A Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily Somatropin Product in Adults With Growth Hormone Deficiency

Completed · Phase 3 · Has a placebo group

Conditions studied: Growth Hormone Deficiency, Endocrine System Diseases, Hormone Deficiency

In brief

A 38-week dosing trial of lonapegsomatropin, a long-acting growth hormone product, administered once-a-week versus placebo-control. A daily somatropin product arm is also included to assist clinical judgement on the trial results. A total of 264 adults (males and females) with growth hormone deficiency were included. Randomization occurred in a 1:1:1 ratio (lonapegsomatropin: placebo: daily somatropin product). This is a global trial conducted in, but not limited to, the United States, Europe, and Asia.

Key facts

Study ID

NCT04615273

Run by

Ascendis Pharma Endocrinology Division A/S

People needed

264

Starts

2020-12-03

Expected to finish

2023-12-01

Last updated by the study team

2026-07-07

Who can join

Age: 23 and older, up to 80. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age between 23 and 80 years, inclusive, at screening.
• Adult Growth Hormone Deficiency (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).
• Participants with childhood-onset GHD must have had GH axis re-assessed at final height.
• In participants with TBI as a cause of GHD, GHD must be confirmed by GH -stimulation testing performed at least 12 months after the injury.
• A. For all countries except Japan: participants must have satisfied at least one of the following criteria:
• Insulin tolerance test: peak growth hormone (GH) <=5 ng/mL
• Glucagon stimulation test according to body mass index (BMI)
• i. BMI <=30 kg/m\^2: peak GH <=3 ng/mL
• ii. BMI >30 kg/m\^2: peak GH <=1 ng/mL
• Three or four pituitary axis deficiencies (i.e., adrenal, thyroid, gonadal, and/or vasopressin; not including GH) with insulin-like growth factor-1 standard deviation score (IGF-1 SDS) <= -2.0 at screening
• Macimorelin test: peak GH <=2.8 ng/mL
• Growth hormone releasing hormone (GHRH) + arginine test according to BMI:
• i. BMI <25 kg/m\^2, peak GH <11 ng/mL
• ii. BMI >=25-<=30 kg/m\^2, peak GH <8 ng/mL
• iii. BMI >30 kg/m\^2, peak GH <4 ng/mL
• B. For Japan only: Participants with AGHD and deficiency of at least one non-GH pituitary hormones need to satisfy one of the following GH stimulation tests. Participants with GHD without additional non-GH pituitary hormone deficiencies with or without and evidence of intracranial structure disorder need to satisfy at least 2 of the following stimulation tests:
• Insulin tolerance test: peak GH <=1.8 ng/mL
• Glucagon test: peak GH <=1.8 ng/mL
• Growth Hormone Releasing Peptide-2 (GHRP-2) tolerance test: peak GH <=9 ng/mL
• IGF-1 SDS <= -1.0 at screening as measured by central laboratory.
• hGH treatment naïve or no exposure to hGH therapy or GH secretagogue for at least 12 months prior to screening.
• For participants on hormone replacement therapies for any hormone deficiencies other than GH (e.g., adrenal, thyroid, estrogen, testosterone) must be on adequate and stable doses for >=6 weeks prior to and throughout screening.
• For participants not on glucocorticoid replacement therapy, documentation of adequate adrenal function at screening defined as: morning (6:00-10:00 AM) serum cortisol >15.0 mcg/dL (measured at central laboratory) and/or adrenocorticotropic hormone (ACTH) stimulation test or insulin tolerance test with serum cortisol >18.0 mcg/dL at or within 90 days prior to screening.
• For males not on testosterone replacement therapy: morning (6:00 - 10:00AM) total testosterone within normal limits for age.

