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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00958841




Registration number
NCT00958841
Ethics application status
Date submitted
22/07/2009
Date registered
11/08/2009
Date last updated
15/06/2016

Titles & IDs
Public title
Study of Pasireotide in Patients With Rare Tumors of Neuroendocrine Origin
Scientific title
An Open Label, Multicenter, Single Arm Study of Pasireotide LAR in Patients With Rare Tumors of Neuroendocrine Origin
Secondary ID [1] 0 0
2008-007348-32
Secondary ID [2] 0 0
CSOM230D2203
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pancreatic Neoplasm 0 0
Pituitary Neoplasm 0 0
Nelson Syndrome 0 0
Ectopic ACTH Syndrome 0 0
Condition category
Condition code
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders
Neurological 0 0 0 0
Other neurological disorders
Cancer 0 0 0 0
Pancreatic
Cardiovascular 0 0 0 0
Other cardiovascular diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - pasireotide LAR

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.


Treatment: Drugs: 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.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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.
Timepoint [1] 0 0
6 months
Secondary outcome [1] 0 0
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.
Timepoint [1] 0 0
6 months
Secondary outcome [2] 0 0
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.
Timepoint [2] 0 0
6 months
Secondary outcome [3] 0 0
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.
Timepoint [3] 0 0
Baseline, month 6
Secondary outcome [4] 0 0
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.
Timepoint [4] 0 0
Baseline, month 6
Secondary outcome [5] 0 0
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.
Timepoint [5] 0 0
Baseline, month 6

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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
Purpose of the study
Treatment
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Westmead
Recruitment hospital [2] 0 0
Novartis Investigative Site - Fitzroy
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
3065 - Fitzroy
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Massachusetts
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
United States of America
State/province [4] 0 0
Washington
Country [5] 0 0
Argentina
State/province [5] 0 0
Buenos Aires
Country [6] 0 0
Brazil
State/province [6] 0 0
CE
Country [7] 0 0
Brazil
State/province [7] 0 0
MG
Country [8] 0 0
Brazil
State/province [8] 0 0
SP
Country [9] 0 0
Canada
State/province [9] 0 0
Nova Scotia
Country [10] 0 0
Canada
State/province [10] 0 0
Quebec
Country [11] 0 0
France
State/province [11] 0 0
Angers
Country [12] 0 0
France
State/province [12] 0 0
Bron Cedex
Country [13] 0 0
France
State/province [13] 0 0
Le Kremlin Bicetre
Country [14] 0 0
France
State/province [14] 0 0
Lille Cedex
Country [15] 0 0
France
State/province [15] 0 0
Marseille cedex 05
Country [16] 0 0
France
State/province [16] 0 0
Pessac Cedex
Country [17] 0 0
France
State/province [17] 0 0
Reims
Country [18] 0 0
France
State/province [18] 0 0
Strasbourg
Country [19] 0 0
Germany
State/province [19] 0 0
Berlin
Country [20] 0 0
Germany
State/province [20] 0 0
Erlangen
Country [21] 0 0
Germany
State/province [21] 0 0
Frankfurt
Country [22] 0 0
Germany
State/province [22] 0 0
Muenchen
Country [23] 0 0
Germany
State/province [23] 0 0
Ulm
Country [24] 0 0
Germany
State/province [24] 0 0
Würzburg
Country [25] 0 0
Italy
State/province [25] 0 0
AN
Country [26] 0 0
Italy
State/province [26] 0 0
FE
Country [27] 0 0
Italy
State/province [27] 0 0
PD
Country [28] 0 0
Italy
State/province [28] 0 0
PI
Country [29] 0 0
Italy
State/province [29] 0 0
RM
Country [30] 0 0
Mexico
State/province [30] 0 0
Distrito Federal
Country [31] 0 0
Russian Federation
State/province [31] 0 0
Moscow
Country [32] 0 0
Russian Federation
State/province [32] 0 0
Saint-Petersburg
Country [33] 0 0
Spain
State/province [33] 0 0
Andalucia
Country [34] 0 0
Spain
State/province [34] 0 0
Catalunya
Country [35] 0 0
Thailand
State/province [35] 0 0
Bangkok

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study will assess the effectiveness and safety of pasireotide long-acting release in
patients who have rare tumors of neuroendocrine origin.
Trial website
https://clinicaltrials.gov/show/NCT00958841
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT00958841