A Phase 2 interventional study of Pasireotide with or without cabergoline in Cushings Disease, sponsored by Novartis Pharmaceuticals. Terminated at 29 sites in 16 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-09-18.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 2, Interventional, and Treatment
The main purpose of this prospective, multicenter, open-label phase II study, was to evaluate the efficacy and safety of pasireotide alone or in combination with cabergoline in patients with Cushing's disease.
This was an open-label, multi-center, international, non-comparative study with adult patients with confirmed diagnosis of Cushing's disease. Given the fact that CD patients may need a multimodality treatment approach, the trial design aimed to mimic CD treatment by using a medical stepwise approach. Therefore, the whole patient population started treatment with Pasireotide and only in patients within this population who did not achieve biochemical control, cabergoline was added.
The whole patient population had never received pasireotide or had received it in the past (reasons of discontinuation not related to safety).
Core Phase
Extension Phase
Novartis had a local transition plan in order to ensure that all trial patients had access to the study medication without any delay in their treatment
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Adult patients with confirmed diagnosis of ACTH-dependent Cushing's disease as evidenced by all of the following:
Patients on medical treatment for Cushing's disease the following must have been completed before screening assessments were performed
Exclusion criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Child-Pugh class
A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
pasireotide alone or with cabergoline
Drug: Pasireotide with or without cabergoline
Drug pages: Pasireotide · Cabergoline
The trial consisted of Pasireotide-untreated patients who started pasireotide 0.6mg twice a day for 8 weeks. If biochemical control was not achieved by the end of the 8 weeks, and the 0.6mg dose is well-tolerated, the dose was increased to 0.9mg twice a day for another 8 weeks. If biochemical control is not achieved, cabergoline was added and patients began combination treatment with cabergoline at the starting dose of 0.5mg once a day for 8 weeks. If biochemical control is still not achieved at the end of the third 8 week period, the dose of cabergoline was increased to 1.0mg once a day. Patients could also immediately start the combination treatment by adding cabergoline 0.5mg once a day at study entry to their current maximal tolerated dose of pasireotide. Patients continued with the combination treatment for 8 weeks. If biochemical control was not not achieved by the end of the 8 week period, the dose of cabergoline was increased to 1mg once a day.
Percentage of Responders With Mean Urinary Free Cortisol (mUFC) ≤ 1.0xULN Collected or Imputed at Week 35
Participants who attained mUFC ≤ 1.0 x ULN (upper limit of normal) with pasireotide alone or in combination with cabergoline. The 24h-UFC concentration results from three samples, collected during the screening period, were averaged to obtain the Baseline urinary free cortisol level. mean 24h-UFC was determined from two 24-hour urine collections collected on two consecutive days that occurred before the visit. Imputation: subjects who completed the end of treatment visit at Week 35, but had missing evaluation of mean urinary free cortisol (mUFC). The last available mUFC assessment at or after previous visit (week) before last week was carried forward as the last week mUFC assessment.
Time frame: Baseline up to week 35
Mean Urinary Free Cortisol (mUFC) at Scheduled Visits
Mean value of mUFC at each scheduled visit was determined from two 24-hour urine collections collected on two consecutive days that occurred before the visit when UFC was measured.
Time frame: Baseline, weeks 2, 4, every 4 or 5 weeks during core, every 8 weeks during extension
Percentage of Responders With Mean Urinary Free Cortisol (mUFC) ≤ 1.0xULN
Actual mean value of mUFC at each scheduled visit was determined from two 24-hour urine collections collected on two consecutive days that occurred before the visit when UFC was measured.
Time frame: Baseline up to week 235
Percentage of Participants Who Attain mUFC ≤ 1.0 x ULN or Have at Least 50% Reduction From Baseline in mUFC
Actual mean value of mUFC at each scheduled visit was determined from two 24-hour urine collections collected on two consecutive days that occurred before the visit when UFC was measured.
Time frame: Baseline up to week 235
Duration (Weeks) of Controlled or Partially Controlled Response
Duration of controlled or partially controlled response is defined as the period starting from the date of patient's first normalization (mUFC ≤ 1.0 x ULN) or at least 50% reduction from baseline up to the date when the patient's mUFC \> 1.0 x ULN and the reduction from baseline falls to less than 50% from the first time
Time frame: from the date patient's first normalization (mUFC ≤ 1.0xULN) or at least 50% reduction from baseline up to the date when the patient's mUFC > 1.0 x ULN
Plasma Adrenocorticotropic Hormone (ACTH)
A pre-dose blood draw for plasma ACTH sampling was taken at visits. Samples were analyzed by a central laboratory.
Time frame: Baseline, weeks 2, 4, every 4 or 5 weeks during core, every 8 weeks during extension
Serum Cortisol Levels
A pre-dose blood draw for an 8 am fasting serum cortisol measurement were taken at visits. Samples were analyzed by a central laboratory.
