Clinical Trial: Study of Pasireotide in Patients With Rare Tumors of Neuroendocrine Origin

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

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

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 Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Percentage of Responders at Month 6 - Pooled Pancreatic NETs (PNETs) [ Time Frame: 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.


Original Primary Outcome: Change in disease specific primary biochemical tumor markers [ Time Frame: 6 months ]

Current Secondary Outcome:

  • Percentage of Responders at Month 6 - Individual NETs [ Time Frame: 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.
  • Percentage of Responders With Probability of Success at Month 6 - Individual NETs [ Time Frame: 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 [ Time Frame: 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.
  • PiNETs: Number of Patients Attaining Normalization or a More Than 50% Reduction in Primary Biochemical Tumor Marker [ Time Frame: 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.
  • Nelson's Syndrome: Number of Patients Attaining Normalization or a More Than 50% Reduction in Primary Biochemical Tumor Marker [ Time Frame: 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.


Original Secondary Outcome:

  • Change in disease related symptoms [ Time Frame: 6 months ]
  • Change in disease related symptoms [ Time Frame: assessed monthly, analyzed between baseline & month 6 ]


Information By: Novartis

Dates:
Date Received: July 22, 2009
Date Started: September 2009
Date Completion:
Last Updated: June 15, 2016
Last Verified: June 2016