Phase 2
N=19
Extension Study to Assess the Safety and Efficacy of Pasireotide in Participants With Cushing's Disease
Cushing Disease
Bottom Line
View on ClinicalTrials.gov: NCT00171951 ↗Enrolled (actual)
19
Serious AEs
14.8%
Results posted
Jun 2021
Primary outcome: Primary: Percentage of Responders With Mean Urinary Free Cortisol (UFC) Within Normal Limits — 22.2 percentage of responders
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Pasireotide (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Jul 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Responders With Mean Urinary Free Cortisol (UFC) Within Normal Limits |
22.2 | — |
| PRIMARY Change From Baseline in Mean Urinary Free Cortisol (UFC) |
1219.74; -710.57; -801.85; -1202.1; -1241.1; -2417.0 | — |
| SECONDARY Number of Participants Who Had At Least One Adverse Event (AE) |
19; 8 | — |
| SECONDARY Change From Baseline in Serum Cortisol Levels |
723.60; -25.96; -150.60; -66.06; -227.53; -166.00 | — |
| SECONDARY Plasma Trough Concentrations (Ctrough) of Pasireotide in UFC Responders |
7.0; 17.4 | — |
| SECONDARY Change From Baseline in Plasma Adrenocorticotropic Hormone (ACTH) Levels |
13.72; -1.28; -3.36; -3.60; -4.50; 0.00 | — |
| SECONDARY Change From Baseline in Gene-expression and Protein in Blood and Urine for Biomarker Development |
— | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- Participants who have completed the 15 days of Pasireotide treatment in the CSOM230B2208 study and have achieved normalization of 24-hour urinary free cortisol. Participants who did not achieve normalization of 24 -hour urinary free cortisol may be enrolled if in the opinion of the investigator the participant is getting significant clinical benefits from treatment with Pasireotide .
- The participant did not experience any unacceptable adverse events of tolerability issues during the original 15 day treatment.
- Female participants of childbearing potential who have not undergone clinically documented total hysterectomy and/or ovariectomy or tubal ligation must agree to use barrier contraception throughout the course of the extension study, and for one month after the study has ended.
Exclusion Criteria
- Participant who have developed poorly controlled diabetes mellitus as indicated by ketoacidosis or hemoglobin (Hgb) A1C (HgbA1C) > 10 since starting [study CSOM230B2208].
- Participant with persistent alanine aminotransferase (ALT)/ aspartate transaminase (AST) or alkaline phosphatase levels more than 2.5X upper limit of normal (ULN), serum creatinine > 2.0 X ULN, serum bilirubin > 2 X ULN.
- Participant with abnormal coagulation (Prothrombin time (PT) and partial thromboplastin time (PTT) elevated by 30% above normal limits), white blood cells (WBC) <3.0x1'000'000'000/L; Hgb <12.0g/dL for females, Hgb <13.0g/dL for males; PLT <100x1'000'000'000/L.
Other protocol-defined inclusion / exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT00171951). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.