Back to resultsAn Extension Study to Assess the Long-Term Safety and Efficacy of Pasireotide in Participants With Acromegaly
Primary Purpose
Acromegaly
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Pasireotide
Sponsored by
About this trial
This is an interventional treatment trial for Acromegaly focused on measuring Acromegaly, Pasireotide, GH, IGF-1
Eligibility Criteria
Inclusion Criteria: Participants who have completed all four treatment regimens in the core study CSOM230B2201 (NCT00088582) and achieved biochemical control in growth hormone (GH) and insulin-like growth factor-1 (IGF-1) levels after at least one month of pasireotide administration at any of the three doses. Participants who did not experience any unacceptable adverse events or tolerability issues during the core study CSOM230B2201. Exclusion Criteria: Participants who experienced or developed compression of the optic chiasm causing any visual field defect during the core study CSOM230B2201. Participants who required a surgical intervention for relief of any sign or symptom associated with tumor compression during the core study CSOM230B2201. Participants who experienced or developed congestive heart failure, unstable angina, sustained ventricular tachycardia, ventricular fibrillation or acute myocardial infraction during the core study CSOM230B2201. Other protocol-defined inclusion/exclusion criteria may apply.
Sites / Locations
- Cedars Sinai Medical Center Dept. of Pituitary Ctr.
- University of Michigan Health System StudyCoordinatorCSOM230B2201E1
- NYU / VA Medical Center
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Pasireotide s.c. Overall
Arm Description
Participants received pasireotide as a daily subcutaneous (s.c) injection, every 12 hours at 9:00 AM and 9:00 PM at the dose at which the biochemical control was achieved (either 200, 400, or 600 microgram (μg)) for as long as the participant benefited from the treatment, and there were no safety or tolerability concerns (median duration of 22.7 months).
Outcomes
Primary Outcome Measures
Percentage of Participants With Growth Hormone (GH) and Insulin-like Growth Factor 1 (IGF-1) Observed Response by Dose Class
A participant was a responder to a dose level if the mean GH level after dosing (t30, t60, t90, and t120) was below/equal to 2.5 microgram/litre (μg/L), and if the mean of IGF-1 of the two pre-dose values (t-30, t-1) was within normal limits for age-sex matched controls. If three or more of t30, t60, t90, or t120 were missing, mean GH was considered missing. If either t-30 or t-1 was missing, mean IGF-1 was considered missing. Pasireotide incident dose classes were defined by total daily doses ranges (<1200 μg/d, 1200 to <1500 μg/d, ≥ 1500 μg/d).
Secondary Outcome Measures
Time to Tumor Response
Time to tumor response was defined as time from Sandostatin baseline (core study baseline) to at least 20% decrease in tumor volume.
Summary Magnetic Resonance Imaging (MRI) Pituitary Tumor Volumes
Pituitary Tumor Volumes were assessed by MRI. Core study baseline was defined as the last non-missing observation prior to the start of Sandostatin s.c. treatment.
Percentage of Participants With Symptoms of Acromegaly
Participants scored the following symptoms of acromegaly: Headache, perspiration, paresthesia, fatigue, osteoarthralgia, and carpal tunnel syndrome on a 5-point scale (0 = None/absent, 1 = Mild, 2 = Moderate, 3 = Severe, 4 = Very severe).
Percentage of Participants With Sleep Apnea Symptoms as Assessed by Epworth Sleepiness Scale by Situation
Sleep apnea symptoms were assessed using the Epworth Sleepiness Scale (ESS). The ESS is a self-administered questionnaire with 8 questions. It provides a measure of a person's general level of daytime sleepiness, or average sleep propensity in daily life. Percentage of participants were reported in 8 different situations: sitting and reading; watching TV; sitting, inactive in a public place; passenger in a car, an hour without break; lying down to rest in the afternoon; sitting and talking to someone; sitting quietly after a lunch without alcohol; and in a car, stopped a few minutes in the traffic. The participants were rated: 0 = would never doze, 1 = slight chance of dozing, 2 = moderate chance of dozing, 3 = high chance of dozing. Higher scores indicate more severe daytime sleepiness.