You may not qualify if…

• Known Prader-Willi Syndrome and/or other genetic diseases that may have an impact on an endpoint.
• Diabetes mellitus at screening if any of the following criteria are met:
• Poorly controlled diabetes, defined as HbA1c >7.5% at screening.
• 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
• Change in diabetes regimen (includes dose adjustment) within <90 days prior and throughout screening
• Use of any diabetes drugs other than metformin and/or dipeptidyl peptidase-4 (DPP-4) inhibitors for a cumulative duration of greater than 4 weeks within 12 months prior to screening
• Diabetes-related complications at screening (i.e., 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)
• Active malignant disease or history of malignancy. Exceptions to this exclusion criterion:
• Resection of in situ carcinoma of the cervix uteri
• Complete eradication of squamous cell or basal cell carcinoma of the skin
• Participants 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 participant's file (based on a Magnetic Resonance Imaging (MRI) result for intracranial malignant tumors)
• Evidence of growth of pituitary adenoma or other benign intracranial tumor within the last 12 months before screening.
• Participants with acromegaly without remission / with documented remission less than 24 months prior to screening.
• Participants with Cushing's disease without remission / with documented remission less than 24 months prior to screening.
• Participants with prior cranial irradiation or hypothalamic-pituitary surgery: the procedure was to take place less than 12 months prior to screening.
• Estimated glomerular filtration rate (eGFR) <60 mL/min/1.73m\^2 determined based on Modification of Diet in Renal Disease (MDRD) equation.
• Hepatic transaminases (i.e., aspartate aminotransferase [AST] or alanine aminotransferase [ALT]) >3 times the upper limit of normal.
• Heart failure New York Heart Association (NYHA) class 3 or greater (NYHA 1994).
• Q-T interval, corrected by Fridericia's method (QTcF) >= 451 milliseconds on 12-lead electrocardiogram (ECG) at screening.
• Poorly controlled hypertension, defined as supine systolic blood pressure >159 mmHg and/or supine diastolic blood pressure >95 mmHg at screening.
• Cerebrovascular accident within 5 years prior to screening.
• Anabolic steroids (other than gonadal steroid replacement therapy) or oral/intravenous/intramuscular corticosteroids within 90 days prior to or throughout screening.
• Currently using or have used within 26 weeks prior to screening any weight-loss or appetite-suppressive medications including orlistat, zonisamide, lorcaserin, bupropion, topiramate, sibutramine, stimulants, glucagon-like peptide 1 (GLP-1) receptor agonists, sodium-dependent glucose cotransporters (SGLT-2) inhibitors or medications that affects IGF-1 or GH measurements including cabergoline at doses above 0.5 mg weekly or bromocriptine at doses above 20 mg weekly.
• Known history of hypersensitivity and/or idiosyncrasy to any of the test compounds (somatropin) or excipients employed in this trial.
• Known history of neutralizing anti-hGH antibodies.

Where it is running

• Ascendis Pharma Investigational Site — Phoenix, Arizona, United States
• Ascendis Pharma Investigational Site — Fresno, California, United States
• Ascendis Pharma Investigational Site — Los Angeles, California, United States
• Ascendis Pharma Investigational Site — Los Angeles, California, United States
• Ascendis Pharma Investigational Site — Palo Alto, California, United States
• Ascendis Pharma Investigational Site — Torrance, California, United States
• Ascendis Pharma Investigational Site — Chicago, Illinois, United States
• Ascendis Pharma Investigational Site — Indianapolis, Indiana, United States
• Ascendis Pharma Investigational Site — Boston, Massachusetts, United States
• Ascendis Pharma Investigational Site — Dearborn, Michigan, United States
• Ascendis Pharma Investigational Site — Rochester, Minnesota, United States
• Ascendis Pharma Investigational Site — St Louis, Missouri, United States
• Ascendis Pharma Investigational Site — Las Vegas, Nevada, United States
• Ascendis Pharma Investigational Site — Reno, Nevada, United States
• Ascendis Pharma Investigational Site — New York, New York, United States
• Ascendis Pharma Investigational Site — New York, New York, United States
• Ascendis Pharma Investigational Site — Morehead City, North Carolina, United States
• Ascendis Pharma Investigational Site — Portland, Oregon, United States
• Ascendis Pharma Investigational Site — Pittsburgh, Pennsylvania, United States
• Ascendis Pharma Investigational Site — Dallas, Texas, United States
• Ascendis Pharma Investigational Site — San Antonio, Texas, United States
• Ascendis Pharma Investigational Site — Seattle, Washington, United States
• Ascendis Pharma Investigational Site — Yerevan, Armenia
• Ascendis Pharma Investigational Site — Saint Leonards, New South Wales, Australia
• Ascendis Pharma Investigational Site — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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