AcromegalyPhase 2NCT00171730
An Extension Study to Assess the Long-Term Safety and Efficacy of Pasireotide in Participants With Acromegaly
A Phase 2 study testing Pasireotide in people with Acromegaly, run by Novartis Pharmaceuticals at 10 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Acromegaly
- What is being tested?
- Pasireotide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 10 sitesin 7 countries
- Study dates
- 24 Aug 2004to 6 Dec 2013
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
10 sites in 7 countries.
10 sites in 7 countries
United States3 sites
University of Michigan Health System StudyCoordinatorCSOM230B2201E1
Ann Arbor, Michigan, 48109
Cedars Sinai Medical Center Dept. of Pituitary Ctr.
Los Angeles, California, 90048
NYU / VA Medical Center
New York, New York, 10010
Germany2 sites
Novartis Investigative Site
Essen, 45122
Novartis Investigative Site
München, 80336
About this study
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
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.
- Conditions
- Acromegaly
- Being tested
- Pasireotide
- Keywords
- Acromegaly, Pasireotide, GH, IGF-1
- Medical subject headings
- Acromegaly
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PasireotideDrug
Also called SOM230
| Study group | Role | What they receive |
|---|---|---|
| Pasireotide s.c. Overall | Experimental | 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). Receives: Drug: Pasireotide |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Month 9 (Month 9 visit is at the completion of six months in this extension study)
Secondary outcomes (5)
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.
Measured: Core study baseline to at least a 20% decrease in pituitary tumor volume (up to approximately 114 months)
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.
Measured: Core study baseline, Months 9, 27, 63, 75 and 99
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).
Measured: Core study baseline till the last assessment of the extension study (up to approximately 114 months)
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.
Measured: Core study baseline till the last assessment of the extension study (up to approximately 114 months)
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).
Measured: From start of study drug treatment up to end of study (approximately 111 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2005 (estimated)
- Study start24 Aug 2004
- Primary completion6 Dec 2013
- Study completion6 Dec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for AcromegalyStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis PharmaceuticalsNovartis, Study chair, Novartis
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00171730
- Sponsor's protocol ID
- CSOM230B2201E1
- Other IDs
- 2004-002849-12, 2004
- Status last verified
- Sep 2021
- First submitted
- 13 Sep 2005
- Last updated
- 5 Sep 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 23529827
Study at a glance
From the registry record, last updated by the sponsor on 5 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00171730
- Acronym / sponsor ID
- CSOM230B2201E1
- Conditions
- Acromegaly
- Keywords
- Acromegaly, Pasireotide, GH, IGF-1
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 24 Aug 2004 (actual)
- Primary completion
- 6 Dec 2013 (actual)
- Study completion
- 6 Dec 2013 (actual)
- First posted
- 15 Sep 2005 (estimated)
- Results
- Posted 2 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pasireotide s.c. Overall | Experimental | Drug: PasireotideParticipants 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). |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PasireotideAlso: SOM230 | Drug | Pasireotide s.c. Overall |
Primary outcome
Percentage of Participants With Growth Hormone (GH) and Insulin-like Growth Factor 1 (IGF-1) Observed Response by Dose Class
Time frame: 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).
5 secondary outcomes
Time to Tumor Response
Time frame: Core study baseline to at least a 20% decrease in pituitary tumor volume (up to approximately 114 months)
Summary Magnetic Resonance Imaging (MRI) Pituitary Tumor Volumes
Time frame: Core study baseline, Months 9, 27, 63, 75 and 99
Percentage of Participants With Symptoms of Acromegaly
Time frame: Core study baseline till the last assessment of the extension study (up to approximately 114 months)
Percentage of Participants With Sleep Apnea Symptoms as Assessed by Epworth Sleepiness Scale by Situation
Time frame: Core study baseline till the last assessment of the extension study (up to approximately 114 months)
Percentage of Participants With One or More Adverse Events (AEs)
Time frame: From start of study drug treatment up to end of study (approximately 111 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Sep 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 23529827Petersenn 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.
Related trials for Acromegaly
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RecruitingObservational study
An Observational Study of Octreotide Subcutaneous Depot in Patients With Acromegaly
Acromegaly
RecruitingPhase 3
A Study to Assess the Efficacy and Safety of Debio 4126 in Participants With Acromegaly Previously Treated With Somatostatin Analogs
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RecruitingObservational study
An Investigation of Pituitary Tumors and Related Hypothalmic Disorders
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Recruiting
Managing Pasireotide-associated Hyperglycaemia in Acromegaly With Ketogenic Diet
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
Novartis study chair
Novartis
10 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pasireotide in people with Acromegaly, run by Novartis Pharmaceuticals at 10 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Germany, Australia, Belgium, France and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Jun 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants With Growth Hormone (GH) and Insulin-like Growth Factor 1 (IGF-1) Observed Response by Dose Class (time frame: Month 9 (Month 9 visit is at the completion of six months in this extension study)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00171730, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Sep 2021.