E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Acromegaly is a rare, serious condition characterized by chronic hypersecretion of GH (growth hormone) caused in over 95% of patients by a GH-secreting pituitary adenoma. Clinical manifestations are due to excess GH and elevated IGF-I concentrations and/or the central pressure effects of the tumor mass. Clinical features comprise structural and functional changes occurring in practically all organs. Cardiovascular disease is the main reason for morbidity and increased mortality of patients. |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 7.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10000599 |
|
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
•To assess the long-term safety and efficacy of SOM230 in patients with acromegaly |
|
E.2.2 | Secondary objectives of the trial |
•To assess PK exposure of SOM230 in patients with acromegaly •To investigate gene-expression and protein changes in blood and urine for biomarker development
|
|
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
•Male or female patients aged between 18 and 80 years. •Patients must have completed all four treatment regimens in [study 2201], and achieved biochemical control (as defined above) after at least one month of SOM230 administration at any of the three doses •The patient did not experience any unacceptable adverse events or tolerability issues during the original SOM230 treatments. •Patients who entered the original protocol with diabetes mellitus may be included, but it is recommended that the glucose levels and treatment for diabetes are monitored throughout the trial. If a patient developed diabetes or glucose intolerance during [study 2201] that did not resolve following cessation of treatment with SOM230, they are not eligible for the extension protocol. •Karnofsky Performance Status ≥ 60 •Written informed consent to participate in the extension study has been obtained •Female patients of child bearing potential who have not undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation must agree to use barrier contraception throughout the course of the extension study, and for one month after the study has ended
|
|
E.4 | Principal exclusion criteria |
•Compression of the optic chiasm causing any visual field defect. •Patient required a surgical intervention for relief of any sign or symptom associated with tumor compression. •Congestive heart failure (NYHA Class III or IV), unstable angina, sustained ventricular tachycardia, ventricular fibrillation, or acute myocardial infarction . •Symptomatic cholelithiasis. •Liver disease such as cirrhosis, chronic active hepatitis or chronic persistent hepatitis, or persistent ALT, AST, or alkaline phosphatase levels more than twice the upper limit of normal; or direct bilirubin more than 10% of the upper limit of normal. •A hypersensitivity to SOM230. •Abnormal clinical laboratory values considered by the Investigator or the Sponsor’s Medical Monitor to be clinically significant and which could affect the interpretation of the study results. •Female patients who are pregnant or lactating, or are of childbearing potential and not practicing a medically acceptable and highly effective method for birth control. If oral contraception is used, the 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 one month after the study has ended. •Patients who have developed glucose intolerance or diabetes mellitus that does not resolve once SOM230 treatment is stopped.
|
|
E.5 End points |
E.5.1 | Primary end point(s) | |
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
|
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
Response rate based on the mean GH levels after dosing and the mean IGF-1 levels before dosing |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 1 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 1 |