A Phase 3 interventional study of Pasireotide sub-cutaneous in Cushing's Disease, sponsored by Novartis Pharmaceuticals. Completed at 65 sites in 11 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2018-06-19.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 3, Interventional, and Treatment
Among 37 studies in Pituitary ACTH Hypersecretion with a site in United States — 8 open to participants now, 10 under way, none of them phase 3. Pituitary ACTH Hypersecretion · United States.
Among the 37 studies in Pituitary ACTH Hypersecretion with a site in United States, this one is the 7th largest by enrollment of the 29 interventional ones, is one of 4 that started in 2011, is one of 11 in phase 3, and was the 14th registered.
64 studies on the registry are indexed under Pituitary ACTH Hypersecretion; 16 are open to participants now.
This study's enrollment of 104 is above the median of 44 across 40 interventional studies indexed under Pituitary ACTH Hypersecretion.
Browse Pituitary ACTH Hypersecretion studies →8 of the 37 studies in Pituitary ACTH Hypersecretion with a site in United States are open now, in 1 country. Each count lists the studies.
Of the 12 Pituitary ACTH Hypersecretion studies recruiting now, 7 (58%) set no upper age limit and 2 (17%) stop at 75 or younger; 6 take children under 18; 5 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →37 studies; the count open now beside each. This study's row is marked.
First in 1997; this study’s year is marked. 4 started in 2011.
Median enrollment 31 across the 29 interventional studies, 196 across the 8 observational. Of the 24 that completed or were terminated, 18 (75%) have results posted.
23 of 37 have an industry lead sponsor. The 10 most frequent:
Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
Novartis Pharmaceuticals has 8 studies filed under Pituitary ACTH Hypersecretion — fewer than ten, so there is no comparison to draw.
4 of the studies in Pituitary ACTH Hypersecretion with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT03364803Memorial Sloan Kettering Cancer CenterCollecting Information About Treatment Results for Patients With Cushing's Syndrome | 2026-11-28 |
| NCT03604198Corcept TherapeuticsExtension Study to Evaluate the Safety of Long-Term Use of Relacorilant in Patients With Cushing Syndrome | 2026-12 |
| NCT07335315Mayo ClinicEvaluation of Intraoperative Contrast Enhanced Ultrasound for the Identification of Pituitary Adenoma in Cushing's Disease Compared to Other Pituitary Tumors | 2027-01 |
| NCT06471829H. Lundbeck A/SA Trial of Lu AG13909 in Adult Participants With Cushing's Disease | 2027-08-10 |
Among the 37 studies in Pituitary ACTH Hypersecretion with a site in United States, from their registry updates:
This study provided access to pasireotide sc in patients with Cushing's disease.and provided additional information for safety and efficacy of pasireotide s.c.
Purpose of this study was to give access to pasireotide sc for patients with Cushing's disease as no medical treatment for Cushing's disease was approved at the time of the study initiation. The study population consisted of patients with persistent or recurrent Cushing's disease or patients with de novo Cushing's disease that were not considered candidates for pituitary surgery (poor surgery candidates, surgically unapproachable tumor, patients with no visible pituitary tumor, patients who refused surgery). A confirmed Cushing's disease diagnosis was required.
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.
For patients on previous medical treatment for Cushing's disease the following must be completed before screening assessments are performed
Exclusion criteria:
Patients who have clinically significant impairment in cardiovascular function or are at risk thereof, as evidenced by congestive heart failure ( Class III or IV), unstable angina, sustained ventricular tachycardia, clinically significant bradycardia, high grade AV block, history of acute MI less than one year prior to study entry
Patients who have any current or prior medical condition that can interfere with the conduct of the study or the evaluation of its results, such as
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.
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.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
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.
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.
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.
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.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
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.
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.
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 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.
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 drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
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.
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.
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.
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.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
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.
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.
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.
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.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
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.
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.
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.
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.
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.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
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.
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.
Pasireotide sub-cutaneous was supplied in 1 ml ampoules containing 600 μg pasireotide per 1 ml of solution and was administered BID. Starting dose was 600 μg for glucose impaired metabolism patients. Mean daily dose category is defined on the mean daily dose considering the following grouping rule: 600 μg bid group includes all patients whose mean daily dose \< 1500 μg /day.
