A Phase 2 interventional study of Pasireotide in Cushing Disease, sponsored by Novartis Pharmaceuticals. Completed at 8 sites in 5 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-06-02.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 2, Interventional, and Treatment
Cushing's disease is a rare serious condition that is caused by an adrenocorticotropic hormone (ACTH) secreting pituitary adenoma. This study assessed the long-term safety and efficacy of pasireotide in participants with Cushing's disease.
64 studies on the registry are indexed under Pituitary ACTH Hypersecretion; 16 are open to participants now.
This study's enrollment of 19 is below the median of 44 across 40 interventional studies indexed under Pituitary ACTH Hypersecretion.
Browse Pituitary ACTH Hypersecretion studies →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.
Counted across the registry records on this site, refreshed daily.
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.
Exclusion Criteria:
Other protocol-defined inclusion / exclusion criteria may apply.
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.
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.
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).
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.
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.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
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.
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.
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.
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.
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.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
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.
Participants received pasireotide 600 micrograms (μg) twice daily (BID) subcutaneously (SC) to achieve or maintain urinary free cortisol (UFC) normalization. If UFC levels were increased at any time, participants received 900 μg BID SC, until no safety or tolerability concerns were observed as per investigators assessment. If the participant was unable to tolerate the 900 μg BID, dosing of 600 μg three times a day was given.
Drug: Pasireotide
Pasireotide 600 μg or 900 μg was administered as an SC injection.
Also known as: SOM230
Percentage of Responders With Mean Urinary Free Cortisol (UFC) Within Normal Limits
A participant was considered a responder if the mean UFC from the two 24-hour urine samples collected at Month 6 was within normal limits. The normal range for UFC is 55 to 276 nmol/day.
Time frame: Month 6
Change From Baseline in Mean Urinary Free Cortisol (UFC)
24-hour urine samples were collected to obtain mean UFC measurements. A negative mean change from baseline indicates improvement.
Time frame: Core Baseline, Days 14/15 (Core study), Months 6, 12, 24 and 102
Number of Participants Who Had At Least One Adverse Event (AE)
An AE was any undesirable sign, symptom or medical condition occurring after starting study drug even if the event is not considered to be related to study drug. AEs were assessed according to incident dose group: Pasireotide 1200 μg sc total daily dose (TDD), Pasireotide 1800 μg SC TDD and Pasireotide SC Any Dose. The incident dose for an AE was the last total daily dose administered on or prior to the AE onset date.
Time frame: Up to approximately 106 months
Change From Baseline in Serum Cortisol Levels
Blood samples were withdrawn to obtain the serum cortisol levels. A negative change from baseline indicates improvement.
Time frame: Core Baseline, Day 15 (Core study), Months 6, 12, 24, and Month 105 (end of the study)
Plasma Trough Concentrations (Ctrough) of Pasireotide in UFC Responders
Participants with Cushing's disease were considered responders if mean UFC levels from the 24-hour urine collections at Day 15 (Core study) and at extension Month 6, were within normal limits. Ctrough levels of pasireotide were measured at Day 15 of Core study and Month 6.
Time frame: Day 15 (Core study) and Month 6
Change From Baseline in Plasma Adrenocorticotropic Hormone (ACTH) Levels
Blood samples were withdrawn to obtain the ACTH levels. A negative change from baseline indicates improvement.
Time frame: Core Baseline, Day 15 (Core study), Months 6, 12, 24 and Month 105 (end of the study)
Change From Baseline in Gene-expression and Protein in Blood and Urine for Biomarker Development
Time frame: Baseline to end of the study
| Milestone | Pasireotide 600 μg BID SC or Ramp up Dose 900 μg BID SC |
|---|---|
| Started | 19 |
| Completed | 3 |
| Not completed | 16 |
| Withdrew: Adverse event | 1 |
| Withdrew: Abnormal laboratory values | 2 |
| Withdrew: Unsatisfactory therapeutic effect | 3 |
| Withdrew: Participants withdrew consent | 3 |
| Withdrew: New cancer therapy | 5 |
| Withdrew: Reason not specified | 2 |
A participant was considered a responder if the mean UFC from the two 24-hour urine samples collected at Month 6 was within normal limits. The normal range for UFC is 55 to 276 nmol/day.
| percentage of responders | Pasireotide 600 μg BID SC or Ramp up Dose 900 μg BID SC |
|---|---|
| Percentage of Responders With Mean Urinary Free Cortisol (UFC) Within Normal Limits | 22.2 (6.4 to 47.6) |
24-hour urine samples were collected to obtain mean UFC measurements. A negative mean change from baseline indicates improvement.
