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Study of Management of Pasireotide-induced Hyperglycemia in Adult Patients With Cushing's Disease or Acromegaly

Registration Number
NCT02060383
Lead Sponsor
Novartis Pharmaceuticals
Brief Summary

The study was designed to investigate the optimal management of hyperglycemia developed during pasireotide treatment in participants with Cushing's disease or Acromegaly, which was not manageable with metformin.

This was a Phase IV, multi-center, randomized, open-label study. Eligible patients started pasireotide subcutaneously (s.c.) for Cushing's disease and pasireotide LAR (long-acting release) for Acromegaly.

Participants being treated with pasireotide s.c or LAR at screening were eligible as long as they met protocol criteria during the screening period. If previously normo-glycemic participants experienced an increase in their fasting blood glucose and met the criteria for diabetes while on pasireotide, they started anti-diabetic treatment using metformin. If they continued to have elevated blood glucose above target on metformin within the first 16 weeks, they were randomized in a 1:1 ratio to receive treatment with incretin based therapy or insulin for approximately 16 weeks.

Participants who continued to receive clinical benefit after completing the Core Phase could enter an optional Extension Phase if pasireotide was not commercially available in their country or a local access program was not available to provide drug. Patients continued in the Extension Phase until the last participant randomized in the Core Phase completed 16 weeks of treatment post-randomization.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
249
Inclusion Criteria
  • Patients greater than or equal to 18 years old
  • Confirmed diagnosis of Cushing's disease or acromegaly
Exclusion Criteria
  • Patients who require surgical intervention
  • Patients receiving DPP-4 inhibitors or GLP-1 receptor agonists within 4 weeks prior to study entry
  • HbA1c > 10 % at screening
  • Known hypersensitivity to somatostatin analogues Other protocol-defined inclusion/exclusion criteria may apply.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Non-Randomized ArmPasireotide s.c.This arm represents the non-randomized participants: Cushing's Disease (CD) or Acromegaly participants, who received pasireotide s.c. or LAR (long-acting release) respectively, but who were not randomized to the Incretin or Insulin arms. For the purpose of analysis, this non-randomized arm is further split into 3 groups: * Baseline insulin group (BL insulin) includes participants who were receiving insulin at study entry * Oral antidiabetic drugs (OAD) group includes participants who developed hyperglycemia that was controlled by metformin and/or other background anti-diabetic treatment * No OAD group includes participants who did not receive any anti-diabetic medication during the core phase of the trial
Incretin based therapy (randomized group)Pasireotide LARParticipants randomized to the incretin based arm started with sitagliptin once daily. If sitagliptin did not control the participant's hyperglycemia, sitagliptin was stopped and participants switched to liraglutide once daily. If despite treatment with liraglutide, hyperglycemia was not controlled then the participant was eligible for rescue therapy with addition of insulin.
Incretin based therapy (randomized group)Pasireotide s.c.Participants randomized to the incretin based arm started with sitagliptin once daily. If sitagliptin did not control the participant's hyperglycemia, sitagliptin was stopped and participants switched to liraglutide once daily. If despite treatment with liraglutide, hyperglycemia was not controlled then the participant was eligible for rescue therapy with addition of insulin.
Insulin (randomized group)Pasireotide s.c.Participants randomized to the insulin arm started with once daily dose of basal insulin. The dose was up or down titrated at the discretion of the investigator. If blood glucose levels remained uncontrolled on basal insulin, participant switched to basal insulin plus prandial insulin.
Non-Randomized ArmInsulinThis arm represents the non-randomized participants: Cushing's Disease (CD) or Acromegaly participants, who received pasireotide s.c. or LAR (long-acting release) respectively, but who were not randomized to the Incretin or Insulin arms. For the purpose of analysis, this non-randomized arm is further split into 3 groups: * Baseline insulin group (BL insulin) includes participants who were receiving insulin at study entry * Oral antidiabetic drugs (OAD) group includes participants who developed hyperglycemia that was controlled by metformin and/or other background anti-diabetic treatment * No OAD group includes participants who did not receive any anti-diabetic medication during the core phase of the trial
