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Weekly shot could replace daily hormone injections for growth hormone deficiency

NCT ID NCT04615273

Disease control Sponsor: Ascendis Pharma Endocrinology Division A/S Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 07, 2026 · Last updated Jul 08, 2026 · Updated 1 time

Summary

This phase 3 trial investigates whether a once-weekly injection of lonapegsomatropin, a long-acting growth hormone, can safely and effectively treat adults with growth hormone deficiency. The study compares the weekly drug against a placebo and a standard daily growth hormone product. Researchers measure changes in body fat and lean muscle mass over 38 weeks to assess the treatment's impact.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
lonapegsomatropin (a long-acting growth hormone)
What this could lead to
If successful, this could offer adults with growth hormone deficiency a more convenient once-weekly injection instead of daily shots, potentially improving treatment adherence and quality of life.
What could go wrong
This is a phase 3 trial, so while promising, the long-acting hormone may not prove as effective or safe as daily therapy. Side effects from growth hormone treatment, such as joint pain or fluid retention, remain possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

264 people

The number who actually took part.

Started

Dec 2020

Finished

Dec 2023

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

23 to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria 1. Age between 23 and 80 years, inclusive, at screening. 2. 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: 1. Insulin tolerance test: peak growth hormone (GH) \<=5 ng/mL 2. 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 3. 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 4. Macimorelin test: peak GH \<=2.8 ng/mL 5. 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: 1. Insulin tolerance test: peak GH \<=1.8 ng/mL 2. Glucagon test: peak GH \<=1.8 ng/mL 3. Growth Hormone Releasing Peptide-2 (GHRP-2) tolerance test: peak GH \<=9 ng/mL 3. IGF-1 SDS \<= -1.0 at screening as measured by central laboratory. 4. hGH treatment naïve or no exposure to hGH therapy or GH secretagogue for at least 12 months prior to screening. 5. 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. 6. 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. 7. For males not on testosterone replacement therapy: morning (6:00 - 10:00AM) total testosterone within normal limits for age. 8. On a stable diet and exercise regime at screening with no intention to modify diet or exercise pattern during the trial, i.e., 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. Fundoscopy at screening without signs/symptoms of intracranial hypertension or diabetic retinopathy above stage 2 / moderate or above or any other retinal disease contraindicated to growth hormone therapy. For participants with a diagnosis of diabetes mellitus at screening, this must be documented with a fundus photograph. 11. Able and willing to provide a written informed consent and authorization for protected health information (PHI) disclosure in accordance with Good Clinical Practice (GCP). 12. Serum free thyroxine (fT4) in the normal range at screening as measured by central laboratory. Exclusion Criteria 1. Known Prader-Willi Syndrome and/or other genetic diseases that may have an impact on an endpoint. 2. Diabetes mellitus at screening if any of the following criteria are met: 1. Poorly controlled diabetes, defined as HbA1c \>7.5% at screening. 2. 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 3. Change in diabetes regimen (includes dose adjustment) within \<90 days prior and throughout screening 4. 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 5. 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) 3. Active malignant disease or history of malignancy. Exceptions to this exclusion criterion: 1. Resection of in situ carcinoma of the cervix uteri 2. Complete eradication of squamous cell or basal cell carcinoma of the skin 3. 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) 4. Evidence of growth of pituitary adenoma or other benign intracranial tumor within the last 12 months before screening. 5. Participants with acromegaly without remission / with documented remission less than 24 months prior to screening. 6. Participants with Cushing's disease without remission / with documented remission less than 24 months prior to screening. 7. Participants with prior cranial irradiation or hypothalamic-pituitary surgery: the procedure was to take place less than 12 months prior to screening. 8. Estimated glomerular filtration rate (eGFR) \<60 mL/min/1.73m\^2 determined based on Modification of Diet in Renal Disease (MDRD) equation. 9. Hepatic transaminases (i.e., aspartate aminotransferase \[AST\] or alanine aminotransferase \[ALT\]) \>3 times the upper limit of normal. 10. Heart failure New York Heart Association (NYHA) class 3 or greater (NYHA 1994). 11. Q-T interval, corrected by Fridericia's method (QTcF) \>= 451 milliseconds on 12-lead electrocardiogram (ECG) at screening. 12. Poorly controlled hypertension, defined as supine systolic blood pressure \>159 mmHg and/or supine diastolic blood pressure \>95 mmHg at screening. 13. Cerebrovascular accident within 5 years prior to screening. 14. Anabolic steroids (other than gonadal steroid replacement therapy) or oral/intravenous/intramuscular corticosteroids within 90 days prior to or throughout screening. 15. 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. 16. Known history of hypersensitivity and/or idiosyncrasy to any of the test compounds (somatropin) or excipients employed in this trial. 17. Known history of neutralizing anti-hGH antibodies. 18. Inability to undergo scanning by dual-energy x-ray absorptiometry (DXA) or a non-interpretable DXA scan at screening. 19. Female who is pregnant, breast-feeding or intends to become pregnant or is of childbearing potential (i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile) and not using adequate contraceptive methods. 20. Male participants must use a condom, or his female partner of childbearing potential must use an effective form of contraception as described above, from the beginning of screening to the last trial visit. 21. Known substance abuse or known (or previous) eating disorders, including anorexia nervosa, bulimia and severe gastrointestinal disease affecting normal eating (as judged by the investigator). 22. 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. 23. Participation in another interventional clinical trial involving an investigational compound within 26 weeks prior to screening or in parallel to this trial. 24. Currently using or have used within the last 3 days prior to screening: biotin \>0.03 mg/day from supplements 25. Known history of positive results of tests for human immunodeficiency virus (HIV) antibodies or hepatitis B and/or C (exceptions if vaccinated towards Hepatitis B virus and Hepatitis C virus). 26. Any of the following: acute critical illness, and complications following open heart surgery, abdominal surgery, multiple accidental traumas, acute respiratory failure, or similar conditions within 180 days prior to screening.

