Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Pasireotide LAR · 6 trials · 6 indications
Percentage of participants who achieved biochemical control defined as GH \<1μg/L and IGF-1 \<ULN at week 36.
Percentage of participants who achieved biochemical control defined as GH \<1μg/L and IGF-1 \<ULN at week 36, for participants who had been up-titrated with pasireotide LAR 60 mg.
Percentage of participants who achieved biochemical control defined as GH \<1μg/L and IGF-1 \<ULN at week 36.
Percentage of participants who achieved biochemical control defined as GH \<1μg/L and IGF-1 \<ULN at week 36, overall by baseline diabetic status.
Percentage of participants who achieved biochemical control defined as GH \<1μg/L and IGF-1 \<ULN at week 36, by previous treatment and overall - last observation carried forward (LOCF)
Percentage of participants that attained a mean urinary free cortisol (mUFC) \<= 1.0 x upper limit of normal (ULN) at Month 7 regardless of dose up-titration at Month 4. Patients who discontinued before month 4 evaluations classed as non-responders. For patients missing month 7 mUFC assessments, the last available mUFC assessment at or after month 4 was carried forward as the month 7 mUFC assessment value.
Patients with Complete Response (CR), Partial Response (PR), or Stable Disease (SD) at Month 9 were to be considered as "progression-free" based on RECIST v1.1. Patients with missing tumor assessment, or with overall lesion response "unknown" at Month 9 were considered as "non progression-free", unless any of the following assessments at Week 48 or Week 52 indicate CR, PR, or SD, in which case the patient was to be considered as progression-free at Month 9. Patients discontinuing the study for any reason prior to the 9 month assessment were to be considered as "non progression-free".
Tumor volume was evaluated by MRI. MRIs were performed and processed according to the guidelines of the central evaluator's facility.The pituitary adenomas were measured by a neuro-radiologist, using manual tracing together with the imaging analysis software. The largest diameter at any plan determined the maximum diameter of the tumor. A change ≥ 20% in the original volume of the tumor was considered to be clinically significant. Evaluable participants required tumor volume assessment at baseline and at week 24.
Percentage of participants with a reduction of mean growth hormone (GH) levels to \< 2.5 µg/L and the normalization of insulin-like growth factor-1 (IGF-1) to within normal limits (age and sex related) at 3 months across all doses
Frequency of dose-limiting toxicities (DLTs) at each dose level associated with q28 days administration of pasireotide LAR during the first 2 treatment cycles.
| Arm | Type | Description |
|---|---|---|
| Pasireotide LAR | EXPERIMENTAL | Patients who qualify for the core phase of the study will be treated with pasireotide LAR 40 mg initially. Patients not achieving biochemical control can be up-titrated to pasireotide LAR 60 mg. |
| 10 mg LAR dose | EXPERIMENTAL | Randomization was stratified based on Screening mUFC to ensure balanced distribution of disease severity in the two dose arms. |
| 30 mg LAR dose | EXPERIMENTAL | Randomization was stratified based on Screening mUFC to ensure balanced distribution of disease severity in the two dose arms. |
| Everolimus | EXPERIMENTAL | Everolimus 10 mg taken orally (p.o) once daily starting on Day 1 |
| Pasireotide LAR and Everolimus Combination | EXPERIMENTAL | Pasireotide LAR 60 mg i.m. injected once every 28 days + Everolimus 10 mg p.o. daily starting on Day 1 |
| Pasireotide LAR 20mg | EXPERIMENTAL | Enrolled patients were randomized to 20mg pasireotide LAR. |
| Pasireotide LAR 40mg | EXPERIMENTAL | Enrolled patients were randomized to 40mg pasireotide LAR. |
| Pasireotide LAR 60mg | EXPERIMENTAL | Enrolled patients were randomized to 60mg pasireotide LAR. |
| Name | Type | Description |
|---|---|---|
| Pasireotide LAR | DRUG | Pasireotide 40 mg and 60 mg. Pasireotide 20 mg which was allowed for dose decrease in case of adverse event. |
| SOM230 LAR 30 mg | DRUG | starting dose of 30 mg i.m. administered once every 28 days for 4 months, followed by dose up-titration or continuation of starting dose. |
| SOM230 LAR 10 mg | DRUG | starting does of SOM230 LAR 10 mg i.m. administered once every 28 days for 4 months, followed by dose up-titration or continuation of the starting dose. |
| Everolimus | DRUG | 10 mg tables administered orally once a day |
| Pasireotide LAR and Everolimus Combination | DRUG | Pasireotide LAR 60 mg i.m. injected once every 28 days + Everolimus 10 mg p.o. daily |
Inclusion Criteria: * Written informed consent * Male and female patients ≥18 years * Patients with confirmed diagnosis of inadequately controlled acromegaly (mean GH concentration ≥1 μg/L and sex- and age-adjusted IGF-1 \>1.3 x ULN) * Patients treated with octreotide LAR (30 mg or 40 mg) or lanreo...
Pasireotide LAR is an investigational small molecule being studied for several endocrine conditions, including Cushing's Disease, Acromegaly, Neuroendocrine Tumors, Neuroendocrine Carcinoma of the Lung and Thymus, and Non-functioning Pituitary Adenoma. It is developed by Novartis AG and is in Phase 3 clinical development.
Pasireotide LAR is a somatostatin analog that targets somatostatin receptors. It is being studied for its ability to inhibit hormone secretion in conditions like Cushing's Disease and acromegaly. The drug is designed to bind to these receptors and modulate their activity, though specific receptor subtypes are not detailed here.
Pasireotide LAR is developed by Novartis AG, a multinational pharmaceutical company traded on the New York Stock Exchange under the ticker symbol NVS. The company is conducting clinical trials to evaluate the drug's safety and efficacy across multiple endocrine indications.
Pasireotide LAR is in Phase 3 clinical development, specifically for Cushing's Disease. It has completed Phase 1 and Phase 2 trials for other indications, including Neuroendocrine Tumors and Non-functioning Pituitary Adenoma. The drug is investigational and not yet approved by regulatory authorities.
Pasireotide LAR has been studied in several clinical trials, including NCT01374906 for Cushing's Disease, NCT01283542 for Non-functioning Pituitary Adenoma, NCT01364415 for Neuroendocrine Tumors, and NCT01563354 for Neuroendocrine Carcinoma of the Lung and Thymus. All trials listed are completed.
Pasireotide LAR is a long-acting release formulation of pasireotide, also known as SOM230. The LAR version is designed for monthly administration, while other formulations may be given more frequently. Both are being developed by Novartis for similar endocrine conditions.