Time frame: Baseline, weeks 2, 4, every 4 or 5 weeks during core, every 8 weeks during extension
Sitting Systolic Blood Pressure at Week 35
The mean arterial blood pressure determinations were obtained in the sitting position. Three measurements were taken with 5 minute intervals and the mean was used for study specific procedures
Time frame: Baseline and week 35
Sitting Diastolic Blood Pressure at Week 35
The mean arterial blood pressure determinations were obtained in the sitting position. Three measurements were taken with 5 minute intervals and the mean was used for study specific procedures
Time frame: Baseline and week 35
Body Weight at Week 35
Weight was was one of the measures of clinical symptoms of CD
Time frame: Baseline and week 35
Body Mass Index at Week 35
Body mass index was one of the measurements of clinical symptoms of CD. Body mass index=weight in kg divided by the square of the body height (in meters)
Time frame: Baseline and week 35
Waist Circumference at Week 35
Waist circumference was one of the measurements of clinical signs of CD
Time frame: Baseline and week 35
LDL, HDL and Total Cholesterol at Week 35
LDL=Low density lipoprotein, HDL=high density llipoprotein and total protein were analyzed by a central laboratory
Time frame: Baseline and week 35
Mean Scores of Cushing QoL Standardized Score at Week 17 and 35
Patients who completed nine or more items for the 12-item Cushing's syndrome QoL questionaire were considered evaluable for that assessment. Raw scores were obtained for the 12 items using the following scoring: 1) always or very much, 2) often or quite a bit, 3) sometimes or somewhat, 4) rarely or very little, 5) never or not at all. Then the standardized score, Y, was calculated for each patient as follows: Y = 100\* (X - n) / n\*5 - n\*1) = 100 \* (X - n) / 4\*n where X denoted the raw score and n the number of questions answered by the patient. The higher the score, the better the QoL. The best possible standardized score was 100 and the worst possible standardized score was 0
Time frame: Baseline and week 17 and 35
Mean Scores of SF-12v2 Domain Scores at Week 17 and 35
SF-12v2 General Health Survey is a general patient reported outcome instrument over time. It is scored to provide eight health domain scores (Bodily Pain (BP), General Health (GH), Physical Functioning (PF), Role-Physical (RP), Social Functioning (SF), Role-Emotional (RE), Vitality (VT) and Mental Health (MH)). These eight domain scores can be combined to form two summary scores reflecting overall physical and mental health: the Physical Component Summary (PCS) and the Mental Component Summary (MCS). The analyses reported here focus on PCS and MCS scores. The domain scores use a norm-based score, which standardizes the scores with respect to the mean and standard deviation of a nationally representative sample of United States (US) adults. These are the scores on the original scale which have not been transformed in any way. The possible range of scores is 0 to 100, with higher scores representing better outcomes.
Time frame: Baseline, week 17 and 35
Number of Participants With Improvement in Clinical Symptom of Hypercortisolism From Baseline - Facial Rubor
Clinical symptoms include: facial rubor, hirsutism, striae, and supraclavicular and dorsal fat pad. Two photographs, one frontal and one lateral from the shoulders up were taken to assess facial plethora, supraclavicular and dorsal fat pads. Two photographs, frontal and dorsal of the trunk with patient in standing position were taken to assess striae, and hirsutism. The photographs were assessed by a qualified physician at the site. Improvement=decrease in severity of symptom since baseline
Time frame: Baseline, weeks 2, 4, every 4 or 5 weeks during core, every 16 weeks during extension
Number of Participants With Improvement in Clinical Symptom of Hypercortisolism From Baseline - Hirsutism
Clinical symptoms include: facial rubor, hirsutism, striae, and supraclavicular and dorsal fat pad. Two photographs, one frontal and one lateral from the shoulders up were taken to assess facial plethora, supraclavicular and dorsal fat pads. Two photographs, frontal and dorsal of the trunk with patient in standing position were taken to assess striae, and hirsutism. The photographs were assessed by a qualified physician at the site. Improvement=decrease in severity of symptom since baseline
Time frame: Baseline, weeks 2, 4, every 4 or 5 weeks during core, every 16 weeks during extension
Number of Participants With Improvement in Clinical Symptom of Hypercortisolism From Baseline - Striae
Clinical symptoms include: facial rubor, hirsutism, striae, and supraclavicular and dorsal fat pad. Two photographs, one frontal and one lateral from the shoulders up were taken to assess facial plethora, supraclavicular and dorsal fat pads. Two photographs, frontal and dorsal of the trunk with patient in standing position were taken to assess striae, and hirsutism. The photographs were assessed by a qualified physician at the site. Improvement=decrease in severity of symptom since baseline
Time frame: Baseline, weeks 2, 4, every 4 or 5 weeks during core, every 16 weeks during extension
Number of Participants With Improvement in Clinical Symptom of Hypercortisolism From Baseline - Supraclavicular Fat Pad
Clinical symptoms include: facial rubor, hirsutism, striae, and supraclavicular and dorsal fat pad. Two photographs, one frontal and one lateral from the shoulders up were taken to assess facial plethora, supraclavicular and dorsal fat pads. Two photographs, frontal and dorsal of the trunk with patient in standing position were taken to assess striae, and hirsutism. The photographs were assessed by a qualified physician at the site. Improvement=decrease in severity of symptom since baseline