Drug: Pasireotide sub-cutaneous
Pasireotide sub-cutaneous was supplied in 1 ml ampoules containing 900 μg pasireotide per 1 ml of solution and was administered BID. Starting dose was 900 μg. Mean daily dose category is defined on the mean daily dose considering the following grouping rule: 900 μg bid group includes all patients whose mean daily dose ≥ 1500 μg /day
Drug: Pasireotide sub-cutaneous
Pasireotide sub-cutaneous was supplied in 1 ml ampoules containing 900 μg, 600 μg, or 300 μg pasireotide per 1 ml of solution and was administered BID. Starting dose was 900 μg and 600 μg for glucose impaired metabolism patients
Also known as: SOM230 sub-cutaneous
Percentage of Patients With a Drug-related Adverse Event That is Recorded as Grade 3 or 4 or as a Serious Adverse Event (SAE)
Only AEs occurring on or after the start of study treatment and no more than 28 days after the discontinuation of study treatment. A patient with multiple occurrences of an AE under one treatment is counted only once in the AE category for that treatment. A patient with multiple severity grades for an AE while on a treatment, is only counted under the maximum grade.
Time frame: Baseline up to approximately 256 weeks
Percentage of Patients With Mean Urinary Free Cortisol (UFC) ≤ Upper Limit of Normal (ULN)
The 24h-UFC concentration results from three samples during screening were averaged to obtain baseline. After baseline, mean 24h UFC was determined at week 24. At Week 4, 8, 16 and 20, mean 24h UFC was determined from two 24 hour urine collections collected on two consecutive days occurring before the visit. At Week 12, 24 and 48, the mean 24h-UFC from three 24 hour urine collections, collected over the week before the visit, was determined. After Week 24, the mean 24h UFC was determined at 12-week intervals until end of study visit, from two 24 hour collections during two consecutive days prior to each respective visit (except at Week 48). UFC was determined by liquid chromatography tandem mass spectroscopy (LC/MS/MS). The normal ranges were determined by the central laboratory's own reference range. All samples, including screening samples, were analyzed by a central laboratory.
Time frame: Baseline, week 12, 24 and 48
Percentage of Patients Achieving a Reduction of Mean UFC ≥ 50% From Baseline
The 24h-UFC concentration results from three samples during screening were averaged to obtain baseline. After baseline, mean 24h UFC was determined at week 24. At Week 4, 8, 16 and 20, mean 24h UFC was determined from two 24 hour urine collections collected on two consecutive days occurring before the visit. At Week 12, 24 and 48, the mean 24h-UFC from three 24 hour urine collections, collected over the week before the visit, was determined. After Week 24, the mean 24h UFC was determined at 12-week intervals until end of study visit, from two 24 hour collections during two consecutive days prior to each respective visit (except at Week 48). UFC was determined by liquid chromatography tandem mass spectroscopy (LC/MS/MS). The normal ranges were determined by the central laboratory's own reference range. All samples, including screening samples, were analyzed by a central laboratory.
Time frame: Baseline, week 12, 24 and 48
Percent Change in Cushing Quality of Life and Work Productivity and Activity Impairment-General Health (WPAI-GH) Scores
A 12-item Cushing's syndrome HRQoL questionnaire (CushingQoL, cf. Webb et al 2008) was implemented and patients who completed 9 or more items at a visit were considered evaluable for that visit. The standardized scores were calculated as follows: 1) Obtain raw scores, denoted by X, as the sum of all the ratings on all the HRQoL questions for a single patient and the score can range from 12 (worst HRQoL) to 60 points (best HRQoL). Therefore, the lower the score, greater the negative impact on HRQoL and 2) obtain standardized score, Y, for a single patient • Y = 100 (X-12) / (60-12) = 100 (X-12)/48. For example, if a patient answers all 12 items with 'Sometimes' or 'Somewhat', X = 36 and Y = 100 ∙ 24/48 = 50 The WPAI-GH questionnaire was used to assess work productivity and activity impairment. However, there was very limited baseline data and therefore the results and outcomes of the objective, 'change from baseline in WPAI-GH scores' are not included.
