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

  Show 42 Study Locations
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:
pituitary neoplasm
Rare tumors of neuroendocrine origin
NETs of the pancreatic
Pituitary NETs
EAS Tumors
Nelson's Syndrome
Pancreatic neoplasm
Nelson Syndrome
ectopic ACTH syndrome
pasireotide LAR
PNETs
PiNETS

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

ClinicalTrials.gov processed this record on August 24, 2016