Time frame: Baseline, weeks 2, 4, every 4 or 5 weeks during core, every 8 weeks during extension
Number of Participants With Improvement in Clinical Symptom of Hypercortisolism From Baseline - Dorsal Fat Pad
Clinical symptoms include: facial rubor, hirsutism, striae, and supraclavicular and dorsal fat pad. Two photographs, one frontal and one lateral from the shoulders up were taken to assess facial plethora, supraclavicular and dorsal fat pads. Two photographs, frontal and dorsal of the trunk with patient in standing position were taken to assess striae, and hirsutism. The photographs were assessed by a qualified physician at the site. Improvement=decrease in severity of symptom since baseline
Time frame: Baseline, weeks 2, 4, every 4 or 5 weeks during core, every 16 weeks during extension
Number of Participants With Shift From Mild to Severe in Clinical Signs of Hypercortisolism
Facial rubor, hirsutism, striae, and supraclavicular and dorsal fat pad were assessed. Two photographs, one frontal and one lateral from the shoulders up will be taken to assess facial plethora, supraclavicular and dorsal fat pads. Two photographs, frontal and dorsal of the trunk with patient in standing position will be taken to assess striae, and hirsutism. The photographs must be assessed by a qualified physician at the site. Improvement=decrease in severity of symptom since baseline
Time frame: Baseline, weeks 1, 2, 4, every 4 or 5 weeks during core, every 8 weeks during extension
Number of Patients With Shift From Standing Easily to Not Being Able to Stand
To test proximal muscle strength patients should be placed in a low seated position (for instance on an examination room stool). They should be asked to extend the arms in front of them. From this seated position patients will be asked to stand up. Patients will be evaluated using the following scale: 3. - completely unable to stand 2. - able to stand only by using arms as assistance 1. - able to stand after several efforts without using arms as assistance 0. - able to stand easily with arms extended
Time frame: Baseline up to week 267
| Milestone | All Patients |
|---|---|
| Started | 68 |
| Completed | 52 |
| Not completed | 16 |
| Withdrew: Adverse event | 7 |
| Withdrew: Unsatisfactory therapeutic effect | 3 |
| Withdrew: Death | 2 |
| Withdrew: Protocol violation | 2 |
| Withdrew: Abnormal lab value | 1 |
| Withdrew: Withdrawal by subject | 1 |
| Milestone | All Patients |
|---|---|
| Started | 29 |
| Completed | 12 |
| Not completed | 17 |
| Withdrew: Unsatisfactory therapeutic effect | 8 |
| Withdrew: Withdrawal by subject | 4 |
| Withdrew: Adverse event | 3 |
| Withdrew: Death | 1 |
| Withdrew: Protocol violation | 1 |
Participants who attained mUFC ≤ 1.0 x ULN (upper limit of normal) with pasireotide alone or in combination with cabergoline. The 24h-UFC concentration results from three samples, collected during the screening period, were averaged to obtain the Baseline urinary free cortisol level. mean 24h-UFC was determined from two 24-hour urine collections collected on two consecutive days that occurred before the visit. Imputation: subjects who completed the end of treatment visit at Week 35, but had missing evaluation of mean urinary free cortisol (mUFC). The last available mUFC assessment at or after previous visit (week) before last week was carried forward as the last week mUFC assessment.
| percentage of responders | All Patients |
|---|---|
| Percentage of Responders With Mean Urinary Free Cortisol (mUFC) ≤ 1.0xULN Collected or Imputed at Week 35 | 50.0 (37.6 to 62.4) |
Mean value of mUFC at each scheduled visit was determined from two 24-hour urine collections collected on two consecutive days that occurred before the visit when UFC was measured.
| nmol/24h | All Patients |
|---|---|
| Baseline | 501.6 ± 488.66 |
| Core Week 2 n-8 | 217.0 ± 100.69 |
| Core Week 4 n=59 | 242.1 ± 203.47 |
| Core Week 8 n=58 | 230.0 ± 195.21 |
| Core Week 13 n=51 | 231.0 ± 240.98 |
| Core Week 17 n=57 | 310.3 ± 429.64 |
| Core Week 22 n=50 | 214.0 ± 202.80 |
| Core Week 26 n=54 | 211.6 ± 173.58 |
| Core Week 31 n=46 | 154.3 ± 104.16 |
| Core Week 35 n=45 | 184.8 ± 140.13 |
| Ext Week 43 n=22 | 136.1 ± 91.13 |
| Ext Week 51 n=20 | 156.8 ± 108.91 |
| Ext Week 59 n=24 | 157.7 ± 111.63 |
| Ext Week 67 n=17 | 213.8 ± 194.99 |
| Ext Week 75 n=18 | 157.6 ± 166.28 |
| Ext Week 83 n=20 | 157.9 ± 134.98 |
| Ext Week 91 n=18 | 180.0 ± 302.36 |
| Ext Week 99 n=13 | 222.5 ± 375.67 |
| Ext Week 107 n=12 | 118.5 ± 122.01 |
| Ext Week 115 n=13 | 126.0 ± 80.09 |
| Ext Week 123 n=13 | 147.5 ± 157.77 |
| Ext Week 131 n=11 | 76.4 ± 49.92 |
| Ext Week 139 n=9 | 92.9 ± 73.18 |
| Ext Week 147 n=9 | 90.1 ± 55.31 |
| Ext Week 155 n=4 | 76.3 ± 39.05 |
| Ext Week 163 n=6 | 141.1 ± 142.42 |
| Ext Week 171 n=6 | 89.5 ± 61.87 |
| Ext Week 179 n=4 | 61.3 ± 43.06 |
| Ext Week 187 n=4 | 89.9 ± 52.44 |
| Ext Week 195 n=3 | 46.1 ± 40.21 |
| Ext Week 203 n=4 | 194.0 ± 259.15 |
| Ext Week 211 n=3 | 107.3 ± 37.68 |
| Ext Week 219 n=2 | 70.6 ± 46.74 |
| Ext Week 227 n=2 | 47.1 ± 7.99 |
| Ext Week 235 n=3 | 62.0 ± 23.83 |
| Ext Week 243 n=1 | 117.7 |
| Ext Week 251 n=1 | 202.9 |
| Ext Week 267 n=1 | 249.0 |
Actual mean value of mUFC at each scheduled visit was determined from two 24-hour urine collections collected on two consecutive days that occurred before the visit when UFC was measured.
