Clinical trial: Roll-over Study in Patients With Endogenous Cushing's Syndrome for LCI699 (NCT03606408)

Eligibility criteria:
Inclusion Criteria:

          -  Patient is currently participating in a Global Novartis-sponsored study receiving
             osilodrostat for any type of endogenous CS and has fulfilled all their requirements in
             the parent study.

          -  Patient is currently benefiting from treatment with osilodrostat, as determined by the
             Investigator.

          -  Patient has demonstrated compliance, as assessed by the Investigator, with the parent
             study protocol requirements.

          -  Willingness and ability to comply with scheduled visits and treatment plans.

          -  Written informed consent obtained prior to enrolling into the roll-over study before
             evaluating the applicability of the subject's participating in the study. -- If
             consent cannot be expressed in writing, it must be formally documented and witnessed,
             ideally via an independent trusted witness.

        Exclusion Criteria:

          -  Patient has been permanently discontinued from osilodrostat study treatment in a
             parent Novartis-sponsor study.

          -  Patients who are receiving osilodrostat in combination with unapproved or experimental
             treatments for any type of endogenous CS.

          -  Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a
             female after conception and until the termination of gestation, confirmed by a
             positive hCG laboratory test. Pregnant or nursing (lactating) women

          -  Women of child-bearing potential, defined as all women physiologically capable of
             becoming pregnant, unless they are using highly effective methods of contraception
             during dosing and for 1 week of study after stopping medication. Highly effective
             contraception methods include:

               -  Total abstinence (when this is in line with the preferred and usual lifestyle of
                  the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal,
                  post-ovulation methods) and withdrawal are not acceptable methods of
                  contraception

               -  Female sterilization (have had surgical bilateral oophorectomy with or without
                  hysterectomy), total hysterectomy, or tubal ligation. at least six weeks before
                  taking study treatment. In case of oophorectomy alone, only when the reproductive
                  status of the woman has been confirmed by follow up hormone level assessment

               -  Male sterilization (at least 6 months prior to baseline). The vasectomized male
                  partner should be the sole partner for that subject

               -  Use of oral, (estrogen and progesterone), injected or implanted hormonal methods
                  of contraception or placement of an intrauterine device (IUD) or intrauterine
                  system (IUS), or other forms of hormonal contraception that have comparable
                  efficacy (failure rate <1%), for example hormone vaginal ring or transdermal
                  hormone contraception.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 18 Years old.
Subject must be at most 75 Years

Patient description (medical language): A 47-year-old man comes to the office due to weight gain and fatigue.  He is not able to lift heavy objects or climb stairs. Family history is positive for DM type 2 and HTN in his father.  Blood pressure is 165/90 mm Hg and pulse is 85/min.  On physical examination, there is symmetric proximal muscle weakness of the upper and lower extremities.  Fasting plasma glucose level is 138 mg/dL and 24-hour urinary cortisol is twice the upper normal limit.  Further evaluation reveals that high-dose, but not low-dose, dexamethasone suppresses serum cortisol levels.  Serum ACTH levels are high-normal. This patient's findings are consistent with endogenous Cushing Syndrome.

Patient description (patient's own words): I'm a 47-year-old man. I went to the clinic because I had been gaining weight and had increased fatigue over the past few weeks. I was not able to lift heavy objects or even climb the stairs. My dad was diagnosed with type 2 diabetes and high blood pressure. During the exam, they took my blood pressure, which was 165/90 mm Hg, and my pulse was 85/min. They found out that I have weaknesses in both of my arms and legs. I also did a blood test on an empty stomach, and I had a glucose level of 138 mg/dL, and twice the upper normal limit of cortisol. I had high-normal levels of ACTH. The doctor said I suffer from endogenous Cushing Syndrome.

Does the patient match the trial criteria? 0