Study of Pasireotide in Patients With Rare Tumors of Neuroendocrine Origin

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Primary Purpose

Status

Terminated

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

pasireotide LAR

About this trial

This is an interventional treatment trial for Pancreatic Neoplasm focused on measuring Rare tumors of neuroendocrine origin, NETs of the pancreatic, Pituitary NETs, EAS Tumors, Nelson's Syndrome, Pancreatic neoplasm, pituitary neoplasm, Nelson Syndrome, ectopic ACTH syndrome, pasireotide LAR, PNETs, PiNETS

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

pasireotide LAR 60mg

Arm Description

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.

Outcomes

Primary Outcome Measures

Percentage of Responders at Month 6 - Pooled Pancreatic NETs (PNETs)

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

Percentage of Responders at Month 6 - Individual NETs

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.

Percentage of Responders With Probability of Success at Month 6 - Individual NETs

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

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.

PiNETs: Number of Patients Attaining Normalization or a More Than 50% Reduction in Primary Biochemical Tumor Marker

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.

Nelson's Syndrome: Number of Patients Attaining Normalization or a More Than 50% Reduction in Primary Biochemical Tumor Marker

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.

Full Information

NCT ID

NCT00958841

First Posted

July 22, 2009

Last Updated

June 15, 2016

Sponsor

Novartis Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT00958841

Brief Title

Study of Pasireotide in Patients With Rare Tumors of Neuroendocrine Origin

Official Title

An Open Label, Multicenter, Single Arm Study of Pasireotide LAR in Patients With Rare Tumors of Neuroendocrine Origin

Study Type

Interventional

2. Study Status

Record Verification Date

June 2016

Overall Recruitment Status

Terminated

Why Stopped

Slow recruitment rate into this study with rare tumors of neuroendocrine origin (enrollment issues)

Study Start Date

September 2009 (undefined)

Primary Completion Date

June 2015 (Actual)

Study Completion Date

June 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Novartis Pharmaceuticals

4. Oversight

Data Monitoring Committee

No

5. Study Description

Brief Summary

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pancreatic Neoplasm, Pituitary Neoplasm, Nelson Syndrome, Ectopic ACTH Syndrome

Keywords

Rare tumors of neuroendocrine origin, NETs of the pancreatic, Pituitary NETs, EAS Tumors, Nelson's Syndrome, Pancreatic neoplasm, pituitary neoplasm, Nelson Syndrome, ectopic ACTH syndrome, pasireotide LAR, PNETs, PiNETS

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

118 (Actual)

8. Arms, Groups, and Interventions

Arm Title

pasireotide LAR 60mg

Arm Type

Experimental

Arm Description

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.

Intervention Type

Drug

Intervention Name(s)

pasireotide LAR

Other Intervention Name(s)

SOM230

Intervention Description

Investigational drug pasireotide LAR was supplied in vials with 20 mg or 40 mg powder and 2 mL vehicle was supplied in ampoules for reconstitution.

Primary Outcome Measure Information:

Title

Percentage of Responders at Month 6 - Pooled Pancreatic NETs (PNETs)

Description

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.

Time Frame

6 months

Secondary Outcome Measure Information:

Title

Percentage of Responders at Month 6 - Individual NETs

Description

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.

Time Frame

6 months

Title

Percentage of Responders With Probability of Success at Month 6 - Individual NETs

Description

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.

Time Frame

6 months

Title

PNETs: Number of Patients Attaining Normalization or a More Than 50% Reduction in Primary Biochemical Tumor Marker

Description

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.

Time Frame

Baseline, month 6

Title

PiNETs: Number of Patients Attaining Normalization or a More Than 50% Reduction in Primary Biochemical Tumor Marker

Description

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.

Time Frame

Baseline, month 6

Title

Nelson's Syndrome: Number of Patients Attaining Normalization or a More Than 50% Reduction in Primary Biochemical Tumor Marker

Description

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.

Time Frame

Baseline, month 6

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

No

Eligibility Criteria

Inclusion Criteria: Male and Female Patients at least 18 years old Patient who have rare tumors of neuroendocrine origin, such as tumors of the: pancreas pituitary glands Nelson syndrome 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Novartis Pharmaceuticals

Organizational Affiliation

Novartis Pharmaceuticals

Official's Role

Study Director

Facility Information:

Facility Name

Cedars Sinai Medical Center Cedars Sinai 4

City

Los Angeles

State/Province

California

ZIP/Postal Code

90048

Country

United States

Facility Name

Cedars Sinai Medical Center The Pituitary Center (3)

City

Los Angeles

State/Province

California

ZIP/Postal Code

90048

Country

United States

Facility Name

Stanford University Medical Center SC

City

Stanford

State/Province

California

ZIP/Postal Code

94304

Country

United States

Facility Name

Dana Farber Cancer Institute Deptof DanaFarberCancerInst(5)