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Conditions

The condition(s) this trial relates to.

endocrine system disorder Failure to Thrive isolated congenital growth hormone deficiency pituitary dwarfism

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Endocrine system diseases Growth hormone deficiency Hormone deficiency

Contacts and locations

Locations

  • Ascendis Pharma Investigational Site

    Birmingham, Alabama, 35205, United States

  • Ascendis Pharma Investigational Site

    Phoenix, Arizona, 85048, United States

  • Ascendis Pharma Investigational Site

    Fresno, California, 93720, United States

  • Ascendis Pharma Investigational Site

    Los Angeles, California, 90048, United States

  • Ascendis Pharma Investigational Site

    Los Angeles, California, 90095, United States

  • Ascendis Pharma Investigational Site

    Palo Alto, California, 94304, United States

  • Ascendis Pharma Investigational Site

    Torrance, California, 90509, United States

  • Ascendis Pharma Investigational Site

    Chicago, Illinois, 60611, United States

  • Ascendis Pharma Investigational Site

    Indianapolis, Indiana, 46202, United States

  • Ascendis Pharma Investigational Site

    Boston, Massachusetts, 02114, United States

  • Ascendis Pharma Investigational Site

    Dearborn, Michigan, 48126, United States

  • Ascendis Pharma Investigational Site

    Rochester, Minnesota, 55905, United States

  • Ascendis Pharma Investigational Site

    St Louis, Missouri, 63110, United States

  • Ascendis Pharma Investigational Site

    Las Vegas, Nevada, 89148, United States

  • Ascendis Pharma Investigational Site

    Reno, Nevada, 89511, United States

  • Ascendis Pharma Investigational Site

    New York, New York, 10017, United States

  • Ascendis Pharma Investigational Site

    New York, New York, 10021, United States

  • Ascendis Pharma Investigational Site

    Morehead City, North Carolina, 28557, United States

  • Ascendis Pharma Investigational Site

    Portland, Oregon, 97239, United States

  • Ascendis Pharma Investigational Site

    Pittsburgh, Pennsylvania, 15212, United States

  • Ascendis Pharma Investigational Site

    Dallas, Texas, 75390, United States

  • Ascendis Pharma Investigational Site

    San Antonio, Texas, 78232, United States

  • Ascendis Pharma Investigational Site

    Seattle, Washington, 98108, United States

  • Ascendis Pharma Investigational Site

    Yerevan, 0075, Armenia

  • Ascendis Pharma Investigational Site

    Saint Leonards, New South Wales, 2065, Australia

  • Ascendis Pharma Investigational Site

    Sydney, New South Wales, 2109, Australia

  • Ascendis Pharma Investigational Site

    Box Hill, Victoria, 3128, Australia

  • Ascendis Pharma Investigational Site

    Fitzroy, Victoria, 3065, Australia

  • Ascendis Pharma Investigational Site

    Parkville, Victoria, 3050, Australia

  • Ascendis Pharma Investigational Site

    Perth, Western Australia, 6009, Australia

  • Ascendis Pharma Investigational Site

    Vancouver, British Columbia, V6Z 1Y6, Canada

