregon Health and Science University SC - Portland

Hokkaido

  • Novartis Investigative Site - Sapporo

Trial Details

FieldValue
Enrollment Target 31 participants
Start Date 2011-03-23
Est. Completion 2019-10-22
Phase Phase 2
Novartis Pharmaceuticals

1,375 total trials

What the finished NCT01331239 record still lists

NCT01331239 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 31 participants, a relatively small participant target, below the 389-participant average among 8 other Cushing Disease trials with a reported enrollment target (92% lower).

The record links to 2 conditions, with Cushing Disease appearing as the primary indexed condition, and to 1 intervention - of which LCI699 is the first listed.

NCT01331239 lists 10 locations in 6 states (Other, Illinois, Massachusetts).

Frequently Asked Questions

What is clinical trial NCT01331239 about?

NCT01331239 is a clinical study titled "Safety and Efficacy of LCI699 in Cushing's Disease Patients". 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...

What is the current status of trial NCT01331239?

This trial is currently completed. It is a Phase 2 study. The enrollment target is 31 participants. The study started on 2011-03-23. Estimated completion is 2019-10-22.

What conditions does trial NCT01331239 study?

This clinical trial studies the following conditions: Cushing Disease, Cushing's Disease.

What interventions are being tested in trial NCT01331239?

The interventions under investigation include: LCI699 (DRUG).

Who is sponsoring clinical trial NCT01331239?

This trial is sponsored by Novartis Pharmaceuticals, which has 1,375 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT01331239 being conducted?

This trial has 10 study locations across Illinois, Massachusetts, Ohio, Oregon, Hokkaido. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Learn More About Clinical Trials

Similar trials for Cushing Disease

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT01331239's enrollment target sits among peer trials

31 6th of 8 higher than 3 of 8 other Cushing Disease trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Cushing Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Related registry views

Source: ClinicalTrials.gov NCT01331239, the US trial registry maintained by the National Library of Medicine. NCT01331239 (small enrollment · multi site footprint · completed) retrieved and formatted by PlainTrial, see methodology.