Insulin (randomized group)Pasireotide LARParticipants randomized to the insulin arm started with once daily dose of basal insulin. The dose was up or down titrated at the discretion of the investigator. If blood glucose levels remained uncontrolled on basal insulin, participant switched to basal insulin plus prandial insulin.
Non-Randomized ArmPasireotide LARThis arm represents the non-randomized participants: Cushing's Disease (CD) or Acromegaly participants, who received pasireotide s.c. or LAR (long-acting release) respectively, but who were not randomized to the Incretin or Insulin arms. For the purpose of analysis, this non-randomized arm is further split into 3 groups: * Baseline insulin group (BL insulin) includes participants who were receiving insulin at study entry * Oral antidiabetic drugs (OAD) group includes participants who developed hyperglycemia that was controlled by metformin and/or other background anti-diabetic treatment * No OAD group includes participants who did not receive any anti-diabetic medication during the core phase of the trial
Incretin based therapy (randomized group)InsulinParticipants randomized to the incretin based arm started with sitagliptin once daily. If sitagliptin did not control the participant's hyperglycemia, sitagliptin was stopped and participants switched to liraglutide once daily. If despite treatment with liraglutide, hyperglycemia was not controlled then the participant was eligible for rescue therapy with addition of insulin.
Insulin (randomized group)InsulinParticipants randomized to the insulin arm started with once daily dose of basal insulin. The dose was up or down titrated at the discretion of the investigator. If blood glucose levels remained uncontrolled on basal insulin, participant switched to basal insulin plus prandial insulin.
Incretin based therapy (randomized group)SitagliptinParticipants randomized to the incretin based arm started with sitagliptin once daily. If sitagliptin did not control the participant's hyperglycemia, sitagliptin was stopped and participants switched to liraglutide once daily. If despite treatment with liraglutide, hyperglycemia was not controlled then the participant was eligible for rescue therapy with addition of insulin.
Incretin based therapy (randomized group)LiraglutideParticipants randomized to the incretin based arm started with sitagliptin once daily. If sitagliptin did not control the participant's hyperglycemia, sitagliptin was stopped and participants switched to liraglutide once daily. If despite treatment with liraglutide, hyperglycemia was not controlled then the participant was eligible for rescue therapy with addition of insulin.
Incretin based therapy (randomized group)MetforminParticipants randomized to the incretin based arm started with sitagliptin once daily. If sitagliptin did not control the participant's hyperglycemia, sitagliptin was stopped and participants switched to liraglutide once daily. If despite treatment with liraglutide, hyperglycemia was not controlled then the participant was eligible for rescue therapy with addition of insulin.
Insulin (randomized group)MetforminParticipants randomized to the insulin arm started with once daily dose of basal insulin. The dose was up or down titrated at the discretion of the investigator. If blood glucose levels remained uncontrolled on basal insulin, participant switched to basal insulin plus prandial insulin.
Non-Randomized ArmMetforminThis arm represents the non-randomized participants: Cushing's Disease (CD) or Acromegaly participants, who received pasireotide s.c. or LAR (long-acting release) respectively, but who were not randomized to the Incretin or Insulin arms. For the purpose of analysis, this non-randomized arm is further split into 3 groups: * Baseline insulin group (BL insulin) includes participants who were receiving insulin at study entry * Oral antidiabetic drugs (OAD) group includes participants who developed hyperglycemia that was controlled by metformin and/or other background anti-diabetic treatment * No OAD group includes participants who did not receive any anti-diabetic medication during the core phase of the trial
Primary Outcome Measures
NameTimeMethod
Change in HbA1c From Randomization to Approximately 16 WeeksRandomization, 16 weeks

Absolute change in HbA1c from randomization to end of core phase (16 weeks) in incretin based therapy arm and insulin arm, and mean difference of change in HbA1c between the two treatment groups based on an ANOVA model using treatment (Incretin, Insulin) and the two randomization stratification factors (Disease: Cushing's disease vs Acromegaly; Baseline glycemic status: HbA1c \<7% vs HbA1c ≥ 7%) as fixed effects. For Participants who discontinued the study or required rescue treatment before the time of assessing the primary endpoint, the last HbA1c assessment collected 8 weeks (56 days) after randomization (and prior to or on the date of start of rescue treatment) was carried forward. If the participant discontinued the study or used rescue treatment within 8 weeks after randomization, it was considered missing.