Time frame: Baseline, week 12, 24 and 48
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Blood Pressure (BP)
Standing systolic and diastolic BP based on 1 assessment and sitting systolic and diastolic BP was mean of 3 assessments.
Time frame: Baseline, week 12, 24 and 48
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Pulse
Change from baseline is shown as: Percent change from baseline (BL) =((Post BL value - BL value)/ BL value)\*100
Time frame: Baseline, week 12, 24 and 48
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Temperature
degrees celius
Time frame: Baseline week 12, 24 and 48
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Body Mass Index (BMI)
Percent change in patients reducing by at least one class level. Class levels: \<25.0, 25.0 to \<30.0, ≥ 30.0. Percent change from baseline (BL) =((Post BL value - BL value)/ BL value)\*100
Time frame: Baseline, week 12, 24 and 48
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Weight
Clinically relevant threshold (at any time point) was reduction of ≥ 5%
Time frame: Baseline, week 12, 24 and 48
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Muscle Strength
Direct observation of ability to stand unaided: 0=able to stand easily with arms extended, 1=able to stand after several efforts without using arms as assistance, 2=able to stand only by using arms as assistance 3=completely unable to stand
Time frame: Baseline, week 12, 24 and 48
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Waist Circumference
Clinically relevant threshold (at any time point). Reduction of ≥ 5%, Reduction of ≥ 10%
Time frame: Baseline, week 12, 24 and 48
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Hirsutism
Change from baseline is shown as: Percent change from baseline (BL) =((Post BL value - BL value)/ BL value)\*100. Ferriman-Gallway scoring was used: 0=minimum and 36 was maximum in females only.
Time frame: Baseline, week 12, 24 and 48
Percent Change From Baseline in Growth Hormone (GH) Values
Descriptive summary of the effect of pasireotide on GH.
Time frame: Baseline, week 12, 24 and 48
Percent Change From Baseline in Insulin Growth Factor - 1 (IGF - 1) Values
Descriptive summary of the effect of pasireotide on IGF-1
Time frame: Baseline, week 12, 24 and 48
| Milestone | Pasireotide 600 μg | Pasireotide 900 μg |
|---|---|---|
| Started | 49 | 55 |
| Completed | 21 | 19 |
| Not completed | 28 | 36 |
| Withdrew: Lack of efficacy | 3 | 23 |
| Withdrew: Adverse event | 12 | 8 |
| Withdrew: Withdrawal by subject | 10 | 4 |
| Withdrew: Abnormal laboratory value(s) | 1 | 0 |
| Withdrew: Death | 0 | 1 |
| Withdrew: Condition no longer requires study drug | 1 | 0 |
| Withdrew: Lost to follow-up | 1 | 0 |
Only AEs occurring on or after the start of study treatment and no more than 28 days after the discontinuation of study treatment. A patient with multiple occurrences of an AE under one treatment is counted only once in the AE category for that treatment. A patient with multiple severity grades for an AE while on a treatment, is only counted under the maximum grade.