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02215

Country

United States

Facility Name

Mount Sinai School of Medicine Study Coordinator

City

New York

State/Province

New York

ZIP/Postal Code

10029

Country

United States

Facility Name

Swedish Medical Center Dept.ofSwedishMedicalCtr.(2)

City

Seattle

State/Province

Washington

Country

United States

Facility Name

Novartis Investigative Site

City

Buenos Aires

ZIP/Postal Code

C1264AAA

Country

Argentina

Facility Name

Novartis Investigative Site

City

Westmead

State/Province

New South Wales

ZIP/Postal Code

2145

Country

Australia

Facility Name

Novartis Investigative Site

City

Fitzroy

State/Province

Victoria

ZIP/Postal Code

3065

Country

Australia

Facility Name

Novartis Investigative Site

City

Fortaleza

State/Province

CE

ZIP/Postal Code

60430-370

Country

Brazil

Facility Name

Novartis Investigative Site

City

Belo Horizonte

State/Province

MG

ZIP/Postal Code

30130-100

Country

Brazil

Facility Name

Novartis Investigative Site

City

Botucatu

State/Province

SP

ZIP/Postal Code

18618-970

Country

Brazil

Facility Name

Novartis Investigative Site

City

Halifax

State/Province

Nova Scotia

ZIP/Postal Code

B3H 2Y9

Country

Canada

Facility Name

Novartis Investigative Site

City

Montreal

State/Province

Quebec

ZIP/Postal Code

H2L 2W5

Country

Canada

Facility Name

Novartis Investigative Site

City

Angers

ZIP/Postal Code

49033

Country

France

Facility Name

Novartis Investigative Site

City

Bron Cedex

ZIP/Postal Code

69677

Country

France

Facility Name

Novartis Investigative Site

City

Le Kremlin Bicetre

ZIP/Postal Code

94275

Country

France

Facility Name

Novartis Investigative Site

City

Lille Cedex

ZIP/Postal Code

59037

Country

France

Facility Name

Novartis Investigative Site

City

Marseille cedex 05

ZIP/Postal Code

13385

Country

France

Facility Name

Novartis Investigative Site

City

Pessac Cedex

ZIP/Postal Code

33604

Country

France

Facility Name

Novartis Investigative Site

City

Reims

ZIP/Postal Code

51092

Country

France

Facility Name

Novartis Investigative Site

City

Strasbourg

ZIP/Postal Code

67098

Country

France

Facility Name

Novartis Investigative Site

City

Berlin

ZIP/Postal Code

10098

Country

Germany

Facility Name

Novartis Investigative Site

City

Berlin

ZIP/Postal Code

13353

Country

Germany

Facility Name

Novartis Investigative Site

City

Erlangen

ZIP/Postal Code

91054

Country

Germany

Facility Name

Novartis Investigative Site

City

Frankfurt

ZIP/Postal Code

60590

Country

Germany

Facility Name

Novartis Investigative Site

City

Muenchen

ZIP/Postal Code

80804

Country

Germany

Facility Name

Novartis Investigative Site

City

Ulm

ZIP/Postal Code

89081

Country

Germany

Facility Name

Novartis Investigative Site

City

Würzburg

ZIP/Postal Code

97080

Country

Germany

Facility Name

Novartis Investigative Site

City

Ancona

State/Province

AN

ZIP/Postal Code

60126

Country

Italy

Facility Name

Novartis Investigative Site

City

Cona

State/Province

FE

ZIP/Postal Code

44100

Country

Italy

Facility Name

Novartis Investigative Site

City

Padova

State/Province

PD

ZIP/Postal Code

35128

Country

Italy

Facility Name

Novartis Investigative Site

City

Pisa

State/Province

PI

ZIP/Postal Code

56124

Country

Italy

Facility Name

Novartis Investigative Site

City

Roma

State/Province

RM

ZIP/Postal Code

00168

Country

Italy

Facility Name

Novartis Investigative Site

City

México

State/Province

Distrito Federal

ZIP/Postal Code

14269

Country

Mexico

Facility Name

Novartis Investigative Site

City

Moscow

ZIP/Postal Code

115478

Country

Russian Federation

Facility Name

Novartis Investigative Site

City

Moscow

ZIP/Postal Code

117036

Country

Russian Federation

Facility Name

Novartis Investigative Site

City

Saint-Petersburg

ZIP/Postal Code

197341

Country

Russian Federation

Facility Name

Novartis Investigative Site

City

Malaga

State/Province

Andalucia

ZIP/Postal Code

29010

Country

Spain

Facility Name

Novartis Investigative Site

City

Barcelona

State/Province

Catalunya

ZIP/Postal Code

08036

Country

Spain

Facility Name

Novartis Investigative Site

City

Bangkok

ZIP/Postal Code

10330

Country

Thailand

Facility Name

Novartis Investigative Site

City

Bangkok

ZIP/Postal Code

10700

Country

Thailand

Pancreatic Neoplasm, Pituitary Neoplasm, Nelson Syndrome

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial