Study of Pasireotide in Patients With Rare Tumors of Neuroendocrine Origin

This study has been terminated.
(Slow recruitment rate into this study with rare tumors of neuroendocrine origin (enrollment issues))
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00958841
First received: July 22, 2009
Last updated: June 15, 2016
Last verified: June 2016
  Purpose
This study will assess the effectiveness and safety of pasireotide long-acting release in patients who have rare tumors of neuroendocrine origin.

Condition Intervention Phase
Pancreatic Neoplasm
Pituitary Neoplasm
Nelson Syndrome
Ectopic ACTH Syndrome
Drug: pasireotide LAR
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label, Multicenter, Single Arm Study of Pasireotide LAR in Patients With Rare Tumors of Neuroendocrine Origin

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Percentage of Responders at Month 6 - Pooled Pancreatic NETs (PNETs) [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline, month 6 ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline, month 6 ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline, month 6 ] [ Designated as safety issue: No ]
    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.


Enrollment: 118
Study Start Date: September 2009
Study Completion Date: June 2015
Primary Completion Date: June 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: pasireotide LAR 60mg
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.
Drug: pasireotide LAR
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.
Other Name: SOM230

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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:

    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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00958841

  Hide Study Locations
Locations
United States, California
Cedars Sinai Medical Center Cedars Sinai 4
Los Angeles, California, United States, 90048
Cedars Sinai Medical Center The Pituitary Center (3)
Los Angeles, California, United States, 90048
Stanford University Medical Center SC
Stanford, California, United States, 94304
United States, Massachusetts
Dana Farber Cancer Institute Deptof DanaFarberCancerInst(5)
Boston, Massachusetts, United States, 02215
United States, New York
Mount Sinai School of Medicine Study Coordinator
New York, New York, United States, 10029
United States, Washington
Swedish Medical Center Dept.ofSwedishMedicalCtr.(2)
Seattle, Washington, United States
Argentina
Novartis Investigative Site
Buenos Aires, Argentina, C1264AAA
Australia, New South Wales
Novartis Investigative Site
Westmead, New South Wales, Australia, 2145
Australia, Victoria
Novartis Investigative Site
Fitzroy, Victoria, Australia, 3065
Brazil
Novartis Investigative Site
Fortaleza, CE, Brazil, 60430-370
Novartis Investigative Site
Belo Horizonte, MG, Brazil, 30130-100
Novartis Investigative Site
Botucatu, SP, Brazil, 18618-970
Canada, Nova Scotia
Novartis Investigative Site
Halifax, Nova Scotia, Canada, B3H 2Y9
Canada, Quebec
Novartis Investigative Site
Montreal, Quebec, Canada, H2L 2W5
France
Novartis Investigative Site
Angers, France, 49033
Novartis Investigative Site
Bron Cedex, France, 69677
Novartis Investigative Site
Le Kremlin Bicetre, France, 94275
Novartis Investigative Site
Lille Cedex, France, 59037
Novartis Investigative Site
Marseille cedex 05, France, 13385
Novartis Investigative Site
Pessac Cedex, France, 33604
Novartis Investigative Site
Reims, France, 51092
Novartis Investigative Site
Strasbourg, France, 67098
Germany
Novartis Investigative Site
Berlin, Germany, 10098
Novartis Investigative Site
Berlin, Germany, 13353
Novartis Investigative Site
Erlangen, Germany, 91054
Novartis Investigative Site
Frankfurt, Germany, 60590
Novartis Investigative Site
Muenchen, Germany, 80804
Novartis Investigative Site
Ulm, Germany, 89081
Novartis Investigative Site
Würzburg, Germany, 97080
Italy
Novartis Investigative Site
Ancona, AN, Italy, 60126
Novartis Investigative Site
Cona, FE, Italy, 44100
Novartis Investigative Site
Padova, PD, Italy, 35128
Novartis Investigative Site
Pisa, PI, Italy, 56124
Novartis Investigative Site
Roma, RM, Italy, 00168
Mexico
Novartis Investigative Site
México, Distrito Federal, Mexico, 14269
Russian Federation
Novartis Investigative Site
Moscow, Russian Federation, 115478
Novartis Investigative Site
Moscow, Russian Federation, 117036
Novartis Investigative Site
Saint-Petersburg, Russian Federation, 197341
Spain
Novartis Investigative Site
Malaga, Andalucia, Spain, 29010
Novartis Investigative Site
Barcelona, Catalunya, Spain, 08036
Thailand
Novartis Investigative Site
Bangkok, Thailand, 10330
Novartis Investigative Site
Bangkok, Thailand, 10700
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00958841     History of Changes
Other Study ID Numbers: CSOM230D2203  2008-007348-32 
Study First Received: July 22, 2009
Results First Received: April 5, 2016
Last Updated: June 15, 2016
Health Authority: United States: Food and Drug Administration
Brazil: Ministry of Health
Canada: Health Canada
Denmark: Danish Medicines Agency
Finland: Finnish Medicines Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: BfArM
Israel: Ministry of Health
Italy: The Italian Medicines Agency
Norway: Norwegian Medicines Agency
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Spain: Spanish Agency of Medicines
Sweden: Medical Products Agency
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Novartis:
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

Additional relevant MeSH terms:
Syndrome
Neoplasms
Pituitary Diseases
Pancreatic Neoplasms
Cardiac Complexes, Premature
Pituitary Neoplasms
Nelson Syndrome
ACTH Syndrome, Ectopic
Cushing Syndrome
Disease
Pathologic Processes
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Endocrine System Diseases
Digestive System Neoplasms
Neoplasms by Site
Endocrine Gland Neoplasms
Digestive System Diseases
Pancreatic Diseases
Arrhythmias, Cardiac
Heart Diseases
Cardiovascular Diseases
Hypothalamic Neoplasms
Supratentorial Neoplasms
Brain Neoplasms
Central Nervous System Neoplasms
Nervous System Neoplasms
ACTH-Secreting Pituitary Adenoma

ClinicalTrials.gov processed this record on July 28, 2016