| percentage of responders | All Patients |
|---|---|
| Baseline | 4.4 (0.9 to 12.4) |
| Core Week 17 n=57 | 28.1 (17.0 to 41.5) |
| Core Week 35 n=45 | 48.9 (33.7 to 64.2) |
| Ext Week 43 n=22 | 63.6 (40.7 to 82.8) |
| Ext Week 67 n=17 | 47.1 (23.0 to 72.2) |
| Ext Week 91 n=18 | 61.1 (35.7 to 82.7) |
| Ext Week 115 n=13 | 76.9 (46.2 to 95.0) |
| Ext Week 139 n=9 | 77.8 (40.0 to 97.2) |
| Ext Week 163 n=6 | 66.7 (22.3 to 95.7) |
| Ext Week 187 n=4 | 75.0 (19.4 to 99.4) |
| Ext Week 211 n=3 | 66.7 (9.4 to 99.2) |
| Ext Week 235 n=3 | 100 (29.2 to 100.0) |
Actual mean value of mUFC at each scheduled visit was determined from two 24-hour urine collections collected on two consecutive days that occurred before the visit when UFC was measured.
| percentage of responders | All Patients |
|---|---|
| Baseline | 4.4 (0.9 to 12.4) |
| Core Week 17 n=57 | 54.4 (40.7 to 67.6) |
| Core Week 35 n=45 | 68.9 (53.4 to 81.8) |
| Ext Week 43 n=22 | 86.4 (65.1 to 97.1) |
| Ext Week 67 n=17 | 76.5 (50.1 to 93.2) |
| Ext Week 91 n=18 | 83.3 (58.6 to 96.4) |
| Ext Week 115 n=13 | 92.3 (64.0 to 99.8) |
| Ext Week 139 n=9 | 77.8 (40.0 to 97.2) |
| Ext Week 163 n=6 | 83.3 (35.9 to 99.6) |
| Ext Week 187 n=4 | 100 (39.8 to 100.0) |
| Ext Week 211 n=3 | 66.7 (9.4 to 99.2) |
| Ext Week 235 n=3 | 100 (29.2 to 100.0) |
Duration of controlled or partially controlled response is defined as the period starting from the date of patient's first normalization (mUFC ≤ 1.0 x ULN) or at least 50% reduction from baseline up to the date when the patient's mUFC \> 1.0 x ULN and the reduction from baseline falls to less than 50% from the first time
| weeks | All Patients |
|---|---|
| Core n=36 | 13.1 (9.3 to 22.4) |
| Extension n=20 | 22.0 (8.1 to 70.1) |
A pre-dose blood draw for plasma ACTH sampling was taken at visits. Samples were analyzed by a central laboratory.
| pmol/L | All Patients |
|---|---|
| Baseline | 16.3 ± 16.3 |
| Core Week 2 n-62 | 12.4 ± 9.91 |
| Core Week 4 n=63 | 14.2 ± 12.50 |
| Core Week 8 n=61 | 13.3 ± 11.90 |
| Core Week 13 n=53 | 11.9 ± 10.98 |
| Core Week 17 n=58 | 12.3 ± 9.03 |
| Core Week 22 n=49 | 13.7 ± 13.82 |
| Core Week 26 n=55 | 12.4 ± 12.09 |
| Core Week 31 n=49 | 12.1 ± 11.02 |
| Core Week 35 n=40 | 11.0 ± 8.71 |
| Ext Week 43 n=23 | 11.5 ± 8.12 |
| Ext Week 51 n=21 | 10.4 ± 6.80 |
| Ext Week 59 n=23 | 10.7 ± 6.89 |
| Ext Week 67 n=22 | 11.0 ± 7.23 |
| Ext Week 75 n=22 | 9.1 ± 4.16 |
| Ext Week 83 n=19 | 9.8 ± 8.20 |
| Ext Week 91 n=19 | 10.6 ± 8.34 |
| Ext Week 99 n=16 | 11.3 ± 8.50 |
| Ext Week 107 n=13 | 9.3 ± 6.61 |
| Ext Week 115 n=15 | 9.5 ± 7.22 |
| Ext Week 123 n=14 | 10.6 ± 8.24 |
| Ext Week 131 n=12 | 8.2 ± 6.01 |
| Ext Week 139 n=10 | 10.0 ± 7.01 |
| Ext Week 147 n=9 | 10.4 ± 7.02 |
| Ext Week 155 n=9 | 10.4 ± 7.02 |
| Ext Week 163 n=5 | 10.0 ± 4.85 |
| Ext Week 171 n=5 | 7.4 ± 3.36 |
| Ext Week 179 n=5 | 7.0 ± 3.32 |
| Ext Week 187 n=5 | 8.6 ± 5.32 |
| Ext Week 195 n=4 | 9.0 ± 4.76 |
| Ext Week 203 n=4 | 8.0 ± 2.94 |
| Ext Week 211 n=3 | 11.0 ± 4.58 |
| Ext Week 219 n=2 | 6.0 ± 0.00 |
| Ext Week 227 n=2 | 7.0 ± 1.41 |
| Ext Week 235 n=3 | 10.3 ± 3.21 |
| Ext Week 243 n=1 | 6.0 |
| Ext Week 251 n=1 | 7.0 |
| Ext Week 267 n=1 | 4.0 |
A pre-dose blood draw for an 8 am fasting serum cortisol measurement were taken at visits. Samples were analyzed by a central laboratory.
