Lack of growth hormone in the body
Conditions
Interventions
Randomized subjects will receive the study drug as assigned according to
randomization and dosing group.
Lonapegsomatropin will be provided as a lyophilized powder in single-use glass
vials requiri
Sponsors
Ascendis Pharma Endocrinology Division A/S
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: 1. Age between 23 and 75 years, inclusive, at screening. 2. AGHD Diagnosis Criteria For adult-onset AGHD: documented history of structural hypothalamic-pituitary disease, hypothalamic-pituitary surgery, cranial irradiation, 1-4 non-GH pituitary hormone deficiencies, a proven genetic cause of GHD, or traumatic brain injury (TBI). Subjects with childhood-onset GHD must have had GH axis re assessed at final height. In subjects with TBI as a cause of GHD, GHD must be confirmed by GH stimulation testing performed at least 12 months after the injury. For all subjects, documentation of test results must be available before randomization. Stimulation test protocols and results are subject to review and approval by the Medical Monitor. A. For all countries except Japan: Subjects must satisfy at least one of the following criteria: a. Insulin tolerance test: peak GH =25-=6 weeks prior to and throughout screening. 6. For subjects not on glucocorticoid replacement therapy, documentation of adequate adrenal function at screening defined as: morning (6:00-10:00AM) serum cortisol >15.0 ng/mL (measured at central laboratory) and/or Adrenocorticotrophic Hormone (ACTH) stimulation test or ITT with serum cortisol >18.0 ng/mL at or within 26 weeks prior to screening. 7. For males not on testosterone replacement therapy: morning (6:00 10:00AM) total testosterone within normal limits for age as measured by the central laboratory at screening. 8. On a stable diet and exercise regime at screening with no intention to modify diet or exercise pattern during the trial, ie, no weight reduction program intended during the trial or within the last 90 days prior to or through screening. 9. No plans to undergo bariatric surgery during the trial. 10. Normal fundoscopy
Exclusion criteria
Exclusion criteria: 1. Known Prader-Willi Syndrome and/or other genetic diseases that may have an impact on an endpoint; individual cases to be discussed by the Investigator with the Medical Monitor. 2. Diabetes mellitus at screening if any of the following criteria are met: a. Poorly controlled diabetes, defined as HbA1c >7.5% at screening according to central laboratory b. Diabetes mellitus (defined as HbA1c >=6.5% and/or fasting plasma glucose >=126 mg/dL and/or plasma glucose >=200 mg/dL two hours after oral glucose tolerance test) diagnosed <26 weeks prior to screening c. Change in diabetes regimen (includes dose adjustment) within <90 days prior and throughout screening d. Use of any diabetes drugs other than metformin and/or DPP 4 inhibitors for a cumulative duration of greater than 4 weeks within 12 months prior to screening e. Diabetes-related complications at screening (ie, nephropathy as judged by the investigator, neuropathy requiring pharmacological treatment, retinopathy stage 2 / moderate and above within 90 days prior to screening or during screening) 3. 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 c. Subjects with GHD attributed to treatment of intracranial malignant tumors or leukemia, provided that a recurrence-free survival period of at least 5 years prior to screening is documented in the subject*s file based on a Magnetic Resonance Imaging (MRI) result 4. Evidence of growth of pituitary adenoma or other benign intracranial tumor within the last 12 months before screening. 5. Subjects with acromegaly without remission / with documented remission less than 24 months prior to screening. 6. Subjects with Cushing*s disease without remission / with documented remission less than 24 months prior to screening. 7. Subjects with prior cranial irradiation or hypothalamic-pituitary surgery: the procedure took place less than 12 months prior to screening. 8. Any disease or condition that, in the judgement of the investigator, may make the subject unlikely to comply with the requirements of the trial or any condition that presents undue risk from the investigational product or procedures. 9. Participation in another interventional clinical trial involving an investigational compound within 26 weeks prior to screening or in parallel to this trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary Efficacy Endpoint: Change from baseline in trunk percent fat (as assessed by dual-energy x-ray absorptiometry [DXA]) at Week 38. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Efficacy Endpoints: • Change from baseline in trunk fat mass at Week 38 (as assessed by DXA) • Change from baseline in total body lean mass at Week 38 (as assessed by DXA) | — |
Countries
Netherlands
Outcome results
None listed