| percentage of participants | 600 µg Bid - All Grades | 600 μg - Grades 3/4 | 900 μg - All Grades | 900 μg - Grades 3/4 | All Patients - All Grades | All Patients - Grades 3/4 |
|---|---|---|---|---|---|---|
| Any primary system organ class | 53.1 | 53.1 | 29.1 | 27.3 | 40.4 | 39.4 |
| Metabolism and nutrition disorders | 26.5 | 26.5 | 12.7 | 10.9 | 19.2 | 18.3 |
| Gastrointestinal disorders | 18.4 | 18.4 | 7.3 | 7.3 | 12.5 | 12.5 |
| Investigations | 8.2 | 8.2 | 3.6 | 3.6 | 5.8 | 5.8 |
| Hepatobiliary disorders | 2.0 | 2.0 | 5.5 | 5.5 | 3.8 | 3.8 |
| Endocrine disorders | 6.1 | 6.1 | 0 | 0 | 2.9 | 2.9 |
| Gen disorders,admin site conditions | 4.1 | 4.1 | 0 | 0 | 1.9 | 1.9 |
| Ear and labyrinth disorders | 2.0 | 2.0 | 0 | 0 | 1.0 | 1.0 |
| Infections and infestations | 2.0 | 2.0 | 0 | 0 | 1.0 | 1.0 |
| Musculoskeletal and connective tissue disorders | 2.0 | 2.0 | 0 | 0 | 1.0 | 1.0 |
| Neoplasms benign, malignant and unspecified | 2.0 | 2.0 | 0 | 0 | 1.0 | 1.0 |
| Nervous system disorders | 2.0 | 2.0 | 0 | 0 | 1.0 | 1.0 |
| Psychiatric disorders | 0 | 0 | 1.8 | 1.8 | 1.0 | 1.0 |
| Respiratory, thoracic, mediastinal disorders | 0 | 0 | 1.8 | 0 | 1.0 | 0 |
The 24h-UFC concentration results from three samples during screening were averaged to obtain baseline. After baseline, mean 24h UFC was determined at week 24. At Week 4, 8, 16 and 20, mean 24h UFC was determined from two 24 hour urine collections collected on two consecutive days occurring before the visit. At Week 12, 24 and 48, the mean 24h-UFC from three 24 hour urine collections, collected over the week before the visit, was determined. After Week 24, the mean 24h UFC was determined at 12-week intervals until end of study visit, from two 24 hour collections during two consecutive days prior to each respective visit (except at Week 48). UFC was determined by liquid chromatography tandem mass spectroscopy (LC/MS/MS). The normal ranges were determined by the central laboratory's own reference range. All samples, including screening samples, were analyzed by a central laboratory.
| percentage of participants | Pasireotide 600 μg | Pasireotide 900 μg | All Patients |
|---|---|---|---|
| Week 12 | 77.8 (57.74 to 91.38) | 38.5 (23.36 to 55.38) | 54.5 (41.81 to 66.86) |
| Week 24 | 68.2 (45.13 to 86.14) | 29.2 (12.62 to 51.09) | 47.8 (32.89 to 63.05) |
| Week 48 | 70.0 (34.75 to 93.33) | 18.2 (2.28 to 51.78) | 42.9 (21.82 to 65.98) |
| Week 24 (LOCF) | 54.1 (36.92 to 70.51) | 28.6 (16.59 to 43.26) | 39.5 (29.15 to 50.66) |
| Week 48 (LOCF) | 51.4 (34.40 to 68.08) | 22.4 (11.78 to 36.62) | 34.9 (24.92 to 45.92) |
The 24h-UFC concentration results from three samples during screening were averaged to obtain baseline. After baseline, mean 24h UFC was determined at week 24. At Week 4, 8, 16 and 20, mean 24h UFC was determined from two 24 hour urine collections collected on two consecutive days occurring before the visit. At Week 12, 24 and 48, the mean 24h-UFC from three 24 hour urine collections, collected over the week before the visit, was determined. After Week 24, the mean 24h UFC was determined at 12-week intervals until end of study visit, from two 24 hour collections during two consecutive days prior to each respective visit (except at Week 48). UFC was determined by liquid chromatography tandem mass spectroscopy (LC/MS/MS). The normal ranges were determined by the central laboratory's own reference range. All samples, including screening samples, were analyzed by a central laboratory.