| nmol/L | All Patients |
|---|---|
| Baseline | 738.6 ± 332.53 |
| Core Week 2 n-62 | 626.1 ± 281.57 |
| Core Week 4 n=64 | 663.6 ± 292.38 |
| Core Week 8 n=61 | 649.0 ± 297.11 |
| Core Week 13 n=53 | 628.8 ± 269.43 |
| Core Week 17 n=58 | 683.0 ± 282.28 |
| Core Week 22 n=49 | 625.4 ± 216.29 |
| Core Week 26 n=55 | 632.7 ± 278.75 |
| Core Week 31 n=49 | 597.5 ± 247.60 |
| Core Week 35 n=40 | 538.2 ± 205.40 |
| Ext Week 43 n=23 | 525.5 ± 178.63 |
| Ext Week 51 n=21 | 512.2 ± 243.59 |
| Ext Week 59 n=21 | 547.5 ± 193.67 |
| Ext Week 67 n=22 | 495.4 ± 213.03 |
| Ext Week 75 n=21 | 458.5 ± 170.88 |
| Ext Week 83 n=19 | 419.5 ± 141.26 |
| Ext Week 91 n=19 | 501.2 ± 235.89 |
| Ext Week 99 n=16 | 470.9 ± 248.0 |
| Ext Week 107 n=13 | 463.4 ± 189.27 |
| Ext Week 115 n=15 | 501.7 ± 212.27 |
| Ext Week 123 n=14 | 441.6 ± 150.98 |
| Ext Week 131 n=12 | 413.8 ± 325.5 |
| Ext Week 139 n=10 | 404.0 ± 90.87 |
| Ext Week 147 n=10 | 585.4 ± 216.92 |
| Ext Week 155 n=8 | 472.5 ± 122.96 |
| Ext Week 163 n=6 | 617.0 ± 212.11 |
| Ext Week 171 n=5 | 648.6 ± 243.79 |
| Ext Week 179 n=5 | 599.0 ± 388.78 |
| Ext Week 187 n=5 | 609.8 ± 292.85 |
| Ext Week 195 n=4 | 418.8 ± 138.69 |
| Ext Week 203 n=4 | 514.0 ± 212.27 |
| Ext Week 211 n=3 | 551.3 ± 339.87 |
| Ext Week 219 n=2 | 482.0 ± 25.46 |
| Ext Week 227 n=2 | 324.5 ± 177.48 |
| Ext Week 235 n=3 | 384.7 ± 165.17 |
| Ext Week 243 n=1 | 679.0 |
| Ext Week 251 n=1 | 574.0 |
| Ext Week 267 n=1 | 638.0 |
The mean arterial blood pressure determinations were obtained in the sitting position. Three measurements were taken with 5 minute intervals and the mean was used for study specific procedures
| mmHg | All Patients |
|---|---|
| Baseline | 125.9 ± 14.3 |
| Week 35 n=41 | 119.5 ± 18.6 |
The mean arterial blood pressure determinations were obtained in the sitting position. Three measurements were taken with 5 minute intervals and the mean was used for study specific procedures
| mmHg | All Patients |
|---|---|
| Baseline | 81.8 ± 9.12 |
| Week 35 n=41 | 77.2 ± 12.42 |
Weight was was one of the measures of clinical symptoms of CD
| kg | All Patients |
|---|---|
| Baseline | 82.2 ± 19.1 |
| Week 35 n=41 | 75.6 ± 20.4 |
Body mass index was one of the measurements of clinical symptoms of CD. Body mass index=weight in kg divided by the square of the body height (in meters)
| kg/m2 | All Patients |
|---|---|
| Baseline | 31.3 ± 6.6 |
| Week 35 n=41 | 28.4 ± 6.8 |
Waist circumference was one of the measurements of clinical signs of CD
| cm | All Patients |
|---|---|
| Baseline | 104.1 ± 19.1 |
| Week 35 n=41 | 99.1 ± 18.2 |
LDL=Low density lipoprotein, HDL=high density llipoprotein and total protein were analyzed by a central laboratory
| mmol/L | All Patients |
|---|---|
| HDL Baseline | 1.5 ± 0.3 |
| HDL Week 35 n=41 | 1.5 ± 0.4 |
| LDL Baseline | 3.2 ± 1.0 |
| LDL Week 35 n=41 | 2.8 ± 1.0 |
| Total Baseline | 69.4 ± 3.7 |
| Total Week 35 n=41 | 68.6 ± 4.8 |
Patients who completed nine or more items for the 12-item Cushing's syndrome QoL questionaire were considered evaluable for that assessment. Raw scores were obtained for the 12 items using the following scoring: 1) always or very much, 2) often or quite a bit, 3) sometimes or somewhat, 4) rarely or very little, 5) never or not at all. Then the standardized score, Y, was calculated for each patient as follows: Y = 100\* (X - n) / n\*5 - n\*1) = 100 \* (X - n) / 4\*n where X denoted the raw score and n the number of questions answered by the patient. The higher the score, the better the QoL. The best possible standardized score was 100 and the worst possible standardized score was 0
| scores on a scale | All Patients |
|---|---|
| Baseline Mean | 41.6 ± 20.2 |
| Week 17 n=54 | 46.3 ± 19.7 |
| Week 35 n=40 | 47.6 ± 20.8 |
SF-12v2 General Health Survey is a general patient reported outcome instrument over time. It is scored to provide eight health domain scores (Bodily Pain (BP), General Health (GH), Physical Functioning (PF), Role-Physical (RP), Social Functioning (SF), Role-Emotional (RE), Vitality (VT) and Mental Health (MH)). These eight domain scores can be combined to form two summary scores reflecting overall physical and mental health: the Physical Component Summary (PCS) and the Mental Component Summary (MCS). The analyses reported here focus on PCS and MCS scores. The domain scores use a norm-based score, which standardizes the scores with respect to the mean and standard deviation of a nationally representative sample of United States (US) adults. These are the scores on the original scale which have not been transformed in any way. The possible range of scores is 0 to 100, with higher scores representing better outcomes.
