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Study Summary
No. 2008-0048:.......Endocrine......Steven G. Waguespack......Endocrine Neoplasia & Hormonal Disorders
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Study Summary Title
Study Summary
Number:
2008-0048
Study Title:A multicenter, randomized, blinded study to assess safety and efficacy of pasireotide LAR
vs. octreotide LAR in patients with active acromegaly
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Physician New Patient Referral
Name:Steven G. WaguespackPatients Call:800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Dept:Endocrine Neoplasia & Hormonal DisordersReferring MD
Call:
800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Phone:713-792-2841
Contact us about clinical trials
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General Information
Disease Group:EndocrineSupported By:Novartis Pharmaceuticals Corporation
Phase of Study:Phase IIIReturn
Visit:
There will be 18 outpatient visits for the main study. The extension study
will require more visits as outlined in the protocol.
Treatment
Agents:
Octreotide
SOM230
Home Care:N/A
Treatment Loc:Both at MDACC & outside MDACC at one or more Collaborating Sites or Institutions
Estimated
Length of Stay
in Houston:
N/A
Description/
Intervention:
This goal of this clinical research study is to compare an investigational
treatment for acromegaly against the standard treatment for acromegaly.
Pasireotide long-acting release (LAR), which is also known as som230 LAR, will
be compared against Sandostatin® LAR (octreotide LAR).

Researchers want to learn if pasireotide LAR can help to control acromegaly.
The safety of pasireotide LAR will also be studied.
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Study Objectives / Outcomes
Primary Objective:
1) To compare the proportion of patients with a reduction of mean growth hormone (GH) level to < 2.5 µg/L and the normalization of insulin-like growth factor 1 (IGF-1) to within normal limits (sex and age related) between the two treatment groups at 12 months.

Secondary Objectives:
1) To compare the effect of pasireotide LAR vs. octreotide LAR on reduction of GH to < 2.5 µg/L
alone at 12 months
2) To compare the effect of pasireotide LAR vs. octreotide LAR on tumor volume at 12 months
3) To compare the effect of pasireotide LAR vs. octreotide LAR on normalization of IGF-1 alone at 12 months
4) To assess the effect of pasireotide LAR vs. octreotide LAR on symptoms of
acromegaly, specifically: ring size, headache, fatigue, perspiration, paresthesias,
osteoarthralgia at 12 months
5) To assess the effect of pasireotide LAR vs. octreotide LAR on health related quality of
Life at 12 months
6) To assess the effect of pasireotide LAR vs. octreotide LAR on prolactin (PRL) levels at 12
months
7) To assess the overall safety and tolerability of pasireotide LAR vs. octreotide LAR at 12 months
8) To assess pasireotide and octreotide plasma exposure (trough concentrations)
9) To assess the effect of pasireotide LAR vs. octreotide LAR on the proportion of patients
with a reduction of mean GH level to < 2.5 µg/L and on the normalization of IGF-1 to
within normal limits (age and sex related) at months 6 and 9
10)To evaluate the effect of pasireotide LAR vs. octreotide LAR on duration of
response for patients achieving a reduction of mean GH level to < 2.5 µg/L
and the normalization of IGF-1 to within normal limits (age and sex
related) at month 12
11) To assess the effect of pasireotide LAR vs. octreotide LAR on change from baseline in
mean GH at 12 months
12) To assess the effect of pasireotide LAR vs. octreotide LAR on time to first achievement of
mean GH < 2.5 µg/L and the normalization of IGF-1 to within normal limits (age and
sex related).
    Exploratory Objectives:
    1) To explore patient demographics (e.g. age, BMI, sex) as pharmacokinetic (PK) covariates on pasireotide/octreotide trough concentrations, if applicable
    2) To explore the correlation between pasireotide/octreotide trough concentrations and pharmacodynamic (PD)/safety markers, if applicable
    3) To explore GH nadir post-oral glucose tolerance test (oGTT) with 75 g of glucose at 12 months (required only for US sites, but not applicable for diabetic patients; any other sites who have the capacity to perform thistest should also perform it for non-diabetic patients.)
    4) For patients who have had previous pituitary surgery, correlation of the expression of different somatostatin receptor subtypes with treatment response to pasireotide LAR or octreotide LAR will be explored if tissue samples are available, the patient provides consent and local legislation allows shipment of samples to the central facility.
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    Study Status Information
    Study Activation / Registration Date:
    IRB Review and Approval Date:04/17/2008
    Study Type:Phase Iii
    Recruitment Status:Closed
    Projected Accrual:330
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    Enrollment Eligibility
    If you do not meet the enrollment eligibility, there may be other treatment options for you. Please Contact the Referral Office for more information.

