An Extension Study to Assess the Long-Term Safety and Efficacy of Pasireotide in Participants With Acromegaly
- Registration Number
- NCT00171730
- Lead Sponsor
- Novartis Pharmaceuticals
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 30
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Pasireotide s.c. Overall Pasireotide 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).
- Primary Outcome Measures
Name Time Method Percentage of Participants With Growth Hormone (GH) and Insulin-like Growth Factor 1 (IGF-1) Observed Response by Dose Class Month 9 (Month 9 visit is at the completion of six months in this extension study) 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
Name Time Method Percentage of Participants With Sleep Apnea Symptoms as Assessed by Epworth Sleepiness Scale by Situation Core study baseline till the last assessment of the extension study (up to approximately 114 months) 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 to Tumor Response Core study baseline to at least a 20% decrease in pituitary tumor volume (up to approximately 114 months) 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 Core study baseline, Months 9, 27, 63, 75 and 99 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 Core study baseline till the last assessment of the extension study (up to approximately 114 months) 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 One or More Adverse Events (AEs) From start of study drug treatment up to end of study (approximately 111 months) 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).
Trial Locations
- Locations (4)
NYU / VA Medical Center
🇺🇸New York, New York, United States
Novartis Investigative Site
🇨🇭Lausanne, Switzerland
Cedars Sinai Medical Center Dept. of Pituitary Ctr.
🇺🇸Los Angeles, California, United States
University of Michigan Health System StudyCoordinatorCSOM230B2201E1
🇺🇸Ann Arbor, Michigan, United States