Percentage of Participants With One or More Adverse Events (AEs)
An AE was any undesirable sign, symptom or medical condition that occurred after starting study drug even if the event was not considered to be related to study drug. Percentage of participants with any AE were categorized by pasireotide incident dose classes, which were defined by total daily doses ranges (<1200 μg/d, 1200 to <1500 μg/d, ≥ 1500 μg/d).
Full Information
NCT ID
NCT00171730
First Posted
September 13, 2005
Last Updated
September 1, 2021
Sponsor
Novartis Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT00171730
Brief Title
An Extension Study to Assess the Long-Term Safety and Efficacy of Pasireotide in Participants With Acromegaly
Official Title
Extension to a Multi-Center, Randomized, Crossover, Open Label, Dose Finding Study to Compare the Safety, Efficacy, and Pharmacokinetics/Pharmacodynamics (PK/PD) Relationship of Multiple Doses of Pasireotide (SOM230) (200, 400, and 600 μg Bid) and Doses of Open Label Sandostatin® (SMS) (100 μg Tid) in Acromegalic Patients
Study Type
Interventional
2. Study Status
Record Verification Date
September 2021
Overall Recruitment Status
Completed
Study Start Date
August 24, 2004 (Actual)
Primary Completion Date
December 6, 2013 (Actual)
Study Completion Date
December 6, 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Acromegaly is a rare, serious condition characterized by chronic hypersecretion of growth hormone (GH), generally caused by a GH-secreting pituitary adenoma. The study assessed the long-term safety and efficacy of pasireotide in participants with acromegaly.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acromegaly
Keywords
Acromegaly, Pasireotide, GH, IGF-1
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Pasireotide s.c. Overall
Arm Type
Experimental
Arm Description
Participants received pasireotide as a daily subcutaneous (s.c) injection, every 12 hours at 9:00 AM and 9:00 PM at the dose at which the biochemical control was achieved (either 200, 400, or 600 microgram (μg)) for as long as the participant benefited from the treatment, and there were no safety or tolerability concerns (median duration of 22.7 months).
Intervention Type
Drug
Intervention Name(s)
Pasireotide
Other Intervention Name(s)
SOM230
Primary Outcome Measure Information:
Title
Percentage of Participants With Growth Hormone (GH) and Insulin-like Growth Factor 1 (IGF-1) Observed Response by Dose Class
Description
A participant was a responder to a dose level if the mean GH level after dosing (t30, t60, t90, and t120) was below/equal to 2.5 microgram/litre (μg/L), and if the mean of IGF-1 of the two pre-dose values (t-30, t-1) was within normal limits for age-sex matched controls. If three or more of t30, t60, t90, or t120 were missing, mean GH was considered missing. If either t-30 or t-1 was missing, mean IGF-1 was considered missing. Pasireotide incident dose classes were defined by total daily doses ranges (<1200 μg/d, 1200 to <1500 μg/d, ≥ 1500 μg/d).
Time Frame
Month 9 (Month 9 visit is at the completion of six months in this extension study)
Secondary Outcome Measure Information:
Title
Time to Tumor Response
Description
Time to tumor response was defined as time from Sandostatin baseline (core study baseline) to at least 20% decrease in tumor volume.
Time Frame
Core study baseline to at least a 20% decrease in pituitary tumor volume (up to approximately 114 months)
Title
Summary Magnetic Resonance Imaging (MRI) Pituitary Tumor Volumes
Description
Pituitary Tumor Volumes were assessed by MRI. Core study baseline was defined as the last non-missing observation prior to the start of Sandostatin s.c. treatment.
Time Frame
Core study baseline, Months 9, 27, 63, 75 and 99
Title
Percentage of Participants With Symptoms of Acromegaly
Description
Participants scored the following symptoms of acromegaly: Headache, perspiration, paresthesia, fatigue, osteoarthralgia, and carpal tunnel syndrome on a 5-point scale (0 = None/absent, 1 = Mild, 2 = Moderate, 3 = Severe, 4 = Very severe).
