4-stage LCI699 study with optional extension in Cushing's Disease
Research type
Research Study
Full title
A Phase III, multi-center, double-blind, randomized withdrawal study of LCI699 following a 24 week, single-arm,open-label dose titration and treatment period to evaluate the safety and efficacy of LCI699 for the treatment of patients with Cushing’s disease
IRAS ID
158613
Contact name
Peter Trainer
Contact email
Sponsor organisation
Novartis
Eudract number
2013-004766-34
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 7 months, 18 days
Research summary
This study is a Phase III, double-blind, randomised withdrawal study of LCI699 following a 24 week, open-label dose titration and treatment period to evaluate the safety and efficacy of LCI699 for the treatment of patients with Cushing’s disease.
Once eligibility has been confirmed, patients will enter the study. The study has four periods plus an optional extension period:
Period 1: Single-arm, open-label (Week 1 to Week 12), is the individual patient dose titration period. The dose is increased based on the average (mean) of three 24-hour Urinary Free Cortisol (UFC) values as measured by the central lab. Urine samples are collected every two weeks during dose titration and the dose is increased if mUFC (mean UFC) is above normal.
Period 2: (Week 13 to Week 24) is the period to assess the efficacy (how well it works) and safety of LCI699 at the therapeutic (most effective) dose determined during dose titration period.
Period 3: Double-blind, placebo-controlled randomised withdrawal (Week 26 to Week 34). Patients will be randomised at the week 26 visit based on urine samples collected at week 24. Eligible patients are randomised in a double-blinded fashion at Week 26 at a 1:1 ratio either to continue treatment with LCI699 at the same dose or to placebo.
Period 4: Single-arm, open-label therapy (Week 34 to Week 48). At Week 34, patients continue open-label treatment until Week 48 based on mUFC result.
Optional Extension Period: Patients who wish to enter the extension period must be re-consented at week 48. Patients who enter the extension period will do so without interruption of study drug or assessments. After discontinuing the study treatment, the patient will be followed for 28-day safety follow-up visit.
REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
14/NW/1294
Date of REC Opinion
20 Nov 2014
REC opinion
Further Information Favourable Opinion