  • Ascendis Pharma Investigational Site

    Halifax, Nova Scotia, B3H 1V7, Canada

  • Ascendis Pharma Investigational Site

    Copenhagen, 2100, Denmark

  • Ascendis Pharma Investigational Site

    Lyon, 69677, France

  • Ascendis Pharma Investigational Site

    Marseille, 13385, France

  • Ascendis Pharma Investigational Site

    Nantes, 44093, France

  • Ascendis Pharma Investigational Site

    Paris, 75013, France

  • Ascendis Pharma Investigational Site

    Tbilisi, 0144, Georgia

  • Ascendis Pharma Investigational Site

    Tbilisi, 0159, Georgia

  • Ascendis Pharma Investigational Site

    München, Bavaria, 80336, Germany

  • Ascendis Pharma Investigational Site

    Athens, Attica, 10676, Greece

  • Ascendis Pharma Investigational Site

    Athens, Attica, 11527, Greece

  • Ascendis Pharma Investigational Site

    Thessaloniki, Central Macedonia, 54636, Greece

  • Ascendis Pharma Investigational Site

    Thessaloniki, 546 42, Greece

  • Ascendis Pharma Investigational Site

    Beersheba, 8410100, Israel

  • Ascendis Pharma Investigational Site

    Haifa, 31048, Israel

  • Ascendis Pharma Investigational Site

    Petah Tikva, 4941480, Israel

  • Ascendis Pharma Investigational Site

    Tel Aviv, 6423906, Israel

  • Ascendis Pharma Investigational Site

    Genova, 16132, Italy

  • Ascendis Pharma Investigational Site

    Rome, 00161, Italy

  • Ascendis Pharma Investigational Site

    Rome, 00168, Italy

  • Ascendis Pharma Investigational Site

    Rozzano, 20089, Italy

  • Ascendis Pharma Investigational Site

    Kobe, Hyōgo, 650-0047, Japan

  • Ascendis Pharma Investigational Site

    Kawasaki, Kanagawa, 211-8533, Japan

  • Ascendis Pharma Investigational Site

    Yokohama, Kanagawa, 222-0036, Japan

  • Ascendis Pharma Investigational Site

    Yokohama, Kanagawa, 236-004, Japan

  • Ascendis Pharma Investigational Site

    Matsumoto, Nagano, Japan, Japan

  • Ascendis Pharma Investigational Site

    Kashihara, Nara, 634-8522, Japan

  • Ascendis Pharma Investigational Site

    Ishikawa, Okinawa, 920-0293, Japan

  • Ascendis Pharma Investigational Site

    Suita, Osaka, 565-0871, Japan

  • Ascendis Pharma Investigational Site

    Chiba, 260-8677, Japan

  • Ascendis Pharma Investigational Site

    Fukuoka, 812-8582, Japan

  • Ascendis Pharma Investigational Site

    Kagoshima, 890-8520, Japan

  • Ascendis Pharma Investigational Site

    Kawasaki, 216-8511, Japan

  • Ascendis Pharma Investigational Site

    Nagakute, 480-1195, Japan

  • Ascendis Pharma Investigational Site

    Okayama, 700-8558, Japan

  • Ascendis Pharma Investigational Site

    Osaka, 550-0006, Japan

  • Ascendis Pharma Investigational Site

    Tokyo, 108-8329, Japan

  • Ascendis Pharma Investigational Site

    Yamagata, 990-9585, Japan

  • Ascendis Pharma Investigational Site

    George Town, 10450, Malaysia

  • Ascendis Pharma Investigational Site

    Kota Bharu, 16150, Malaysia

  • Ascendis Pharma Investigational Site

    Malacca, 75400, Malaysia