    Inclusion Criteria:1) Male or female patients of at least 18 years of age

    2) Patients with active acromegaly demonstrated by a lack of suppression of GH nadir to < 1 µg/L after an oral tolerance test with 75 g of glucose (OGTT) (not applicable for diabetic patients) or a mean GH concentration of a 5-point profile within a 2 hour time period of > 5 µg/L AND an elevated circulating IGF-1 concentration (age and sex adjusted)

    3) Patients who have undergone one or more pituitary surgeries, but have not been treated medically or de-novo patients presenting a visible pituitary adenoma on MRI and who refuse pituitary surgery or for whom pituitary surgery is contraindicated

    4) Patients with a known history or new diagnosis of impaired fasting glucose or diabetes mellitus may be included, however blood glucose and antidiabetic treatment must be monitored closely throughout the trial and adjusted as necessary

    5) Patients for whom written informed consent to participate in the study has been obtained prior to any study related activity.

    Exclusion Criteria:1) Patients who are being or were treated with octreotide, lanreotide or dopamine agonists with the exception of a single dose of short-acting octreotide or short-acting dopamine agonists. In case of a single dose of short-acting octreotide, the dose should not be used to predict the response to the octreotide treatment. The single dose of short acting octreotide or short-acting dopamine agonists should not be administered in the 3 days prior to randomization.

    2) Patients who are being or were treated with GH antagonists

    3) De-novo patients not having a visible pituitary adenoma on MRI

    4) Patients who have received pasireotide (SOM 230) prior to randomization

    5) Patients with compression of the optic chiasm causing any visual field defect for whom surgical intervention is indicated.

    6) Patients who require a surgical intervention for relief of any sign or symptom associated with tumor compression

    7) Patients who have undergone major surgery/surgical therapy for any cause within 4 weeks of visit 1

    8) Patients who have received radiotherapy (except for pituitary irradiation) for any reason in the 4 weeks prior to visit 1 must have recovered from any side effect of radiotherapy

    9) Patients who have received pituitary irradiation within the last ten years prior to visit 1

    10) Patients who are hypothyroid and not adequately treated with stable doses of thyroid hormone replacement therapy

    11) Diabetic patients on antidiabetic medications whose fasting blood glucose is poorly controlled as evidenced by HbA1c >8%

    12) Patients with symptomatic cholelithiasis

    13) Patients with abnormal coagulation (PT and/or APTT elevated by 30% above normal limits) or patients receiving anticoagulants that affect PT (prothrombin time) or APTT (activated partial thromboplastin time)

    14) 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

    15) Patients with risk factors for torsade de pointes, i.e. patients with a baseline QTc > 450 ms, hypokalemia, hypomagnesemia, hypocalcemia, family history of long QT syndrome, or patients receiving a concomitant medication known to prolong QT interval

    16) Patients with confirmed central hypothyroidism, central hypoadrenalism and diabetes insipidus, unless they are adequately treated with stable doses of hormone replacement therapy for a minimum of three months prior to study entry (first dose of study medication). Patients with confirmed central hypogonadism unless they are adequately treated with stable doses of hormone replacement therapy for a minimum of three months prior to study entry (first dose of study medication) except in cases where hormones replacement therapy is not indicated.

    17) Patients with liver disease such as cirrhosis, chronic active hepatitis or chronic persistent hepatitis, or patients with ALT and/or AST more than 2 x ULN, serum creatinine > 2.0 x ULN, serum bilirubin > 2 x ULN, serum albumin < 0.67 x LLN, [Chinese patients need to have normal serum creatinine level (China only)].

    18) Patients with WBC <3 x 109/L; Hgb < 90 % LLN; PLT <100 x 109/L

    19) Patients who have any current or prior medical condition that may interfere with the conduct of the study or the evaluation of its results in the opinion of the Investigator or the Sponsor's Medical Monitor

    20) Female patients who are pregnant or lactating, or are of childbearing potential and not practicing a medically acceptable method of birth control. Female patients must use one contraception method and the partner should use a condom. If oral contraception is used, the female patient must have been practicing this method for at least two months prior to the enrollment and must agree to continue the oral contraceptive throughout the course of the study and for three months after the last study drug administration. Male patients who are sexually active are required to use condoms.

    21) History of immunocompromise, including a positive HIV test result (ELISA and Western blot). An HIV test will not be required; however, previous medical history will be reviewed

    22) Patients who have participated in any clinical investigation with an investigational drug within 1 month prior to first dosing

    23) Known hypersensitivity to somatostatin analogues or any other component of the pasireotide LAR or octreotide LAR formulations

    24) Patients with active malignant disease within the last five years (with the exception of basal cell carcinoma or carcinoma in situ of the cervix)

    25) Patients with the presence of active or suspected acute or chronic uncontrolled infection

    26) Patients with a history of non-compliance to medical regimens or who are considered potentially unreliable or will be unable to complete the entire study

    27) Patients with a history of alcohol or drug abuse in the 6 month period prior to receiving the study drug

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    Links
    Registration Number: NCT00600886
    Study Information on Clinical Trials Registry (clinicaltrials.gov)

    Other Links:
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    Results


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