E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Growth hormone deficiency in adults |
|
E.1.1.1 | Medical condition in easily understood language |
Growth hormone deficiency in adults |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Hormonal diseases [C19] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10056438 |
E.1.2 | Term | Growth hormone deficiency |
E.1.2 | System Organ Class | 10014698 - Endocrine disorders |
|
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 demonstrate the efficacy of once weekly dosing of NNC0195-0092 compared to placebo after 34 weeks of treatment in adults with growth hormone deficiency |
|
E.2.2 | Secondary objectives of the trial |
1. To evaluate the clinical safety of once weekly dosing of NNC0195-0092 during 34 weeks of treatment in adults with growth hormone deficiency
2. To evaluate the efficacy and safety of NNC0195-0092 for up to 86 weeks of treatment in adults with growth hormone deficiency (i.e. during the main and extension periods of the trial) |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female of at least 23 years of age and not more than 79 years of age at the time of signing informed consent
2. hGH treatment naïve or no exposure to hGH or GH secretagogues for at least 180 days prior to randomisation with any registered or investigational hGH or GH secretagogue product (if only used in connection with stimulation tests for diagnosis of GHD, subjects can be included)
3. If applicable, hormone replacement therapies for any other hormone deficiencies, adequate and stable for at least 90 days prior to randomisation as judged by the investigator
4. FOR ALL COUNTRIES EXCEPT JAPAN:
- Confirmed diagnosis of adult growth hormone deficiency. Subjects must satisfy at least one of the following criteria, and documentation of test results must be available before randomisation (either from subject´s file or new test):
a. Insulin tolerance test (ITT) or glucagon test: a peak GH response of < 3 ng/mL (3 μg/L)
b. Growth hormone releasing hormone (GHRH) + arginine test according to body mass index (BMI)
i. BMI< 25 kg/m^2, a peak GH < 11 ng/mL (μg/L)
ii. BMI 25–30 kg/m^2, a peak GH < 8 ng/mL (8 μg/L)
iii. BMI > 30 kg/m^2, a peak GH < 4 ng/mL (4 μg/L )
c. Three or more pituitary hormone deficiencies and IGF-I SDS < -2.0
FOR JAPAN ONLY: Confirmed diagnosis of adult growth hormone deficiency (subjects with adult onset AGHD need to satisfy at least one of the following criteria, subjects with a history of childhood GHD need to satisfy at least 2 of the following criteria):
a. ITT test: a peak GH of ≤ 1.8 ng/mL (assay using recombinant GH standard)
b. glucagon test: a peak GH of ≤ 1.8 ng/mL (assay using recombinant GH standard)
c. GHRP-2 tolerance test: a peak GH of ≤ 9 ng/mL (assay using recombinant GH standard) |
|
E.4 | Principal exclusion criteria |
Active malignant disease or history of malignancy. Exceptions to this exclusion criterion:
a) Resection of in situ carcinoma of the cervix uteri
b) Complete eradication of squamous cell or basal cell carcinoma of the skin
Subjects with GHD attributed to treatment of intracranial malignant tumours or leukaemia, provided that a recurrence-free survival period of at least 5 years is documented in the subject’s file |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Change in truncal fat percentage |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
From baseline to end of main treatment period (Week 34) |
|
E.5.2 | Secondary end point(s) |
Key secondary endpoints for efficacy
Changes in the following key variables will be used to address the primary objective:
1. Change in truncal fat mass (kg)
2. Change in truncal lean body mass (kg)
Key secondary endpoints for safety
The following key endpoints will be used to support the secondary objectives of evaluation of safety:
3. Incidence of adverse events, including injection site reactions
4. Occurrence of anti-NNC0195-0092 antibodies |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. + 2. From baseline to end of main treatment period (Week 34)
3. + 4. In both the main trial period (up to week 35) and extension trial period (up to week 88) (including follow-up visits/washout periods) |
|
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 | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
|
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Placebo-controlled (double blind) and active-controlled (open) |
|
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Yes |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 3 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 18 |
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 |
Australia |
Brazil |
European Union |
India |
Japan |
Malaysia |
Russian Federation |
South Africa |
Turkey |
Ukraine |
United States |
|
E.8.7 | Trial has a data monitoring committee | No |
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
|
|
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 | 2 |
E.8.9.1 | In the Member State concerned days | 19 |
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 | 2 |
E.8.9.2 | In all countries concerned by the trial days | 19 |