| scores on a scale | All Patients |
|---|---|
| Baseline Bodily pain scale | 42.9 ± 11.93 |
| Week 17 n=52 Bodily pain scale | 45.1 ± 10.75 |
| Week 35 n=40 Bodily pain scale | 44.9 ± 10.79 |
| Baseline n=67 General health scale: | 38.9 ± 9.23 |
| Week 17 n=52 General health scale: | 40.9 ± 8.52 |
| Week 35 n=40 General health scale: | 40.7 ± 8.94 |
| Baseline n=67 Mental component | 42.4 ± 10.17 |
| Week 17 n=52 Mental component | 41.9 ± 9.68 |
| Week 35 n=40 Mental component | 42.1 ± 8.32 |
| Baseline n=67 Mental health | 41.9 ± 10.48 |
| Week 17 n=52 Mental health | 43.0 ± 9.54 |
| Week 35 n=40 Mental health | 42.4 ± 8.44 |
| Baseline n=67 Physical component summary | 42.7 ± 9.03 |
| Week 17 n=52 Physical component summary | 44.0 ± 8.52 |
| Week 35 n=40 Physical component summary | 43.4 ± 9.59 |
| Baseline n=67 Physical functioning | 42.1 ± 10.17 |
| Week 17 n=52 Physical functioning | 41.9 ± 8.93 |
| Week 35 n=40 Physical functioning | 42.1 ± 11.10 |
| Baseline n=67 Role emotional | 39.7 ± 11.67 |
| Week 17 n=52 Role emotional | 38.0 ± 9.95 |
| Week 35 n=40 Role emotional | 38.7 ± 10.70 |
| Baseline n=67 Role physical scale: | 42.3 ± 10.45 |
| Week 17 n=52 Role physical scale: | 42.8 ± 8.73 |
| Week 35 n=40 Role physical scale: | 41.3 ± 9.63 |
| Baseline n=67 Social functioning | 42.2 ± 11.22 |
| Week 17 n=52 Social functioning | 43.0 ± 8.70 |
| Week 35 n=40 Social functioning | 43.3 ± 9.66 |
| Baseline n=67 Vitality scale: | 47.2 ± 10.02 |
| Week 17 n=52 Vitality scale: | 45.7 ± 10.29 |
| Week 35 n=40 Vitality scale: | 45.6 ± 8.78 |
Clinical symptoms include: facial rubor, hirsutism, striae, and supraclavicular and dorsal fat pad. Two photographs, one frontal and one lateral from the shoulders up were taken to assess facial plethora, supraclavicular and dorsal fat pads. Two photographs, frontal and dorsal of the trunk with patient in standing position were taken to assess striae, and hirsutism. The photographs were assessed by a qualified physician at the site. Improvement=decrease in severity of symptom since baseline
| participants | All Patients |
|---|---|
| Core Week 1 n=40 | 5 |
| Core Week 2 n=37 | 8 |
| Core Week 4 n=38 | 8 |
| Core Week 8 n=37 | 12 |
| Core Week 13 n=32 | 11 |
| Core Week 17 n=32 | 11 |
| Core Week 22 n=28 | 10 |
| Core Week 26 n=31 | 14 |
| Core Week 31 n=32 | 15 |
| Core Week 35 n=26 | 13 |
| Ext Week 43 n=11 | 2 |
| Ext Week 59 n=11 | 3 |
| Ext Week 75 n=12 | 3 |
| Ext Week 91 n=10 | 3 |
| Ext Week 107 n=9 | 3 |
| Ext Week 123 n=10 | 2 |
| Ext Week 139 n=7 | 2 |
| Ext Week 155 n=4 | 0 |
| Ext Week 171 n=3 | 0 |
| Ext Week 187 n=3 | 0 |
| Ext Week 203 n=4 | 0 |
| Ext Week 219 n=2 | 0 |
| Ext Week 235 n=3 | 0 |
| Ext Week 251 n=1 | 0 |
| Ext Week 267 n=1 | 0 |
Clinical symptoms include: facial rubor, hirsutism, striae, and supraclavicular and dorsal fat pad. Two photographs, one frontal and one lateral from the shoulders up were taken to assess facial plethora, supraclavicular and dorsal fat pads. Two photographs, frontal and dorsal of the trunk with patient in standing position were taken to assess striae, and hirsutism. The photographs were assessed by a qualified physician at the site. Improvement=decrease in severity of symptom since baseline
| participants | All Patients |
|---|---|
| Core Week 1 n=35 | 5 |
| Core Week 2 n-33 | 3 |
| Core Week 4 n=32 | 4 |
| Core Week 8 n=32 | 4 |
| Core Week 13 n=27 | 4 |
| Core Week 17 n=28 | 3 |
| Core Week 22 n=25 | 5 |
| Core Week 26 n=27 | 8 |
| Core Week 31 n=28 | 9 |
| Core Week 35 n=23 | 7 |
| Ext Week 43 n=10 | 2 |
| Ext Week 59 n=9 | 2 |
| Ext Week 75 n=10 | 3 |
| Ext Week 91 n=9 | 2 |
| Ext Week 107 n=8 | 1 |
| Ext Week 123 n=9 | 2 |
| Ext Week 139 n=7 | 2 |
| Ext Week 155 n=4 | 2 |
| Ext Week 171 n=4 | 2 |
| Ext Week 187 n=3 | 2 |
| Ext Week 203 n=4 | 2 |
| Ext Week 219 n=2 | 1 |
| Ext Week 235 n=3 | 1 |
| Ext Week 251 n=1 | 0 |
| Ext Week 267 n=1 | 0 |