| percentage of participants | Pasireotide 600 μg | Pasireotide 900 μg | All Patients |
|---|---|---|---|
| Week 12 | 74.1 (53.72 to 88.89) | 48.7 (32.42 to 65.22) | 59.1 (46.29 to 71.05) |
| Week 24 | 68.2 (45.13 to 86.14) | 33.3 (15.63 to 55.32) | 50.00 (34.90 to 65.10) |
| Week 48 | 60.0 (26.24 to 87.84) | 54.5 (23.38 to 83.25) | 57.1 (34.02 to 78.18) |
| Week 24 (LOCF) | 56.8 (39.49 to 72.90) | 38.8 (25.20 to 53.76) | 46.5 (35.68 to 57.59) |
| Week 48 (LOCF) | 51.4 (34.40 to 68.08) | 42.9 (28.82 to 57.79) | 46.5 (35.68 to 57.59) |
A 12-item Cushing's syndrome HRQoL questionnaire (CushingQoL, cf. Webb et al 2008) was implemented and patients who completed 9 or more items at a visit were considered evaluable for that visit. The standardized scores were calculated as follows: 1) Obtain raw scores, denoted by X, as the sum of all the ratings on all the HRQoL questions for a single patient and the score can range from 12 (worst HRQoL) to 60 points (best HRQoL). Therefore, the lower the score, greater the negative impact on HRQoL and 2) obtain standardized score, Y, for a single patient • Y = 100 (X-12) / (60-12) = 100 (X-12)/48. For example, if a patient answers all 12 items with 'Sometimes' or 'Somewhat', X = 36 and Y = 100 ∙ 24/48 = 50 The WPAI-GH questionnaire was used to assess work productivity and activity impairment. However, there was very limited baseline data and therefore the results and outcomes of the objective, 'change from baseline in WPAI-GH scores' are not included.
| percent change in score | Pasireotide 600 μg | Pasireotide 900 μg | All Patients |
|---|---|---|---|
| Week 12 | 21.3 ± 47.03 | 100.8 ± 252.25 | 67.1 ± 197.09 |
| Week 24 | 36.7 ± 59.25 | 119.7 ± 321.61 | 82.3 ± 243.19 |
| Week 48 | 24.0 ± 37.76 | 42.3 ± 60.75 | 34.4 ± 52.29 |
Standing systolic and diastolic BP based on 1 assessment and sitting systolic and diastolic BP was mean of 3 assessments.
| percent change of mmhg | Pasireotide 600 μg | Pasireotide 900 μg | All Patients |
|---|---|---|---|
| Sitting systolic Week 12 | -6.8 ± 12.04 | -1.4 ± 14.40 | -3.8 ± 13.61 |
| Sitting systolic Week 24 | -7.4 ± 8.97 | -5.7 ± 11.09 | -6.5 ± 10.13 |
| Sitting systolic Week 48 | -5.1 ± 8.57 | -4.7 ± 12.55 | -4.9 ± 10.87 |
| Standing systolic Week 12 | -7.9 ± 12.49 | -3.6 ± 15.38 | -5.5 ± 14.27 |
| Standing systolic Week 24 | -10.9 ± 10.30 | -6.7 ± 14.09 | -8.6 ± 12.62 |
| Standing systolic Week 48 | -6.5 ± 9.45 | -4.4 ± 13.86 | -5.3 ± 12.10 |
| Sitting diastolic Week 12 | -4.6 ± 15.13 | -0.2 ± 14.40 | -2.1 ± 14.79 |
| Sitting diastolic Week 24 | -6.2 ± 7.29 | -3.8 ± 15.83 | -4.9 ± 12.65 |
| Sitting diastolic Week 48 | -3.3 ± 10.38 | -4.3 ± 13.93 | -3.8 ± 12.37 |
| Standing diastolic Week 12 | -5.5 ± 16.02 | -1.9 ± 17.63 | -3.5 ± 16.93 |
| Standing diastolic Week 24 | -7.6 ± 10.30 | -5.3 ± 16.74 | -6.3 ± 14.18 |
| Standing diastolic Week 48 | -2.1 ± 13.78 | -4.4 ± 14.13 | -3.4 ± 13.83 |
