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Phase 2 N=33 Randomized Treatment

Efficacy and Safety of Pasireotide LAR (Long-acting Release) in Japanese Patients With Acromegaly or Pituitary Gigantism

Acromegaly · Pituitary Gigantism

Enrolled (actual)
33
Serious AEs
33.3%
Results posted
Sep 2019
Primary outcome: Primary: Total-group Response Rate at Month 3 — 18.2 Percentage of participants

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Pasireotide LAR (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Novartis Pharmaceuticals
Primary completion
Apr 2017

Outcome Measures

OutcomeResultp-value
PRIMARY
Total-group Response Rate at Month 3
18.2
SECONDARY
Response Rate at Month 3 by Randomized Dose Level
9.1; 36.4; 9.1
SECONDARY
GH Response at Month 3 by Randomized Dose
27.3; 45.5; 18.2
SECONDARY
IGF-1 Response at Month 3 by Randomized Dose
18.2; 45.5; 9.1
SECONDARY
Total-group Response Rate (GH & IGF-1) Over Time (Core Phase)
18.2; 21.2; 21.2; 15.2
SECONDARY
Percentage of Overall Participants With the Reduction of GH Levels to <2.5 ug/L by Visit (Core Phase)
30.3; 33.3; 30.3; 36.4
SECONDARY
Percentage of Overall Participants With the Normalization of IGF-1 by Visit (Core Phase)
24.2; 33.3; 39.4; 27.3
SECONDARY
Summary of Pasireotide LAR PK Parameters of Ctrough & Cmax by Randomized Dose Level
5.36; 8.41; 10.8; 5.86; 10.7; 15.0
SECONDARY
Summary of Pasireotide LAR PK Parameter of Accumulation Ratio Randomized Dose Level
1.33; 1.85; 1.64
SECONDARY
Change of Tumor Volume From Baseline
-21.0; -1.00
SECONDARY
Change in Mean GH Levels From Baseline
-7.60; -5.98; -7.27; -8.83; -8.90; -15.68
SECONDARY
Change in Ring Size From Baseline
-1.00; -2.00; -2.00; -2.00
SECONDARY
Number of Participants With Acromegaly Symptoms or Pituitary Gigantism (Core Phase)
9; 6; 10; 1; 4; 0
SECONDARY
Change From Baseline in Prolactin
-43.00; 0.00; 0.00; 0.00
SECONDARY
Total-group Response Rate by Visit (Extension Phase)
12.5; 30.4
SECONDARY
Percentage of Overall Participants With the Reduction of Mean GH Levels to <2.5 ug/L by Visit (Extension Phase)
41.7; 60.9
SECONDARY
Percentage of Overall Participants With the Normalization of IGF-1 by Visit (Extension Phase)
16.7; 30.4
SECONDARY
Change From Baseline in Mean GH by Visit and SSA Uncontrolled Status (Extension Phase)
-8.84; -6.62; -6.71; -5.04; -8.15; -6.35

Summary

To evaluate efficacy, safety, pharmacokinetics and pharmacodynamics of pasireotide LAR in Japanese patients with active acromegaly or pituitary gigantism. Primary objective was to assess the total-group efficacy of pasireotide LAR on the reduction of mean GH levels to < 2.5 µg/L and the normalization of insulin-like growth factor-1 (IGF-1) at 3 months of study treatment.

Eligibility Criteria

Inclusion Criteria

  • Patients with medication naïve acromegaly or pituitary gigantism
  • Patients with inadequately controlled acromegaly or pituitary gigantism

Exclusion Criteria

  • Diabetic patients whose blood glucose is poorly controlled as evidenced by HbA1c >8%
  • Patients who have congestive heart failure (NYHA Class III or IV), unstable angina, sustained ventricular tachycardia, ventricular fibrillation, clinically significant bradycardia, advanced heart block or a history of acute myocardial infarction within the six months preceding enrollment
  • Patients with risk factors for torsade de pointes, i.e. patients with a baseline QTcF > 470 ms, hypokalemia, hypomagnesemia, hypocalcemia, family history of long QT syndrome, or patients receiving a concomitant medication known to prolong QT interval
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01673646). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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