| nanomoles per 24 hours (nmol/24h) | Pasireotide 600 μg BID SC or Ramp up Dose 900 μg BID SC |
|---|---|
| Core Baseline | 1219.74 ± 752.9 |
| Change from Core Baseline to Day 14/Day 15 | -710.57 ± 740.6 |
| Change from Core Baseline to Month 6 | -801.85 ± 819.4 |
| Change from Core Baseline to Month 12 | -1202.1 ± 1084.1 |
| Change from Core Baseline to Month 24 | -1241.1 ± 925.6 |
| Change from Core Baseline to Month 102 | -2417.0 ± NA |
An AE was any undesirable sign, symptom or medical condition occurring after starting study drug even if the event is not considered to be related to study drug. AEs were assessed according to incident dose group: Pasireotide 1200 μg sc total daily dose (TDD), Pasireotide 1800 μg SC TDD and Pasireotide SC Any Dose. The incident dose for an AE was the last total daily dose administered on or prior to the AE onset date.
| Participants | Pasireotide 600 μg BID SC | Pasireotide 900 μg BID SC |
|---|---|---|
| Number of Participants Who Had At Least One Adverse Event (AE) | 19 | 8 |
Blood samples were withdrawn to obtain the serum cortisol levels. A negative change from baseline indicates improvement.
| nanomoles per liters (nmol/L) | Pasireotide 600 μg BID SC or Ramp up Dose 900 μg BID SC |
|---|---|
| Core Baseline | 723.60 ± 221.5 |
| Change from Core Baseline to Day 15 | -25.96 ± 125.1 |
| Change from Core Baseline to Month 6 | -150.60 ± 308.9 |
| Change from Core Baseline to Month 12 | -66.06 ± 301.9 |
| Change from Core Baseline to Month 24 | -227.53 ± 283.1 |
| Change from Core Baseline to Month 105 | -166.00 ± NA |
Participants with Cushing's disease were considered responders if mean UFC levels from the 24-hour urine collections at Day 15 (Core study) and at extension Month 6, were within normal limits. Ctrough levels of pasireotide were measured at Day 15 of Core study and Month 6.
| nanograms per milliliter (ng/mL) | Pasireotide 600 μg BID SC or Ramp up Dose 900 μg BID SC |
|---|---|
| Day 15 (Core Study) | 7.0 ± 4.3 |
| Month 6 | 17.4 ± 11.6 |
Blood samples were withdrawn to obtain the ACTH levels. A negative change from baseline indicates improvement.
| picomoles per litre (pmol/L) | Pasireotide 600 μg BID SC or Ramp up Dose 900 μg BID SC |
|---|---|
| Core Baseline | 13.72 ± 11.0 |
| Change from Core Baseline to Day 15 | -1.28 ± 7.1 |
| Change from Core Baseline to Month 6 | -3.36 ± 9.4 |
| Change from Core Baseline to Month 12 | -3.60 ± 15.2 |
| Change from Core Baseline to Month 24 | -4.50 ± 8.3 |
| Change from Core Baseline to Month 105 | 0.00 ± NA |
No measurements were reported for this outcome.
Collected over Up to approximately 106 months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Pasireotide 600 μg BID SC | 0/19 (0%) | 2/19 (10.5%) | 19/19 (100%) |
| Pasireotide 900 μg BID SC | 0/8 (0%) | 2/8 (25%) | 8/8 (100%) |
| Event | Pasireotide 600 μg BID SC | Pasireotide 900 μg BID SC |
|---|---|---|
| Urinary tract infectionInfections and infestations | 0/19 | 1/8 |
| Skin injuryInjury, poisoning and procedural complications | 0/19 | 1/8 |
| Type 2 diabetes mellitusMetabolism and nutrition disorders | 0/19 | 1/8 |
| ConvulsionNervous system disorders | 1/19 | 0/8 |
| PneumothoraxRespiratory, thoracic and mediastinal disorders | 1/19 | 0/8 |
| Event | Pasireotide 600 μg BID SC | Pasireotide 900 μg BID SC |
|---|---|---|
| DiarrhoeaGastrointestinal disorders | 12/19 | 2/8 |
| NauseaGastrointestinal disorders | 12/19 | 1/8 |
| HyperglycaemiaMetabolism and nutrition disorders | 11/19 | 3/8 |
| Abdominal painGastrointestinal disorders | 8/19 | 2/8 |
| CholelithiasisHepatobiliary disorders | 0/19 | 3/8 |
| HeadacheNervous system disorders | 7/19 | 0/8 |
| Injection site painGeneral disorders | 6/19 | 0/8 |
| FatigueGeneral disorders | 5/19 | 0/8 |
| Injection site pruritusGeneral disorders | 5/19 | 0/8 |
| HypertensionVascular disorders | 2/19 | 2/8 |
Intent-to-treat (ITT) population included all participants who completed 15 days of treatment in Core study and experienced significant clinical benefit from treatment and received at least 1 dose in the Extension Period.
| Age, Continuous(years) | Pasireotide 600 μg BID SC or Ramp up Dose 900 μg BID SC |
|---|---|
| Mean | 43.0 ± 11.62 |
| Sex: Female, Male(Participants) | Pasireotide 600 μg BID SC or Ramp up Dose 900 μg BID SC |
|---|---|
| Female | 17 |
| Male | 2 |
Ran from Aug 2004 to primary completion in Jul 2013, 106 months.
Results were first posted in Jun 2021, 7 years 10 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 2021. 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.
Studies of the drug tested here: Pasireotide
Pituitary ACTH Hypersecretion →