Change from baseline is shown as: Percent change from baseline (BL) =((Post BL value - BL value)/ BL value)\*100
| percent change in bpm | Pasireotide 600 μg | Pasireotide 900 μg | All Patients |
|---|---|---|---|
| Sitting pulse Week 12 | 2.3 ± 18.93 | -7.5 ± 11.50 | -3.2 ± 15.87 |
| Sitting pulse Week 24 | -1.8 ± 17.05 | -2.6 ± 18.07 | -2.2 ± 17.46 |
| Sitting pulse Week 48 | 2.9 ± 16.39 | 3.7 ± 15.46 | 3.3 ± 15.65 |
degrees celius
| percent change in celius | 600 µg Bid - All Grades | 900 μg - All Grades | All Patients - All Grades |
|---|---|---|---|
| Week 12 | 0.1 ± 1.26 | -0.3 ± 1.06 | -0.1 ± 1.17 |
| Week 24 | -0.1 ± 1.15 | -0.1 ± 1.26 | -0.1 ± 1.20 |
| Week 48 | 0.03 ± 1.47 | -0.1 ± 1.19 | 0.1 ± 1.31 |
Percent change in patients reducing by at least one class level. Class levels: \<25.0, 25.0 to \<30.0, ≥ 30.0. Percent change from baseline (BL) =((Post BL value - BL value)/ BL value)\*100
| percent change in kg/m2 | Pasireotide 600 μg | Pasireotide 900 μg | All Patients |
|---|---|---|---|
| Week 12 | -4.2 ± 3.70 | -4.8 ± 5.38 | -4.5 ± 4.69 |
| Week 24 | -7.3 ± 5.46 | -5.2 ± 6.51 | -6.1 ± 6.10 |
| Week 48 | -8.0 ± 6.82 | -6.3 ± 7.88 | -7.0 ± 7.39 |
Clinically relevant threshold (at any time point) was reduction of ≥ 5%
| percent change in kg | Pasireotide 600 μg | Pasireotide 900 μg | All Patients |
|---|---|---|---|
| Week 12 | -4.2 ± 3.70 | -4.8 ± 5.38 | -4.5 ± 4.69 |
| Week 24 | -7.3 ± 5.46 | -5.2 ± 6.51 | -6.1 ± 6.10 |
| Week 48 | -8.0 ± 6.82 | -6.3 ± 7.88 | -7.0 ± 7.39 |
Direct observation of ability to stand unaided: 0=able to stand easily with arms extended, 1=able to stand after several efforts without using arms as assistance, 2=able to stand only by using arms as assistance 3=completely unable to stand
| percent change in scores | Pasireotide 600 μg | Pasireotide 900 μg | All Patients |
|---|---|---|---|
| Week 12 | -34.6 ± 62.53 | -53.7 ± 46.98 | -42.4 ± 56.28 |
| Week 24 | -28.6 ± 48.80 | -47.6 ± 50.40 | -38.1 ± 48.67 |
| Week 48 | -30.0 ± 44.72 | -75.0 ± 50.0 | -50.0 ± 50.00 |
Clinically relevant threshold (at any time point). Reduction of ≥ 5%, Reduction of ≥ 10%
| percent change of centimeters | Pasireotide 600 μg | Pasireotide 900 μg | All Patients |
|---|---|---|---|
| Week 12 | -2.0 ± 4.29 | -2.9 ± 6.70 | -2.5 ± 5.72 |
| Week 24 | -5.8 ± 6.28 | -3.1 ± 5.48 | -4.4 ± 5.96 |
| Week 48 | -5.1 ± 5.74 | -4.1 ± 5.64 | -4.6 ± 5.62 |
Change from baseline is shown as: Percent change from baseline (BL) =((Post BL value - BL value)/ BL value)\*100. Ferriman-Gallway scoring was used: 0=minimum and 36 was maximum in females only.
| percent change in scores | Pasireotide 600 μg | Pasireotide 900 μg | All Patients |
|---|---|---|---|
| Week 12 | -12.2 ± 24.57 | -8.2 ± 35.30 | -10.0 ± 30.88 |
| Week 24 | -21.2 ± 32.72 | -16.2 ± 46.99 | 18.4 ± 40.79 |
| Week 48 | -18.2 ± 30.12 | 9.6 ± 143.83 | -2.2 ± 110.20 |
Descriptive summary of the effect of pasireotide on GH.
