Study of Pasireotide in Patients With Rare Tumors of Neuroendocrine Origin

Phase 2

Terminated

Conditions: Pancreatic Neoplasm
Pituitary Neoplasm
Nelson Syndrome
Ectopic ACTH Syndrome

Interventions: Drug: pasireotide LAR

Registration Number: NCT00958841

Lead Sponsor: Novartis Pharmaceuticals

Brief Summary: This study will assess the effectiveness and safety of pasireotide long-acting release in patients who have rare tumors of neuroendocrine origin.

Detailed Description: Not available

Recruitment & Eligibility

Status: TERMINATED

Sex: All

Target Recruitment: 118

Inclusion Criteria

Male and Female Patients at least 18 years old
Patient who have rare tumors of neuroendocrine origin, such as tumors of the:
1. pancreas
2. pituitary glands
3. Nelson syndrome
4. ectopic-ACTH secreting tumor
Patients who have failed standard of care treatment or for whom no standard of care treatment exist
Signed Informed Consent

Exclusion Criteria

Patients with active gallbladder disease
Patients with any ongoing or planned anti-neoplastic or interferon therapy
Poorly controlled diabetes mellitus
Female patients who are pregnant or lactating, or are of childbearing potential and not practicing a medically acceptable method of birth control

Other protocol-defined inclusion/exclusion criteria may apply.

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
pasireotide LAR 60mg	pasireotide LAR	Patients received pasireotide LAR at 60 mg approximately once every 28 days for 6 months during the core treatment period and additional treatment cycles up to a total of 48 months during the extension phase.

Primary Outcome Measures

Name	Time	Method
Percentage of Responders at Month 6 - Pooled Pancreatic NETs (PNETs)	6 months	The primary efficacy endpoint was defined as the percentage of responders at Month 6 among pooled PNET patients (insulinoma, gastrinoma, VIPoma, and glucagonoma). A responder was defined as a patient who either attained normalization or had a greater than 50% reduction from baseline of the level of the primary biochemical tumor marker at Month 6 (M6). Four insulinoma pts were excluded from analysis because of unavailability of normal ranges for the associated primary biochemical tumor marker (insulin-to-glucose ratio). One patient with VIPoma with a normal baseline was also excluded. As a result, only 20 out of 25 patients with PNET were included in the assessment of the primary endpoint, which was less than the planned sample size of 34. Therefore, the primary objective could not be assessed with sufficient power. Patients with missing Month 6 assessment were considered as non-responders. Responder analyses are reported only for indications with minimum of 6 patients.

Secondary Outcome Measures

Name	Time	Method
PiNETs: Number of Patients Attaining Normalization or a More Than 50% Reduction in Primary Biochemical Tumor Marker	Baseline, month 6	Specific primary biochemical tumor markers were used to assess the efficacy of pasireotide in PNETs. A Month 6 responder was defined as the patients who either attained normalization or greater than 50% reduction from baseline in the level of the primary biochemical tumor marker at Month 6.
Percentage of Responders With Probability of Success at Month 6 - Individual NETs	6 months	Percentage of responders for each of the 10 NET indications considered in the study. Responder analyses were performed for an indication only if there were at least 6 patients in the efficacy analyzable set. For all other individual indications, the numbers of patients in the efficacy analyzable sets were less than 6 and therefore no responder analyses were carried out for these indications. The probability of success was a chance that the true responder rate was greater than 15%) for the indications gastrinoma, prolactinoma, and Nelson's syndrome.
PNETs: Number of Patients Attaining Normalization or a More Than 50% Reduction in Primary Biochemical Tumor Marker	Baseline, month 6	Specific primary biochemical tumor markers were used to assess the efficacy of pasireotide in PNETs. A Month 6 responder was defined as the patients who either attained normalization or greater than 50% reduction from baseline in the level of the primary biochemical tumor marker at Month 6. One gastrinoma patient had a missing primary tumor marker value at Month 6, but had a Month 5 assessment done on Day 141, which fell within the allowed window period for Month 6.
Nelson's Syndrome: Number of Patients Attaining Normalization or a More Than 50% Reduction in Primary Biochemical Tumor Marker	Baseline, month 6	Six patients with Nelson's syndrome met the responder's criteria of attaining normalization or a reduction of more than 50% in primary tumor marker at Month 6.
Percentage of Responders at Month 6 - Individual NETs	6 months	Percentage of responders for each of the 10 NET indications considered in the study. Responder analyses were performed for an indication only if there were at least 6 patients in the efficacy analyzable set. For all other individual indications, the numbers of patients in the efficacy analyzable sets were less than 6 and therefore no responder analyses were carried out for these indications.

Trial Locations

Locations (7): Cedars Sinai Medical Center The Pituitary Center (3)
🇺🇸
Los Angeles, California, United States
Cedars Sinai Medical Center Cedars Sinai 4
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Los Angeles, California, United States
Mount Sinai School of Medicine Study Coordinator
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New York, New York, United States
Stanford University Medical Center SC
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Stanford, California, United States
Dana Farber Cancer Institute Deptof DanaFarberCancerInst(5)
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Boston, Massachusetts, United States
Novartis Investigative Site
🇹🇭
Bangkok, Thailand
Swedish Medical Center Dept.ofSwedishMedicalCtr.(2)
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Seattle, Washington, United States