Time Frame
Core study baseline till the last assessment of the extension study (up to approximately 114 months)
Title
Percentage of Participants With Sleep Apnea Symptoms as Assessed by Epworth Sleepiness Scale by Situation
Description
Sleep apnea symptoms were assessed using the Epworth Sleepiness Scale (ESS). The ESS is a self-administered questionnaire with 8 questions. It provides a measure of a person's general level of daytime sleepiness, or average sleep propensity in daily life. Percentage of participants were reported in 8 different situations: sitting and reading; watching TV; sitting, inactive in a public place; passenger in a car, an hour without break; lying down to rest in the afternoon; sitting and talking to someone; sitting quietly after a lunch without alcohol; and in a car, stopped a few minutes in the traffic. The participants were rated: 0 = would never doze, 1 = slight chance of dozing, 2 = moderate chance of dozing, 3 = high chance of dozing. Higher scores indicate more severe daytime sleepiness.
Time Frame
Core study baseline till the last assessment of the extension study (up to approximately 114 months)
Title
Percentage of Participants With One or More Adverse Events (AEs)
Description
An AE was any undesirable sign, symptom or medical condition that occurred after starting study drug even if the event was not considered to be related to study drug. Percentage of participants with any AE were categorized by pasireotide incident dose classes, which were defined by total daily doses ranges (<1200 μg/d, 1200 to <1500 μg/d, ≥ 1500 μg/d).
Time Frame
From start of study drug treatment up to end of study (approximately 111 months)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Participants who have completed all four treatment regimens in the core study CSOM230B2201 (NCT00088582) and achieved biochemical control in growth hormone (GH) and insulin-like growth factor-1 (IGF-1) levels after at least one month of pasireotide administration at any of the three doses.
Participants who did not experience any unacceptable adverse events or tolerability issues during the core study CSOM230B2201.
Exclusion Criteria:
Participants who experienced or developed compression of the optic chiasm causing any visual field defect during the core study CSOM230B2201.
Participants who required a surgical intervention for relief of any sign or symptom associated with tumor compression during the core study CSOM230B2201.
Participants who experienced or developed congestive heart failure, unstable angina, sustained ventricular tachycardia, ventricular fibrillation or acute myocardial infraction during the core study CSOM230B2201.
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
First Name & Middle Initial & Last Name & Degree
Novartis
Organizational Affiliation
Novartis
Official's Role
Study Chair
Facility Information:
Facility Name
Cedars Sinai Medical Center Dept. of Pituitary Ctr.
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
University of Michigan Health System StudyCoordinatorCSOM230B2201E1
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
NYU / VA Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10010
Country
United States
Facility Name
Novartis Investigative Site
City
Woolloongabba
State/Province
Queensland
ZIP/Postal Code
4102
Country
Australia
Facility Name
Novartis Investigative Site
City
Edegem
ZIP/Postal Code
2650
Country
Belgium
Facility Name
Novartis Investigative Site
City
Toulouse Cédex 4
ZIP/Postal Code
31043
Country
France
Facility Name
Novartis Investigative Site
City
Essen
ZIP/Postal Code
45122
Country
Germany
Facility Name
Novartis Investigative Site
City
Muenchen
ZIP/Postal Code
80336
Country
Germany
Facility Name
Novartis Investigative Site
City
Napoli
ZIP/Postal Code
80131
Country
Italy
Facility Name
Novartis Investigative Site
City
Lausanne
ZIP/Postal Code
1011
Country
Switzerland
12. IPD Sharing Statement
Citations:
PubMed Identifier
23529827
Citation
Petersenn S, Farrall AJ, De Block C, Melmed S, Schopohl J, Caron P, Cuneo R, Kleinberg D, Colao A, Ruffin M, Hermosillo Resendiz K, Hughes G, Hu K, Barkan A. Long-term efficacy and safety of subcutaneous pasireotide in acromegaly: results from an open-ended, multicenter, Phase II extension study. Pituitary. 2014 Apr;17(2):132-40. doi: 10.1007/s11102-013-0478-0. Erratum In: Pituitary. 2014 Apr;17(2):141. Block, Christophe [corrected to De Block, Christophe].
Results Reference
result
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An Extension Study to Assess the Long-Term Safety and Efficacy of Pasireotide in Participants With Acromegaly
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