|E.1 Medical condition or disease under investigation
|Medical condition(s) being investigated
|Isolated Adult Growth Hormone Deficiency
|E.1.2 Medical condition or disease under investigation
|Growth hormone deficiency
|Condition being studied is a rare disease
|E.2 Objective of the trial
|Main objective of the trial
|The primary objective of this trial is to determine the effects of 52 weeks of GH replacement therapy on visceral fat mass in adult patients with isolated growth hormone deficiency.
|Secondary objectives of the trial
|The secondary objectives of this trial are to determine the change in quality of life related to growth hormone substitution assessed by disease and non-disease specific questionnaires (QoL-AGHDA, SF 36, and EQ-5D), assessment of cognitive function, determination of body fat by anthropometric measurements, assessment of cardiovascular risk markers and assessment of the effect of GH replacement therapy on visceral fat mass in different subgroups of adult patients with isolated growth hormone deficiency. Patients suffering from isolated GHD due to surgery and / or irradiation of pituitary adenoma and suprasellar tumors will represent one subgroup and patients with a history of traumatic brain injury (TBI) or subarachnoid hemorrhage (SAH) will represent the other subgroup.
|Trial contains a sub-study
|Principal inclusion criteria
|1. Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative) has been informed of all pertinent aspects of the trial.
2. Males and females between 18 and 65 years of age.
3. Women of childbearing potential must have a negative pregnancy test at entry.
4. If female be not of child-bearing potential (i.e. surgically sterile or postmenopausal for at least one years), or be non-pregnant and using an acceptable method of birth control (such as implants, injectables, combined oral contraceptives, hormonal IUDs, sexual abstinence or vasectomised partner) for at least one month prior to the screening visit if necessary, for the duration of the study period and one month after patients last visit.
5. Prior to screening visit, an isolated GH deficiency must be determined either by a previously performed GH stimulation test or the measurement of an IGF-I value below -2 SD according to age- and sex-adjusted reference data. At participating study site, isolated GH deficiency has to be confirmed by an ITT-Test (peak GH ≤ 3 ng/mL respective in patients under 25 years of age <5ng/mL) at screening visit which has to be carried out no sooner than 12 months after surgery and / or irradiation of pituary adenoma or suprasellar tumors or incident of TBI or SAH.
6. Clinical and/or laboratory determination following clinical standard methods defined by Medical Societies for exclusion of ACTH, TSH, and LH, FSH deficiencies. Prolactin deficiency only, if detectable at all, should not be considered.
7. Patients with the following clinical diagnoses underlying isolated GHD determined in adulthood: pituitary damage resulting from functioning and non-functioning pituitary adenoma and / or suprasellar tumors (minimum time span one year between operation and study entry), a history of cranial irradiation, traumatic brain injury (history of trauma with subsequent prolonged hospitalization, minimum one year before entry into study), subarachnoid hemorrhage.
8. Patients with a QoL-AGHDA score of 8 or higher at screening visit.
9. Subjects are willing and able to comply with scheduled visits and laboratory tests.
|Principal exclusion criteria
|1. Isolated Growth hormone deficiency by childhood onset.
2. Women who are pregnant or lactating, or who are planning to become pregnant.
3. Severe renal, hepatic or cardiac diseases that cause clinical relevant metabolic changes regarding visceral fat mass or may influence, in the judgment of the investigator, cardiovascular risk factors and anthropometric parameters to be assessed in this study.
4. Diabetes mellitus type 1 or 2.
5. Anterior pituitary disease other than described under point 5 of the inclusion criteria.
6. BMI > 35.
7. Active malignancies or in anamnesis within the last 5 years.
8. GH treatment during the last 12 months.
9. Systemic pharmacological therapy with steroid corticoids for more than one week in the month before study entry, L-thyroxin, testosterone and oral estrogens other than for contraceptive purpose. Low-dose sexual hormone substitution for postmenopausal women as well as low-dose L-thyroxin for treatment of struma is permitted in case secondary hormone deficiencies are excluded.
10. Participation in any other clinical trial with investigational drugs within the past 6 months before the current study begins and/or during study participation.
11. Known drug or alcohol abuse.
13. Hospitalized patients. Stay in rehabilitation facility will not be considered as hospitalization.
14. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study.
15. Presence of any other conditions listed as contraindications or warnings in the local SPC of Genotropin®.
16. Patients with contraindication for clinical diagnostic procedures (especially ITT and MRI).
|E.5 End points
|Primary end point(s)
|The primary endpoint of this study is the change of visceral fat mass assessed by magnetic resonance imaging scanning (MRI) after 52 weeks.
|E.6 and E.7 Scope of the trial
|Scope of the trial
|Trial type and phase
|Human pharmacology (Phase I)
|First administration to humans
|Other trial type description
|Therapeutic exploratory (Phase II)
|Therapeutic confirmatory (Phase III)
|Therapeutic use (Phase IV)
|E.8 Design of the trial
|Other trial design description
|The second stage of this study is an open label design of 6 months.
| Comparator of controlled trial
|Other medicinal product(s)
The trial involves single site in the Member State concerned
| The trial involves multiple sites in the Member State concerned
|Number of sites anticipated in Member State concerned
|The trial involves multiple Member States
|Number of sites anticipated in the EEA
|E.8.6 Trial involving sites outside the EEA
|Trial being conducted both within and outside the EEA
|Trial being conducted completely outside of the EEA
| Information not present in EudraCT
|Trial has a data monitoring committee
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|This trial will be conducted in Germany only. End of Trial is defined as Last Subject Last Visit.
|E.8.9 Initial estimate of the duration of the trial
|In the Member State concerned years
|In the Member State concerned months
|In the Member State concerned days