| percent change of µg/L | Pasireotide 600 μg | Pasireotide 900 μg | All Patients |
|---|---|---|---|
| Week 12 | -17.3 ± 109.89 | -20.90 ± 156.60 | -19.3 ± 137.33 |
| Week 24 | -22.2 ± 62.43 | -26.2 ± 105.44 | -24.4 ± 88.70 |
| Week 48 | 23.1 ± 127.30 | -1.0 ± 134.97 | 9.7 ± 130.32 |
Descriptive summary of the effect of pasireotide on IGF-1
| percent change of ng/ml | Pasireotide 600 μg | Pasireotide 900 μg | All Patients |
|---|---|---|---|
| Week 12 | -53.4 ± 23.66 | -57.8 ± 23.93 | -55.9 ± 23.76 |
| Week 24 | -49.2 ± 26.56 | -56.2 ± 26.55 | -53.1 ± 26.53 |
| Week 48 | -41.8 ± 35.72 | -52.1 ± 21.87 | -47.6 ± 28.71 |
Collected over Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to approximately 256 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| 600 µg Bid | 0/49 (0%) | 13/49 (26.5%) | 49/49 (100%) |
| 900 µg Bid | 1/55 (1.8%) | 17/55 (30.9%) | 54/55 (98.2%) |
| All Patients | 1/104 (1%) | 30/104 (28.8%) | 103/104 (99%) |
| Event | 600 µg Bid | 900 µg Bid | All Patients |
|---|---|---|---|
| Cholecystitis acuteHepatobiliary disorders | 0/49 | 3/55 | 3/104 |
| DiarrhoeaGastrointestinal disorders | 2/49 | 1/55 | 3/104 |
| Diabetes mellitusMetabolism and nutrition disorders | 2/49 | 1/55 | 3/104 |
| HyperglycaemiaMetabolism and nutrition disorders | 2/49 | 0/55 | 2/104 |
| HypokalaemiaMetabolism and nutrition disorders | 0/49 | 2/55 | 2/104 |
| Adrenal insufficiencyEndocrine disorders | 1/49 | 0/55 | 1/104 |
| Glucocorticoid deficiencyEndocrine disorders | 1/49 | 0/55 | 1/104 |
| HyperprolactinaemiaEndocrine disorders | 1/49 | 0/55 | 1/104 |
| Pituitary-dependent Cushing's syndromeEndocrine disorders | 1/49 | 0/55 | 1/104 |
| Abdominal painGastrointestinal disorders | 1/49 | 0/55 | 1/104 |
| Event | 600 µg Bid | 900 µg Bid | All Patients |
|---|---|---|---|
| DiarrhoeaGastrointestinal disorders | 28/49 | 23/55 | 51/104 |
| NauseaGastrointestinal disorders | 22/49 | 26/55 | 48/104 |
| CholelithiasisHepatobiliary disorders | 8/49 | 22/55 | 30/104 |
| HyperglycaemiaMetabolism and nutrition disorders | 19/49 | 22/55 | 41/104 |
| HeadacheNervous system disorders | 12/49 | 19/55 | 31/104 |
| Diabetes mellitusMetabolism and nutrition disorders | 9/49 | 14/55 | 23/104 |
| FatigueGeneral disorders | 11/49 | 12/55 | 23/104 |
| Abdominal painGastrointestinal disorders | 10/49 | 9/55 | 19/104 |
| Oedema peripheralGeneral disorders | 9/49 | 3/55 | 12/104 |
| Blood glucose increasedInvestigations | 4/49 | 9/55 | 13/104 |
| Age, Continuous(years) | Pasireotide 600 μg | Pasireotide 900 μg | Total |
|---|---|---|---|
| Mean | 45.5 ± 13.14 | 39.9 ± 12.55 | 42.5 ± 13.07 |
| Sex: Female, Male(Participants) | Pasireotide 600 μg | Pasireotide 900 μg | Total |
|---|---|---|---|
| Female | 37 | 47 | 84 |
| Male | 12 | 8 | 20 |
| Race (NIH/OMB)(Participants) | Pasireotide 600 μg | Pasireotide 900 μg | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 6 | 15 | 21 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 3 | 2 | 5 |
| White | 39 | 36 | 75 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 1 | 2 | 3 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Undecided
Ran from Aug 2011 to primary completion in Jan 2017, 65 months.
Results were first posted in Jun 2018, 1 year 4 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in May 2018. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Pituitary ACTH Hypersecretion →