Secondary Outcome Measures
NameTimeMethod
Percentage of Participants in the Incretin-based Arm Who Required Anti-diabetic Rescue Therapy With InsulinRandomization to up to 16 weeks

The percentage of participants who received anti-diabetic rescue therapy in incretin based therapy is summarized.

Change in HbA1c From Randomization (R) Over Time Per Randomized ArmRandomization (R), Week (W) 4 post R, W 8 post R, W 16 post R, end of Core phase (up to week 16 post R)

Absolute change in HbA1c overtime from randomization (i.e. start of randomized antidiabetic treatment) to end of core phase per randomized arm

Change in FPG (Fasting Plasma Glucose) From Randomization Until End of Core PhaseRandomization, R(randomization) Week 2, R-Week 4, R-Week 6, R-Week 8, R-Week 10, R-Week 12, R-Week 14, R-Week 16, end of Core phase

Absolute change in fasting glucose overtime from randomization (i.e. start of randomized antidiabetic treatment) to end of core phase per randomized arm

Absolute Change in FPG From Baseline to End of Core PhaseBaseline, Up to 32 weeks (end of Core Phase)

Absolute change in FPG from baseline to end of core phase in the incretin based therapy arm and the insulin arm.

Absolute Change in HbA1c From Baseline to End of Core PhaseBaseline, up to 32 weeks (end of Core phase)

Absolute change in HbA1c from baseline to end of core phase in the incretin based therapy arm and the insulin arm

Percentage of Participants With ≤ 0.3% HbA1c Increase to End of Core PhaseRandomization, up to 16 weeks

Percentage of participants with ≤ 0.3% HbA1c increase in the incretin based therapy arm and the insulin arm.

Trial Locations

Locations (16)

LA Biomedical Research at Harbor UCLA Medical Center SC - SOM230B2219

🇺🇸

Torrance, California, United States

Diabetes and Endocrine Associates La Mesa Location

🇺🇸

Multiple Locations, California, United States

East Coast Institute for Research East Coast Inst. for Res(ECIR)

🇺🇸

Jacksonville, Florida, United States

Coastal Metabolic Research Centre SC

🇺🇸

Ventura, California, United States

Washington University SC - SOM230B2411

🇺🇸

Saint Louis, Missouri, United States

Robert Wood Johnson Medical School - Rutgers SC

🇺🇸

New Brunswick, New Jersey, United States

The Mount Sinai Hospital SC

🇺🇸

New York, New York, United States

Lenox Hill Hospital/Manhattan Eye, Ear and Throat Hospital SC

🇺🇸

New York, New York, United States

Columbia University Medical Center New York Presbyterian Neuroendocrine Unit

🇺🇸

New York, New York, United States

Virginia Endocrinology Research SC-2

🇺🇸

Chesapeake, Virginia, United States

Allegheny Endocrinology Associates SC

🇺🇸

Pittsburgh, Pennsylvania, United States

Baylor College of Medicine Ben Taub General Hosp.

🇺🇸

Houston, Texas, United States

Swedish Medical Center Dept.ofSeattle Neuroscience(2)

🇺🇸

Seattle, Washington, United States

Great Falls Clinic

🇺🇸

Great Falls, Montana, United States

Novartis Investigative Site

🇹🇷

Antalya, Turkey

Vanderbilt Clinical Trials Center SOM230B2219

🇺🇸

Nashville, Tennessee, United States

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