Clinical symptoms include: facial rubor, hirsutism, striae, and supraclavicular and dorsal fat pad. Two photographs, one frontal and one lateral from the shoulders up were taken to assess facial plethora, supraclavicular and dorsal fat pads. Two photographs, frontal and dorsal of the trunk with patient in standing position were taken to assess striae, and hirsutism. The photographs were assessed by a qualified physician at the site. Improvement=decrease in severity of symptom since baseline
| participants | All Patients |
|---|---|
| Core Week 1 n=40 | 5 |
| Core Week 2 n-37 | 2 |
| Core Week 4 n=38 | 5 |
| Core Week 8 n=37 | 8 |
| Core Week 13 n=32 | 7 |
| Core Week 17 n=32 | 7 |
| Core Week 22 n=28 | 7 |
| Core Week 26 n=31 | 9 |
| Core Week 31 n=32 | 12 |
| Core Week 35 n=26 | 8 |
| Ext Week 43 n11= | 1 |
| Ext Week 59 n=11 | 1 |
| Ext Week 75 n=12 | 1 |
| Ext Week 91 n=10 | 1 |
| Ext Week 107 n=9 | 0 |
| Ext Week 123 n=10 | 0 |
| Ext Week 139 n=7 | 0 |
| Ext Week 155 n=4 | 1 |
| Ext Week 171 n=3 | 0 |
| Ext Week 187 n=3 | 0 |
| Ext Week 203 n=4 | 1 |
| Ext Week 219 n=2 | 0 |
| Ext Week 235 n=3 | 1 |
| Ext Week 251 n=1 | 0 |
| Ext Week 267 n=1 | 1 |
Clinical symptoms include: facial rubor, hirsutism, striae, and supraclavicular and dorsal fat pad. Two photographs, one frontal and one lateral from the shoulders up were taken to assess facial plethora, supraclavicular and dorsal fat pads. Two photographs, frontal and dorsal of the trunk with patient in standing position were taken to assess striae, and hirsutism. The photographs were assessed by a qualified physician at the site. Improvement=decrease in severity of symptom since baseline
| participants | All Patients |
|---|---|
| Core Week 1 n=40 | 6 |
| Core Week 2 n-37 | 7 |
| Core Week 4 n=37 | 6 |
| Core Week 8 n=37 | 8 |
| Core Week 13 n=32 | 8 |
| Core Week 17 n=32 | 9 |
| Core Week 22 n=28 | 9 |
| Core Week 26 n=31 | 10 |
| Core Week 31 n=32 | 12 |
| Core Week 35 n=26 | 11 |
| Ext Week 43 n=11 | 1 |
| Ext Week 59 n=11 | 0 |
| Ext Week 75 n=12 | 1 |
| Ext Week 91 n=10 | 1 |
| Ext Week 107 n=9 | 0 |
| Ext Week 123 n=10 | 1 |
| Ext Week 139 n=7 | 1 |
| Ext Week 155 n=4 | 1 |
| Ext Week 171 n=3 | 1 |
| Ext Week 187 n=3 | 1 |
| Ext Week 203 n=4 | 1 |
| Ext Week 219 n=2 | 1 |
| Ext Week 235 n=3 | 2 |
| Ext Week 251 n=1 | 0 |
| Ext Week 267 n=1 | 0 |
Clinical symptoms include: facial rubor, hirsutism, striae, and supraclavicular and dorsal fat pad. Two photographs, one frontal and one lateral from the shoulders up were taken to assess facial plethora, supraclavicular and dorsal fat pads. Two photographs, frontal and dorsal of the trunk with patient in standing position were taken to assess striae, and hirsutism. The photographs were assessed by a qualified physician at the site. Improvement=decrease in severity of symptom since baseline
| participants | All Patients |
|---|---|
| Core Week 1 n=40 | 6 |
| Core Week 2 n-37 | 4 |
| Core Week 4 n=37 | 5 |
| Core Week 8 n=37 | 5 |
| Core Week 13 n=32 | 4 |
| Core Week 17 n=32 | 8 |
| Core Week 22 n=28 | 8 |
| Core Week 26 n=31 | 12 |
| Core Week 31 n=32 | 13 |
| Core Week 35 n=26 | 9 |
| Ext Week 43 n=11 | 3 |
| Ext Week 59 n=10 | 4 |
| Ext Week 75 n=12 | 5 |
| Ext Week 91 n=10 | 3 |
| Ext Week 107 n=9 | 2 |
| Ext Week 123 n=10 | 3 |
| Ext Week 139 n=7 | 3 |
| Ext Week 155 n=4 | 1 |
| Ext Week 171 n=3 | 1 |
| Ext Week 187 n=3 | 2 |
| Ext Week 203 n=4 | 1 |
| Ext Week 219 n=2 | 1 |
| Ext Week 235 n=3 | 2 |
| Ext Week 251 n=1 | 0 |
| Ext Week 267 n=1 | 0 |
Facial rubor, hirsutism, striae, and supraclavicular and dorsal fat pad were assessed. Two photographs, one frontal and one lateral from the shoulders up will be taken to assess facial plethora, supraclavicular and dorsal fat pads. Two photographs, frontal and dorsal of the trunk with patient in standing position will be taken to assess striae, and hirsutism. The photographs must be assessed by a qualified physician at the site. Improvement=decrease in severity of symptom since baseline
| participants | All Patients |
|---|---|
| Core Week 4 Facial rubor baseline n=17 | 1 |
| Core Week 35 Facial rubor baseline n=17 | 1 |
| Ext Week 43 Facial rubor baseline n=17 | 1 |
| Ext Week 59 Facial rubor baseline n=17 | 1 |
| Ext Week 67 Facial rubor baseline n=17 | 1 |
| Ext Week 83 Facial rubor baseline n=17 | 1 |