  • Ascendis Pharma Investigational Site

    Putrajaya, 62250, Malaysia

  • Ascendis Pharma Investigational Site

    Leiden, 2300, Netherlands

  • Ascendis Pharma Investigational Site

    Palmerston North, Manawatu-Wanganui, 4440, New Zealand

  • Ascendis Pharma Investigational Site

    Wellington, 6021, New Zealand

  • Ascendis Pharma Investigational Site

    Krakow, 31-501, Poland

  • Ascendis Pharma Investigational Site

    Lodz, 93-338, Poland

  • Ascendis Pharma Investigational Site

    Poznan, 60-355, Poland

  • Ascendis Pharma Investigational Site

    Warsaw, 03-242, Poland

  • Ascendis Pharma Investigational Site

    Wroclaw, 50-367, Poland

  • Ascendis Pharma Investigational Site

    Bucharest, 11868, Romania

  • Ascendis Pharma Investigational Site

    Iași, 700106, Romania

  • Ascendis Pharma Investigational Site

    Timișoara, 300723, Romania

  • Ascendis Pharma Investigational Site

    Belgrade, 11000, Serbia

  • Ascendis Pharma Investigational Site

    Kragujevac, 34000, Serbia

  • Ascendis Pharma Investigational Site

    Bratislava, 82606, Slovakia

  • Ascendis Pharma Investigational Site

    Ľubochňa, 3491, Slovakia

  • Ascendis Pharma Investigational Site

    Seoul, 03722, South Korea

  • Ascendis Pharma Investigational Site

    Seoul, 05278, South Korea

  • Ascendis Pharma Investigational Site

    Seoul, 06591, South Korea

  • Ascendis Pharma Investigational Site

    Suwon, 443-721, South Korea

  • Ascendis Pharma Investigational Site

    Alicante, 3010, Spain

  • Ascendis Pharma Investigational Site

    Barcelona, 8035, Spain

  • Ascendis Pharma Investigational Site

    Barcelona, 8041, Spain

  • Ascendis Pharma Investigational Site

    Madrid, 28006, Spain

  • Ascendis Pharma Investigational Site

    Santiago de Compostela, 15706, Spain

  • Ascendis Pharma Investigational Site

    Seville, 41013, Spain

  • Ascendis Pharma Investigational Site

    Ankara, 06560, Turkey (Türkiye)

  • Ascendis Pharma Investigational Site

    Antalya, 07070, Turkey (Türkiye)

  • Ascendis Pharma Investigational Site

    Aydin, 09010, Turkey (Türkiye)

  • Ascendis Pharma Investigational Site

    Izmir, 35100, Turkey (Türkiye)

  • Ascendis Pharma Investigational Site

    İzmit, 41001, Turkey (Türkiye)

  • Ascendis Pharma Investigational Site

    Kayseri, 38039, Turkey (Türkiye)

  • Ascendis Pharma Investigational Site

    Ivano-Frankivsk, 76008, Ukraine

  • Ascendis Pharma Investigational Site

    Kharkiv, 61103, Ukraine

  • Ascendis Pharma Investigational Site

    Kyiv, 03115, Ukraine

  • Ascendis Pharma Investigational Site

    Kyiv, 04001, Ukraine

  • Ascendis Pharma Investigational Site

    Kyiv, 04114, Ukraine

  • Ascendis Pharma Investigational Site

    Vinnytsia, 21010, Ukraine

  • Ascendis Pharma Investigational Site

    Cardiff, CF14 4XW, United Kingdom

  • Ascendis Pharma Investigational Site

    Coventry, CV2 2DX, United Kingdom

  • Ascendis Pharma Investigational Site

    Leeds, LS9 7TF, United Kingdom

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