| Ext Week 91 Facial rubor baseline n=17 | 1 |
| Ext Week 99 Facial rubor baseline n=17 | 1 |
| Ext Week 107 Facial rubor baseline n=17 | 1 |
| Ext Week 115 Facial rubor baseline n=17 | 1 |
| Ext Week 123 Facial rubor baseline n=17 | 1 |
| Core Week 1 Hirsutism baseline n=12 | 1 |
| Core Week 2 Hirsutism baseline n=12 | 1 |
| Ext Week 59 Striae baseline n=10 | 1 |
| Ext Week 99 Striae baseline n=10 | 1 |
| Ext Week 107 Striae baseline n=10 | 1 |
| Ext Week 115 Striae baseline n=10 | 1 |
| Ext Week 123 Striae baseline n=10 | 1 |
| Ext Week 99 Supraclavicular fat pad BL n=17 | 1 |
| Ext Week 107 Supraclavicular fat pad BL n=17 | 1 |
To test proximal muscle strength patients should be placed in a low seated position (for instance on an examination room stool). They should be asked to extend the arms in front of them. From this seated position patients will be asked to stand up. Patients will be evaluated using the following scale: 3. - completely unable to stand 2. - able to stand only by using arms as assistance 1. - able to stand after several efforts without using arms as assistance 0. - able to stand easily with arms extended
| participants | All Patients |
|---|---|
| Core Week 26 | 1 |
| Core Week 35 | 1 |
Collected over Adverse events were collected from first dose of study treatment until end of study treatment plus 28 days post treatment, up to maximum duration of 272 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| All Patients | 3/68 (4.4%) | 15/68 (22.1%) | 67/68 (98.5%) |
| Event | All Patients |
|---|---|
| Abdominal painGastrointestinal disorders | 2/68 |
| CholelithiasisHepatobiliary disorders | 2/68 |
| AnaemiaBlood and lymphatic system disorders | 1/68 |
| Coronary artery diseaseCardiac disorders | 1/68 |
| Glucocorticoid deficiencyEndocrine disorders | 1/68 |
| Intra-abdominal haematomaGastrointestinal disorders | 1/68 |
| DeathGeneral disorders | 1/68 |
| Multiple organ dysfunction syndromeGeneral disorders | 1/68 |
| Bile duct stoneHepatobiliary disorders | 1/68 |
| CholecystitisHepatobiliary disorders | 1/68 |
| Event | All Patients |
|---|---|
| NauseaGastrointestinal disorders | 35/68 |
| HyperglycaemiaMetabolism and nutrition disorders | 35/68 |
| DiarrhoeaGastrointestinal disorders | 30/68 |
| CholelithiasisHepatobiliary disorders | 22/68 |
| HeadacheNervous system disorders | 20/68 |
| DizzinessNervous system disorders | 19/68 |
| FatigueGeneral disorders | 13/68 |
| Back painMusculoskeletal and connective tissue disorders | 13/68 |
| Diabetes mellitusMetabolism and nutrition disorders | 12/68 |
| Abdominal painGastrointestinal disorders | 11/68 |
| Age, Customized(participants) | All Patients |
|---|---|
| < 65 years | 64 |
| ≥ 65 years | 4 |
| Sex: Female, Male(Participants) | All Patients |
|---|---|
| Female | 60 |
| Male | 8 |
| Race/Ethnicity, Customized(Participants) | All Patients |
|---|---|
| Caucasian | 44 |
| Asian | 9 |
| Native American | 5 |
| Black | 2 |
| Other | 8 |
| Cushing's disease status(Participants) | All Patients |
|---|---|
| De novo | 10 |
| Persistent/recurrent | 58 |
| Clinical symptoms of facial rubor(Participants) | All Patients |
|---|---|
| None | 10 |
| Mild | 17 |
| Moderate | 11 |
| Severe | 4 |
| Not done | 26 |
| Clinical symptoms of hirsutism(Participants) | All Patients |
|---|---|
| None | 13 |
| Mild | 12 |
| Moderate | 7 |
| Severe | 3 |
| Not done | 33 |
| Clinical symptoms of striae(Participants) | All Patients |
|---|---|
| None | 20 |
| Mild | 10 |
| Moderate | 4 |
| Severe | 8 |
| Not done | 26 |
| Clinical symptoms of supraclavicular fat pad(Participants) | All Patients |
|---|---|
| None | 8 |
| Mild | 17 |
| Moderate | 13 |
| Severe | 4 |
| Not done | 26 |
1 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Ran from Mar 2014 to primary completion in Sep 2016, 29 months.
Results were first posted in Sep 2020, 4 years after primary completion.
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This study is terminated, as verified in Aug 2020. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Pasireotide · Cabergoline
Adrenocortical Hyperfunction →